RegulationOrphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (United States), Breakthrough Therapy (United States)

Structure/Sequence

Sequence Code 319369934

Source: *****

Clinical Trials associated with Olipudase alfa

NCT06192576

/ RecruitingNot Applicable

A Prospective Observational Study to Assess the Long-term Safety and Immunogenicity of Olipudase Alfa Therapy During Routine Clinical Care in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency

US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care for acid sphingomyelinase deficiency (ASMD), but the study methodology remains non-interventional, as the additional collection of data from participants will not dictate treatment. The total overall study duration will be 5 years. The follow-up period will be a minimum of 1 year to a maximum of 3 years. The enrollment period will be up to 4 years, to allow a minimum of 1 year of follow-up for the last participant enrolled.

Start Date16 Apr 2024

Sponsor / Collaborator

Sanofi

NCT05359276

/ CompletedNot Applicable

Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France

Primary Objective:
To describe the lung, spleen and liver outcomes of olipudase alfa
Secondary Objectives:
* To describe the patient's characteristics
* To describe conditions of olipudase alfa use
* To describe safety data related to the use of olipudase alfa
* To describe complementary effectiveness outcomes parameters

Start Date10 Jun 2022

Sponsor / Collaborator

Sanofi

NCT06949358

/ CompletedPhase 2

A Long-Term Follow-up Study to Evaluate Safety and Tolerability of Olipudase Alfa in Patients Who Completed the DFI12712 or the LTS13632 Study in France

This is an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632 Study in France until olipudase alfa reimbursement is granted in France.
Study and treatment duration:
The period between the patient's completion of Study DFI12712 or LTS13632 and olipudase alfa reimbursement is available in France.
In case reimbursement will not be obtained, this study will end 5 years after starting.
Visit frequency: every 2 weeks.

Start Date18 Nov 2021

Sponsor / Collaborator

Sanofi

100 Clinical Results associated with Olipudase alfa

100 Translational Medicine associated with Olipudase alfa

100 Patents (Medical) associated with Olipudase alfa

Literatures (Medical) associated with Olipudase alfa

01 Jan 2026·JOURNAL OF INHERITED METABOLIC DISEASE

Natural‐History Mapping of Lysosomal Storage Disorders ( LSDs ): Gaucher Disease as a Model for Precision Care

Review

Author: Vaishnaw, Maya ; Mistry, Pramod. K. ; Ain, Noor Ul

ABSTRACT:

Natural‐history datasets have become pivotal for drug development and for shaping clinical‐practice guidelines in rare diseases, yet many lysosomal storage disorders would benefit from deep phenotyping and modern analytic methods. Our objective was to integrate the past decade of genomic, cellular, treatment‐outcome, and regulatory advances into a practical framework for capturing and interpreting natural‐history data, using Gaucher disease (GD) as a paradigm. We reviewed more than 300 peer‐reviewed articles (2005–2025), FDA guidance documents, and output from large real‐world registries. Particular attention was paid to long‐read GBA sequencing, biomarkers such as lyso‐Gb1, emerging newborn screening programs, and preliminary observational work that points toward multi‐state disease modeling. Key observations include: (i) Whole‐gene sequencing has expanded genotype–phenotype maps, revealing more than 70 recombinant GBA alleles that confound panel tests; (ii) registry trajectories suggest that formal multi‐state models could capture treatment‐modified courses and silent endpoints—monoclonal gammopathy, malignancy, Parkinson's disease, pulmonary arterial hypertension—better than current summary statistics; (iii) lyso‐Gb1 outperforms legacy biomarkers and now serves as a second‐tier newborn‐screening marker; (iv) Robust natural‐history evidence has already underpinned regulatory approvals across several lysosomal disorders—including olipudase alfa for ASMD, cerliponase alfa for CLN2, vestronidase alfa for MPS VII, and sebelipase alfa for infantile‐onset LAL‐D—demonstrating that well‐curated registries can serve as viable external controls for future LSD submissions. The convergence of deep phenotyping, genotype‐aware analytics, and systematic biomarker capture promises to transform natural‐history registries from descriptive archives into predictive engines. Gaucher disease offers a working template that, when extended across the LSD spectrum, can accelerate precision care and the development of next‐generation therapeutics.Trial Registration:ClinicalTrials.gov identifier: NCT00358943, NCT03291223.

01 Dec 2025·DRUGS

Current and Emerging Treatments for Acid Sphingomyelinase Deficiency

Review

Author: Silva Alves, Tamires ; Fonseca Siqueira, Ana Luíza ; Giugliani, Roberto

Acid sphingomyelinase deficiency is an ultra-rare disease characterised by generalised storage of sphingomyelin, caused by deficiency of the lysosomal enzyme acid sphingomyelinase, owing to the presence of biallelic pathogenic variants in the SMPD1 gene. The main disease manifestations are in the liver, spleen, lung and bone - with some patients having also involvement of the central nervous system. Patients show variable degrees of anaemia, thrombocytopenia and lipid abnormalities, among other findings. Clinically, acid sphingomyelinase deficiency spans from an acute neurovisceral form, with neurological involvement and early death (type A), to a chronic visceral disease, with no or minimal neurological manifestations (type B). An intermediate form, with chronic neurovisceral involvement, is presented by some patients (type A/B). Diagnosis involves the measurement of biomarkers, an assay of enzyme activity and genetic testing. Until a few years ago, treatment was mainly dependent on symptomatic management and bone marrow or solid organ (liver and/or lung) transplantation. In 2022, a specific enzyme replacement therapy with olipudase alfa was approved, and the results available indicate that it changed the therapeutic landscape for patients with acid sphingomyelinase deficiency type B and A/B. Research is being developed to address the needs of patients with acid sphingomyelinase deficiency type A, with gene therapy remaining as a promising approach.

01 Sep 2025·JOURNAL OF INHERITED METABOLIC DISEASE

Long‐Term Safety and Clinical Outcomes With Olipudase Alfa Enzyme Replacement Therapy in Children and Adolescents With Acid Sphingomyelinase Deficiency

Article

Author: Mengel, Eugen ; Srivastava, Shruti ; Witters, Peter ; Guffon, Nathalie ; Ganesh, Jaya ; Scarpa, Maurizio ; Diaz, George A. ; Armstrong, Nicole M. ; Jones, Simon A. ; Giugliani, Roberto ; Kim, Yong

ABSTRACT:

Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disease characterized by hepatosplenomegaly, pulmonary dysfunction, dyslipidemia, and growth deficits. Olipudase alfa (recombinant‐human ASM) is the only treatment for non‐central‐nervous‐system ASMD manifestations in children and adults. An open‐label long‐term study followed 4 to 8 years of olipudase alfa treatment in 20 children and adolescents with ASMD (baseline age (years) 1.5–17.5). At final assessments, splenomegaly and hepatomegaly were reduced relative to baseline (mean percent decrease in spleen and liver volumes 78% ± 1.2% and 59.8% ± 1.5%, respectively). Baseline splenomegaly was severe (spleen volume > 15 MN [multiples of normal]) or moderate (5–15 MN) ( n = 12 and n = 8, respectively) versus mild/absent (< 5 MN) ( n = 12) or moderate ( n = 8) at final assessment. Baseline hepatomegaly was severe (liver volume > 2.5 MN) ( n = 10) or moderate (1.25–2.5 MN) ( n = 10) versus mild/absent (< 1.25 MN) ( n = 19) or moderate ( n = 1) at final assessment. Among nine individuals able to perform assessments, diffusing capacity of the lung for carbon monoxide (DL CO ) impairment was severe (< 40%) ( n = 1), moderate (40%–60%) ( n = 4), or mild (60%–80%) ( n = 4) at baseline versus absent ( n = 4), mild ( n = 4) or moderate ( n = 1) at final assessment. Mean percent increase in DL CO was 53.7% ± 6.5%. At baseline, 10/20 children had clinical short stature (height Z ‐scores ≤ − 2) at baseline versus 0/20 at the final assessment. Atherogenic lipid profiles and liver function tests normalized within 2 years and remained stable. Adverse events were mostly mild or moderate, with 4 individuals experiencing 7 serious adverse events, all recovered/resolved. Interpretation: Enzyme replacement therapy with olipudase alfa in children and adolescents with chronic ASMD was well‐tolerated with clinically meaningful improvements in multiple disease parameters. Trial Registration: NCT02004704.

