This Week in Biotech #33
Catch up on the latest biotech breakthroughs and upcoming trends (Dec 13-19).
Welcome to This Week in Biotech by Biotech Blueprint! So glad you’re here.
This is the final edition for the year. This Week in Biotech will resume on Friday, Jan. 10, 2025.
It has been a fantastic year for Biotech Blueprint. Thank you all so much for subscribing and for your continued support. Wishing you peaceful holidays and all the best for 2025!
THIS WEEK’S KEY TAKEAWAYS 🔑
As 2024 draws to a close, California’s dairy industry finds itself at the epicenter of an emerging public health crisis. H5N1, traditionally the scourge of poultry farmers, has made a leap to dairy cattle across 16 states. Governor Gavin Newsom’s emergency declaration this Wednesday betrays a growing unease among public health officials. With 34 human cases already confirmed in California, this zoonotic pivot is getting concerning.
This past week was remarkably busy for FDA decisions, with several significant approvals that span rare diseases to oncology. Let’s break down what happened. Just on Dec. 19th alone, we saw three major decisions:
Tryngolza’s approval as the first ever treatment for familial chylomicronemia syndrome (first treatment option for a condition previously managed through diet alone)
Zealand Pharma’s glepaglutide rejection for short bowel syndrome
Mesoblast’s approval for Ryoncil as the first MSC therapy in the US
Go back just a few days earlier to December 13th, and we had two more notable approvals:
Neurocrine’s Crenessity for congenital adrenal hyperplasia - the first new treatment in 70 years
Checkpoint’s Unloxcyt for advanced skin cancer
Throw in Xcovery’s Ensacove approval for ALK-positive lung cancer and J&J’s setback with their amivantamab rejection, and you’ve got an extraordinarily busy period for the FDA. Seven major decisions in just a week is quite remarkable, especially considering three of them were first-in-class treatments (Tryngolza, Ryoncil, Crenessity).
The obesity market continues to draw deep-pocketed suitors. Merck’s acquisition of Hansoh Pharma’s oral GLP-1 candidate (still in preclinical stages) for up to $2B speaks volumes about the industry’s appetite for alternatives to injectable treatments. With the market projected to reach $100B by 2030, the race to develop more convenient delivery methods has become a high-stakes gambit.
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MARKET UPDATES
🔹 On Dec. 17, Tonix Pharmaceuticals (TNXP) announced that the FDA has accepted its New Drug Application (NDA) for TNX-102 SL, a non-opioid treatment for fibromyalgia. This marks a key step toward potentially becoming the first new drug for fibromyalgia in over 15 years. The NDA is supported by positive phase 3 trial data showing significant pain reduction in patients. Following the news, Tonix’s stock surged over 55%, reflecting investor optimism ahead of a potential 2025 approval.
🔹 On Dec. 16, Sangamo Therapeutics’ (SGMO) stock surged by 24% following news that Roche would halt development of its phase 3 hemophilia A gene therapy candidate, dirloctocogene samoparvovec (SPK-8011). Roche’s decision was driven by its strategy to focus on a different hemophilia A treatment targeting enhanced function factor VIII (FVIII). Sangamo is advancing its own gene therapy for hemophilia A, giroctocogene fitelparvovec, which is licensed to Pfizer. Roche had acquired dirloctocogene samoparvovec through its purchase of Spark Therapeutics in 2019.
🔹 Morgan Stanley upgraded PTC Therapeutics (PTCT) to Overweight from Equal Weight, increasing the price target from $45 to $67. The firm is optimistic about PTC’s outlook as it heads into the new year, following several positive updates, a stark contrast to the regulatory challenges the company faced with nearly every program just over a year ago. As 2025 approaches, Morgan Stanley sees the potential approval and launch of sepiapterin, with a PDUFA date of Jul. 29, as a key area of focus for investors. Last Friday, Morgan Stanley also downgraded Immuneering (IMRX) and Amicus Therapeutics (FOLD).
