This Week in Biotech #28
Catch up on the latest biotech breakthroughs and upcoming trends (Nov 13-14).
Welcome to This Week in Biotech by Biotech Blueprint.
Thank you to everyone who responded to the poll on Wednesday. Based on your feedback, I’ll be moving to a once-weekly newsletter, sent on toward the end of the week, starting next week. The posts might be a bit longer but I will do my best to only highlight the important and most interesting stories each week.
🎙️ PODCAST
Also available on Spotify.
THIS WEEK’S KEY TAKEAWAYS 🔑
The GLP-1 market continues to capture attention. Amgen faced a stock drop of 7%, equivalent to a $12B loss in market value, after Cantor Fitzgerald analysts raised concerns about potential bone density issues with its investigational drug, MariTide. Amgen responded promptly, asserting no observed link between MariTide and bone mineral density changes, and expressing continued confidence in its dual GLP-1 therapy. Meanwhile, Metsera secured $215M in Series B funding, led by prominent investors, validating its pipeline of monthly GLP-1 therapies and oral peptide delivery platforms. Rhythm Pharmaceuticals also reported positive phase 3 results in The Lancet, showing significant BMI improvement in 83% of young children with Bardet-Biedl syndrome using their MC4R agonist.
Separately, the biotech sector experienced turbulence due to reports that president-elect Trump intends to nominate vaccine critic Robert F. Kennedy Jr. as HHS Secretary. Vaccine makers like BioNTech, Novavax, and Emergent BioSolutions dropped 7%. Concerns are also rising around Kennedy’s associates, such as the Means siblings, regarding their potential influence on health policy and public health.
MARKET UPDATES
🔹 Vaccine makers saw their stock prices drop on Nov. 14 evening following reports that president-elect Donald Trump nominated Robert F. Kennedy Jr., a known vaccine critic, as the Secretary of Health and Human Services (HHS). Leading decliners included BioNTech, Novavax, and Emergent BioSolutions, which fell by 7%. Other major pharmaceutical companies such as Pfizer, Moderna, GSK, Sanofi, and J&J also faced losses, with AstraZeneca and Merck experiencing minor declines.
🔹❗On Nov. 13, Amgen (AMGN) issued a statement regarding its investigational weight-loss drug, MariTide, following phase 1 trial results, affirming that there is no observed link between the drug and changes in bone mineral density or any bone-related safety concerns. The company remains optimistic about MariTide’s potential, despite a temporary 7% stock drop (equal to $12B in market value) due to Cantor Fitzgerald analysts citing early-stage data that hinted at possible bone health risks. Amgen emphasized its confidence in MariTide’s safety profile, noting that upcoming phase 2 topline data will provide further clarity. MariTide works by targeting the GLP-1 and GIP hormones associated with appetite control and fat storage, aiming to offer faster weight loss with less frequent dosing than existing treatments like Wegovy and Zepbound. Early studies have shown a promising 14.5% weight reduction, positioning MariTide as a competitive option in the $150B weight-loss market.
🔹 On Nov. 14, Nuvectis Pharma reported promising interim data from its phase 1b study of NXP800, a novel treatment for platinum-resistant, ARID1a-mutated ovarian cancer. The study, which has fast track and orphan drug designations from the FDA, demonstrated NXP800’s anti-tumor activity, with six patients showing stable disease and one experiencing tumor shrinkage. Despite the positive results, the company’s stock fell almost 43% following the news.
🔹 Earlier this week, Syros Pharmaceuticals announced that its phase 3 SELECT-MDS-1 trial of tamibarotene with azacitidine for myelodysplastic syndrome failed to meet its primary endpoint, leading to the study’s discontinuation and a loan default with Oxford Finance LLC. As a result, Syros’ shares plummeted by 85% on Nov. 13, and H.C. Wainwright downgraded the company to Neutral from Buy.
BIOTECH NEWS
🔹 Eisai and Biogen received a positive opinion from the European Medicines Agency (EMA) recommending approval of lecanemab for treating early Alzheimer’s disease in certain patients. This recommendation follows a previous negative opinion in July 2024, which Eisai appealed. Lecanemab, an amyloid-beta antibody, demonstrated significant efficacy in the phase 3 Clarity AD trial, showing a 31% reduction in cognitive decline at 18 months. If the European Commission approves, lecanemab could become available in Europe within 67 days, adding to its approvals in several countries, including the U.S. and Japan.
