Headlines

• Geron Corporation (NASDAQ:GERN) announced FDA approval of its drug RYTELO (imetelstat) for treating adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) (LINK)

• MAIA Biotechnology (NASDAQ:MAIA) shares closed up 16% FDA approval of Geron Corp’s imetelstat, a telomerase inhibitor for certain blood cancers. This validation boosted MAIA’s prospects for its own lead candidate THIO, designed to target telomeres in cancer cells. (LINK)

• Eisai Co., Ltd. and Biogen Inc. (Nasdaq: BIIB) announced that the U.S. FDA has accepted sBLA for monthly intravenous (IV) maintenance (Leqembi). The Prescription Drug User Fee Act (PDUFA) action date is set for January 25, 2025. lso submitted rolling BLA for subq autoinjector. More Below. (LINK)

• Biomea Fusion, Inc. (NASDAQ:BMEA) announced that the FDA has placed a full clinical hold on the Phase 1/2 trials evaluating its covalent menin inhibitor BMF-219 (LINK)

• Tevogen Bio Holdings Inc. (NASDAQ:TVGN) announced that its investigational COVID-19 immunotherapy TVGN 489 retains activity against the highly mutated FLiRT variants. More below (LINK)

• BioCardia, Inc. (NASDAQ:BCDA) announced that the United States Patent Office (USPTO) has granted Patent titled “Radial and Transendocardial Delivery Catheter,” with a patent term expiring in 2036. (LINK)

• Lilly (NYSE: LLY) announced that its drug tirzepatide was superior to placebo for resolving metabolic-associated steatohepatitis (MASH). More Below. (LINK)

Regulatory

FDA

• Geron Corporation (NASDAQ:GERN) announced FDA approval of its drug RYTELO (imetelstat) for treating adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have transfusion-dependent anemia. RYTELO targets patients needing 4 or more RBC units over 8 weeks who have not responded to, lost response to, or are ineligible for erythropoiesis-stimulating agents (ESAs). The approval is based on clinical trial data showing significant reductions in transfusion needs. (LINK)

• Biomea Fusion, Inc. (NASDAQ:BMEA) announced that the FDA has placed a full clinical hold on the Phase 1/2 trials (COVALENT-111 and COVALENT-112) evaluating its covalent menin inhibitor BMF-219 for the treatment of type 2 and type 1 diabetes. Despite the clinical hold, the company will continue collecting data on the safety and efficacy of BMF-219 during this period. (LINK)

• GSK (LSE/NYSE:GSK) announced that the US FDA has approved an expanded age indication for Arexvy, making it the first RSV vaccine for adults aged 50-59 at increased risk of severe outcomes. Over 13 million US adults in this age group have medical conditions that heighten their risk for severe RSV. The clinical development program continues to evaluate the safety and immunogenicity of Arexvy in adults aged 18 and older, with data read-outs expected in the second half of 2024. (LINK)

• Eisai Co., Ltd. and Biogen Inc. (Nasdaq: BIIB) announced that the U.S. FDA has accepted Eisai’s Supplemental Biologics License Application (sBLA) for monthly intravenous (IV) maintenance dosing of lecanemab-irmb (U.S. brand name: LEQEMBI®) for the treatment of early Alzheimer’s disease (AD). The Prescription Drug User Fee Act (PDUFA) action date is set for January 25, 2025. The sBLA is supported by data from Phase 2 and Phase 3 studies, showing that sustained treatment can prolong the benefits by continuously removing toxic protofibrils. LEQEMBI is currently approved in the U.S., Japan, China, and South Korea, with applications submitted in multiple other regions. (LINK)

Pipeline Updates

• Tevogen Bio Holdings Inc. (NASDAQ:TVGN) announced that its investigational COVID-19 immunotherapy TVGN 489 retains activity against the highly mutated FLiRT variants. The company reported that all patients treated with TVGN 489 experienced prompt clinical improvement and a >99-100% reduction in viral load within 14 days, indicating the therapy’s potential efficacy against emerging strains. (LINK)

• Eisai Co., Ltd. and Biogen Inc. (Nasdaq: BIIB) announced that the U.S. FDA has accepted Eisai’s Supplemental Biologics License Application (sBLA) for monthly intravenous (IV) maintenance dosing of lecanemab-irmb (U.S. brand name: LEQEMBI®). The Prescription Drug User Fee Act (PDUFA) action date is set for January 25, 2025. LEQEMBI is indicated for the treatment of Alzheimer's disease (AD) in patients with mild cognitive impairment or mild dementia, collectively referred to as early AD.