News (Medical) associated with Olipudase alfa

13 Nov 2025

·hlstherapeutics.com

HLS Therapeutics Announces Q3 2025 Financial Results

Adjusted EBITDA grew 19% in Q3 and 25% year-to-date Cash from operations grew 67% in Q3 and 121% year-to-date Announced new credit facility with lower rates, increased flexibility and reduced F/X risk Bempedoic acid approval expected in Q4 2025; preparations underway for Q2 2026 launch TORONTO, Nov. 13, 2025 /CNW/ - HLS Therapeutics Inc. ("HLS" or the "Company") (TSX: HLS), a pharmaceutical company focused on addressing unmet needs in the treatment of psychiatric disorders and cardiovascular disease, announces its financial results for the three and nine months ended September 30, 2025 ("Q3 2025" and "year-to-date"). All amounts are in thousands of United States ("U.S.") dollars unless otherwise stated. Q3 & YEAR-TO-DATE 2025 FINANCIAL HIGHLIGHTS (comparisons are to the respective 2024 periods) Q3 2025 revenue was $13.5 million, Adjusted EBITDA1 was $4.9 million and cash from operations was $2.5 million, compared to $14.1 million, $4.1 million and $1.5 million, respectively. Year-to-date 2025 revenue was $40.3 million, Adjusted EBITDA was $13.9 million and cash from operations was $10.6 million, compared to $41.1 million, $11.1 million and $4.8 million, respectively. In local currency, combined Canadian product sales for Vascepa and Clozaril were down 4% in Q3 2025 and up 2% year-to-date. For the year-to-date period, unfavorable foreign exchange negatively impacted revenue by approximately $0.8 million in fiscal 2025. U.S. sales of Clozaril in Q3 2025 were flat year-over-year and were up 1% year-to-date. Excluding cost of sales, operating expenses decreased 22% in Q3 2025 and 20% year-to-date. Vascepa contributed $0.6 million to Adjusted EBITDA in Q3 2025, its most profitable quarter to date. Q3 2025 CORPORATE HIGHLIGHTS Secured new credit agreement with improved terms, reducing annual interest expense by approximately $1.5 million. Made principal repayments on the Company's long-term debt totalling $2.5 million ($13.9 million year-to-date). Repurchased $0.8 million of common stock in Q3 2025. The Company has purchased 517,776 common shares at a cost of $1.8 million since launching its buyback on March 17, 2025. "We are pleased with the solid growth in profitability and cash flow we delivered in Q3 and throughout 2025," said Craig Millian, CEO at HLS. "Adjusted EBITDA increased 19% in Q3 and 25% year-to-date, and builds on the consistent improvement achieved over the past two years. Our U.S. Clozaril business has outperformed expectations, but, in addition to FX impacts, our Canadian business has faced headwinds in 2025 that have impacted sales performance." "Vascepa prescriptions have grown 24% year-to-date, and the product achieved its highest level of profitability this quarter. Overall, prescription growth fell short of our targets, due in part to the sales force transition in the first half. This transition was necessary to strengthen our capabilities and prepare for the bempedoic acid launch, and the team is now fully deployed. Clozaril continues to generate strong profit and cash flow and has successfully maintained its leadership for over 20 years in a generic market. Importantly, while patient numbers are down 1% versus prior year, they are still up 1% since year-end 2023. Although patient numbers have remained relatively stable, inventory fluctuations and regional competitive pressures can impact financial results year-to-year, and we have experienced that in 2025. We've adjusted guidance accordingly in our outlook." "Looking to the future, with the expected approval of bempedoic acid this month, we are excited to launch this important therapy in Canada in the first half of next year. Bempedoic acid addresses a significant unmet need for more than half a million Canadians who require additional LDL-cholesterol lowering beyond currently available options. We will leverage the strong operational synergies with Vascepa and anticipate significant growth potential for this combined cardiovascular franchise as we build HLS into a leading Canadian CV company." "We remain committed to operational efficiency to further optimize profitability, while a strengthening balance sheet will enable HLS to pursue additional growth opportunities beyond bempedoic acid. We expect growth in both revenue and Adjusted EBITDA next year and will provide detailed guidance when year-end results are reported in March." UPDATED 2025 OUTLOOKRevenue projections for the Company's Canadian product portfolio are denominated in local currency to account for ongoing FX rate fluctuations. 2025 financial targets have been updated and are as follows: Consolidated Adjusted EBITDA of $19.5-20.5 million (17-23% growth) is unchanged Consolidated revenue of $55-56 million, consisting of: Vascepa revenue growth in the mid-teen percentages (compared to prior estimate of 18-26% growth) Canada Clozaril revenue decline of 4-5% (compared to prior estimate of flat year-over-year revenue) U.S. Clozaril revenue decline of 2-4% year-over-year is unchanged Royalty revenue of $0.6-0.75 million (50-60% decline) is unchanged Vascepa revenue growth in the mid-teen percentages (compared to prior estimate of 18-26% growth) Canada Clozaril revenue decline of 4-5% (compared to prior estimate of flat year-over-year revenue) U.S. Clozaril revenue decline of 2-4% year-over-year is unchanged Royalty revenue of $0.6-0.75 million (50-60% decline) is unchanged Future results could be impacted by continued exchange rate volatility. Q3 & YEAR-TO-DATE 2025 FINANCIAL REVIEWThe Company's Management's Discussion and Analysis and Consolidated Financial Statements for the three and nine months ended September 30, 2025, are available at the Company's website and at its profile at SEDAR+. Revenue Three months ended September 30, Nine months ended September 30, 2025 2024 2025 2024 Product sales Canada 10,519 11,087 30,748 30,878 United States 2,813 2,803 9,033 8,907 13,332 13,890 39,781 39,785 Royalty revenue 179 195 524 1,292 13,511 14,085 40,305 41,077 Revenue for Q3 2025 decreased 4%, due primarily to lower Clozaril Canada revenue, lower royalty revenues and FX rate fluctuations, offset in part by revenue increases from Vascepa and Clozaril U.S. For the year-to-date period, revenue decreased 2% with flat product revenue offset by FX rate fluctuations and a 59% drop in royalty revenue. For the year-to-date period, unfavorable foreign exchange negatively impacted revenue by approximately $0.8 million in fiscal 2025. Product sales – Canada 000's of CAD Three months ended September 30, Nine months ended September 30, 2025 2024 % change 2025 2024 % change Clozaril 8,222 9,013 (8.8) % 24,740 26,009 (4.9) % Vascepa 6,207 6,077 2.1 % 18,069 15,955 13.2 % Other 65 27 173 54 14,494 15,117 (4.1) % 42,982 42,018 2.3 % Canadian product sales in local currency decreased 4% in Q3 2025 due to lower Clozaril Canada sales that were largely attributable to competitive dynamics and inventory burn at select hospitals. This was partially offset by 2% growth in Vascepa. For the year-to-date period, Canadian product sales increased 2%, with a 13% increase in Vascepa sales offset, in part, by a 5% decline in Clozaril Canada sales. The timing of orders can impact quarterly results, and for this reason, the Company views year-to-date revenue as a more relevant measure for the comparison of year-over-year revenue performance. Product Sales – United States In the U.S. market, Clozaril revenue for Q3 2025 was up slightly year-over-year, while revenue for the