🔹❗ PTC Therapeutics announced the FDA’s accelerated approval of Kebilidi (eladocagene exuparvovec), the first U.S. gene therapy for AADC deficiency, a severe neurological disorder caused by a deficiency in dopamine production. Kebilidi provides gene replacement therapy by delivering a functioning DDC gene into the brain’s putamen via a minimally invasive neurosurgical procedure. This therapy is approved for both children and adults and is aimed at stimulating dopamine production, which is critical for motor development. The approval, based on positive clinical trials showing motor improvements, requires long-term follow-up for continued efficacy verification. AADC deficiency is a rare, often life-shortening disorder that impacts motor, physical, and mental health, frequently resulting in severe complications from infancy. The drug carries risks linked to the neurosurgical procedure, including potential adverse events like dyskinesia, respiratory and cardiac arrest, and neuroinflammation, underscoring the need for patient monitoring post-administration.
🔹 Kronos Bio is discontinuing its development of istisociclib, a CDK9 inhibitor, after an unfavorable risk-benefit profile was observed in treating platinum-resistant high-grade serous ovarian cancer. Following this decision, the company’s board approved a plan to explore strategic alternatives to enhance shareholder value, potentially including a merger, acquisition, or sale of assets. Kronos will also cut expenses as it evaluates options for its remaining preclinical assets, such as two p300 KAT inhibitors, KB-9558 (oncology) and KB-7898 (autoimmune disease), which are nearing investigational new drug readiness. Kronos reported a cash position of $124.9M as of Sept. 30, and posted a Q3 net loss of $14.1M. The company has engaged a financial advisor to assist in the strategic review but noted that there is no assurance of any specific outcome.
🔹 Metsera has secured $215M in Series B financing to accelerate its portfolio of innovative therapies. Led by Wellington Management and Venrock, with participation from major investors like Fidelity and T. Rowe Price, the funding will support the advancement of Metsera’s clinical-stage products. Key developments include phase 2 trials for MET-097i, a long-acting GLP-1 receptor agonist designed for monthly dosing, and MET-233i, an ultra-long-acting amylin analog. Additionally, Metsera has initiated trials for MET-002, an oral GLP-1 RA peptide. The company’s proprietary platforms enable extended peptide half-lives and scalable oral peptide delivery.
🔹 On Nov. 13, Adaptimmune Therapeutics is undergoing significant restructuring to streamline its operations and focus on its highest-potential programs, particularly within its sarcoma franchise. The company plans a 33% workforce reduction in early 2025, aiming to achieve approximately $300M in cost savings over the next four years and reach operating breakeven by 2027. Adaptimmune is concentrating its resources on the commercialization of Tecelra for MAGE-A4+ synovial sarcoma and the development of lete-cel for advanced synovial sarcoma and myxoid/round cell liposarcoma, targeting $400M in peak annual sales for these therapies. The company will discontinue less promising programs, such as the SURPASS-3 Phase 2 trial for ovarian cancer, while continuing preclinical development of key assets like ADP-600 and ADP-520, with plans to seek partnerships to further these programs.
🔹 On Nov. 13, BioNTech announced plans to acquire Biotheus, a biotech company specializing in novel antibodies for cancer and inflammatory diseases, for $800M plus potential milestone payments of up to $150M. This acquisition will grant BioNTech full global rights to BNT327/PM8002, a bispecific antibody targeting PD-L1 and VEGF-A, designed to treat solid tumors by overcoming resistance to current checkpoint inhibitors. BioNTech aims to accelerate BNT327/PM8002 development, with multiple clinical trials set for late 2024 and 2025. The acquisition will also add Biotheus’ pipeline and manufacturing capabilities, expanding BioNTech’s R&D presence in China and bolstering its global oncology strategy.
CLINICAL TRIAL UPDATES
🔹 On Nov. 14, AC Immune announced promising interim results from its phase 2 trial of ACI-7104.056, an active immunotherapy targeting alpha-synuclein (a-syn) in patients with early-stage Parkinson’s disease. The therapy generated strong immune responses, with a 16-fold increase in anti-a-syn antibodies in treated patients compared to placebo, and 100% of treated patients showed a response against the target antigen. The drug has been well-tolerated so far, with no significant safety issues aside from mild injection site reactions and headaches. Based on further data expected in early 2025, the trial may expand to include up to 150 patients to assess motor and non-motor symptom progression and biomarker outcomes, potentially advancing the therapy towards a pivotal study.
