This Week in Biotech #11
Catch up on the latest biotech breakthroughs and upcoming trends (Sept 13-17).
Welcome to the Wednesday edition of This Week in Biotech by Biotech Blueprint!
In today’s edition I focused on the European Society for Medical Oncology (ESMO) Congress 2024. See the ESMO section below packed with updates from the conference that started last Friday and ended yesterday.
ESMO CONFERENCE UPDATES
🎯 iTeos, GSK announced interim data from the GALAXIES Lung-201 study, showing that the combination of belrestotug and dostarlimab outperfromed dostarlimab (Jemperli) alone in PD-L1 high non-small cell lung cancer patients in phase 2 trial. The results support the ongoing GALAXIES Lung-301 phase 3 trial.
🎯 Incyte shared that its PD-1 inhibitor retifanlimab (Zynyz), combined with chemotherapy, significantly improved progression-free survival in patients with anal carcinoma in a phase 3 trial. The combination reduced the risk of disease progression or death by 37% compared to chemotherapy alone. The data support Incyte’s plan to file a supplemental Biologics License Application for retifanlimab by the end of 2024.
🎯 Pfizer presented positive phase 2 results for ponsegromab, a monoclonal antibody targeting GDF-15, in patients with cancer cachexia (extreme weight loss). The study met its primary endpoint, showing a significant increase in body weight across all doses, with a 5.6% increase at the highest dose over 12 weeks. Additional improvements were observed in appetite, physical activity, muscle mass, and cachexia symptoms. Ponsegromab was well-tolerated, with no major safety concerns. Based on these results, registration-enabling studies are planned for 2025.
🎯 Eisai presented phase 3 LEAP-012 trial data where the combination of Lenvima (lenvatinib) and Keytruda (pembrolizumab) with transarterial chemoembolization (TACE) significantly improved survival in patients with unresectable, non-metastatic hepatocellular carcinoma. After a median follow-up of 25.6 months, this regimen reduced the risk of disease progression or death by 34% compared to TACE alone. Although there was a trend toward improved overall survival, the data did not reach statistical significance at this interim analysis.
🎯 Bristol Myers Squibb has ended its $150M partnership with Immatics, returning full development and commercialization rights for the bispecific molecule IMA401 to Immatics. The decision is part of BMS’s portfolio prioritization. Immatics presented phase 1 data at ESMO, showing promising results in heavily pre-treated patients with solid tumors. Immatics plans to optimize treatment schedules and explore combination therapies with checkpoint inhibitors.
🎯 Bayer’s phase 3 ARANOTE trial demonstrated that NUBEQ (darolutamide) combined with androgen deprivation therapy (ADT) significantly improved radiological progression-free survival in patients with metastatic prostate cancer, with a 46% reduction in the risk of progression or death compared to placebo plus ADT. The results were also consistent with NUBEQA’s established safety profile.
🎯 BeyondSpring presented interim phase 2 results for their study of plinabulin and docetaxel combined with Keytruda (pembrolizumab) in metastatic non-small cell lung cancer patients. The study showed a median progression-free survival of 8.63 months and a disease control rate of 89.5%, significantly improving on historical controls. The combination was well tolerated.
🎯 Jazz Pharmaceuticals presented updated phase 2 trial results for zanidatamab, a dual HER2-targeted bispecific antibody, used with chemotherapy for HER2-positive metastatic gastroesophageal cancer (mGEA). The treatment showed a confirmed objective response rate of 84% and a median progression-free survival of 15.2 months. The Kaplan-Meier estimated overall survival was 59% at 30 months. The ongoing phase 3 trial will further evaluate its effectiveness, with results expected in mid-2025. In a separate Phase 2 cohort, zanidatamab also demonstrated a 91% confirmed objective response rate in HER2-positive metastatic colorectal cancer.
🎯 PDS Biotechnology presented results from their VERSATILE-002 phase 2 trial of Versamune HPV combined with Keytruda (pembrolizumab) for HPV16-positive recurrent/metastatic head and neck cancer. The data showed a median overall survival of 30 months, significantly higher than the 12-18 months reported for Keytruda alone. The objective response rate was 36%, with 21% of patients achieving 90-100% tumor shrinkage and 9% having a complete response. PDS Biotech plans to advance this therapy through a phase 3 trial starting this year. The treatment, which has received Fast Track designation from the FDA, was well-tolerated with manageable adverse events.