year-to-date period increased 1% compared to the same prior year period last year. Royalty revenues As expected, royalty revenue has declined since the sale of the Xenpozyme royalty interest in Q2 2024. Royalty revenue for the three and nine months ended September 30, 2025, was down 8% and 59%, respectively, compared to the same periods last year. HLS has one remaining royalty interest which generated $0.18 million in revenue in Q3 2025. Operating Expenses Three months ended September 30, Nine months ended September 30, 2025 2024 2025 2024 Cost of product sales 2,537 2,235 7,440 6,312 Selling and marketing 2,669 4,208 8,545 13,295 Medical, regulatory and patient support 1,272 1,439 4,074 4,124 General and administrative 2,111 2,077 6,334 6,255 8,589 9,959 26,393 29,986 With operating expenses, HLS continues to demonstrate strong operational discipline, helping to drive increases in Adjusted EBITDA and cash flow. For the three and nine months ended September 30, 2025, operating expenses, comprising sales and marketing, G&A, and medical, regulatory, and patient support, were down 22% and 20% compared to the same periods last year. This was primarily due to lower selling and marketing expenses following the Company's discontinuation of co-promotional activities with its marketing partner in August 2024 as well as several staff vacancies during 2025 that have now been filled. Cost of product sales increased for the three and nine months ended September 30, 2025, due primarily to higher Vascepa sales volumes. Adjusted EBITDA1 Three months ended September 30, Nine months ended September 30, 2025 2024 2025 2024 Net loss for the period (3,918) (4,844) (11,095) (16,632) Stock-based compensation 785 511 1,901 1,194 Amortization and depreciation 5,511 5,508 16,354 17,283 Finance and related costs, net 2,027 2,389 5,689 7,998 Other costs (income) 534 621 861 (2,740) Income tax expense (recovery) (17) (59) 202 3,988 Adjusted EBITDA 4,922 4,126 13,912 11,091 Adjusted EBITDA for the three and nine months ended September 30, 2025, increased 19% and 25%, respectively, compared to the same periods last year. The increases were primarily due to the ongoing focus on cost management and were partially offset by FX fluctuations and the previously mentioned decline in royalty revenue. In Q3 2025, the direct brand contribution from Clozaril to Adjusted EBITDA was $6.3 million, while the direct brand contribution from Vascepa to Adjusted EBITDA improved from negative $0.6 million in Q3 2024 to positive $0.6 million. Year-to-date, the direct brand contribution from Clozaril to Adjusted EBITDA was $19.2 million, while the direct brand contribution from Vascepa to Adjusted EBITDA improved from negative $3.8 million to positive $0.7 million. Net LossQ3 2025 net loss was ($3.9) million, or ($0.12) per share, compared to a net loss of ($4.8) million, or ($0.15) per share, in Q3 2024. Year-to-date net loss was ($11.1) million, or ($0.35) per share, compared to a net loss of ($16.6) million, or ($0.52) per share, in the same period last year. Net loss improved in both periods of 2025 due primarily to the previously noted factors that have positively impacted Adjusted EBITDA this year. Cash from Operations and Financial PositionCash generated from operations for the three and nine months ended September 30, 2025, was $2.5 million and $10.6 million, compared to $1.5 million and $4.8 million in the same periods last year. This represents year-over-year increases of 67% and 121%, respectively. Total borrowings under the credit agreement at September 30, 2025, were $53.1 million compared to $67.4 million at December 31, 2024, representing a reduction of 21%. Year-to-date, HLS has made debt principal repayments of $13.9 million and under its share buyback program, has purchased 517,776 shares at a cost of $1.8 million since launching the buyback on March 17, 2025. Cash was $10.8 million at September 30, 2025, compared to $17.5 million at December 31, 2024. The decrease reflects the debt principal repayments, and the share buyback activity described previously, offset, in part, by the significant increase in cash from operations. Q3 2025 CONFERENCE CALLHLS will hold a conference call today at 8:30 am Eastern Time to discuss its Q3 and year-to-date 2025 financial results. The call will be hosted by Mr. Craig Millian, CEO, Mr. John Hanna, CFO and Mr. Brian Walsh, CCO. To view the slides that accompany management's discussion, please use the webcast link. CONFERENCE ID: 19299 DATE: Thursday, November 13, 2025 TIME: 8:30 a.m. ET WEBCAST LINK: https://app.webin ar.net/0d539rj9qEn TRADITIONAL DIAL-IN NUMBER: 1-888-699-1199 or 1-416-945-7677 RAPIDCONNECT: To instantly join the conference call by phone, please use the following URL to easily register and be connected into the conference call automatically: https://emportal.ink/4pZWp5i TAPED REPLAY: 1-888-660-6345 or 1-289-819-1450 REPLAY CODE: 19299# The taped replay will be available for seven days and the archived webcast will be available for 365 days. A link to the live audio webcast and replay of the conference call will also be available on the events page of the investors section of HLS Therapeutics' website at www.hlstherapeutics.com. ABOUT HLS THERAPEUTICS INC.Formed in 2015, HLS is a pharmaceutical company focused on the acquisition and commercialization of late-stage development, commercial stage promoted and established branded pharmaceutical products in the North American markets. HLS's focus is on products targeting the central nervous system and cardiovascular therapeutic areas. HLS's management team is composed of seasoned pharmaceutical executives with a strong track record of success in these therapeutic areas and at managing products in each of these lifecycle stages. For more information visit: www.hlstherapeutics.com 1CAUTIONARY NOTE REGARDING NON-IFRS MEASURESThis press release refers to certain non-IFRS measures. These measures are not recognized measures under IFRS, do not have a standardized meaning prescribed by IFRS and are therefore unlikely to be comparable to similar measures presented by other companies. Rather, these measures are provided as additional information to complement those IFRS measures by providing further understanding of HLS's results of operations from management's perspective. Accordingly, they should not be considered in isolation nor as a substitute for analysis of HLS's financial information reported under IFRS. HLS uses non-IFRS measures to provide investors with supplemental measures of its operating performance and thus highlight trends in its core business that may not otherwise be apparent when relying solely on IFRS financial measures. HLS also believes that securities analysts, investors and other interested parties frequently use non-IFRS measures in the evaluation of issuers. HLS's management also uses non-IFRS measures in order to facilitate operating performance comparisons from period to period, prepare annual operating budgets and assess HLS's ability to meet its future debt service, capital expenditure and working capital requirements. In particular, management uses Adjusted EBITDA as a measure of HLS's performance. To reconcile net income (loss) for the period with Adjusted EBITDA, each of (i) "stock-based compensation", (ii) "amortization and depreciation", (iii) "finance and related costs, net", (iv) "other costs (income)", and (v) "income tax expense (recovery)" appearing in the Consolidated Statement of Net Income (Loss) are added to net income (loss) for the period to determine Adjusted EBITDA. Adjusted EBITDA does not have any