Nivolumab

LONDON, May 15, 2024 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its financial results for the first quarter ended March 31, 2024, and provided an update on recent corporate highlights. The Company reported cash and cash equivalents of $48.7 million as of March 31, 2024 and continues to expect this to fund its operations into 2026. “2024 is off to an exciting start following the completion of enrollment by our partner Ultragenyx in both the Orbit and Cosmic studies of setrusumab in Osteogenesis Imperfecta (OI) along with the continued advancement of the pre-launch activities in Mereo’s European territories. These include further identification of patients who could potentially benefit from setrusumab treatment, the ongoing dialogues with the HTA’s and Payors in Europe to support both rapid adoption and efficient reimbursement following a potential European approval, and SATURN,” said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo. “Further, in parallel with the ongoing partnering discussions for alvelestat, we have completed the initial validation work for the St. George’s Respiratory Questionnaire (SGRQ) instrument in the Alpha-1 Antitrypsin Deficiency-associated Lung Disease (AATD-LD) population.” First Quarter 2024 Highlights, Recent Developments and Anticipated Milestones Setrusumab (UX143) Enrollment completed in the Phase 3 Orbit and Cosmic studies of setrusumab in OI, conducted by our partner Ultragenyx. The Phase 3 portion of the Orbit Phase 2/3 trial completed enrollment with 158 patients aged 5 to 25 years old. Patients were randomized 2:1 to receive setrusumab or placebo, and the study has a primary efficacy endpoint of annualized clinical fracture rate. Additional longer-term safety and efficacy data from the Phase 2 portion of the Orbit study are expected in the second half of 2024. The Cosmic study was initiated in the second half of 2023 and completed enrollment with 69 patients. Cosmic is a Phase 3 open-label, randomized study in patients aged 2 to <7 years evaluating setrusumab compared to bisphosphonates on reduction in total annualized clinical fracture rate. The initial results from the IMPACT Survey, a joint initiative between the Osteogenesis Imperfecta Foundation, Osteogenesis Imperfecta Federation Europe and its members, and Mereo, were recently made available. IMPACT is the largest ever burden of disease survey on the impact of OI on patients, physicians and caregivers. Additional information is available at The Company continues to invest in pre-launch activities and other studies to generate further evidence that will inform coverage, pricing and reimbursement decisions in Mereo’s European territory. These include SATURN (Systematic Accumulation of Treatment practices and Utilization, Real world evidence, and Natural history data for OI) which is expected to provide a coordinated data set across multiple treatment centers for OI across European countries, to support pricing and reimbursement decisions. The Company’s patient identification activities are continuing, with a focus on adult patients in the key five European countries where these activities were previously initiated, and adult and pediatric patients in additional European countries. Alvelestat (MPH-966) Mereo remains on-track to submit the completed initial validation work supporting the use of the SGRQ-Total Score as the primary efficacy endpoint to the FDA, alongside the detailed Phase 3 study protocol in the first half of 2024. The content validation of the SGRQ using semi-structured interviews with patients with AATD-LD from several sites in the United States has been completed. The analysis concluded that the current SGRQ instrument is fit for purpose with valid content measures for patients with AATD-LD and is suitable for use as a key Clinical Outcomes Assessment endpoint. The global Phase 3 study, which is supported by positive data from the ASTRAEUS and ATALANTa studies, is expected to enroll approximately 220 early- and late-stage patients with the severe Pi*ZZ genotype and confirmed emphysema, with a treatment period of 18 months. If the Phase 3 trial is successful, it is expected to support full approvals of alvelestat in the U.S. and Europe. Mereo continues to actively engage with multiple potential partners for the development and commercialization of alvelestat and aims to initiate the Phase 3 study with a partner around the end of 2024. Etigilimab (MPH-313) Etigilimab in combination with nivolumab, is being studied in an ongoing investigator-led single-arm, two-stage, open-label Phase 1b/2 trial in a subtype of platinum-resistant recurrent ovarian cancer (clear cell ovarian cancer) at the MD Anderson Cancer Center, financed by the Cancer Focus Fund. Based on the results to-date, the study has been expanded from the initial 10 patients to 20 patients and an update may be provided by the investigator in the second half of 2024 or early 2025. First Quarter 2024 Financial Results Total research and development (R&D) expenses decreased by $1.3 million, or 25%, from $5.3 million in the first quarter of 2023 to $4.0 million in the first quarter of 2024. The decrease was primarily due to a $1.8 million reduction in R&D expenses for etigilimab, partially offset