🎯 Exact Sciences presented promising data on their blood-based colorectal cancer (CRC) screening test. The test demonstrated 88.3% sensitivity for CRC and 31.2% for advanced precancerous lesions. The study involved over 3,000 blood samples and aims to close the screening gap with a cost-effective, non-invasive option. Final validation and FDA approval are anticipated by early 2025. The news led to Exact’s stock increasing by 6%.
🎯 In the HIMALAYA phase 3 trial, AstraZeneca’s Imfinzi (durvalumab) combined with Imjudo (tremelimumab) demonstrated significant overall survival benefits for patients with liver cancer. After five years, 19.6% of patients on the STRIDE regimen (a single dose of Imjudo followed by Imfinzi) were alive compared to 9.4% with sorafenib. The safety profile remained consistent with previous findings.
🎯 The NIAGARA phase 3 trial results reveal that AstraZeneca’s Imfinzi (durvalumab) combined with neoadjuvant chemotherapy significantly improves outcomes in bladder cancer. Compared to chemotherapy alone, the Imfinzi regimen reduced the risk of disease progression, recurrence, or death by 32% and the risk of death by 25%.
🎯 Exelixis released final phase 3 data from the CABINET trial, showing that cabozantinib (CABOMETYX) significantly improves survival in patients with advanced neuroendocrine tumors. Exelixis filed supplemental New Drug Application to the FDA, with a decision expected by April 2025. Additionally, Exelixis presented data from the CONTACT-02 trial, which evaluated cabozantinib with Tecentriq in metastatic prostate cancer. Though the combination showed a trend toward improved overall survival, it was not statistically significant. A submission for FDA approval in this indication is planned by year-end.
🎯 Scorpion Therapeutics presented promising phase 1/2 data for STX-478, a mutant-selective PI3Kα inhibitor for advanced solid tumors. STX-478 showed a 23% overall response rate in breast cancer and 21% across all tumors. Tumor reductions were observed in 72% of patients, with circulating tumor DNA decline in 86%. The drug was well-tolerated, with no major toxicities. Enrollment for further trials, including combination therapies, is ongoing.
🎯 23andMe Therapeutics announced preclinical results for its antibody 23ME-01473, targeting ULBP6. In a mouse model of non-small cell lung cancer, the antibody inhibited tumor growth and demonstrated elevated ULBP6 levels in squamous cell carcinomas and some adenocarcinomas. The phase 1 trial, which began in March 2024, aims to assess the safety, pharmacokinetics, and efficacy of 23ME-01473.
MARKET UPDATES
🔹 JMP Securities initiated coverage of Rhythm Pharmaceuticals (RYTM) with a “Market Outperform” rating due to its potential in addressing rare genetic forms of obesity, which are not targeted by popular GLP-1 weight loss drugs like Wegovy and Zepbound. Rhythm’s lead product, Imcivree (setmelanotide), is already FDA-approved for chronic weight management in specific genetic obesity disorders and is under FDA priority review for a label expansion. JMP set a $64 price target for RYTM. As a result the stock increased by 7.4%.
🔹 Rocket Pharmaceuticals (RCKT) announced the completion of enrollment for its phase 2 trial of RP-A501, a gene therapy for Danon disease, leading to a stock surge of 13.5% as of Sept. 17. The trial aims to improve heart function in affected patients using a one-time gene therapy.
🔹 J.P. Morgan upgraded BioNTech (BNTX) from “Underweight” to “Neutral,” raising the price target from $91 to $125, based on promising updates from BioNTech’s oncology pipeline at the ESMO 2024 event. This optimism centers on BNT327/PM8002, an anti-VEGF-A/PD-L1 bispecific drug showing positive early trial results. The parallels to successful competitor ivonescimab support the potential of BNT327.
🔹 On Sept. 16, BioMarin Pharmaceuticals’ stock fell by approximately 17% following positive results from Ascendis Pharma’s TransCon CNP (navepegritide) for treatment of achondroplasia (bone growth disorder that results in dwarfism). The drug improved annualized growth velocity to 5.89 cm/year compared to 4.41 cm/year with placebo. This once-weekly dosing advantage could challenge BioMarin’s daily-administered Voxzogo (vosoritide), leading to a 19% increase in Ascendis’ stock.
🔹 On Friday, Sept. 13, Zenas BioPharma, MBX Biosciences, and Bicara Therapeutics all went public on Nasdaq, marking a busy day for biotech IPOs. Bicara raised approximately $315M by offering 17.5 million shares at $18 each, exceeding its initial target, to fund its phase 2/3 trial of ficerafusp alfa for head and neck cancer. Zenas increased its offering to $225M for studying obexelimab in autoimmune diseases, while MBX raised $163.2M for its hypoparathyroidism therapy, MBX 2109.