standardized meaning prescribed by IFRS and is not necessarily comparable to similar measures presented by other companies. Adjusted EBITDA should not be considered in isolation or as a substitute for net income (loss) prepared in accordance with IFRS as issued by the IASB. FORWARD LOOKING INFORMATIONThis release includes forward-looking statements regarding HLS and its business. Such statements are based on the current expectations and views of future events of HLS's management. In some cases the forward-looking statements can be identified by words or phrases such as "may", "will", "expect", "plan", "anticipate", "intend", "potential", "estimate", "believe" or the negative of these terms, or other similar expressions intended to identify forward-looking statements, including, among others, statements with respect to HLS's pursuit of additional product and pipeline opportunities in certain therapeutic markets, statements regarding growth opportunities, expectations regarding financial performance, and the NCIB and ASPP. The forward-looking events and circumstances discussed in this release may not occur and could differ materially as a result of known and unknown risk factors and uncertainties affecting HLS, including risks relating to the specialty pharmaceutical industry, risks related to the regulatory approval process, economic factors and many other factors beyond the control of HLS. Forward-looking statements and information by their nature are based on assumptions and involve known and unknown risks, uncertainties and other factors which may cause HLS's actual results, performance or achievements, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statement or information. Accordingly, readers should not place undue reliance on any forward-looking statements or information. A discussion of the material risks and assumptions associated with this release can be found in the Company's Annual Information Form dated March 12, 2025, and Management's Discussion and Analysis dated November 12, 2025, both of which have been filed on SEDAR+ and can be accessed at www.sedarplus.ca. Accordingly, readers should not place undue reliance on any forward-looking statements or information. Except as required by applicable securities laws, forward-looking statements speak only as of the date on which they are made and HLS undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, or otherwise. HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION Unaudited [in thousands of U.S. dollars] As at As at September 30, 2025 December 31, 2024 ASSETS Current Cash 10,778 17,456 Accounts receivable 8,183 7,454 Inventories 7,106 9,058 Income taxes recoverable 80 71 Other current assets 1,389 1,361 Total current assets 27,494 35,400 Property, plant and equipment 1,267 997 Intangible assets 109,675 122,122 Deferred tax asset 1,247 857 Other non-current assets 545 528 Total assets 140,228 159,904 LIABILITIES AND SHAREHOLDERS' EQUITY Current Accounts payable and accrued liabilities 10,587 8,709 Provisions 7,725 8,367 Debt and other liabilities 4,754 5,317 Income taxes payable 390 152 Total current liabilities 23,456 22,545 Debt and other liabilities 51,109 61,944 Deferred tax liability 3,924 4,074 Total liabilities 78,489 88,563 Shareholders' equity Share capital 256,351 260,595 Contributed surplus 15,909 15,136 Accumulated other comprehensive loss (7,698) (10,210) Deficit (202,823) (194,180) Total shareholders' equity 61,739 71,341 Total liabilities and shareholders' equity 140,228 159,904 HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF LOSS Unaudited [in thousands of U.S. dollars, except per share amounts] Three months ended September 30, Nine months ended September 30, 2025 2024 2025 2024 Revenue 13,511 14,085 40,305 41,077 Expenses Cost of product sales 2,537 2,235 7,440 6,312 Selling and marketing 2,669 4,208 8,545 13,295 Medical, regulatory and patient support 1,272 1,439 4,074 4,124 General and administrative 2,111 2,077 6,334 6,255 Stock-based compensation 785 511 1,901 1,194 Amortization and depreciation 5,511 5,508 16,354 17,283 Finance and related costs, net 2,027 2,389 5,689 7,998 Other costs (income) 534 621 861 (2,740) Loss before income taxes (3,935) (4,903) (10,893) (12,644) Income tax expense (recovery) (17) (59) 202 3,988 Net loss for the period (3,918) (4,844) (11,095) (16,632) Net loss per share: Basic and diluted $(0.12) $(0.15) $(0.35) $(0.52) HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS Unaudited [in thousands of U.S. dollars] Three months ended September 30, Nine months ended September 30, 2025 2024 2025 2024 Net loss for the period (3,918) (4,844) (11,095) (16,632) Item that may be reclassified subsequently to net loss Unrealized foreign currency translation adjustment (1,240) 1,158 2,512 (2,178) Comprehensive loss for the period (5,158) (3,686) (8,583) (18,810) HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF CHANGES IN SHAREHOLDERS' EQUITY Unaudited [in thousands of U.S. dollars] Notes Share capital Contributed surplus Accumulated other comprehensive loss Deficit Total Balance as at December 31, 2024 260,595 15,136 (10,210) (194,180) 71,341 Shares repurchased 7 (4,244) — — 2,452 (1,792) Stock option expense 7, 10 — 773 — — 773 Net loss for the period — — — (11,095) (11,095) Unrealized foreign currency translation adjustment — — 2,512 — 2,512 Balance as at September 30, 2025 256,351 15,909 (7,698) (203,261) 61,739 Balance as at December 31, 2023 262,127 13,865 (2,838) (175,457) 97,697 Shares repurchased (1,532) — — 932 (600) Change in share purchase obligation — 300 — — 300 Stock option expense 7, 10 — 742 — — 742 Net loss for the period — — — (16,632) (16,632) Unrealized foreign currency translation adjustment — — (2,178) — (2,178) Balance as at September 30, 2024 260,595 14,907 (5,016) (191,157) 79,329 HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS Unaudited [in thousands of U.S. dollars] Three months ended June 30, Nine months ended September 30, 2025 2024 2025 2024 OPERATING ACTIVITIES Net loss for the period (3,918) (4,844) (11,095) (16,632) Adjustments to reconcile net loss to cash provided by operating activities Stock-based compensation 785 511 1,901 1,194 Amortization and depreciation 5,511 5,508 16,354 17,283 Gain on royalty sale — — — (3,381) Accreted interest expense 1,331 353 1,941 912 Foreign exchange (432) — (432) — Fair value adjustment on financial assets and liabilities — 18 — 523 Deferred income taxes (62) (47) (540) 3,753 Net change in non-cash working capital balances related to operations (739) 2 2,494 1,146 Cash provided by operating activities 2,476 1,501 10,623 4,798 INVESTING ACTIVITIES Intangible asset acquisition — — (1,000) — Other intangible additions (215) — (215) — Additions to property, plant and equipment (64) — (172) (2) Proceeds from royalty sale — — — 13,250 Payment of purchase consideration — (1,500) — (1,500) Cash provided by (used in) investing activities (279) (1,500) (1,387) 11,748 FINANCING ACTIVITIES Shares repurchased (783) — (1,792) (600) Repayment of old credit agreement borrowing (56,000) (14,965) (67,441) (18,533) New credit agreement borrowing 57,236 — 57,236 — Repayment of new credit agreement borrowing (2,518) — (2,518) — Debt costs (1,220) — (1,220) (1,191) Lease payments (159) (131) (453) (386) Cash used in financing activities (3,444) (15,096) (16,188) (20,710) Net decrease in cash during the period (1,247) (15,095) (6,952) (4,164) Foreign currency translation (128) 120 274 (248) Cash, beginning of period 12,153 32,515 17,456 21,952 Cash, end of period 10,778 17,540 10,778 17,540 SOURCE HLS Therapeutics Inc.

