lipoprotein(a)

-More than 35 authorized treatment centers (ATCs) activated globally for CASGEVY™ and approximately 20 patients have had cells collected across all regions as of mid-July- -Clinical trials ongoing for next generation CAR T product candidates, CTX112™ and CTX131™ targeting CD19 and CD70 respectively, across multiple indications- -Clinical trials opened for CTX112 in systemic lupus erythematosus (SLE) and for CTX131 in hematological malignancies- -Clinical trials ongoing for in vivo gene editing product candidates, CTX310™ and CTX320™ targeting ANGPTL3 and LPA, respectively- -Clinical trial ongoing for CTX211™, an allogeneic, hypoimmune, gene-edited, stem cell derived product candidate for the treatment of Type 1 Diabetes (T1D)- -Strong balance sheet with approximately $2 billion in cash, cash equivalents, and marketable securities as of June 30, 2024- ZUG, Switzerland and BOSTON, Aug. 05, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the second quarter ended June 30, 2024. “In addition to the continued momentum of CASGEVY’s launch, we are making significant progress across the rest of our pipeline,” said Samarth Kulkarni, Ph.D., Chief Executive Officer and Chairman of CRISPR Therapeutics. “We continue to advance our next generation CD19-directed CAR T cell program, CTX112, which has the potential to be best-in-class in both oncology and autoimmune indications. We have opened the clinical trial for CTX131 in hematologic malignancies, and continue to dose-escalate with our in vivo directed programs, CTX310 and CTX320. We are making outstanding progress across our early stage discovery efforts and are well-positioned to realize our mission of bringing multiple transformative medicines to patients in need.” Recent Highlights and Outlook Hemoglobinopathies and CASGEVY™ (exagamglogene autotemcel [exa-cel]) CRISPR Therapeutics has two next generation approaches with the potential to significantly expand the addressable population with SCD and TDT. The Company continues to advance its internally developed targeted conditioning program, an anti-CD117 (c-Kit) antibody-drug conjugate (ADC), through preclinical studies. Additionally, the Company has ongoing research efforts to enable in vivo editing of hematopoietic stem cells. This work could obviate the need for conditioning altogether, expand geographic reach, and enable the treatment of multiple additional other diseases beyond SCD and TDT.Enrollment has been completed in two global Phase 3 studies of CASGEVY in people 5 to 11 years of age with SCD or TDT and the trials are ongoing.In June, positive long-term data from CLIMB-111, CLIMB-121 and the long-term follow-up study of CASGEVY were presented at the 2024 Annual European Hematology Association Congress. These long-term data from more than 100 patients dosed with CASGEVY, with the longest follow-up of more than five years, confirm the transformative, consistent, and durable clinical benefits of CASGEVY over time.The French National Authority for Health (HAS) approved Vertex’s request for the implementation of an early access program (EAP) for the use of CASGEVY in indicated patients with SCD. HAS previously approved the implementation of an EAP for CASGEVY in indicated patients with TDT in the first quarter of 2024.As of mid-July, more than 35 authorized treatment centers (ATCs) have been activated globally, including centers in all regions where CASGEVY is approved, and approximately 20 patients have had cells collected across all regions.CASGEVY is approved in the U.S., Great Britain, the European Union (EU), the Kingdom of Saudi Arabia (KSA), and the Kingdom of Bahrain (Bahrain) for the treatment of both sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), and launches are ongoing. Regulatory submissions for CASGEVY have been completed in both SCD and TDT in Switzerland and Canada where it received Priority Review. CASGEVY is the first therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals established in 2015. As part of an amendment to the collaboration agreement in 2021, Vertex now leads global development, manufacturing, regulatory and commercialization of CASGEVY with support from CRISPR Therapeutics. Immuno-Oncology and Autoimmune Diseases CTX131 is currently in an ongoing clinical trial in solid tumors. In addition, the Company has opened a clinical trial for CTX131 in hematologic malignancies including T cell lymphomas (TCL). Allogeneic CAR T approaches for TCL may have greater potential to meet the unmet need in this patient population given the patients’ own T cells are not suitable for autologous manufacturing.CRISPR Therapeutics opened a clinical trial for CTX112 in systemic lupus erythematosus (SLE), with the potential to expand into additional autoimmune indications in the future. Early clinical studies conducted by third parties have shown that CD19-directed autologous CAR T therapy can produce long-lasting remissions in multiple autoimmune indications by deeply depleting B cells. The Company’s first generation allogeneic CD19-directed CAR T program has demonstrated effective depletion of B cells in oncology settings, which supports the potential for CTX112 in autoimmune diseases.CTX112 is being developed for both oncology and autoimmune indications. In oncology settings, CTX112 is in a Phase 1/2 trial for CD19 positive relapsed or refractory B-cell malignancies, and the Company expects to report preliminary clinical data this year.CRISPR Therapeutics’ next generation allogeneic CAR T candidates reflect the Company’s mission of innovating continuously to bring potentially transformative medicines to patients as quickly as possible. Clinical trials are ongoing for the Company’s next generation CAR T product candidates, CTX112™ and CTX131™, targeting CD19 and CD70, respectively, across multiple indications. CTX112 and CTX131 both contain novel potency edits