by increases of $0.3 million of R&D expenses for both setrusumab and alvelestat. The reduction in etigilimab expenses was primarily due to the winding down and completion during 2023 of the open label Phase 1b/2 basket study in combination with an anti-PD-1 in a range of tumor types. Program expenses for setrusumab are in relation to increases in ongoing activities in Europe, and input into development, regulatory and manufacturing plans with our partner, Ultragenyx, as the global development program is funded by Ultragenyx pursuant to our license and collaboration agreement. Program expenses for alvelestat primarily include the preparatory work for the Phase 3 study, including manufacturing and drug formulation activities, SGRQ validation activities and regulatory interactions. General and administrative expenses decreased by $0.5 million, or 8%, from $6.4 million in the first quarter of 2023 to $5.9 million in the first quarter of 2024. The decrease is primarily related to recognition of a $1.7 million reduction in expenses in the first quarter of 2024 for amounts from our depository to reimburse certain expenses incurred by us in respect of our ADR program, partially offset by an increase in employee-related expenses and professional fees. No similar reimbursements from our depository were recognized in the first quarter of 2023. Net loss for the first quarter of 2024 was $9.0 million, compared to $12.1 million during the first quarter of 2023, primarily reflecting an operating loss of $9.9 million. As of March 31, 2024, the Company had cash and cash equivalents of $48.7 million, compared to $57.4 million as of December 31, 2023. The Company’s guidance remains unchanged and it continues to expect, based on current operational plans, that its existing cash and cash equivalents balance will enable it to fund its currently committed clinical trials, operating expenses, and capital expenditure requirements into 2026. This guidance does not include any potential upfront payments associated with a partnership for alvelestat or business development activity around any of the Company’s non-core programs. Total ordinary shares issued as of March 31, 2024 were 701,349,434. Total ADS equivalents as of March 31, 2024 were 140,269,886, with each ADS representing five ordinary shares of the Company. About Mereo BioPharma Mereo BioPharma is a biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. The Company has two rare disease product candidates, setrusumab for the treatment of osteogenesis imperfecta (OI) and alvelestat primarily for the treatment of severe alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD). The Company’s partner, Ultragenyx Pharmaceutical, Inc., has completed enrollment in the Phase 3 portion of a pivotal Phase 2/3 pediatric study in young adults (5 to 25 years old) for setrusumab in OI and in the Phase 3 study in pediatric patients (2 to <7 years old) in the first half of 2024. The partnership with Ultragenyx includes potential additional milestone payments of up to $245 million and royalties to Mereo on commercial sales in Ultragenyx territories. Mereo has retained EU and UK commercial rights and will pay Ultragenyx royalties on commercial sales in those territories. Setrusumab has received orphan designation for osteogenesis imperfecta from the EMA and FDA, PRIME designation from the EMA and has pediatric disease designation from the FDA. Alvelestat has received U.S. Orphan Drug Designation for the treatment of AATD and Fast Track designation from the FDA. Following results from ASTRAEUS and ATALANTa in AATD-lung disease, the Company has aligned with the FDA and the EMA on the primary endpoints for a Phase 3 pivotal study which if successful could enable full approval in both the U.S. and Europe. In addition to the rare disease programs, Mereo has two oncology product candidates in clinical development. Etigilimab (anti-TIGIT) has completed a Phase 1b/2 basket study evaluating its safety and efficacy in combination with an anti-PD-1 in a range of tumor types including three rare tumors and three gynecological carcinomas – cervical, ovarian, and endometrial and is an ongoing Phase 1b/2 investigator led study at the MD Anderson Cancer Center in clear cell ovarian cancer; Navicixizumab, for the treatment of late line ovarian cancer, has completed a Phase 1 study and has been partnered with Feng Biosciences Inc. in a global licensing agreement that includes milestone payments and royalties. Mereo has entered into an exclusive global license agreement with ReproNovo SA for the development and commercialization of leflutrozole, a non-steroidal aromatase inhibitor. Under the terms of the agreement, ReproNovo, a reproductive medicine company, is responsible for all future development and commercialization of leflutrozole. Forward-Looking Statements This press release contains “forward-looking statements” that involve substantial risks and uncertainties. All statements other than statements of historical fact contained herein are forward-looking statements within the meaning of Section 27A of the United States Securities Act of 1933, as amended, and Section 21E of the United States Securities Exchange Act of 1934, as amended. Forward-looking statements usually relate to