Financial Statement

03 Sep 2025

·pharma.economictimes.indiatimes.com

GST waiver for 36 drugs; formulations under 5% slab

New Delhi: Executing the much-awaited overhaul of the Goods and Services Tax (GST) regime, the GST Council has brought a major relief for patients by reducing the indirect tax on medicines to 5 per cent, along with exempting as many as 36 life-saving medicines and other critical drugs. The GST council—a statutory body highest decision making body on indirect taxes led by the Union Finance Ministry at its 56th meeting in the national capital has recommended to reduce the GST on all drugs and medicines (finished formulations) from 12 per cent to 5 per cent under the new two-tier tax structure of 5 and 18 per cent slabs to be effective from September 22. Additionally the body has also recommended to exempt GST on 33 lifesaving drugs and on 3 other medicines used for treatment of cancer, rare diseases and other severe chronic diseases. Speaking at a press briefing after the council 10.5-hour long marathon meet, Finance Minister Nirmala Sitharaman said, “all recommendations have been cleared with zero reservations and have corrected various inverted duty structure problems, classification related issues and that this rationalization exercise will bring predictability and stability of GST.” “These reforms have been carried out with a focus on the common man, and after a rigorous evaluation process in most sectors including healthcare, the rates have come down drastically, she added. Previously, medicines in India were taxed at 5 per cent for life-saving and essential drugs, and at 12 per cent for others but following the announcement all medicines will attract a standard duty of 5 per cent with extensions for several medicines specified at the end of this article. Welcoming the move, Sudarshan Jain, Secretary General, IPA said, “the decision to exempt life-saving and cancer medicines from GST, is a step that will bring direct relief to patients and their families. and, the reduction in GST on a wide range of medicines from 12 per cent to 5 per cent will further help to ease the overall treatment burden and make essential therapies more affordable.” Meanwhile, for companies, the clarity on a 5 per cent slab and exemptions removes long-standing ambiguity, enabling transparent pricing and better market planning. The move will expand access in semi-urban and rural markets, ease litigation, and free up resources for innovation, said Manoj Mishra, Partner and Tax Controversy Management Leader, Grant Thornton Bharat LLP. Notably, on the issue of inverted duty involving APIs and key starting materials—which are currently taxed at 18 per cent against 12 or 5 per cent for finished formulations—further clarity is awaited from the industry. ETPharma earlier reported that, in a letter to the Finance Minister, the national chemist body, the All India Organisation of Chemists and Druggists (AIOCD) had urged, “to bring all medicines including vitamins, probiotics, nutritional, food supplements under the 5 per cent GST slab. The rate rationalization comes days after Prime Minister Narendra Modi had pitched for introducing next-gen reforms across sectors to “increase ease of living for the common man and strengthen the economy.” However, this rationalization brings a revenue shortfall challenge for governments with states likely to bear a greater share of the burden, as they receive over 40 percent of the inter-state inflows in addition to their own SGST collections. Responding to a media query at the media briefing Arvind Shrivastava, Revenue Secretary said that, based on the consumption of FY23-24 the ministry has estimated that the net revenue implication of this proposal will be around Rs 48,000 crore. “However the exercise will trigger buoyancy effects and will have a positive impact on consumer and compliance. And therefore we believe this proposal will be fiscally sustainable both for centers and the states,” Shrivastava noted. ' S.no. Drug Indication Brand name and (Company) 1 Onasemnogene abeparvovec SMA (One-time gene therapy) Zolgensma (Novartis) 2 Asciminib Blood Cancer Scemblix (Novartis) 3 Mepolizumab Asthma Nucala (GSK) 4 Pegylated Liposomal Irinotecan Pancreatic Cancer Onivyde (Ipsen) 5 Daratumumab Cancer Darzalex (J&J subsidiary) 6 Daratumumab subcutaneous Cancer Darzalex Faspro (J&J subsidiary) 7 Teclistamab Blood Cancer Tecvayli (J&J subsidiary) 8 Amivantamab Lung Cancer Rybrevant (J&J subsidiary) 9 Alectinib Lung Cancer Alecensa (Roche) 10 Risdiplam SMA Evrysdi (Roche) 11 Obinutuzumab Blood Cancer Gazyva (Roche) 12 Polatuzumab vedotin Blood Cancer Polivy (Roche) 13 Entrectinib Cancer Rozlytrek (Roche) 14 Atezolizumab Cancer (PD-L1 immunotherapy) Tecentriq (Roche) 15 Spesolimab GPP flares Spevigo (Boehringer Ingelheim) 16 Velaglucerase Alpha Gaucher Vpriv (Takeda) 17 Agalsidase Alfa Fabry disease Replagal (Takeda) 18 Rurioctocog Alpha Pegol Hemophilia Adynovate (Takeda) 19 Idursulphatase Type II Mucopolysaccharidosis Elaprase (Takeda) 20 Alglucosidase Alfa Pompe Myozyme (Sanofi) 21 Laronidase Type I Mucopolysaccharidosis Aldurazyme (Sanofi) 22 Olipudase Alfa ASMD Xenpozyme (Sanofi) 23 Tepotinib Lung Cancer Tepmetko (Maerck KGaA) 24 Avelumab Cancer (PDL-1 immunotherapy) Bavencio (Pfizer) 25 Emicizumab Hemophilia Hemlibra (Roche subsidiary) 26 Belumosudil cGVHD - 27 Miglustat Gaucher Zavesca (J&J subsidiary) 28 Velmanase Alfa Alpha-mannosidosis - 29 Alirocumab Cholesterol lowering drug (LDL-C) Praluent (Sanofi) 30 Evolocumab Cholesterol lowering drug (LDL-C) Repatha (Amgen) 31 Cystamine Bitartrate Cystagon - 32 CI-Inhibitor injection Hereditary Angioedema - 33 Inclisiran Cholesterol lowering drug (LDL-C) Leqvio (Novartis) 34 Agalsidase Beta Fabry disease Fabrazyme (Sanofi) 35 Imiglucerase Gaucher Cerezyme (Sanofi) 36 Eptacog alfa activated recombinant coagulation factor VIIa Hemophilia NovoSeven Note: Above is the list of medicines exempted from GST tax recommended by the 56th GST Council Meet/ Source: PIB By Abhijeet Singh ,

