This Week in Biotech #23
Catch up on the latest biotech breakthroughs and upcoming trends (Oct 24-29).
Welcome to the Wednesday edition of This Week in Biotech by Biotech Blueprint!
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MARKET UPDATES
🔹 On Oct. 29, Coya Therapeutics (COYA) announced phase 2 trial results for its low-dose interleukin-2 (LD IL-2) treatment in Alzheimer’s disease, achieving primary and secondary endpoints in safety and Treg cell enhancement in the every-four-week (q4wks) dosing group. This group showed cognitive stabilization and improvements in Alzheimer’s biomarkers, including Aβ42 levels. However, the higher-frequency dosing group (every two weeks, q2wks) did not show benefits in exploratory endpoints, suggesting that higher dosing may compromise Treg functionality. Coya’s stock took a 27% hit, likely due to mixed efficacy signals between the two dosing regimens.
🔹 On Oct. 28, Monte Rosa Therapeutics (GLUE) entered an exclusive global licensing agreement with Novartis to advance the development and commercialization of MRT-6160, a VAV1-targeting molecular glue degrader for immune-mediated diseases. This deal will grant Monte Rosa a $150M upfront payment, with potential for up to $2.1B in milestone payments, royalties on international sales, and a profit-sharing arrangement for U.S. sales. While Monte Rosa will complete the ongoing Phase 1 trial of MRT-6160, Novartis will take charge of further clinical development, starting with phase 2. The company’s stock went up nearly 94% following the announcement from $6.39 to 9.48 per share.
🔹 On Oct. 26, Vera Therapeutics (VERA) reported 96-week results from its ORIGIN phase 2b study showing that its drug candidate, atacicept, stabilizes kidney function in patients with immunoglobulin A nephropathy. The study, presented at the American Society of Nephrology’s Kidney Week and published in the Journal of the American Society of Nephrology, highlighted atacicept’s potential to slow IgAN progression by reducing proteinuria, hematuria, and a key IgA biomarker associated with kidney disease. The drug showed a consistent and generally favorable safety profile. Following this news, on Oct. 28, Vera Therapeutics’ stock rose initially by 17% before stabilizing at $47.34 per share, reflecting a 6% gain in one day. The company is now preparing to release phase 3 trial results in mid-2025 and plans to submit a biologics license application to the FDA by the end of that year. Shortly after, Vera also announced the pricing of an underwritten public offering of 7,142,858 shares of its Class A common stock at $42.00 per share, aiming to raise approximately $300M before expenses.
🔹 Tyra Biosciences (TYRA) announced positive interim results from their phase 1/2 SURF301 study of TYRA-300, an oral FGFR3-selective inhibitor, showing promising anti-tumor activity in heavily pre-treated metastatic urothelial cancer (mUC) patients. Among those with FGFR3+ mUC receiving ≥90 mg daily, 54.5% achieved partial responses with a 100% disease control rate, and TYRA-300 demonstrated a favorable safety profile with minimal FGFR1/FGFR2-related toxicities. Despite the data, Tyra’s stock dipped 23% on Friday, Oct. 25. Tyra plans to expand trials to additional FGFR3-driven cancers.
BIOTECH NEWS
🔹 Pfizer CEO Albert Bourla defended the company’s turnaround strategy amid pressure from activist hedge fund Starboard Value. Following a better than expected Q3 profit driven by strong sales of the COVID-19 treatment Paxlovid, Bourla highlighted cost-cutting measures and management restructuring efforts, including the appointment of a new chief strategy officer and a planned head of R&D. While Starboard criticized Pfizer’s performance and questioned its drug development strategy, Bourla maintained that recent acquisitions, particularly the $43B buyout of Seagen, were transformational for the company. Despite challenges, including declining sales from COVID products, Pfizer raised its annual profit and sales forecast, anticipating $10.5B from COVID-related sales this year. Paxlovid sales surged to $2.7B, surpassing expectations, while total revenue for the quarter reached $17.7B. Analysts emphasized the need for Pfizer to improve profitability and streamline operations to alleviate ongoing investor concerns.