which can lead to significantly higher CAR T cell expansion and cytotoxicity, potentially representing best-in-class allogeneic CAR T products for these targets. In Vivo CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) platform for the delivery of CRISPR/Cas9 to the liver. The first two in vivo programs utilizing this proprietary platform, CTX310™ and CTX320™, are directed towards validated therapeutic targets associated with cardiovascular disease.CTX310 is currently in an ongoing Phase 1 trial targeting ANGPTL3 in patients with homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (SHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias. Natural loss-of-function mutations in ANGPTL3 are associated with reduced low-density lipoprotein (LDL-C), triglycerides (TG) and atherosclerotic cardiovascular disease (ASCVD) risk without any negative impact on overall health.CTX320 is currently in an ongoing Phase 1 trial targeting LPA in patients with elevated lipoprotein(a) [Lp(a)], which has shown to have an independent association with major adverse cardiovascular events (MACE). Up to 20% of the global population has elevated Lp(a) levels.The Company continues to advance two additional preclinical programs, CTX340™ targeting angiotensinogen (AGT) for the treatment of refractory hypertension and CTX450™ targeting 5’ aminolevulinic acid synthase (ALAS1) for the treatment of acute hepatic porphyrias (AHP). CRISPR Therapeutics has initiated IND/CTA-enabling studies for CTX340, targeting hepatic AGT for hypertension, and expects to initiate both clinical trials in the second half of 2025. Regenerative Medicine CTX211™, an allogeneic, gene-edited, stem cell-derived beta islet cell precursor, is currently in an ongoing Phase 1 clinical trial for the treatment of Type 1 Diabetes (T1D). CRISPR Therapeutics remains committed to its goal of developing a beta-cell replacement product that does not require chronic immunosuppression.Vertex has non-exclusive rights to certain CRISPR Therapeutics’ CRISPR/Cas9 technology to accelerate development of potentially curative cell therapies for T1D. CRISPR Therapeutics remains eligible for development milestones and would receive royalties on any future products resulting from this agreement. Other Corporate Matters In May, CRISPR Therapeutics announced the appointment of Naimish Patel, M.D., as Chief Medical Officer. Dr. Patel brings in-depth experience in successfully accelerating innovation and advancing drug candidates across a breadth of modalities and disease areas. Dr. Patel is an experienced drug developer who has worked across a wide range of disease areas, including his most recent leadership role as the Global Development Therapeutic Area Head of Immunology and Inflammation at Sanofi. In addition, the Company announced the promotions of (i) Julianne Bruno, M.B.A., to Chief Operating Officer; Ms. Bruno previously served as the Company’s Senior Vice President and Head of Programs & Portfolio Management; and (ii) Susan Kim to Senior Vice President, Investor Relations and Corporate Communications; Ms. Kim previously served as the Company’s Vice President of Investor Relations and Corporate Communications. Second Quarter 2024 Financial Results Cash Position: Cash, cash equivalents, and marketable securities were $2,012.8 million as of June 30, 2024, compared to $1,695.7 million as of December 31, 2023. The increase in cash was primarily driven by proceeds from the $280.0 million February 2024 registered direct offering, a $200.0 million milestone payment received from Vertex in connection with the approval of CASGEVY, proceeds from employee option exercises as well as interest income, offset by operating expenses.Revenue: Total collaboration revenue for the second quarter of 2024 was not material. Collaboration revenue for the second quarter of 2023 was $70.0 million. Collaboration revenue recognized in the second quarter of 2023 was primarily attributable to a research milestone achieved during the current quarter in connection with a non-exclusive license agreement with Vertex.R&D Expenses: R&D expenses were $80.2 million for the second quarter of 2024, compared to $101.6 million for the second quarter of 2023. The decrease in R&D expense was primarily driven by reduced variable external research and manufacturing costs.G&A Expenses: General and administrative expenses were $19.5 million for the second quarter of 2024, compared to $19.0 million for the second quarter of 2023.Collaboration Expense: Collaboration expense, net, was $52.1 million for the second quarter of 2024, compared to $44.6 million for the second quarter of 2023. The increase in collaboration expense, net, was primarily attributable to manufacturing and commercial costs under the CASGEVY collaboration with Vertex.Net Loss: Net loss was $126.4 million for the second quarter of 2024, compared to a net loss of $77.7 million for the second quarter of 2023. About CASGEVY™ (exagamglogene autotemcel [exa-cel]) CASGEVY™ is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for certain indications in multiple jurisdictions for eligible patients. About the CRISPR Therapeutics-Vertex Collaboration CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CASGEVY represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing, and commercialization of CASGEVY and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY™. About CTX112 CTX112 is being developed for both oncology and autoimmune indications. CTX112 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 19, or CD19, which incorporates additional edits designed to enhance CAR T potency and reduce CAR T exhaustion. CTX112 is being investigated in an ongoing clinical trial designed to assess safety and efficacy of the product candidate in adult patients with relapsed or refractory CD19-positive B-cell malignancies who have received at least two prior lines of therapy. In addition, the Company has opened a clinical trial of CTX112 in systemic lupus erythematosus. About CTX131 CTX131 is being developed for both solid tumors and hematologic malignancies, including T cell lymphomas (TCL). CTX131 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX131 incorporates additional edits designed to enhance CAR T potency and reduce CAR T exhaustion. CTX131 is being investigated in a clinical trial designed to assess the safety and efficacy of the product candidate in adult patients with relapsed or refractory solid tumors. In addition, we have opened a clinical trial of CTX131 in hematologic malignancies, including TCL. About In Vivo Programs CRISPR Therapeutics has established a proprietary LNP platform for the delivery of CRISPR/Cas9 to the liver. