This Week in Biotech #29
Catch up on the latest biotech breakthroughs and upcoming trends (Nov 15-22).
Welcome to This Week in Biotech by Biotech Blueprint!
THIS WEEK’S KEY TAKEAWAYS 🔑
The biotech sector saw several significant developments this week, headlined by both breakthroughs and setbacks. BridgeBio scored a major win with FDA approval of Attrub for ATTR-CM, showing impressive 42% reduction in mortality and securing a $500M royalty payment - a huge validation of their platform. The MASH/NASH space heated up with both Viking Therapeutics and Novo Nordisk reporting strong data, with Viking’s VK2809 showing particularly impressive 37-55% liver fat reductions and strong NASH resolution rates. However, the field also saw some sobering news: Neurogene’s gene therapy trial for Rett syndrome hit a serious roadblock with a life-threatening adverse event, tanking their stock 60%, while Sage Therapeutics’ dalzanemdor failed in Huntington’s disease, leading to program discontinuation.
Looking ahead, there’s a potential shift brewing in the regulatory landscape with Trump’s nomination of Marty Makary as FDA commissioner. Given Makary’s unconventional views and alignment with RFK Jr.’s MAHA movement, this could signal major changes in how drugs get approved and how the FDA approaches vaccine data transparency. Meanwhile, we’re seeing an evolution in treatment approaches across multiple fields - from Jazz’s novel bispecific antibody for biliary tract cancer to Alnylam’s promising next-gen RNAi therapeutic for ATTR amyloidosis, suggesting a continued push toward more targeted and sophisticated therapeutic modalities.
🎙️ PODCAST
Also available on Spotify.
MARKET UPDATES
🔹 On Nov. 20, Pyxis Oncology (PYXS) announced favorable preliminary phase 1 data for PYX-201, showing a 50% objective response rate in heavily pretreated head and neck cancer patients, including one complete response, and a 26% ORR across six solid tumor types. PYX-201 demonstrated a strong safety profile and dose-dependent efficacy, supporting its potential as both a monotherapy and in combination with Merck’s Keytruda in expansion trials set to begin in Q1 2025. Despite the promising data and a new collaboration with Merck, Pyxis’ stock is down 46%.
🔹 On Nov. 18, Neurogene Inc. (NGNE) provided an update on its phase 1/2 clinical trial of NGN-401, a gene therapy for Rett syndrome, after a participant in the high-dose cohort experienced a rare, life-threatening hyperinflammatory syndrome, a serious adverse event. The participant remains in critical condition. Neurogene has paused enrollment for the high-dose cohort and discontinued its use, while the U.S. FDA approved continuation of the trial with the low-dose cohort. Neurogene is revising the trial protocol and no longer expects to complete low-dose enrollment in Q4 2024. Following news of the SAE, Neurogene’s stock has plunged, currently down 60% over the past five days.
BIOTECH NEWS
🔹 On Nov. 22, BridgeBio’s Attrub, a near-complete Transthyretin (TTR) stabilizer (≥90%), was approved by the FDA for reducing cardiovascular death and hospitalization in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Attruby is the first treatment specifying near-complete TTR stabilization. Phase 3 trials showed a 42% reduction in all-cause mortality and cardiovascular hospitalization by 30 months, with benefits evident by 3 months. A 50% reduction in cardiovascular hospitalization frequency was observed. Mild side effects included diarrhea (11.6%) and abdominal pain (5.5%). BridgeBio offers lifetime access for trial participants and patient support programs. Approval triggers a $500M royalty payment for BridgeBio. Global expansion is planned, with European regulatory review expected in 2025.
🔹 On Nov. 20, Jazz Pharmaceuticals announced the FDA’s accelerated approval of Ziihera (zanidatamab-hrii) for adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer. Ziihera is the first dual HER2-targeted bispecific antibody approved for this condition, showing a 52% objective response rate and a median response duration of 14.9 months in clinical trials. Approval is contingent on confirming clinical benefits in ongoing phase 3 trials. The therapy provides a chemotherapy-free option for this aggressive cancer, addressing a significant unmet need. Jazz will host an investor webcast on Dec. 11 to discuss the treatment and its commercialization strategy.
🔹 On Nov. 18, Halozyme Therapeutics proposed acquiring Evotec for €11.00 per share in cash, valuing Evotec at €2B. However, on Nov. 22, Halozyme withdrew its proposal due to Evotec’s reluctance.
🔹 On Nov. 15, Syndax Pharmaceuticals announced the FDA approval of Revuforj (revumenib), the first menin inhibitor for treating relapsed or refractory acute leukemia with a KMT2A translocation in patients aged 1+ years old. The clinical trial demonstrated a 21% complete remission rate, with a median remission duration of 6.4 months. Revuforj’s safety profile includes common adverse reactions such as nausea, infections, and differentiation syndrome, the latter requiring monitoring and prompt intervention. With FDA designations like Breakthrough Therapy and Priority Review, Syndax plans an imminent U.S. launch for 110 mg and 160 mg tablets, with a pediatric formulation expected by 2025. Support programs, including financial assistance via SyndAccess, are available.
CLINICAL TRIAL UPDATES
🔹 On Nov. 20, Sage Therapeutics announced topline results from the phase 2 DIMENSION study of dalzanemdor (SAGE-718) for cognitive impairment associated with Huntington’s disease, which failed to meet its primary endpoint of improving cognitive function measured by the Symbol Digit Modalities Test at Day 84. Secondary endpoints also showed no statistically significant or clinically meaningful differences compared to placebo. Dalzanemdor was generally well-tolerated, with no new safety signals reported. Based on these findings, Sage will discontinue further development of dalzanemdor and close the related PURVIEW safety study.