🔹 Novartis’s €2.7B acquisition of MorphoSys is facing significant challenges, primarily due to delays in the development of pelabresib, a BET inhibitor that the company had planned to file for regulatory approval. Concerns over toxicity have led Novartis to write off $800M related to the purchase. Originally, a filing was expected this year following promising results from a key trial, but subsequent data indicated that safety issues need to be resolved before moving forward. Additionally, Novartis has discontinued its BCMA-targeting CAR-T project amid a crowded myeloma treatment landscape.
🔹 On Oct. 29, Spero Therapeutics announced disappointing interim results for its SPR720 phase 2a study, which did not meet its primary endpoint for treating nontuberculous mycobacterial pulmonary disease. Analysis revealed minimal efficacy compared to placebo and identified safety concerns, including three cases of reversible grade 3 liver toxicity in patients taking the highest dose. Consequently, Spero has suspended development of SPR720 and will re-evaluate its future. Due to this setback, Spero will cut approximately 39% of its workforce and restructure its operations, extending its cash runway into mid-2026.
🔹 On Oct. 29, Biogen and Neomorph announced a collaboration to develop molecular glue degraders for Alzheimer’s, rare, and immunological diseases. Utilizing Neomorph’s molecular glue platform and Biogen’s expertise, the partnership aims to identify and optimize small-molecule drug candidates for Biogen’s key therapeutic areas. Molecular glue degraders represent a novel approach to treat challenging disease targets by promoting targeted protein degradation, an area with potential for complex neurological and immunological treatments. The agreement includes a total potential consideration of up to $1.45B for Neomorph.
🔹 On Oct. 29, GSK announced its acquisition of CMG1A46, a dual CD19 and CD20 T cell-engager from Chimagen Biosciences, for $300M upfront. CMG1A46, currently in phase I trials, targets uncontrolled B cells in autoimmune diseases like lupus, aiming to address unmet needs in treatment-resistant cases. GSK plans to initiate lupus trials for the drug in 2025, with potential applications in other autoimmune diseases.
🔹 On Oct. 29, Biogen appointed Dr. Daniel Quirk as Chief Medical Officer, effective October 28, 2024. With prior roles at Bristol-Myers Squibb and Pfizer, Quirk brings “extensive medical and leadership experience across immunology, neuroscience, and rare diseases.”
🔹 Resalis Therapeutics secured a strategic equity investment from Sanofi to accelerate the development of its lead candidate, RES-010, which targets obesity through an innovative approach involving the inhibition of miR-22, a non-coding RNA that regulates metabolic processes. RES-010 is a first-in-class antisense oligonucleotide designed to modify disease pathways underlying obesity by addressing lipid biosynthesis, mitochondrial function, and fat storage in the body. Unlike traditional treatments that focus mainly on appetite suppression, RES-010 aims to offer sustained weight loss and reduce fat in key areas such as visceral and hepatic fat. With strong preclinical results, RES-010 is positioned to complement existing obesity treatments, including GLP-1 receptor agonists, by addressing broader biological mechanisms involved in obesity.
🔹 AbbVie announced it will acquire Aliada Therapeutics, a biotech company specializing in therapies for central nervous system diseases, for $1.4B. The acquisition centers on Aliada’s lead candidate, ALIA-1758, an antibody aimed at treating Alzheimer’s disease by targeting pyroglutamate amyloid beta. This compound is designed to cross the blood-brain barrier using Aliada’s proprietary MODEL platform, which also enhances AbbVie’s broader CNS drug development efforts.
🔹 On Oct. 28, Eli Lilly announced that mirikizumab, its IL23p19 antagonist, has shown significant long-term efficacy and safety results for treating both ulcerative colitis (UC) and Crohn’s disease. In multi-year phase 3 studies, over 80% of patients with moderately to severely active UC maintained long-term remission, while more than 50% of those with Crohn’s disease sustained endoscopic remission for up to five years. The long-term safety profiles in both studies were consistent with previously known data for mirikizumab. The drug is currently approved for moderately to severely active UC in the U.S. and is under FDA review for Crohn’s disease.
🔹 On Oct. 25, Revolution Medicines shared initial data from a phase 1/1b study of RMC-9805, a selective RAS G12D inhibitor, showing promising safety and antitumor activity in patients with advanced pancreatic cancer. The study included 179 patients with KRAS G12D mutations, previously treated with standard therapies. RMC-9805 was generally well-tolerated, with most adverse effects mild and no observed dose-limiting toxicities. Among patients receiving the candidate phase 2 dose, 30% showed objective responses, and 80% achieved disease control. This research highlights potential treatment avenues for RAS-driven pancreatic cancer, an area with significant unmet needs.