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apo(a), a critical component of lipoprotein(a) [Lp(a)]), targeting two validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include CTX340 and CTX450, targeting angiotensinogen (AGT) for refractory hypertension and 5’-aminolevulinate synthase 1 (ALAS1) for acute hepatic porphyria (AHP), respectively. About CTX211 CTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of type 1 diabetes (T1D), which incorporates gene edits that aim to make cells hypoimmune and enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose. A Phase 1 clinical trial for CTX211 for the treatment of T1D is ongoing. About CRISPR Therapeutics Since its inception over a decade ago, CRISPR Therapeutics has transformed from a research-stage company advancing programs in the field of gene editing, to a company that recently celebrated the historic approval of the first-ever CRISPR-based therapy and has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018 to investigate the treatment of sickle cell disease or transfusion-dependent beta thalassemia, and beginning in late 2023, CASGEVY™ (exagamglogene autotemcel [exa-cel]) was approved in some countries to treat eligible patients with either of those conditions. The Nobel Prize-winning CRISPR science has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer and Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. To learn more, visit www.crisprtx.com. CRISPR THERAPEUTICS® standard character mark and design logo, CTX112™, CTX131™, CTX211™, CTX310™, CTX320™, CTX340™ and CTX450™ are trademarks and registered trademarks of CRISPR Therapeutics AG. The CASGEVY™ word mark and design are trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. CRISPR Therapeutics Forward-Looking Statement Statements contained 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, statements regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications and expectations regarding data, safety and efficacy generally; (ii) its strategy, goals, anticipated financial performance and the sufficiency of its cash resources; (iii) regulatory submissions and authorizations, including timelines for and expectations regarding additional regulatory agency decisions; (iv) the expected benefits of its collaborations; and (v) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies, including as compared to other therapies. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in its most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. This press release discusses CRISPR/Cas9 gene editing investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities. Investor Contact: Susie Kim +1-617-307-7503 susan.kim@crisprtx.com Media Contact: Rachel Eides +1-617-315-4493 rachel.eides@crisprtx.com CRISPR Therapeutics AG Condensed Consolidated Statements of Operations (Unaudited, In thousands except share data and per share data) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Revenue: Collaboration revenue $— $70,000 $— $170,000 Grant revenue 517 — 1,021 — Total revenue 517 $70,000 $1,021 $170,000 Operating expenses: Research and development 80,165 101,555 156,338 201,490 General and administrative 19,481 19,032 37,434 41,392 Collaboration expense, net 52,131 44,636 99,097 86,828 Total operating expenses 151,777 165,223 292,869 329,710 Loss from operations (151,260) (95,223) (291,848) (159,710)Total other income, net 26,139 18,406 50,860 31,148 Net loss before income taxes (125,121) (76,817) (240,988) (128,562)Provision for income taxes (1,287) (923) (2,011) (2,243)Net loss (126,408) (77,740) (242,999) (130,805)Foreign currency translation adjustment 2 28 (9) 60 Unrealized (loss) gain on marketable securities (1,329) 452 (4,783) 6,679 Comprehensive loss $(127,735) $(77,260) $(247,791) $(124,066)Net loss per common share — basic $(1.49) $(0.98) $(2.92) $(1.66)Basic weighted-average common shares outstanding 84,920,929 79,091,061 83,357,780 78,885,168 Net loss per common share — diluted $(1.49) $(0.98) $(2.92) $(1.66)Diluted weighted-average common shares outstanding 84,920,929 79,091,061 83,357,780 78,885,168 CRISPR Therapeutics AG Condensed Consolidated Balance Sheets Data (Unaudited, in thousands) As of June 30, 2024 December 31, 2023Cash and cash equivalents $484,472 $389,477Marketable securities 1,517,147 1,304,215Marketable securities, non-current 11,216 1,973Working capital 1,882,584 1,799,287Total assets 2,339,853 2,229,571Total shareholders' equity 1,980,949 1,882,803