🔹 On Nov. 19, Viking Therapeutics announced positive final results from its phase 2b VOYAGE study of VK2809, a liver-selective thyroid hormone receptor beta agonist, in patients with NASH or MASH. The 52-week data showed significant reductions in liver fat (37–55%), with 64–88% of patients achieving over 30% fat reduction. The study met key secondary endpoints, with 63–75% of VK2809-treated patients achieving NASH resolution without fibrosis worsening and 40–50% showing both NASH resolution and fibrosis improvement. VK2809 also demonstrated cardioprotective potential by reducing LDL cholesterol, triglycerides, and atherogenic proteins. The treatment exhibited excellent safety and tolerability, with most adverse events mild or moderate and discontinuation rates comparable to placebo.
🔹 On Nov. 19, Novo Nordisk also announced positive phase 3 ESSENCE trial results for semaglutide 2.4 mg in treating MASH. The trial met both primary endpoints, showing statistically significant improvements in liver fibrosis without worsening steatohepatitis and resolution of steatohepatitis without worsening fibrosis. Secondary endpoints demonstrated additional benefits, including improved liver enzyme levels (ALT, AST, GGT) and Enhanced Liver Fibrosis (ELF) scores. Semaglutide was well-tolerated with no new safety concerns. While not yet approved for MASH, these findings highlight semaglutide’s potential in addressing this progressive liver disease, affecting one in 20 U.S. adults. Regulatory filings are expected in 2025.
🔹 On Nov. 18, J&J announced positive phase 3 results for icotrokinra (JNJ-2113), a first-in-class oral peptide targeting the IL-23 receptor, for treating moderate-to-severe plaque psoriasis. The ICONIC-LEAD study met co-primary endpoints, with 74% of patients achieving clear or almost clear skin (IGA 0/1) by week 24, and 64.9% achieving a 90% improvement in the Psoriasis Area and Severity Index (PASI 90). Safety findings aligned with prior phase 2 studies, showing comparable adverse event rates to placebo. Additional studies, including ICONIC-TOTAL and ICONIC-ADVANCE, are ongoing, and results are slated for medical congresses.
🔹 On Nov. 17, Alnylam Pharmaceuticals announced interim phase 1 results for nucresiran (ALN-TTRsc04), its next-generation RNAi therapeutic for transthyretin (ATTR) amyloidosis, at the American Heart Association Scientific Sessions 2024. A single dose of 300 mg or higher demonstrated rapid and durable reductions in serum TTR levels, with mean reductions of over 90% achieved by day 15 and sustained for six months. Peak reductions of over 96% were observed by day 29. The data suggest potential for biannual or annual subcutaneous dosing, representing a novel treatment approach for ATTR amyloidosis. Nucresiran was well-tolerated across all dose levels, with no significant safety concerns or injection site reactions reported.
PUBLIC HEALTH SPOTLIGHT
🔹 On Nov. 22, president-elect Donald Trump nominated Dr. Martin “Marty” Makary, a Johns Hopkins surgeon and health policy critic, as the next FDA commissioner. Known for challenging conventional medical practices, Makary aligns with the “Make America Healthy Again” (MAHA) movement spearheaded by RFK Jr., Trump’s pick for HHS secretary. The movement focuses on reducing chronic diseases by improving the food supply and addressing health care corruption. Makary’s FDA role would involve regulating drugs, food, medical devices, and tobacco, with potential plans to reform vaccine data transparency and overhaul agency funding mechanisms. While a noted advocate for hospital price transparency and critiques of the U.S. health system, Makary has faced controversy over his COVID-19 stances, including opposition to vaccine mandates for children. Both Makary and RFK Jr. require Senate confirmation, which may face hurdles given their unconventional views. Their appointments signal a push for significant shifts in FDA priorities under the Trump administration.
🔹 The CDC anticipates rising levels of COVID-19, respiratory syncytial virus (RSV), and flu cases in the coming weeks, attributed to increased holiday gatherings, travel, and indoor activities. Hospitalizations for these illnesses are also expected to rise, with RSV activity particularly impacting young children in the southern and eastern U.S. Updated COVID-19 vaccines from Pfizer/BioNTech and Moderna, as well as Novavax's traditional protein-based vaccine, received FDA approval earlier this year. Three RSV vaccines, developed by GSK, Moderna, and Pfizer, are also FDA-approved. However, vaccination uptake remains low, with only 11.2% of U.S. adults having received an updated COVID-19 vaccine as of October 5, and 36.9% of adults 75 or older receiving RSV shots.
🔹 On Nov. 15, the first U.S. case of clade I mpox was confirmed in California, involving a traveler from an outbreak area. The patient, exhibiting mild symptoms, is isolating and recovering. The CDC reports that the public risk remains low but advises clinicians to remain vigilant for mpox symptoms, especially in patients with relevant travel or exposure history, and to facilitate testing for clade I mpox. Prevention measures include vaccination, adherence to infection control guidelines, and public health reporting. Clade I mpox, predominantly affecting Central and Eastern Africa, has also been detected in other countries through travel. The CDC emphasizes the importance of ongoing surveillance, risk reduction strategies for travelers, and prompt case management.
Have a wonderful rest of your week and thanks for reading Biotech Blueprint!
👩🏻💻 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.