🔹 On Oct. 25, Iterum Therapeutics received FDA approval for Orlynvah, the first oral penem antibiotic approved in the U.S. for treating UTIs in adults. This approval marks the first FDA-approved product for Iterum and the second new UTI treatment approved in the U.S. in 20 years. Clinical trials showed Orlynvah outperformed ciprofloxacin for fluoroquinolone-resistant infections and demonstrated superiority to Augmentin in specific populations, with good tolerance in both studies.
🔹 On Oct. 24, GSK has announced an $800M investment to expand its Pennsylvania site, marking its largest manufacturing investment in the U.S. The project will double the facility’s size and capacity, adding 200+ jobs and incorporating advanced manufacturing and R&D capabilities for vaccines and medicines. The expansion will include a new vaccines drug substance facility and an R&D pilot plant to support clinical trials. Construction will begin by the end of 2024, with new facilities expected to be operational between 2027 and 2028. According to the company’s statement, Pennsylvania Governor Josh Shapiro welcomed the development, highlighting the state’s role in supporting life sciences and innovation.
🔹 Eisai and Eli Lilly have come under scrutiny by the New York Times for allegedly failing to disclose genetic risk factors to participants in Alzheimer’s drug trials for Leqembi and Kisunla. According to recent reports, the companies tested participants for the APOE4 gene variant, which is associated with both Alzheimer’s and an increased risk of brain injuries, including bleeding and swelling. Although trial consent forms noted general risks associated with the gene, participants were not informed of their individual APOE4 status, which could significantly elevate their risk of serious side effects. Notably, two participants in Eisai’s trial died, and over 100 others experienced brain injuries. Lilly’s trial saw “severe” brain bleeding cases as well. Eisai and Lilly claimed that disclosing APOE4 status might bias trial outcomes, as prior knowledge of the gene variant has been shown to affect memory test performance. Critics, however, argue that this withholding of information undermined informed consent, with consumer advocacy group Public Citizen calling for an FDA investigation, stressing that trial participants should have had the option to opt-out based on their personal risk.
CLINICAL TRIAL UPDATES
🔹 On Oct. 29, Eledon Pharmaceuticals announced positive results from an investigator-initiated trial at UChicago Medicine, showing that 2/3 subjects with type 1 diabetes achieved insulin independence after receiving islet transplants along with the investigational drug tegoprubart as part of their immunosuppression regimen. The treatment was well tolerated, with no unexpected adverse events, and participants showed significant reductions in insulin requirements. These findings suggest that tegoprubart may provide a novel immunosuppressive option that could enhance the outcomes of islet transplantation for type 1 diabetes patients.
🔹 On Oct. 29, Zai Lab reported positive phase 3 trial results for KarXT in treating schizophrenia in China. KarXT achieved a significant reduction in the Positive and Negative Syndrome Scale (PANSS) total score by 9.2 points compared to placebo, meeting its primary endpoint. The trial also achieved all secondary endpoints, showing improvements in positive and negative symptoms with no unexpected safety concerns. Adverse effects such as nausea and dizziness were noted but were consistent with previous studies. Zai Lab holds exclusive rights to BMS’s KarXT in mainland China, Hong Kong, Macau, and Taiwan.
🔹 On Oct. 25, IDEAYA Biosciences presented promising phase 1 data on their MAT2A inhibitor IDE397 in urothelial and non-small cell lung cancer at the EORTC-NCI-AACR Symposium. IDE397 showed an overall response rate of ~33%, including one complete response and eight partial responses among 27 patients. The drug demonstrated a favorable safety profile with no serious adverse events or treatment discontinuations. IDEAYA also shared early success in combining IDE397 with Trodelvy.
🔹 ImmunityBio has initiated dosing for its phase 1 clinical trial of CAR-NK cell therapy (CD19 t-haNK) in B-cell non-Hodgkin lymphoma patients. This marks the company’s first CAR-NK trial for liquid tumors and the first NK cell therapy study conducted in Africa. The QUILT 106 trial in South Africa is designed to test the safety and initial efficacy of CD19 t-haNK cells both as a standalone treatment and in combination with rituximab. Preliminary data is anticipated in late 2025.
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