Conference call scheduled for 4:30 p.m. ET today VK2735 to Advance to Phase 3 for Obesity; End-of-Phase 2 Meeting Planned for 2H24 Oral VK2735 Phase 2 Study in Obesity Expected to Begin 4Q24 Positive Biopsy Results From VK2809 Reported in June; End-of-Phase 2 Meeting in NASH/MASH Planned for 4Q24 Enrollment Completed in Phase 1b Study of VK0214 for X-ALD; Data Expected 2H24 In Vivo Data From Novel Amylin Agonist Program Reported at ADA in June; IND in Obesity Planned for 2025 SAN DIEGO, July 24, 2024 /PRNewswire/ -- Viking Therapeutics, Inc. ("Viking") (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced its financial results for the second quarter and six months ended June 30, 2024, and provided an update on its clinical pipeline and other corporate developments. Highlights from the Quarter Ended June 30, 2024, and Other Recent Events: "The first half of 2024 was marked by strong momentum in the company's pipeline programs, as we reported successful outcomes from three different clinical trials, and began preparing for next steps with each program," stated Brian Lian, Ph.D., chief executive officer of Viking. "The Phase 2 VENTURE study of VK2735 in obesity demonstrated up to an approximately 15% reduction in body weight from baseline following 13 weeks of dosing, as well as promising safety and tolerability. Following receipt of written feedback from an FDA Type C meeting, we are advancing this compound into Phase 3 development. We are currently preparing for an end of Phase 2 meeting with the agency, which we expect to occur later this year. Separately, the Phase 1 study of the oral tablet formulation of VK2735 demonstrated encouraging safety and tolerability, and positive signs of clinical activity, with subjects reporting mean weight loss of up to 5.3% from baseline following 28 days of daily oral dosing. Dose escalation continues, and we expect to initiate a Phase 2 trial for this program later this year. "We also recently reported best-in-class histology data from the Phase 2b VOYAGE study of our thyroid hormone beta receptor agonist VK2809 in biopsy-confirmed NASH and fibrosis, and plan to schedule an end of Phase 2 meeting with the FDA later this year. With our earlier-stage programs, we are happy to report that the Phase 1b study of our second thyroid hormone beta receptor agonist VK0214, for X-linked adrenoleukodystrophy, is fully enrolled and we expect to report the results from this trial later this year. Finally, at the ADA conference in June, we reported promising in vivo data from a novel series of internally developed amylin agonists and expect to file an IND for this program in 2025. To support Viking's maturing pipeline, the company ended the quarter with a strong balance sheet of $942 million, providing the runway to execute key milestones for each of our pipeline programs." Pipeline and Recent Corporate Highlights Reported Positive Clinical Results from Phase 2 VENTURE Study of Subcutaneous VK2735 for Obesity in 1H24; To Advance into Phase 3 Development. VK2735 is a wholly owned dual agonist of the glucagon like peptide-1, or GLP-1 receptor, and the glucose dependent insulinotropic polypeptide, or GIP receptor, for the potential treatment of obesity and other metabolic disorders. During the first quarter of 2024, Viking announced positive top-line results from a Phase 2 study called VENTURE. This study was a randomized, double-blind, placebo-controlled multicenter study designed to evaluate the safety, tolerability, pharmacokinetics, and weight loss efficacy of VK2735, administered subcutaneously, once weekly, for 13 weeks. The trial was designed to enroll adults who are obese (BMI ≥30 kg/m2) or adults who are overweight (BMI ≥27 kg/m2) with at least one weight-related comorbid condition. The VENTURE trial successfully achieved its primary endpoint and all secondary endpoints, with patients receiving VK2735 demonstrating statistically significant reductions in body weight compared with placebo. Additionally, the study showed VK2735 to be safe and well tolerated, with the majority of treatment emergent adverse events (TEAEs) being categorized as mild or moderate. With respect to the primary endpoint, patients receiving VK2735 demonstrated statistically significant reductions in mean body weight from baseline, ranging up to 14.7%, as well as statistically significant reductions in mean body weight relative to placebo, ranging up to 13.1%. Statistically significant differences compared to placebo were observed for all doses starting at Week 1 and were maintained throughout the course of the study. Weight loss in all treated cohorts appeared to be progressive through 13 weeks and did not show evidence of plateauing. The company believes further weight loss could be achieved through extended dosing beyond the 13-week treatment period of this study. VK2735 also demonstrated encouraging safety and tolerability in the VENTURE study, with the majority of observed adverse events (AEs) being reported as mild or moderate. Treatment and study discontinuation rates among VK2735 cohorts were well-balanced compared with placebo. Of gastrointestinal (GI) related AEs, 95% were reported as mild or moderate. Across all cohorts in the VENTURE study, GI-related AEs were most prevalent during the first week of the study, with observed rates generally declining through the remainder of the study. During the second quarter, Viking received written responses to a U.S. Food and Drug Administration (FDA) Type C meeting packet, submitted to the agency earlier in the quarter. Based on agency feedback, the company plans to advance VK2735 into a Phase 3 program for obesity. Viking expects to schedule an end-of-Phase 2 meeting with the FDA later this year. Details on Phase 3 trial design and timing will be disclosed following the end-of-Phase 2 meeting. Reported Positive Clinical Results from Phase 1 Study Evaluating Oral VK2735 for Obesity in 1H24; Expect to Initiate Oral Phase 2 Trial in 4Q24. In parallel with the development of a sub-cutaneous formulation of VK2735, Viking is also developing an oral tablet formulation of this compound. The company believes a tablet formulation could represent an attractive treatment option for patients who are hesitant to initiate injection-based therapy, or for those seeking to maintain the weight loss they have already achieved. A key advantage in this regard is the potential to transition patients from the subcutaneous formulation to an oral formulation which