Gene Therapy

14 Aug 2025

·hlstherapeutics.com

HLS Therapeutics Announces Q2 2025 Financial Results

Adjusted EBITDA grew 21% in Q2 and 29% year-to-date Cash from operations grew 83% in Q2 and 147% year-to-date Made principal repayments on the Company's long-term debt totalling $8.5 million Expanded cardiovascular portfolio by licensing Canadian rights to NEXLETOL® and NEXLIZET® from Esperion Therapeutics TORONTO, Aug. 14, 2025 /CNW/ - HLS Therapeutics Inc. ("HLS" or the "Company") (TSX: HLS), a pharmaceutical company focused on addressing unmet needs in the treatment of psychiatric disorders and cardiovascular disease, announces its financial results for the three and six months ended June 30, 2025 ("Q2 2025" and "year-to-date"). All amounts are in thousands of United States ("U.S.") dollars unless otherwise stated. Q2 & YEAR-TO-DATE 2025 FINANCIAL HIGHLIGHTS (comparisons are to the respective 2024 periods) Q2 2025 revenue was $14.2 million, Adjusted EBITDA1 was $5.2 million and cash from operations was $4.6 million, compared to $14.5 million, $4.3 million and $2.5 million, respectively. Year-to-date 2025 revenue was $26.8 million, Adjusted EBITDA was $9.0 million and cash from operations was $8.1 million, compared to $27 million, $7 million and $3.3 million, respectively. In local currency, combined Canadian product sales for Vascepa and Clozaril were flat in Q2 2025 and were up 6% year-to-date. US sales of Clozaril were up 1% in Q2 2025 and were up 2% year-to-date. Excluding cost of sales, operating expenses decreased 18% in Q2 2025 and 19% year-to-date. Vascepa made a positive contribution to Adjusted EBITDA in Q2 2025 for the third straight quarter. Q2 2025 CORPORATE HIGHLIGHTS Announced in-license agreement with Esperion Therapeutics Inc. (NASDAQ:ESPR) to commercialize NEXLETOL2 and NEXLIZET2 in Canada. Made principal repayments on the Company's long-term debt totalling $8.5 million. Repurchased $0.8 million of common stock in Q2 2025. Year-to-date, the Company has purchased $1.0 million of common stock at an average price of C$4.56 per share. "Our year-to-date results reflect continued execution on our key strategic priorities as we position the Company to resume its growth trajectory," said Craig Millian, Chief Executive Officer at HLS. "We remain on track to achieve full-year guidance, with strong underlying performance demonstrated by 29% year-to-date Adjusted EBITDA growth, robust cash flow generation, and continued balance sheet strengthening through significant debt repayments." "Looking ahead, we're excited to introduce NEXLETOL and NEXLIZET in Canada, addressing an important treatment need for at-risk Canadian patients. These medicines have the potential to more than double the size of our cardiovascular business and will further establish HLS as a leading Canadian-based company focused on cardiovascular risk reduction. With Health Canada approval expected by year-end and launch planned for Q2 2026, we believe HLS is well-positioned for our next phase of growth." Q2 & YEAR-TO-DATE 2025 FINANCIAL REVIEWThe Company's Management's Discussion and Analysis and Consolidated Financial Statements for the three and six months ended June 30, 2025, are available at the Company's website and at its profile at SEDAR+. Revenue Three months ended June 30, Six months ended June 30, 2025 2024 2025 2024 Product sales Canada 10,521 10,637 20,229 19,791 United States 3,502 3,462 6,220 6,104 14,023 14,099 26,449 25,895 Royalty revenue 148 420 345 1,097 14,171 14,519 26,794 26,992 Revenue for Q2 2025 decreased 2%, due primarily to lower royalty revenues and FX rate fluctuations. Year-to-date 2025, revenue was essentially flat with 2% growth in product revenue being offset by a 69% drop in royalty revenue. Product sales – Canada 000's of CAD Three months ended June 30, Six months ended June 30, 2025 2024 % change 2025 2024 % change Clozaril 8,589 9,131 (5.9) % 16,518 16,996 (2.8) % Vascepa 5,884 5,407 8.8 % 11,862 9,878 20.1 % Other 76 14 108 27 14,549 14,552 0.0 % 28,488 26,901 5.9 % Canadian product sales in local currency were flat in Q2 2025 and up 6% year-to-date, compared to the same periods last year. The Q2 2024 period benefited from the timing of certain orders shifting from Q1 2024 because of the Easter holiday timing last year. For this reason, the Company views year-to-date revenue as a more relevant measure for the comparison of year-over-year revenue performance. Product Sales – United States In the U.S. market, Clozaril revenue for the three and six months ended June 30, 2025, increased 1% and 2%, respectively, compared to the same prior year periods. Royalty revenues As expected, following the sale of the Xenpozyme royalty interest in Q2 2024, royalty revenue has declined. Royalty revenue for the three and six months ended June 30, 2025, was down 65% and 69%, respectively, compared to the same periods last year. HLS has one remaining royalty interest which generated $0.15 million in revenue in Q2 2025. Operating Expenses Three months ended June 30, Six months ended June 30, 2025 2024 2025 2024 Cost of product sales 2,505 2,303 4,903 4,077 Selling and marketing 3,046 4,561 5,876 9,087 Medical, regulatory and patient support 1,366 1,420 2,802 2,685 General and administrative 2,084 1,977 4,223 4,178 9,001 10,261 17,804 20,027 Cost of product sales increased for the three and six months ended June 30, 2025, due primarily to higher Vascepa sales volumes. Operating expenses in Q2 2025, excluding cost of product sales, decreased by 18% compared to Q2 2024, and by 19% for the year-to-date period. This was primarily due to lower selling and marketing expenses following the Company's discontinuation of co-promotional activities with its marketing partner in August 2024. While selling and marketing expenses will remain below 2024 levels throughout the year, quarterly selling and marketing expenses are expected to be moderately higher for the remainder of 2025 as several staff vacancies have now been filled. Adjusted EBITDA1 Three months ended June 30, Six months ended June 30, 2025 2024 2025 2024 Net loss for the period (2,741) (5,682) (7,177) (11,788) Stock-based compensation 465 427 1,116 683 Amortization and depreciation 5,483 5,856 10,843 11,775 Finance and related costs, net 1,690 2,942 3,662 5,609 Other costs 31 (3,361) 327 (3,361) Income tax expense 242 4,076 219 4,047 Adjusted EBITDA 5,170 4,258 8,990 6,965 Adjusted EBITDA for the three and six months ended June 30, 2025, increased 21% and 29%, respectively, compared to the same periods last year. The increases were primarily due to steady performance from the Company's marketed products and the ongoing focus on cost management and were partially offset by FX fluctuations and the previously mentioned decline in royalty revenue. Excluding royalty revenue, Adjusted EBITDA for the three and six months ended June 30, 2025, would have increased 31% and 47%, respectively, compared to the same periods last year. In Q2 2025, the direct brand contribution from Clozaril to Adjusted EBITDA was $7.1 million, while the direct brand contribution from Vascepa to Adjusted EBITDA improved from negative $1.6 million in Q2 2024 to positive $0.1 million. Year-to-date, the direct brand contribution from Clozaril to Adjusted EBITDA was $12.9 million, while the direct brand contribution from Vascepa to Adjusted EBITDA improved from negative $3.2 million to positive $0.1 million. Net LossQ2 2025 net loss was ($2.7) million, or ($0.09) per share, compared to a net loss of ($5.7) million, or ($0.18) per share, in Q2 2024. Year-to-date net loss was ($7.2) million, or ($0.23) per share, compared to a net loss of ($11.8) million, or ($0.37) per share, in the same period last year. Net loss improved in both periods of 2025 due primarily to the previously noted factors that have positively impacted Adjusted EBITDA this year. Cash from Operations and Financial PositionCash generated from operations for the three and six months ended June 30, 2025, was $4.6 million and $8.1 million, compared to $2.5 million and $3.3 million in the same periods last year. This represents year-over-year increases of 83% and 147%, respectively. Total borrowings under the credit agreement at June 30, 2025, were $56.0 million compared to $67.4 million at December 31, 2024, and $84.9 million at June 30, 2024. This represents a reduction of 34% from June 30, 2024. During Q2 2025, HLS made principal payments on its term loan totaling $8.5 million and spent $0.8 million to purchase shares for cancellation under its Normal Course Issuer Bid. Year-to-date, HLS has made debt principal repayments of $11.4 million and under its share buyback program, has purchased 308,928 shares at a cost of $1 million since launching the buyback on March 17, 2025. Cash was $12.2 million at June 30, 2025, compared to $17.5 million at December 31, 2024. The decrease reflects the debt principal repayments, and the share buyback activity described previously, offset, in part, by the significant increase in cash from operations. 2025 OUTLOOKRevenue projections for the Company's Canadian product portfolio are denominated in local currency to account for ongoing FX rate fluctuations. 