🔹 Editas Medicine (EDIT) announced a shift to focus exclusively on in vivo CRISPR-edited medicines, slashing 65% of its workforce (about 180 jobs) over the next six months. The company is ending development of its reni-cel gene-editing therapy for sickle cell disease after failing to find a commercial partner. Editas is prioritizing in vivo programs, with promising pre-clinical data in hematopoietic stem cells and the liver. The company aims to achieve human proof of concept in two years, extending its cash runway into Q2 2027. Shares fell 10% after the news, and Stifel and Truist downgraded the stock, citing challenges in the company’s path forward without reni-cel.
BIOTECH NEWS
🔹❗On Dec. 19, Ionis Pharmaceuticals announced that the FDA approved Tryngolza (olezarsen) as the first treatment for adults with Familial Chylomicronemia Syndrome (FCS), a rare genetic disorder causing severe hypertriglyceridemia and a high risk of acute pancreatitis. Tryngolza, administered monthly via auto-injector, significantly reduced triglycerides (42.5% at 6 months, 57% at 12 months) and lowered acute pancreatitis events in clinical trials. This approval is based on positive results from the phase 3 Balance trial and provides a critical treatment option for FCS patients, who previously had only diet management.
🔹❗On Dec. 19, the FDA rejected Zealand Pharma’s New Drug Application (NDA) for glepaglutide, a GLP-2 analog for treating short bowel syndrome with intestinal failure. The FDA concluded that the data did not meet the full requirements to establish the efficacy and safety of the proposed dose of glepaglutide. The phase 3 trial showed that twice-weekly glepaglutide significantly reduced parenteral support needs, while once-weekly treatment did not achieve statistical significance. The FDA has recommended an additional clinical trial to confirm the drug’s efficacy and safety.
🔹 On Dec. 19, Mesoblast’s Ryoncil (remestemcel-L) was FDA-approved as the first mesenchymal stromal cell therapy in the U.S., specifically for treating steroid-refractory acute graft versus host disease in children aged 2 months+. In a phase 3 trial, 70% of patients showed a positive response to the treatment. Ryoncil works by modulating the immune system and may have potential for other inflammatory conditions. This approval marks a major milestone for Mesoblast, the first MSC therapy to gain FDA approval.
🔹 On Dec. 18, the FDA approved Xcovery’s ensartinib (Ensacove) for the treatment of adult patients with ALK-positive, locally advanced or metastatic non-small cell lung cancer (NSCLC) who have not received prior ALK-inhibitor therapy. The approval is based on results from the eXALT3 trial, a randomized study of 290 patients, where ensartinib demonstrated a significant improvement in progression-free survival compared to crizotinib. However, there was no significant difference in overall survival. The most common side effects included rash, musculoskeletal pain, fatigue, and nausea. The recommended dose is 225 mg once daily, with or without food, until disease progression or unacceptable toxicity.
🔹 Merck has acquired global rights to HS-10535, an oral GLP-1 receptor agonist from Hansoh Pharma, for obesity treatment. Merck will pay an upfront $112M, with up to $1.9B in potential milestone payments and royalties. HS-10535 is still in pre-clinical stages, and its oral form offers a more convenient alternative to injectable GLP-1 treatments like Eli Lilly’s Zepbound and Novo Nordisk’s Wegovy. The obesity market is expected to reach $100B by 2030, and Merck’s move aims to strengthen its position in this rapidly growing sector, alongside competitors like Lilly, Novo Nordisk, and Amgen, who are also developing GLP-1 therapies.
🔹 Gilead Sciences and Terray Therapeutics have announced a strategic collaboration to discover and develop novel small molecule therapies for multiple targets using Terray’s AI-driven tNova drug discovery platform. This platform combines high-throughput experimentation and computational analysis to identify effective therapeutic compounds. Under the agreement, Terray will use tNova to develop small molecules, with Gilead holding the option to exclusively license and commercialize the compounds.