utilizes the same molecule. Viking believes this may reduce the risk of unexpected safety or tolerability challenges, and could be an attractive option for both patients and clinicians. During the first quarter, Viking reported the initial data from a randomized, double-blind, placebo-controlled Phase 1 trial in healthy adults with a minimum BMI of 30 kg/m2, evaluating once-daily oral doses ranging from 2.5 mg to 40 mg. The primary objective of the study was to evaluate the safety and tolerability of VK2735 administered as an oral tablet once daily for 28 days. The secondary objective was to evaluate the pharmacokinetics of orally administered VK2735 in healthy subjects. Exploratory pharmacodynamic measures included assessments of changes in body weight and other metrics. With respect to safety and tolerability, oral VK2735 was shown to be safe and well tolerated following once daily dosing for up to 28 days, at dose levels up to 40 mg. Among subjects receiving oral VK2735, all TEAEs were reported as mild or moderate in severity, with the majority, 76%, reported as mild. Similarly, all GI-related AEs in this study were reported as mild or moderate, with the majority, 79%, reported as mild. Mild nausea was reported in 14% of subjects receiving VK2735. No vomiting was reported among subjects receiving VK2735. Diarrhea was reported in 3% of VK2735 treated subjects, compared with 20% of subjects receiving placebo. Overall, no clinically meaningful differences were reported for GI AEs among subjects treated with VK2735 compared with placebo. An exploratory assessment of change in body weight showed that subjects receiving oral VK2735 demonstrated dose dependent reductions in body weight, ranging up to 5.3% from baseline. Placebo-adjusted reductions in body weight reached up to 3.3% from baseline. Body weight reductions compared with baseline and placebo were statistically significant at the highest dose evaluated. Weight loss in the 28-day window of this study was progressive at the 20 mg and 40 mg dose levels, with no plateau observed. Given the promising weight loss signal demonstrated after 28 days of once daily, along with the excellent tolerability profile observed thus far, further dose-escalation has been evaluated at daily doses of up to 80 mg; dosing at 100 mg is ongoing. Results from these cohorts will be reported later this year. Viking believes that further benefits from oral dosing of VK2735 might be anticipated from longer dosing periods. To this end, the company plans to initiate a 13 week Phase 2 trial in patients with obesity in the fourth quarter of this year. Reported Positive 52-Week Histologic Data from Phase 2b VOYAGE Study Evaluating VK2809 for the Treatment of NASH and Fibrosis in 2Q24; Primary and Secondary Endpoints Successfully Achieved for Both NASH and Fibrosis with Excellent Safety and Tolerability. VK2809 is an orally available, small molecule agonist of the thyroid hormone receptor that is selective for liver tissue, as well as the beta isoform of the receptor. In the first quarter, Viking announced completion of the Phase 2b VOYAGE study evaluating VK2809 in patients with biopsy-confirmed NASH and fibrosis. This study was a randomized, double-blind, placebo-controlled, multicenter, international trial designed to assess the efficacy, safety and tolerability of VK2809 following 52 weeks of dosing in patients with biopsy-confirmed NASH and fibrosis. Enrollment included patients with at least 8% liver fat content as measured by magnetic resonance imaging, proton density fat fraction, as well as F2 and F3 fibrosis. The primary endpoint of the study evaluated the change in liver fat from baseline to Week 12 in patients treated with VK2809 compared to patients receiving placebo. Secondary and exploratory endpoints assessed histologic changes, such as NASH resolution and fibrosis improvement, following 52 weeks of treatment. In 2023, the company reported that VOYAGE had successfully achieved its primary endpoint, with patients receiving VK2809 demonstrating statistically significant reductions in liver fat content from baseline to Week 12 as compared with placebo. The median relative change from baseline in liver fat among patients treated with VK2809 ranged from 38% to 55% after 12 weeks. In addition, up to 85% of patients receiving VK2809 experienced at least a 30% relative reduction in liver fat. In the second quarter of 2024, Viking announced additional results from the VOYAGE study, demonstrating the successful achievement of the trial's secondary endpoints evaluating histologic changes assessed by hepatic biopsy after 52 weeks of treatment. On the secondary endpoint of NASH resolution without worsening of fibrosis, VK2809-treated patients demonstrated NASH resolution rates ranging from 63% to 75%, compared with 29% for placebo. On the secondary endpoint evaluating the proportion of patients demonstrating at least a one stage improvement in fibrosis with no worsening of NASH, the proportion of VK2809-treated patients demonstrating improvements in fibrosis ranged from 44% to 57%, compared with 34% for placebo. On the secondary endpoint evaluating the proportion of patients experiencing both the resolution of NASH and at least a one-stage improvement in fibrosis, the proportion of VK2809-treated patients achieving both measures ranged from 40% to 50%, compared with 20% for placebo. Consistent with prior studies, patients receiving VK2809 in VOYAGE demonstrated statistically significant improvements in plasma lipids. Placebo-adjusted reductions in LDL-C ranged from 20% to 25%, and reductions in triglycerides and atherogenic proteins such as apolipoprotein B, lipoprotein (a), and apolipoprotein C-III, were significantly improved relative to placebo. These lipids have been correlated with cardiovascular risk, suggesting that that treatment with VK2809 may offer a long-term