2025 financial targets are unchanged and as follows: Vascepa revenue of C$26.5-28.5 million (18-26% growth) Canada Clozaril sales of C$35.5-36 million (flat year-over-year) U.S. Clozaril sales of $12-12.3 million (2-4% decline) Royalty revenue of $0.6-0.75 million (50-60% decline) Consolidated Adjusted EBITDA of $19.5-20.5 million (17-23% growth) Future results could be impacted by continued exchange rate volatility. Q2 2025 CONFERENCE CALLHLS will hold a conference call today at 8:30 am Eastern Time to discuss its Q2 and year-to-date 2025 financial results. The call will be hosted by Mr. Craig Millian, CEO, Mr. John Hanna, CFO and Mr. Brian Walsh, CCO. To view the slides that accompany management's discussion, please use the webcast link. CONFERENCE ID: 66543 DATE: Thursday, August 14, 2025 TIME: 8:30 a.m. ET WEBCAST LINK: https://app.webinar.net/Zo9wjb9V5KR TRADITIONAL DIAL-IN NUMBER: 1-888-699-1199 or 1-416-945-7677 RAPIDCONNECT: To instantly join the conference call by phone, please use the following URL to easily register and be connected into the conference call automatically: https://emportal.ink/4kiYlSu TAPED REPLAY: 1-888-660-6345 or 1-289-819-1450 REPLAY CODE: 66543# The taped replay will be available for 14 days and the archived webcast will be available for 365 days. A link to the live audio webcast of the conference call will also be available on the events page of the investors section of HLS Therapeutics' website at www.hlstherapeutics.com. Please connect at least 15 minutes before the conference call to ensure enough time for any software download required to hear the webcast. ABOUT HLS THERAPEUTICS INC.Formed in 2015, HLS is a pharmaceutical company focused on the acquisition and commercialization of late-stage development, commercial stage promoted and established branded pharmaceutical products in the North American markets. HLS's focus is on products targeting the central nervous system and cardiovascular therapeutic areas. HLS's management team is composed of seasoned pharmaceutical executives with a strong track record of success in these therapeutic areas and at managing products in each of these lifecycle stages. For more information visit: www.hlstherapeutics.com 1CAUTIONARY NOTE REGARDING NON-IFRS MEASURESThis press release refers to certain non-IFRS measures. These measures are not recognized measures under IFRS, do not have a standardized meaning prescribed by IFRS and are therefore unlikely to be comparable to similar measures presented by other companies. Rather, these measures are provided as additional information to complement those IFRS measures by providing further understanding of HLS's results of operations from management's perspective. Accordingly, they should not be considered in isolation nor as a substitute for analysis of HLS's financial information reported under IFRS. HLS uses non-IFRS measures to provide investors with supplemental measures of its operating performance and thus highlight trends in its core business that may not otherwise be apparent when relying solely on IFRS financial measures. HLS also believes that securities analysts, investors and other interested parties frequently use non-IFRS measures in the evaluation of issuers. HLS's management also uses non-IFRS measures in order to facilitate operating performance comparisons from period to period, prepare annual operating budgets and assess HLS's ability to meet its future debt service, capital expenditure and working capital requirements. In particular, management uses Adjusted EBITDA as a measure of HLS's performance. To reconcile net income (loss) for the period with Adjusted EBITDA, each of (i) "stock-based compensation", (ii) "amortization and depreciation", (iii) "finance and related costs, net", (iv) "other costs (income)", and (v) "income tax expense (recovery)" appearing in the Consolidated Statement of Net Income (Loss) are added to net income (loss) for the period to determine Adjusted EBITDA. Adjusted EBITDA does not have any standardized meaning prescribed by IFRS and is not necessarily comparable to similar measures presented by other companies. Adjusted EBITDA should not be considered in isolation or as a substitute for net income (loss) prepared in accordance with IFRS as issued by the IASB. REFERENCES2: NEXLETOL® (bempedoic acid) and NEXLIZET® (bempedoic acid and ezetimibe) are the commercial brand names in the U.S. The brand names in Canada are to be confirmed upon approval by Health Canada. FORWARD LOOKING INFORMATIONThis release includes forward-looking statements regarding HLS and its business. Such statements are based on the current expectations and views of future events of HLS's management. In some cases the forward-looking statements can be identified by words or phrases such as "may", "will", "expect", "plan", "anticipate", "intend", "potential", "estimate", "believe" or the negative of these terms, or other similar expressions intended to identify forward-looking statements, including, among others, statements with respect to HLS's pursuit of additional product and pipeline opportunities in certain therapeutic markets, statements regarding growth opportunities, expectations regarding financial performance, and the NCIB and ASPP. The forward-looking events and circumstances discussed in this release may not occur and could differ materially as a result of known and unknown risk factors and uncertainties affecting HLS, including risks relating to the specialty pharmaceutical industry, risks related to the regulatory approval process, economic factors and many other factors beyond the control of HLS. Forward-looking statements and information by their nature are based on assumptions and involve known and unknown risks, uncertainties and other factors which may cause HLS's actual results, performance or achievements, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statement or information. Accordingly, readers should not place undue reliance on any forward-looking statements or information. A discussion of the material risks and assumptions associated with this release can be found in the Company's Annual Information Form dated March 12, 2025, and Management's Discussion and Analysis dated August 13, 2025, both of which have been filed on SEDAR+ and can be accessed at www.sedarplus.ca. Accordingly, readers should not place undue reliance on any forward-looking statements or information. Except as required by applicable securities laws, forward-looking statements speak only as of the date on which they are made and HLS undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, or otherwise. HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION Unaudited [in thousands of U.S. dollars] As at As at June 30, 2025 December 31, 2024 ASSETS Current Cash 12,153 17,456 Accounts receivable 8,968 7,454 Inventories 6,823 9,058 Income taxes recoverable 80 71 Other current assets 2,011 1,361 Total current assets 30,035 35,400 Property, plant and equipment 1,048 997 Intangible assets 116,047 122,122 Deferred tax asset 1,386 857 Other non-current assets 556 528 Total assets 149,072 159,904 LIABILITIES AND SHAREHOLDERS' EQUITY Current Accounts payable and accrued liabilities 8,339 8,709 Provisions 11,814 8,367 Debt and other liabilities 7,025 5,317 Income taxes payable 411 152 Total current liabilities 27,589 22,545 Debt and other liabilities 50,799 61,944 Deferred tax liability 4,125 4,074 Total liabilities 82,513 88,563 Shareholders' equity Share capital 258,063 260,595 Contributed surplus 14,788 15,136 Accumulated other comprehensive loss (6,458) (10,210) Deficit (199,834) (194,180) Total shareholders' equity 66,559 71,341 Total liabilities and shareholders' equity 149,072 159,904 HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF LOSS Unaudited [in thousands of U.S. dollars, except per share amounts] Three months ended June 30, Six months ended June 30, 2025 2024 2025 2024 Revenue 14,171 14,519 26,794 26,992 Expenses Cost of product sales 2,505 2,303 4,903 4,077 Selling and marketing 3,046 4,561 5,876 9,087 Medical, regulatory and patient support 1,366 1,420 2,802 2,685 General and administrative 2,084 1,977 4,223 4,178 Stock-based compensation 465 427 1,116 683 Amortization and depreciation 5,483 5,856 10,843 11,775 Finance and related costs, net 1,690 2,942 3,662 5,609 Other costs (income) 31 (3,361) 327 (3,361) Loss before income taxes (2,499) (1,606) (6,958) (7,741) Income tax expense 242 4,076 219 4,047 Net loss for the period (2,741) (5,682) (7,177) (11,788) Net loss per share: Basic and diluted $(0.09) $(0.18) $(0.23) $(0.37) HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS Unaudited [in thousands of U.S. dollars] Three months ended June 30, Six months ended June 30, 2025 2024 2025 2024 Net loss for the period (2,741) (5,682) (7,177) (11,788) Item that may be reclassified subsequently to net loss Unrealized foreign currency translation adjustment 3,440 (963) 3,752 (3,336) Comprehensive income (loss) for the period 699 (6,645) (3,425) (15,124) HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF CHANGES IN SHAREHOLDERS' EQUITY Unaudited [in thousands of U.S. dollars] Share capital Contributed surplus Accumulated other comprehensive loss Deficit Total Balance as at December 31, 2024 260,595 15,136 (10,210) (194,180) 71,341 Shares repurchased (2,532) — — 1,523 (1,009) Change in share purchase obligation — (878) — — (878) Stock option expense — 530 — — 530 Net loss for the period — — — (7,177) (7,177) Unrealized foreign currency translation adjustment — — 3,752 — 3,752 Balance as at June 30, 2025 258,063 14,788 (6,458) (199,834) 66,559 Balance as at December 31, 2023 262,127 13,865 (2,838) (175,457) 97,697 Shares repurchased (1,532) — — 932 (600) Change in share purchase obligation — 300 — — 300 Stock option expense — 433 — — 433 Net loss for the period — — — (11,788) (11,788) Unrealized foreign currency translation adjustment — — (3,336) — (3,336) Balance as at June 30, 2024 260,595 14,598 (6,174) (186,313) 82,706 HLS THERAPEUTICS INC. INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS Unaudited [in thousands of U.S. dollars] Three months ended June 30, Six months ended June 30, 2025 2024 2025 2024 OPERATING ACTIVITIES Net loss for the period (2,741) (5,682) (7,177) (11,788) Adjustments to reconcile net loss to cash provided by operating activities Stock-based compensation 465 427 1,116 683 Amortization and depreciation 5,483 5,856 10,843 11,775 Gain on royalty sale — (3,381) — (3,381) Accreted interest expense 302 277 610 559 Fair value adjustment on financial assets and liabilities — 318 — 505 Deferred income taxes (189) 3,861 (478) 3,800 Net change in non-cash working capital balances related to operations 1,284 843 3,233 1,144 Cash provided by operating activities 4,604 2,519 8,147 3,297 INVESTING ACTIVITIES Intangible asset acquisition (1,000) — (1,000) — Additions to property, plant and equipment (87) — (108) (2) Proceeds from royalty sale — 13,250 — 13,250 Cash provided by (used in) investing activities (1,087) 13,250 (1,108) 13,248 FINANCING ACTIVITIES Shares repurchased (833) (317) (1,009) (600) Repayment of credit agreement borrowing (8,481) (1,493) (11,441) (3,568) Debt costs — (658) — (1,191) Lease payments (151) (112) (294) (255) Cash used in financing activities (9,465) (2,580) (12,744) (5,614) Net increase (decrease) in cash during the period (5,948) 13,189 (5,705) 10,931 Foreign currency translation 367 (117) 402 (368) Cash, beginning of period 17,734 19,443 17,456 21,952 Cash, end of period 12,153 32,515 12,153 32,515 SOURCE HLS Therapeutics Inc.