🔹 On Dec. 16, J&J announced that the FDA rejected its Biologics License Application for injectable amivantamab, a treatment for non-small cell lung cancer (NSCLC) with EGFR mutations. The CRL was related to issues found during a routine pre-approval inspection of a manufacturing facility, but it did not pertain to the drug’s formulation, efficacy, or safety data, and the FDA has not requested additional clinical studies. The intravenous (IV) formulation of Rybrevant (amivantamab), currently approved for use in NSCLC, is not affected. J&J expressed confidence in resolving the issue promptly.
🔹 On Dec. 16, Emergent BioSolutions announced a $50M contract option from the BARDA to supply Cyfendus (Anthrax Vaccine Adsorbed, Adjuvanted). Deliveries are expected to be completed by April 2025, following a previous $30M award for 2024. Approved by the FDA in July 2023, Cyfendus is indicated for post-exposure prophylaxis against anthrax in individuals aged 18-65. Anthrax remains a serious public health threat, and this contract boosts preparedness for potential bioterrorism incidents. Emergent’s anthrax portfolio also includes BioThrax and two therapeutic treatments: Anthrasil and raxibacumab.
🔹❗On Dec. 13, Neurocrine Biosciences announced that the FDA has approved Crenessity (crinecerfont), a first-in-class treatment for children and adults with classic congenital adrenal hyperplasia. This approval marks the first new treatment for CAH in 70 years. Crenessity is an oral medication that reduces excess adrenal androgens by targeting the corticotropin-releasing factor type 1 (CRF1) receptor, allowing for reduced glucocorticoid (GC) dosages. It is approved for patients aged 4+ and is expected to be commercially available in about a week. In clinical trials, Crenessity effectively lowered androgen levels and allowed for significant GC dose reductions in both pediatric and adult patients. The medication was well tolerated, with mild to moderate side effects such as headache and fatigue. Crenessity will be provided through PANTHERx Rare pharmacy, and Neurocrine is offering financial support programs for patients. Neurocrine Biosciences has not yet disclosed the price of Crenessity. However, William Blair analyst Myles Minter estimates the cost at $1,116 per dose, which would result in an approximate annual net price of $264,784. The treatment is available in 50 mg and 100 mg capsules, as well as an oral solution. The approval represents a breakthrough for the CAH community, which has struggled for decades with high-dose steroid treatments and their side effects.
🔹❗On Dec. 13, Checkpoint Therapeutics announced the FDA approval of Unloxcyt (cosibelimab-ipdl) for treating adults with metastatic or locally advanced cutaneous squamous cell carcinoma (cSCC) who cannot undergo curative surgery or radiation. This marks the first FDA-approved anti-PD-L1 treatment for advanced cSCC. Unloxcyt works by blocking PD-L1, enhancing the immune response against tumors, and inducing antibody-dependent cell-mediated cytotoxicity. The recommended dose is 1,200 mg every three weeks via intravenous infusion. James Oliviero, CEO of Checkpoint, called the approval a milestone for the company, positioning it in the $1B U.S. cSCC treatment market. cSCC is the second most common skin cancer in the U.S., with 1.8M cases annually. Of these, 40k progress to advanced stages, leading to 15k deaths each year. Unloxcyt’s approval follows positive results from the CK-301-101 study, showing durable responses in advanced cSCC patients.
🔹 AbbVie has agreed to acquire Nimble Therapeutics, including its lead asset, an investigational oral IL23R inhibitor for psoriasis and inflammatory bowel disease (IBD), currently in preclinical development. The acquisition also includes Nimble’s proprietary peptide synthesis platform for discovery and optimization of peptide-based therapeutics for autoimmune diseases. The deal, valued at $200M in cash, allows AbbVie to enhance its immunology portfolio with promising oral peptide therapies.
🔹 On Dec. 13, Gilead Sciences announced that the European Medicines Agency’s CHMP has issued a positive opinion for seladelpar as a treatment for primary biliary cholangitis. The recommendation is based on the RESPONSE phase 3 trial, which showed that seladelpar significantly improved liver function and reduced pruritus (itching) compared to placebo. If approved by the European Commission in early 2025, it will offer a new treatment option for people with primary biliary cholangitis. The drug was accelerated approved by the FDA in Aug. 2024.