cardio-protective benefit. VK2809 also demonstrated an encouraging safety and tolerability profile through 52 weeks of treatment, with minimal differences compared with the previously reported results from 12 weeks. The majority, 94%, of treatment related adverse events among patients receiving VK2809 were reported as mild or moderate. Discontinuations due to adverse events were low and balanced across placebo and treatment arms. VK2809 demonstrated excellent gastrointestinal tolerability through 52 weeks of treatment, with similar rates of nausea, diarrhea, stool frequency, and vomiting among VK2809-treated patients as compared to placebo. The company believes the Phase 2b VOYAGE data demonstrate VK2809's best-in-class efficacy on both NASH resolution and fibrosis improvement, along with the potential for cardiovascular benefit through improvement in plasma lipids. Viking plans to schedule an end of Phase 2 meeting with the FDA in the fourth quarter of 2024. Enrollment Complete for Phase 1b Study of VK0214 in X-ALD: Results Expected 2H24. VK0214 is a novel, orally available thyroid hormone receptor beta agonist that is being evaluated as a potential treatment for X-linked adrenoleukodystrophy (X-ALD), a rare neurogenerative disease for which there are currently no pharmacologic treatment options. The Phase 1b study of VK0214 is enrolling patients with the adrenomyeloneuropathy, or AMN, form of X-ALD, which is the most common form of the disorder. This trial is a randomized, double-blind, placebo-controlled multi-center study in adult male patients with AMN. The primary objectives of the study are to evaluate the safety and tolerability of VK0214 administered orally, once daily for 28 days. The study also includes an evaluation of the pharmacokinetics of VK0214 in AMN patients, as well as an exploratory assessment of changes in plasma levels of very long chain fatty acids. Enrollment in the Phase 1b study of VK0214 in adrenomyeloneuropathy was recently completed and the company expects to announce results in the second half of 2024. Presented Positive Preclinical Data from New, Internally Developed Dual Amylin and Calcitonin Receptor Agonist (DACRA) Program at 84th Scientific Sessions of the American Diabetes Association; Viking DACRAs Demonstrated Significant Weight Loss in Animal Models. During the second quarter, Viking presented preclinical data at the American Diabetes Association's (ADA's) scientific sessions from an internally developed dual amylin and calcitonin receptor agonist program. As the amylin receptor plays an important role in food intake and metabolic control, Viking believes it represents an attractive potential target for therapeutic intervention in obesity. The company's ADA presentation highlighted the effects of treatment on body weight, food intake and metabolic profile in both healthy rats and in diet-induced obese mice. The study demonstrated that Viking's dual amylin and calcitonin receptor agonists reduced food intake in lean rats in the period from 0 – 72 hours following a single subcutaneous dosing. At 72 hours following a single dose, Viking's compounds resulted in up to 8% body weight reductions compared to vehicle-treated animals. In a rodent model of diet-induced obesity, treatment with Viking's compounds for 24 days resulted in up to 10% weight loss from baseline. The company is encouraged by these initial findings and believes that these results, as well as those from other preclinical studies, support the continued development of amylin agonists for obesity. Viking expects to file an IND for this program in 2025. Upcoming Investor Events. Viking management will participate in the following upcoming investor events: BTIG Biotechnology Conference Virtual August 5 - 6 Morgan Stanley 22nd Annual Global Healthcare Conference New York, NY September 4 - 6 Second Quarter and Six Month 2024 Financial Highlights Second Quarter ended June 30, 2024 and 2023 Research and development expenses were $23.8 million for the three months ended June 30, 2024, compared to $13.9 million for the same period in 2023. The increase was primarily due to increased expenses related to manufacturing for the company's drug candidates, clinical studies, pre-clinical studies, salaries and benefits and stock-based compensation. General and administrative expenses were $10.3 million for the three months ended June 30, 2024, compared to $9.8 million for the same period in 2023. The increase was primarily due to increased expenses related to stock-based compensation and services provided by third-party consultants, partially offset by a decrease in expenses related to legal and patent services. For the three months ended June 30, 2024, Viking reported a net loss of $22.3 million, or $0.20 per share, compared to a net loss of $19.2 million, or $0.19 per share, in the corresponding period in 2023. The increase in net loss for the three months ended June 30, 2024, was primarily due to the increase in research and development expenses and general and administrative expenses, noted previously, partially offset by increased interest income, compared to the same period in 2023. Six Months Ended June 30, 2024 and 2023 Research and development expenses for the six months ended June 30, 2024 were $47.9 million compared to $24.9 million for the same period in 2023. The increase was primarily due to increased expenses related to manufacturing for our drug candidates, clinical studies, pre-clinical studies, stock-based compensation, salaries and benefits, services provided by third-party consultants and regulatory services. General and administrative expenses for the six months ended June 30, 2024 were $20.3 million compared to $19.4 million for the same period in 2023. The increase was primarily due to increased expenses related to stock-based compensation, salaries and benefits and services provided by third-party