Financial Statement

100 Deals associated with Olipudase alfa

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KEGG	Wiki	ATC	Drug Bank
D10820	-	-	DB12835

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Acid sphingomyelinase deficiency	Japan	Sanofi KK	28 Mar 2022

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Niemann-Pick Disease, Type A	Phase 3	Netherlands	Genzyme Corp.	20 Jul 2015
Niemann-Pick Disease, Type B	Phase 3	Netherlands	Genzyme Corp.	20 Jul 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02004704 (Pubmed \| J Inherit Metab Dis)	Phase 3	Acid sphingomyelinase deficiency	20	Olipudase alfa	whpdmkatqq(dnonqoxyaj) = Adverse events were mostly mild or moderate, with 4 individuals experiencing 7 serious adverse events, all recovered/resolved. ufxzpwbknj (adkmyorixt )	Positive	01 Sep 2025
ASCEND-Peds/ASCEND (ATS2025)	Not Applicable	Acid sphingomyelinase deficiency \| Lung Diseases, Interstitial	-	Olipudase alfa	ksciqvxbtb(blveoeghdz) = rash urdxrefubj (sjiwsktvky )	Positive	16 May 2025
NCT02004704 (CTgov)	Phase 2	Niemann-Pick Disease, Type A	25	Olipudase Alfa (Olipudase Alfa: Participants From DFI13412 (Adults))	pjqjnwxkna = bfxvcftxiq unkjkqjgzp (wzuobynlia, dnxuiyjhma - orhorfiokt) View more	-	03 Jul 2024
				Olipudase Alfa (Olipudase Alfa: Participants From DFI13803 (Pediatrics))	pjqjnwxkna = aqcbxsitjb unkjkqjgzp (wzuobynlia, kwpjhfrywt - swvfvrzmxx) View more
NCT02004691 (ASCEND, Pubmed \| Orphanet J Rare Dis)	Phase 2/3	Acid sphingomyelinase deficiency	36	Olipudase alfa	jzmujoqsie(wzaedkquav) = 99% of treatment-emergent adverse events were mild or moderate, with one treatment-related serious adverse event (extrasystoles; previously documented cardiomyopathy). No individual discontinued due to an adverse event. tzbeikernl (okdjtahwbq )	Positive	02 Dec 2023
				Placebo
EHA2023	Not Applicable	-	-	Olipudase alfa	gimxsumpnv(ricjawefld) = dxjsplqdlz ibwwocvgyk (lwtilfqiqc, 7.3) View more	-	08 Jun 2023
SSIEM2022	Not Applicable	Acid sphingomyelinase deficiency	60	Olipudase alfa	hzpqnkfuxr(sadccrtvht) = mvbuwygjsb svjjsxjcdp (gdlosoymfk, ±15.6)	-	30 Aug 2022
NCT02004691 (ASCEND, EHA2022)	Phase 2/3	Acid sphingomyelinase deficiency	36	Olipudase alfa	rzkahbsqsr(oirteiyosn) = hnkbrnogab vjsufjkfpq (jftljkrobd, 6.2) View more	-	10 Jun 2022
NCT02004691 (ASCEND, CTgov)	Phase 2/3	Niemann-Pick Disease, Type A	36	placebo (saline) (Placebo)	rizzbgzglc(qfntnagwpy) = aajfetvvkv iepfpkvlto (zeprclqmci, 10.76) View more	-	24 May 2022
				Olipudase alfa (Olipudase Alfa)	rizzbgzglc(qfntnagwpy) = hhsilofnqt iepfpkvlto (zeprclqmci, 10.99) View more
ATS2022	Not Applicable	Acid sphingomyelinase deficiency \| Lung Diseases, Interstitial	-	Olipudase alfa	vzlsxxymqj(wxmwwjnkme) = vpqmrjiudz wnpzfzeeed (mgivpedgry, ±3.0)	-	15 May 2022
				Placebo	vzlsxxymqj(wxmwwjnkme) = jmtukqutyf wnpzfzeeed (mgivpedgry, ±0)
NCT02004691 (ASCEND, Pubmed \| Genet Med) Manual	Phase 2/3	Acid sphingomyelinase deficiency	36	Olipudase alfa	flgfsckyqy(cmzhfugrzy) = ohlcujjvki rcvdcwqwsw (grbmotrbtt ) View more	Positive	26 Apr 2022
				Placebo	flgfsckyqy(cmzhfugrzy) = jwfuoumswd rcvdcwqwsw (grbmotrbtt ) View more

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