🔹 On Sept. 13, Jefferies initiated coverage on Kiniksa Pharmaceuticals with a buy rating and a $40 price target, citing the potential blockbuster status of its lead drug Arcalyst for recurrent pericarditis, projected to reach $1.2B in sales by 2030. The firm also highlighted the promising potential of Kiniksa’s second asset, abiprubart, targeting Sjogren’s disease.
BIOTECH NEWS
🔹 On Sept. 17, all independent directors of 23andMe’s board resigned immediately, citing a lack of a fully financed and actionable proposal from CEO Anne Wojcicki after five months of waiting. The directors expressed differences in strategic direction and noted Wojcicki’s concentrated voting power as a key factor in their decision. The resigning directors include prominent figures such as Roelof Botha, Neal Mohan, and Sandra Hernández.
🔹 On Sept. 17, Novo Nordisk and Korro Bio partnered to develop new genetic medicines using Korro’s OPERA platform and Novo Nordisk’s expertise in cardiometabolic diseases. The collaboration will focus on creating two RNA editing product candidates for cardiometabolic conditions like obesity and diabetes. Korro will receive up to $530M in milestone payments and funding, and will lead preclinical development. Novo Nordisk may advance the programs into clinical trials. The RNA editing technology offers a potential new way to target previously undruggable proteins.
🔹 On Sept. 17, Novartis received FDA approval for Kisqali (ribociclib) to reduce the risk of recurrence in HR+/HER2- stage 2 and 3 early breast cancer. In the phase 3 NATALEE trial, Kisqali showed a 25% reduction in recurrence risk compared to endocrine therapy alone, with benefits observed across all patient subgroups. The trial’s updated data, presented at ESMO 2024, indicated a deepening benefit beyond the initial three-year treatment period. Kisqali is already approved for metastatic breast cancer.
🔹 On Sept. 17, BridgeBio Pharma announced that infigratinib has received breakthrough therapy designation from the FDA for the treatment of children with achondroplasia. This designation, the first of its kind for this condition, accelerates the development and review process for the drug. The designation was granted based on data from the PROPEL 2 clinical trial, which showed significant increases in annualized height velocity and improvements in body proportionality in children treated with infigratinib. The drug also holds orphan drug, fast track, and rare pediatric disease designations from the FDA.
🔹 On Sept. 17, Vigil Neuroscience announced that the FDA has lifted a partial hold on its phase 1 trial of VG-3927. The hold, initially imposed due to concerns about the drug’s safety profile, was resolved after Vigil provided additional data. Despite this setback, early results show VG-3927 is safe and effective, with ongoing dosing in Alzheimer’s patients, including those with TREM2 variants. Full data from the trial is expected in early 2025.
🔹 Eli Lilly announced on Sept. 13 that the FDA approved EBGLYSS (lebrikizumab-lbkz) for treating moderate-to-severe atopic dermatitis in adults and children 12 years and older who have not responded well to topical therapies. EBGLYSS provided skin clearance in 38% of patients and itch relief in 43%, with noticeable improvements as early as two weeks and long-lasting benefits through monthly maintenance dosing. Some serious allergic reactions and eye problems were reported.
🔹 As of Sept. 13, Novavax’s 2024-2025 formula COVID-19 Vaccine is available at major pharmacies across the U.S. for individuals aged 12+. This updated, protein-based vaccine is the only one of its kind available in the U.S. this fall. It targets the JN.1 strain of COVID-19 and shows cross-reactivity with several related strains.
🔹 On Sept. 13, the FDA approved Roche’s Tecentriq Hybreza, a subcutaneous version of the cancer immunotherapy Tecentriq. This new formulation offers quicker and more comfortable administration, taking just 7 minutes compared to the 30-60 minutes required for the intravenous version. It is approved for the same cancer types as intravenous Tecentriq, including liver, lung, skin, and soft tissue cancers. The approval was based on clinical trials showing comparable safety and efficacy to the intravenous form.
🔹 And more good news for Roche/Genentech as the FDA also approved Ocrevus Zunovo on Sept. 13, the first and only twice-yearly 10-minute subcutaneous injection for people with relapsing and primary progressive multiple sclerosis. This allows administration in centers without IV infrastructure, such as doctor’s offices.
Happy Wednesday and thanks for reading Biotech Blueprint!
DISCLAIMER: This content is for informational purposes only. It should not be taken as legal, tax, investment, financial, or other advice. The views expressed here are my own and do not reflect the opinions of any company or institution.
DISCLOSURE: I have no business relationships with any company mentioned in this article.