consultants, partially offset by a decrease in expenses related to legal and patent services. For the six months ended June 30, 2024, Viking reported a net loss of $49.6 million, or $0.46 per share, compared to a net loss of $38.8 million, or $0.44 per share, in the corresponding period in 2023. The increase in net loss for the six months ended June 30, 2024, was primarily due to the increase in research and development expenses and general and administrative expenses, noted previously, partially offset by increased interest income, compared to the same period in 2023. Balance Sheet as of June 30, 2024 At June 30, 2024, Viking held cash, cash equivalents and short-term investments of $942 million, compared to $362 million as of December 31, 2023. Conference Call Management will host a conference call to discuss Viking's second quarter 2024 financial results today at 4:30 pm Eastern. To participate in the conference call, please dial (844) 850-0543 from the U.S. or (412) 317-5199 from outside the U.S. In addition, following the completion of the call, a telephone replay will be accessible until July 31, 2024, by dialing (877) 344-7529 from the U.S. or (412) 317-0088 from outside the U.S. and entering conference ID # 4777459. Those interested in listening to the conference call live via the internet may do so by visiting the Webcasts page of Viking's website at . An archive of the webcast will also be available on the Webcasts page of Viking's website for 30 days. About Viking Therapeutics, Inc. Viking Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel first-in-class or best-in-class therapies for the treatment of metabolic and endocrine disorders, with three compounds currently in clinical trials. Viking's research and development activities leverage its expertise in metabolism to develop innovative therapeutics designed to improve patients' lives. Viking's clinical programs include VK2735, a novel dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors for the potential treatment of various metabolic disorders. Data from a Phase 1 and a Phase 2 trial evaluating VK2735 (dosed subcutaneously) for metabolic disorders demonstrated an encouraging safety and tolerability profile as well as positive signs of clinical benefit. Concurrently, the company is evaluating an oral formulation of VK2735 in a Phase 1 trial. Viking is also developing VK2809, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the treatment of lipid and metabolic disorders. The compound successfully achieved both the primary and secondary endpoints in a recently completed Phase 2b study for the treatment of biopsy-confirmed non-alcoholic steatohepatitis (NASH) and fibrosis. In a Phase 2a trial for the treatment of non-alcoholic fatty liver disease (NAFLD) and elevated LDL-C, patients who received VK2809 demonstrated statistically significant reductions in LDL-C and liver fat content compared with patients who received placebo. The company's newest program is evaluating a series of internally developed dual amylin and calcitonin receptor agonists (or DACRAs) for the treatment of obesity and other metabolic disorders. In the rare disease space, Viking is developing VK0214, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the potential treatment of X-linked adrenoleukodystrophy (X-ALD). VK0214 is currently being evaluated in a Phase 1b clinical trial in patients with the adrenomyeloneuropathy (AMN) form of X-ALD. For more information about Viking Therapeutics, please visit . Forward-Looking Statements This press release contains forward-looking statements regarding Viking Therapeutics, Inc., under the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995, including statements about Viking's expectations regarding its clinical and preclinical development programs, anticipated timing for reporting clinical data and cash resources. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and adversely and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the success, cost and timing of Viking's product candidate development activities and clinical trials, including those for VK2735, VK0214, VK2809, and the company's other incretin receptor agonists; risks that prior clinical and preclinical results may not be replicated; risks regarding regulatory requirements; and other risks that are described in Viking's most recent periodic reports filed with the Securities and Exchange Commission, including Viking's Annual Report on Form 10-K for the year ended December 31, 2023, and subsequent Quarterly Reports on Form 10-Q, including the risk factors set forth in those filings. These forward-looking statements speak only as of the date hereof. Viking disclaims any obligation to update these forward-looking statements except as required by law. SOURCE Viking Therapeutics, Inc.

LIBerate-CVD: 52-Week, Placebo-Controlled Study of Lerodalcibep in Patients with CVD LIBerate-VI: 9-Month Study of Lerodalcibep vs Inclisiran in Very High Risk Patients for CVD CINCINNATI--(BUSINESS WIRE)-- LIB Therapeutics Inc. (LIB), a privately-held, late-stage biopharmaceutical company advancing Lerodalcibep, a novel, once-monthly, LDL-cholesterol lowering, third-generation PCSK9 inhibitor, today announced positive results from two studies in the recently completed Phase 3 LIBerate registration-enabling program were presented during the 92nd European Atherosclerosis Society Congress in Lyon, France, May 26-29, 2024. Phase 3 LIBerate-CVD Study Late-Breaker Oral Presentation by Evan Stein, MD, PhD, May 29 The global Phase 3 LIBerate-CVD study with 922 patients was a randomized, double-blind, placebo-controlled trial to evaluate Lerodalcibep 52-week efficacy and safety in patients with a history of cardiovascular disease or at very high risk, for CVD on stable lipid-lowering therapy. The co-primary efficacy endpoints were the percent change from baseline in LDL-C at