future events and anticipated revenues, earnings, cash flows or other aspects of our operations or operating results. Forward-looking statements are often identified by the words “believe,” “expect,” “anticipate,” “plan,” “intend,” “foresee,” “should,” “would,” “could,” “may,” “estimate,” “outlook” and similar expressions, including the negative thereof. The absence of these words, however, does not mean that the statements are not forward-looking. These forward-looking statements are based on the Company’s current expectations, beliefs and assumptions concerning future developments and business conditions and their potential effect on the Company. While management believes that these forward-looking statements are reasonable as and when made, there can be no assurance that future developments affecting the Company will be those that it anticipates. All of the Company’s forward-looking statements involve known and unknown risks and uncertainties some of which are significant or beyond its control and assumptions that could cause actual results to differ materially from the Company’s historical experience and its present expectations or projections. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical development process; the Company’s reliance on third parties to conduct and provide funding for its clinical trials; the Company’s dependence on enrollment of patients in its clinical trials; and the Company’s dependence on its key executives. You should carefully consider the foregoing factors and the other risks and uncertainties that affect the Company’s business, including those described in the “Risk Factors” section of its Annual Report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in the Company’s subsequent filings with the Securities and Exchange Commission. The Company wishes to caution you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof. The Company undertakes no obligation to publicly update or revise any of our forward-looking statements after the date they are made, whether as a result of new information, future events or otherwise, except to the extent required by law. Mereo BioPharma Contacts: Mereo +44 (0)333 023 7300 Denise Scots-Knight, Chief Executive Officer Christine Fox, Chief Financial Officer Burns McClellan (Investor Relations Adviser to Mereo) +01 646 930 4406 Lee Roth Investors investors@mereobiopharma.com MEREO BIOPHARMA GROUP PLC CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands, except share and per share data) (Unaudited) March 31, December 31, 2024 2023 Assets Current assets: Cash and cash equivalents $ 48,660 $ 57,421 Prepaid expenses and other current assets 3,188 5,156 Research and development incentives receivables 1,648 1,183 Total current assets 53,496 63,760 Property and equipment, net 360 405 Operating lease right-of-use assets 1,109 1,245 Intangible assets 972 1,089 Total assets $ 55,937 $ 66,499 Liabilities Current liabilities: Accounts payable $ 2,455 $ 2,346 Accrued expenses 2,539 5,467 Convertible loan notes – current 4,630 — Operating lease liabilities – current 662 652 Other current liabilities 718 1,021 Total current liabilities 11,004 9,486 Convertible loan notes – non-current — 4,394 Warrant liabilities – non-current 855 412 Operating lease liabilities – non-current 727 906 Other non-current liabilities 513 764 Total liabilities 13,099 15,962 Commitments and contingencies (Note 15) Shareholders’ Equity Ordinary shares, par value £0.003 per share; 701,349,434 shares issued at March 31, 2024 (December 31, 2023: 701,217,089). 2,775 2,775 Treasury shares — (1,230 ) Additional paid-in capital 486,927 486,107 Accumulated deficit (428,581 ) (419,630 ) Accumulated other comprehensive loss (18,283 ) (17,485 ) Total shareholders’ equity 42,838 50,537 Total liabilities and shareholders’ equity $ 55,937 $ 66,499 MEREO BIOPHARMA GROUP PLC CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (In thousands, except share and per share amounts) (Unaudited) Three Months Ended March 31, 2024 2023 Revenue $ — $ — Operating expenses: Cost of revenue — 347 Research and development (3,994 ) (5,307 ) General and administrative (5,906 ) (6,450 ) Loss from operations (9,900 ) (11,410 ) Other income/(expenses) Interest income 617 306 Interest expense (310 ) (800 ) Changes in the fair value of financial instruments (448 ) 542 Foreign currency transaction gain/(loss), net 613 (1,207 ) Other expenses, net — (6 ) Benefit from research and development tax credit 477 499 Net loss before income tax (8,951 ) (12,076 ) Income tax benefit — — Net loss $ (8,951 ) $ (12,076 ) Loss per share – basic and diluted $ (0.01 ) $ (0.02 ) Weighted average shares outstanding – basic and diluted 700,263,490 623,925,635 Net loss $ (8,951 ) $ (12,076 ) Other comprehensive (loss)/income – Foreign currency transaction adjustments, net of tax (798 ) 2,278 Total comprehensive loss $ (9,749 ) $ (9,798 )