CLINICAL TRIAL UPDATES
🔹 Roche’s phase 2b PADOVA study of prasinezumab in early-stage Parkinson’s disease did not meet its primary endpoint but showed a numerically significant delay in motor progression. The drug was well tolerated with no new safety concerns. Despite this, the company announced that these results suggest potential clinical benefit, and Roche will work with health authorities to determine next steps.
🔹 On Dec. 19, Vertex Pharmaceuticals announced positive results from its phase 2 study of suzetrigine, an investigational NaV1.8 pain signal inhibitor, for treating painful lumbosacral radiculopathy (back pain). The study met its primary endpoint, showing a statistically significant and clinically meaningful reduction in pain. The placebo arm showed a similar reduction, though the study was not powered for direct comparison. Suzetrigine was generally well tolerated, with fewer adverse events in the treatment group compared to placebo. Despite a similar response between suzetrigine and placebo, post-hoc analyses indicated variability in the placebo response across study sites, suggesting that future trial designs could better separate the effects of suzetrigine. Vertex plans to move forward with phase 3.
🔹 On Dec. 18, Corvus Pharmaceuticals announced interim data from a phase 1 clinical trial evaluating soquelitinib for treating moderate to severe atopic dermatitis (eczema). The data, which includes complete results from the first cohort (16 patients), showed a favorable safety and efficacy profile. Soquelitinib patients exhibited a 55.9% reduction in the Eczema Area and Severity Index (EASI) score at 28 days, with further improvement to 69.1% at 58 days. In contrast, placebo patients showed minimal improvement. Soquelitinib demonstrated significant efficacy in reducing inflammation, with a potential link to decreases in key cytokines such as IL-5 and IL-17. The drug was well tolerated, and these results suggest its potential as a new treatment for atopic dermatitis and other immune diseases.
🔹 Affimed reported positive clinical data on Dec. 17, from its ongoing AFM24-102 trial, evaluating the combination of AFM24 and atezolizumab in heavily pretreated non-small cell lung cancer (NSCLC) patients. In EGFR wild-type (EGFRwt) patients, the combination achieved a 21% overall response rate, a 76% disease control rate (DCR), and a median progression-free survival of 5.6 months. A post-hoc exposure-response analysis revealed that higher exposure to AFM24 led to significantly better outcomes. Based on these findings, Affimed plans to advance the development of AFM24 at a dose of 720 mg weekly.
🔹 On Dec. 17, Regeneron Pharmaceuticals announced positive results from the phase 3 QUASAR trial for Eylea HD (aflibercept) injection 8 mg, a treatment for macular edema following retinal vein occlusion (RVO). The trial met its primary endpoint, demonstrating that patients treated with Eylea HD every 8 weeks (after initial monthly doses) experienced non-inferior vision gains compared to those receiving the standard Eylea (aflibercept) injection 2 mg every 4 weeks. Safety data for were consistent with previous trials, with no new safety concerns. Regeneron plans to submit a supplementary biologics license application (sBLA) to the FDA in Q1 2025.
🔹 On Dec. 16, PepGen announced a clinical hold from the FDA on its IND application for the CONNECT2-EDO51 phase 2 study of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD). The FDA will issue an official clinical hold letter within 30 days. Despite this, PepGen continues to progress its CONNECT1-EDO51 study in Canada, where the 10 mg/kg dose cohort has been fully enrolled. PGN-EDO51, designed to skip exon 51 of the dystrophin transcript, aims to treat approximately 13% of DMD patients by restoring a functional dystrophin protein. The drug has received both Orphan Drug and Rare Pediatric Disease Designations from the FDA.
🔹 On Dec. 16, Viridian Therapeutics announced positive topline results from the THRIVE-2 phase 3 trial of veligrotug (IV anti-IGF-1R antibody) in chronic thyroid eye disease. The trial met all primary and secondary endpoints, demonstrating high statistical significance with a 56% proptosis responder rate (p < 0.0001), 56% diplopia response rate, and 32% complete diplopia resolution (p = 0.0152). Veligrotug was generally well-tolerated, with a 9.6% placebo-adjusted hearing impairment rate and a 94% treatment completion rate. These results position veligrotug as a potential first-line therapy for TED, with a BLA submission scheduled for the second half of 2025.