Week 52 and the mean of Weeks 50 and 52. Secondary outcomes included achievement of EAS / ESC LDL-C targets, and other lipid and apolipoproteins changes. Patients were randomized 2:1 to a single 300 mg once-monthly, subcutaneous dose of Lerodalcibep or placebo for 52 weeks. Approximately 85% of participants had CVD, and 30% were female. Mean baseline LDL-C was 102 mg/dL (2.64 mmol/L), despite 85% of patients on statins and 15% on ezetimibe. Results: Co-primary endpoint achieved with Lerodalcibep: significant and persistent LDL-C reductions over 52 weeks of 62% (61 mg/dL, 1.61 mmol/L) at Week 52 and 69.4% (70 mg/dL, 1.81 mmol/L) at mean of Weeks 50 and 52 (P<0.0001) During the study, 94% of all patients achieved the more stringent ESC and now global, lipid targets of ≥50% reduction in LDL-C from baseline and LDL-C <55 mg/dL (1.4 mmol/L) Lerodalcibep robustly reduced non-HDL-C 50.5%, Apo B 45%, Lp(a) 33% Lerodalcipeb was well tolerated. Treatment-emergent adverse events were similar between Lerodalcibep and placebo, with the most common being mild to moderate injection site reactions, and no difference in discontinuations: Lerodalcibep (4.2%) vs placebo (3.6%) Lerodalcibep was not associated with in vivo active ADAs or NAbs. There was no attenuation of either free PCSK9 suppression or LDL-C efficacy “LIBerate-CVD is the final key registration-enabling trial of our global Phase 3 LIBerate program for Lerodalcibep,” said Evan Stein, MD, PhD, Chief Scientific Officer at LIB Therapeutics. “The LIBerate program enrolled a diverse global patient population with CVD, at very high and high risk for CVD, heterozygous and homozygous familial hypercholesterolemia (FH) to support a broad label upon approval. We could not have successfully carried out these global trials without the dedication and enthusiasm of the many investigators and patients around the world.” Phase 3 LIBerate-VI (Lerodalcibep vs Inclisiran) Study Oral Moderated Poster by Professor Ulrich Laufs, MD, May 28 The LIBerate-VI study was a randomized, open-label, study comparing efficacy and safety of Lerodalcibep to inclisiran in patients with, or at very high risk for CVD, on high intensity statin requiring additional LDL-C reduction at Day 270 (9 months). The trial was conducted in 21 centers in France, Germany, Norway, Spain and UK, with patients randomized 1:1 to Lerodalcibep 300 mg monthly or inclisiran 284 mg on Day 1 and 90. Primary efficacy endpoints were the percent LDL-C change from baseline at Day 270. Secondary outcomes included achievement of EAS / ESC LDL-C targets, and other lipid and apolipoprotein changes. Of the 166 patients randomized, 160 completed the trial. Approximately 50% of the patients had CVD, 32% were female and 45% had HeFH. Mean baseline LDL-C was 112 mg/dL (2.85 mmol/L) despite nearly all patients on high intensity statins and ~45% on ezetimibe. Both Lerodalcibep and inclisiran were well tolerated with similar rates of adverse events. At Day 270, mean LDL-C, was decreased from baseline with Lerodalcibep by 53% compared to 45.3% with inclisiran (P=0.0319) in the mITT population, and 55.4% and 45.4%, respectively, in the per-protocol population (P=0.005). More patients achieved the EAS/ESC targets with Lerodalcibep than with inclisiran of >50% reduction in LDL-C from baseline (62% vs 50%) and LDL-C target of <55 mg/dL (1.4 mmol/L) (67% vs 50%). 58% of patients on Lerodalcibep versus 42% on inclisiran met both targets. Greater reductions in non-HDL-C (44.9% vs 38.2%), Apo B (38.2% vs 32.1%) and Lp(a) (26% vs 15.8%) were also seen with Lerodalcibep than with inclisiran (all P<0.05). “These Lerodalcibep data provide additional evidence of a potential best in class pro robust, sustained LDL-C lowering activity in a convenient and patient-friendly, once-monthly, single subcutaneous injection stable at ambient temperature that will not require refrigeration at home or in travel,” said David Cory, CEO of LIB Therapeutics. “We look forward to submitting a Biologics License Application to the Food and Drug Administration later this year, followed by a Marketing Authorization Application to the European Medicines Agency.” About Lerodalcibep Lerodalcibep is a novel, potent, small binding protein, third-generation PCSK9 inhibitor, and has been developed as a more convenient, once-monthly, single subcutaneous injection with long-ambient stability. In clinical trials, Lerodalcibep has demonstrated sustained LDL-C reductions and is expected to expand treatment options for the millions of patients around the world with atherosclerotic cardiovascular disease (ASCVD), and those at very high and high risk for ASCVD, including the 30 million individuals with more severe inherited high-cholesterol called familial hypercholesterolemia (FH). The global Phase 3 LIBerate program enrolled a diverse population of over 2,700 patients with CVD, without CVD at very high and high risk for CVD, including heterozygous and homozygous familial hypercholesterolemia. Lerodalcibep was dosed once-monthly for up to 52 weeks in these key registration-enabling, placebo-controlled trials, and over 2,400 patients have continued in the 72-week open-label extension trial. LIB is preparing a biologics license application (BLA) for Lerodalcibep and plans for regulatory submission in 2024, followed by a Marketing Authorization Application to the European Medicines Agency. About LIB Therapeutics Inc. LIB Therapeutics is a privately-held, late-stage biopharmaceutical company dedicated to bringing Lerodalcibep to the millions of patients with cardiovascular disease and to the 30 million individuals with familial hypercholesterolemia (FH) around the world, who require additional large reductions in low density lipoprotein-cholesterol (LDL-C), despite maximally tolerated statins and other lipid lowering agents, to achieve LDL-C goals. For more information, please visit: .