🔹 Immutep has reported positive survival outcomes from its phase 1 INSIGHT-003 trial, which combines its drug eftilagimod alpha (efti) with Keytruda and chemotherapy for treating first-line metastatic non-small cell lung cancer (NSCLC). With a minimum follow-up of 22 months, the trial showed a median overall survival of 32.9 months, median progression-free survival of 12.7 months, and a 24-month overall survival rate of 81%, significantly surpassing historical data for similar treatments. Among all evaluable patients (N=40), overall response rates improved, particularly for patients with low or negative PD-L1 expression, a group generally less responsive to anti-PD-1 therapy alone. INSIGHT-003 is nearing full enrollment, and additional data updates are expected in 2025.
🔹 On Nov. 14, Rhythm Pharmaceuticals announced positive results from its phase 3 VENTURE trial on setmelanotide, an MC4R agonist, published in The Lancet Diabetes & Endocrinology. The study tested setmelanotide in children aged 2-5 with severe obesity caused by Bardet-Biedl syndrome. Among the 12 children, 83% showed improved BMI, and 91% of caregivers noted reduced hunger. Safety was consistent with previous findings, with no new safety concerns reported, and common side effects included skin pigmentation and minor injection site reactions. The drug, already approved for similar use in the EU for children aged 2 and older, is under FDA review for expanded U.S. approval in this younger age group, with a decision expected by Dec. 26, 2024.
🔹 Adaptimmune Therapeutics announced that its cell therapy lete-cel met the primary endpoint in a pivotal phase 2 trial, IGNYTE-ESO, showing promise for treating advanced synovial sarcoma and myxoid/round cell liposarcoma (MRCLS). The trial data, to be presented at the Connective Tissue Oncology Society (CTOS) 2024 Annual Meeting, revealed a 42% response rate in patients, with six complete responses and 21 partial responses out of 64 participants. The median duration of response was over 18 months for synovial sarcoma and over one year for MRCLS. Based on these results, Adaptimmune plans a rolling biologics license application for lete-cel by late 2025. This new therapy targets NY-ESO-1, a tumor antigen prevalent in synovial sarcoma and MRCLS, aiming to significantly expand treatment options for these cancers, which affect younger adults and have limited existing therapies. Safety data showed manageable side effects consistent with lete-cel’s profile, including cytokine release syndrome and cytopenias.
🔹 Gilead has presented comprehensive results from its phase 3 PURPOSE 2 trial on lenacapavir, an injectable HIV-1 capsid inhibitor for pre-exposure prophylaxis (PrEP). The study demonstrated that twice-yearly lenacapavir effectively reduces HIV infection by 96% among a diverse participant group, with only two new infections among 2,179 people, showing superiority to daily Truvada and the background HIV incidence rate. Adherence was high, with most participants receiving injections on schedule. Lenacapavir’s pharmacokinetics were consistent, even in cases where infection occurred, highlighting its potential effectiveness as a long-acting PrEP option. Following successful trials, the FDA recently granted lenacapavir breakthrough therapy designation.
ON THE HORIZON
🔹 Nov. 2024 FDA PDUFAs:
Nov. 4: Journey Medical is expecting decision on DFD-29, a modified-release capsule of minocycline hydrochloride, aimed at treating inflammatory lesions and erythema in adults with rosacea. — APPROVED ✅
Nov. 13: PTC Therapeutics is awaiting FDA decision on its biologics license application for Upstaza (eladocagene exuparvovec), a gene therapy for AADC deficiency. The application has been granted Priority Review. — APPROVED ✅
Nov. 29: BridgeBio Pharma’s acoramidis, an investigational treatment for transthyretin amyloid cardiomyopathy, has a set target action date of Nov. 29. The FDA has indicated that no advisory committee meeting is planned for this application.
Nov. 29: Jazz Pharmaceuticals is awaiting FDA’s decision on zanidatamab, a bispecific antibody targeting HER2, for the treatment of metastatic HER2-positive biliary tract cancer. The FDA has granted the application priority review.
Thanks for reading Biotech Blueprint and have a great weekend.
👩🏻💻 BIOTECH BLUEPRINT CONSULTING
I provide tailored consulting solutions designed to meet the unique challenges of both established companies and startups. My services span a wide range of strategic and technical needs, including:
Research strategy & grant writing
Scientific communication & medical affairs
Data analysis & interpretation
Biotech/pharma innovation & technology assessment
Startup advisory services
I also provide daily Biotech Blueprint newsletters and custom daily analysis (charts & graphs) for individuals and companies.
BOOK A FREE 30-MINUTE CONSULTATION OR A MEET & GREET BELOW.
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.
Nice update. GLP-1 continues to be in the spotlight.