PUBLIC HEALTH SPOTLIGHT
🔹 Governor Gavin Newsom declared a State of Emergency on Dec. 18 to enhance California’s response to the spread of avian influenza A (H5N1), or bird flu, which has affected dairy cows in Southern California and spread to 16 states. This action allows for more flexibility in staffing and resources to support the state’s ongoing efforts to contain the virus, which has already led to 34 confirmed human cases in California. While the risk to the public remains low, the state has implemented the nation’s largest testing and monitoring system and is working closely with federal agencies, including the CDC and USDA. Efforts include distributing personal protective equipment to high-risk farm workers, public education campaigns, and multilingual outreach to ensure timely information and prevent further spread.
ON THE HORIZON
🔹 Dec. 2024 FDA PDUFAs:
Dec. 19: Olezarsen, an investigational RNA-targeted medicine developed by Ionis Pharmaceuticals, is designed to treat familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG) by lowering triglyceride levels. It is currently under Priority Review by the FDA for the treatment of FCS, with a decision expected by Dec. 19, 2024. Olezarsen works by targeting apoC-III, a protein involved in triglyceride metabolism, and aims to reduce the risk of life-threatening acute pancreatitis and other complications related to these conditions. APPROVED ✅
Dec. 20: In Feb. 2024 AstraZeneca and Daiichi Sankyo’s Biologics License Application for datopotamab deruxtecan was accepted in the U.S. for treating patients with previously treated advanced nonsquamous non-small cell lung cancer, based on promising results from the TROPION-Lung01 phase 3 trial. If approved, it could become the first TROP2-directed antibody drug conjugate for lung cancer treatment. ⏳ AWAITING DECISION
Dec. 22: Glepaglutide by Zealand Pharma is a long-acting GLP-2 analog developed for the treatment of adult patients with short bowel syndrome (SBS) who are dependent on parenteral support, aiming to reduce or eliminate the need for such support and improve patients’ quality of life. REJECTED❌
Dec. 27: DCCR (Diazoxide Choline) Extended-Release Tablets are being developed by Soleno Therapeutics for the treatment of hyperphagia, a life-threatening symptom of Prader-Willi Syndrome (PWS), in individuals aged four and older. The drug aims to address the excessive hunger and food-seeking behaviors that significantly impact the quality of life for those with PWS. ⏳ AWAITING DECISION
Dec. 28: Chenodiol is being developed by Mirum Pharmaceuticals as a treatment for cerebrotendinous xanthomatosis (CTX), a rare genetic disorder that affects cholesterol metabolism. ⏳ AWAITING DECISION
Dec. 28: Ensartinib is a next-generation oral tyrosine kinase inhibitor being developed by Xcovery Holdings for the first-line treatment of patients with metastatic ALK-positive non-small cell lung cancer (NSCLC). It is currently being evaluated in a phase 3 study, where it has shown superior progression-free survival compared to crizotinib. Ensartinib also demonstrated a higher intracranial response rate in patients with brain metastases. APPROVED ✅
Dec. 28: Cosibelimab is being developed by Checkpoint Therapeutics as a treatment for adults with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are not eligible for curative surgery or radiotherapy. The drug has shown promising efficacy, with a confirmed objective response rate of 47.4% in a phase 1 trial, and is being reviewed by the FDA for approval. APPROVED ✅
Dec. 30: Crinecerfont, developed by Neurocrine Biosciences, is an investigational drug aimed at treating congenital adrenal hyperplasia (CAH), a rare endocrine disorder caused by 21-hydroxylase deficiency. It works by reducing excess adrenocorticotropic hormone (ACTH) and adrenal androgens, potentially offering a safer treatment option than current therapies, which rely on supraphysiologic glucocorticoid doses. APPROVED ✅
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