SAN DIEGO, May 14, 2024 (GLOBE NEWSWIRE) -- TRACON Pharmaceuticals, Inc. (Nasdaq: TCON), a clinical stage biopharmaceutical company utilizing a cost-efficient, CRO-independent product development platform to advance its pipeline of novel targeted cancer therapeutics and to partner with other life science companies, today announced financial results for the first quarter ended March 31, 2024. The Company will host a conference call and webcast today at 4:30 PM Eastern Time / 1:30 PM Pacific Time. “With ENVASARC fully enrolled we are focused on leveraging our Product Development Platform to generate non-dilutive capital through either an additional license or by replacing a CRO and executing clinical trials for partners at a lower cost compared to a CRO but still at a premium to our costs using a pay for performance model,” said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. “We look forward to reporting the final response assessment data in all patients from the ENVASARC Phase 2 pivotal trial, which are expected in the third quarter.” Recent Corporate Highlights In April, we announced updated interim safety and efficacy data from the ENVASARC Phase 2 pivotal trial in 73 patients treated with single agent envafolimab. The objective response rate (ORR) was 11% by investigator review and 5.5% by blinded independent central review (BICR), all of which were confirmed responses. Envafolimab monotherapy was generally well tolerated and median duration of response by BICR was greater than six months. The primary endpoint of the study is achievement of an ORR by BICR in nine of 82 patients (11%) treated with envafolimab and median duration of response of greater than six months is a key secondary endpoint. In April, the Company announced that the Nasdaq Hearings Panel granted the Company’s request for an extension to demonstrate compliance with all applicable criteria for continued listing on The Nasdaq Capital Market, including the $1.00 bid price and $2.5 million stockholders’ equity requirements, through June 3, 2024, provided the Company execute a reverse stock split and file an S-1, both of which the Company executed in April. The Company continues to consider alternatives to address the $2.5 million stockholders’ equity requirement on or before June 3, 2024. Expected Upcoming Milestone Report the final response assessment data including duration of response in all patients from the ENVASARC Phase 2 pivotal trial, which are expected in the third quarter of 2024. First Quarter 2024 Financial Results Cash, cash equivalents and restricted cash were $8.0 million at March 31, 2024, compared to $8.6 million at December 31, 2023, which is expected to fund the Company late into the third quarter of 2024. Research and development expenses for the first quarter of 2024 were $1.9 million, compared to $5.0 million for the first quarter of 2023. The decrease was primarily related to completing enrollment of the ENVASARC Phase 2 pivotal trial in 2024. General and administrative expenses for the first quarter of 2024 were $1.4 million, compared to $2.3 million for the first quarter of 2023. Net loss for the first quarter of 2024 was $3.2 million, compared to $8.5 million for the first quarter of 2023. Conference Call Details To access the call by phone, please register using this link and you will be provided with dial-in details. A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com. After the live webcast, a replay will remain available on TRACON’s website for 60 days. About Envafolimab Envafolimab (KN035), a single-domain antibody against PD-L1 invented by Alphamab Oncology and licensed by TRACON, is the first approved subcutaneously injected PD-(L)1 inhibitor. Envafolimab was approved by the Chinese NMPA in November 2021 in adult patients with MSI-H/dMMR advanced solid tumors who failed systemic treatment and have no satisfactory alternative treatment options. In December 2019, Alphamab Oncology, 3D Medicines and TRACON entered into a collaboration whereby TRACON has the right to develop and commercialize envafolimab in soft tissue sarcoma in North America. Envafolimab is currently being studied in the ENVASARC Phase 2 pivotal trial in the United States sponsored by TRACON and a Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines. TRACON has received orphan drug designation from the U.S. Food and Drug Administration for envafolimab for patients with soft tissue sarcoma and fast track designation from the U.S. Food and Drug Administration for envafolimab (KN035) for patients with locally advanced, unresectable or metastatic undifferentiated pleomorphic sarcoma (UPS) and myxofibrosarcoma (MFS) who have progressed on one or two prior lines of chemotherapy. About ENVASARC (NCT04480502) The ENVASARC Phase 2 pivotal trial is a multicenter, open label, randomized, non-comparative, parallel cohort study at 30 top cancer centers in the United States and the United Kingdom that began dosing in December 2020. TRACON enrolled patients in ENVASARC with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor. A total of 82 evaluable patients have received treatment with single agent envafolimab at 600 mg every three weeks. The primary endpoint is objective response rate by central review in nine of 82 patients (11%) with duration of response a key secondary endpoint. About TRACON TRACON is a clinical-stage biopharmaceutical company utilizing a cost-efficient, CRO-independent, product development platform to advance its pipeline of novel targeted cancer therapeutics and to partner with other life science companies. The Company’s clinical-stage pipeline includes: Envafolimab, a PD-L1 single-domain antibody given by rapid subcutaneous injection that is being studied in the pivotal ENVASARC trial for sarcoma; YH001, a potential best-in-class CTLA-4 antibody in Phase 1 development; and TRC102, a Phase 2 small molecule drug candidate for the treatment of lung cancer. TRACON is actively seeking additional corporate partnerships through a profit-share or revenue-share partnership, or through franchising TRACON’s product development platform. TRACON believes it can serve as a solution for companies without clinical and commercial capabilities in the United States or who wish to become CRO-independent. To learn more about TRACON and its product pipeline, visit TRACON’s website at www.traconpharma.com. Forward-Looking Statements Statements made in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward‐looking statements. Such statements include, but are not limited to, TRACON’s plans to further develop product candidates; TRACON’s ability to regain compliance with Nasdaq continued listing standards and maintain the listing of its securities on a national securities exchange; TRACON’s plans to further license out its platform or replace CROs and generate non-dilutive capital; expectations regarding the timing and scope of clinical trials and availability of clinical data, including the timing and results of accrual and data from TRACON’s ENVASARC Phase 2 pivotal trial; expected development, regulatory and commercial milestones and timing thereof; potential utility of product candidates; TRACON’s cash runway; and TRACON’s business development strategy and goals, including the ability to enter into additional collaborations or licensing arrangements. Risks that could cause actual results to differ from those expressed in these forward‐looking statements include: the risk that TRACON needs substantial additional capital to continue as a going concern and to enroll or complete its ongoing clinical trials as currently planned, if at all; risks associated with clinical development and regulatory approval of novel pharmaceutical product candidates; whether TRACON or others will be able to complete or initiate clinical trials on TRACON’s expected timelines, if at all, including due to risks associated with geopolitical and macroeconomic events; the fact that future preclinical studies and clinical trials, including ENVARSAC, may not be successful or otherwise consistent with results from prior studies; the fact that TRACON has limited control over whether or when third party collaborators complete on-going trials or initiate additional trials of TRACON’s product candidates; the fact that TRACON’s collaboration agreements are subject to early termination; whether TRACON will be able to enter into additional collaboration agreements or licensing arrangements or arrangements whereby TRACON replaces CROs on favorable terms or at all; potential changes in regulatory requirements in the United States and foreign countries; TRACON’s reliance on third parties for the development of its product candidates, including the conduct of its clinical trials and manufacture of its product candidates; whether TRACON will be able to obtain additional financing; whether TRACON will experience unanticipated costs or other events that cause TRACON’s cash runway to not extend late into the third quarter of 2024; whether TRACON will remain listed on Nasdaq; and other risks described in TRACON’s filings with the Securities and Exchange Commission under the heading “Risk Factors”. All forward‐looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. TRACON undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made except as required by law. TRACON Pharmaceuticals, Inc.Unaudited Condensed Consolidated Statements of Operations(in thousands, except share and per share data) Three Months EndedMarch 31, 2024 2023 Revenue$100 $— Operating expenses: Research and development 1,878 4,969 General and administrative 1,434 2,344 Total operating expenses 3,312 7,313 Loss from operations (3,212) (7,313)Total other income (expense) 44 (1,191)Net loss$(3,168) $(8,504) Loss per share, basic and diluted$(1.33) $(6.76)Weighted-average common shares outstanding, basic and diluted 2,389,519 1,258,096 TRACON Pharmaceuticals, Inc.Unaudited Condensed Consolidated Balance Sheets(in thousands) March 31, December 31, 2024 2023 Assets Current assets: Cash and cash equivalents$7,891 $8,564 Prepaid and other assets 511 526 Total current assets 8,402 9,090 Property and equipment, net 33 37 Restricted Cash 73 73 Other assets 847 905 Total assets$9,355 $10,105 Liabilities and Stockholders’ Deficit Current liabilities: Accounts payable and accrued expenses$10,036 $9,755 Accrued compensation and related expenses 414 427 Total current liabilities 10,450 10,182 Other long-term liabilities 667 732 Commitments and contingencies Stockholders’ deficit: Common stock 3 2 Additional paid-in capital 241,902 239,688 Accumulated deficit (243,667) (240,499)Total stockholders’ deficit (1,762) (809)Total liabilities and stockholders’ deficit$9,355 $10,105 Company Contact:Investor Contact:Charles TheuerBrian RitchieChief Executive OfficerLifeSci Advisors LLC(858) 550-0780(212) 915-2578ctheuer@traconpharma.combritchie@lifesciadvisors.com