• Altimmune, Inc. (NASDAQ:ALT) presented data at the EASL International Liver Congress™ 2024 supporting the potential of pemvidutide in treating Metabolic Dysfunction-Associated Steatohepatitis (MASH). Follow-on analyses from Phase 1 trials in MASLD indicate possible histologic improvements by Q1 2025. A quantitative systems pharmacology model suggests pemvidutide's GLP-1/glucagon dual agonism may enhance MASH resolution and fibrosis improvement over GLP-1 alone. Lipidomic profiling further supports pemvidutide's disease-modifying potential in MASH and associated comorbidities, including cardiovascular disease. Pemvidutide is currently in the Phase 2b IMPACT trial for MASH (LINK)

• Eli Lilly and Company (NYSE: LLY) announced that its drug tirzepatide was superior to placebo for resolving metabolic-associated steatohepatitis (MASH), with more than half of the patients achieving improvement in fibrosis at 52 weeks. Detailed results from the SYNERGY-NASH phase 2 study, involving 190 patients, were presented at the European Association for the Study of the Liver (EASL) Congress 2024 and published in The New England Journal of Medicine (NEJM). The study showed 51.8%, 62.8%, and 73.3% of participants taking 5 mg, 10 mg, and 15 mg of tirzepatide, respectively, achieved an absence of MASH with no worsening of fibrosis, compared to 13.2% for placebo, meeting the primary endpoint. (LINK)

• Y-mAbs Therapeutics, Inc. (NASDAQ:YMAB) announced that preclinical GD2-SADA data will be presented at the Society of Nuclear Medicine & Molecular Imaging (SNMMI) 2024 Annual Meeting in Toronto, Canada. The poster (#241436) characterizes the high-affinity binding properties of the GD2-SADA bispecific fusion protein with lanthanide metals for pre-targeted radioimmunotherapy (PRIT). The data support the potential clinical utility of GD2-SADA in diagnosing and treating GD2-expressing tumors. The GD2-SADA program is advancing through Phase 1 clinical development. (LINK)

• Syndax Pharmaceuticals (NASDAQ:SNDX) announced plans to advance into the Phase 1b portion of its trial evaluating revumenib, an oral menin inhibitor, as monotherapy for relapsed or refractory metastatic microsatellite stable (MSS) colorectal cancer (CRC). This decision follows the Independent Data Monitoring Committee's (IDMC) recommendation based on a favorable safety profile observed in the Phase 1a portion of the trial. (LINK)

• Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) presented new clinical data for ARCT-032 at the 47th Annual European Cystic Fibrosis Conference. Phase 1b interim data showed an early trend of improved lung function, with an average absolute FEV1 response of +4.0% and a relative change of +5.8% on Day 8 after two doses. (LINK)

Deal Flow

IPO / Issuances

• Viridian Therapeutics, Inc. (VRDN) announced that a majority of the independent directors on the Compensation Committee of the company’s Board of Directors approved the grant of non-qualified stock options to purchase an aggregate of 450,000 shares of the company’s common stock to two new employees. The Inducement Grants were approved on June 3, 2024. (LINK)

• Rapport Therapeutics Inc. (RAPP) began trading on Nasdaq on June 7, 2024, after pricing its IPO of 8 million shares at $17 each, raising $136 million. The stock gained $3.80, or 22.4%, closing its first day at $20.80.

• Telix Pharmaceuticals (TLX) is targeting a $200 million initial public offering (IPO) in the U.S. The Nasdaq debut includes 17,000,000 American depository shares (ADSs) and a 30-day option for underwriters to purchase up to 15% more ADSs at the initial price. Telix expects net proceeds of around $183 million, or $211 million if the underwriters’ option is fully exercised. The company will operate under the ticker "TLX," the same symbol used on the Australian Securities Exchange. (LINK)

Dr. S Journal Club

• Tirzepatide for Metabolic Dysfunction–Associated Steatohepatitis with Liver Fibrosis | NEJM. (LINK)