This Week in Biotech #22
Catch up on the latest biotech breakthroughs and upcoming trends (Oct 22-24).
Welcome to the Friday edition of This Week in Biotech by Biotech Blueprint!
🎙️ PODCAST
Also available on Substack, Spotify or Apple Podcasts.
MARKET UPDATES
🔹 On Oct. 24, Zai Lab (ZLAB), a global biopharmaceutical company based in China and the U.S., presented promising data from its phase 1a/b study of ZL-1310, an investigational DLL3-targeted antibody-drug conjugate for small cell lung cancer. The study showed an impressive objective response rate of 74% across all dose levels, indicating strong anti-tumor activity and a favorable safety profile. The stock rose over 20% following the announcement of these encouraging results. Zai Lab aims to continue developing ZL-1310 as both a monotherapy and in combination therapies.
🔹 On Oct. 24, Intellia Therapeutics (NTLA) announced positive results from the phase 2 trial of NTLA-2002, an investigational CRISPR gene-editing therapy for hereditary angioedema. The study showed that a single 50 mg dose reduced monthly attack (severe swelling) rates by 77% to 81%, with eight out of 11 patients becoming attack-free. The treatment was well-tolerated, with no serious adverse events, supporting its potential as a one-time, functional cure for angioedema. These findings will be presented at the 2024 ACAAI meeting and published in The New England Journal of Medicine. However, the stock took a hit, declining by more than 20% following the announcement. Phase 3 trials are now underway.
🔹 On Oct. 23, Viking Therapeutics (VKTX) released its financial results for Q3 2024, highlighting a significant focus on its promising obesity treatment candidate, VK2735. VK2735 is currently in phase 1 trials, and Viking is optimistic about its safety profile, with no recommendations for dose escalation discontinuation reported so far. The company is set to begin a phase 2 trial in Q4 2024, and they anticipate presenting data at Obesity Week, generating excitement around its potential as a leading weight-loss medication. As of the end of September, Viking had approximately $39M in cash, positioning it well for ongoing clinical trials. Additionally, Viking’s stock has seen a remarkable increase of over 250% year-to-date, largely driven by interest in VK2735 as a competitive alternative to existing obesity drugs. Analysts maintain a bullish outlook, with price targets significantly above the current stock price. Following this financial update, the stock went up 20% to $73 per share.
🔹 Monopar Therapeutics’ (MNPR) stock experienced significant stock fluctuations in the past 2 days. On Oct. 22, the company presented promising human clinical dosimetry data at the European Association of Nuclear Medicine Congress, highlighting the potential of its uPAR-targeted radiopharmaceuticals MNPR-101-Zr and MNPR-101-Lu. This led to a dramatic stock price increase of nearly 400% ($5.60 to $28 per share), as investors reacted positively to the encouraging data on tumor uptake and safety. The stock then dipped slightly on Oct. 23. Following the announcement on Oct. 24 of a partnership with Alexion and AstraZeneca to acquire the rights to ALXN-1840, a phase 3 drug candidate for Wilson disease, Monopar’s stock rebounded and is now approaching $20 per share.
🔹 J. P. Morgan downgraded Seres Therapeutics (MCRB) to Underweight from Neutral on Oct. 23, citing challenges in finding a near-term growth driver despite the Vowst sale to Nestle Health Science extending the company’s cash runway to Q4 2025. While the sale alleviates debt pressure, the bank noted a lack of clear catalysts for its SER-155 program, leading to subdued sentiment. As a result, the firm expects Seres shares to underperform as its pipeline remains uncertain.
BIOTECH NEWS
🔹 Precision BioSciences has received approval for its first clinical trial application in the country of Moldova to initiate a first-in-human study for PBGENE-HBV, its in vivo gene editing program aimed at curing chronic hepatitis B. This marks the first global clinical trial for a gene editing therapy targeting HBV. PBGENE-HBV utilizes the Arcus platform to eliminate HBV’s key replication source, cccDNA, and inactivate integrated HBV DNA in hepatocytes.
🔹 Dyno Therapeutics has formed a new strategic partnership with Roche to develop next-generation adeno-associated virus (AAV) gene therapy vectors targeting neurological diseases. This collaboration builds on a prior 2020 partnership between the two companies focused on neurological and liver-directed therapies. Dyno will receive $50M upfront and could earn over $1B in milestone payments, plus royalties. Dyno’s platform uses AI and high-throughput in vivo data to engineer improved AAV capsids for enhanced tissue targeting, immune evasion, and manufacturability. Roche will validate these capsids and handle preclinical, clinical, and commercialization activities.
🔹 On Oct. 24, Lyell Immunopharma announced the acquisition of ImmPACT Bio for $30M and 37.5M shares of Lyell’s stock to strengthen its pipeline of next-generation CAR T-cell therapies. The acquisition adds ImmPACT’s dual-targeting CD19/CD20 CAR T-cell candidate, IMPT-314, to Lyell’s portfolio, focusing on treating large B cell lymphoma. Data from a phase 1-2 trial of IMPT-314 will be presented later in 2024, with a pivotal trial expected in 2025. To prioritize this acquisition, Lyell is discontinuing the development of several other programs, focusing instead on IMPT-314 and LYL119. This shift is expected to fund operations into 2027.
🔹 On Oct. 24, Rigel Pharmaceuticals has issued a “Dear Healthcare Provider” letter regarding a new safety signal for Gavreto (pralsetinib), a treatment for non-small cell lung cancer and advanced thyroid cancer, following discussions with the FDA. The letter highlights the risks of severe adverse reactions, including interstitial lung disease, hypertension, hepatotoxicity, hemorrhagic events, and tumor lysis syndrome, advising healthcare providers to closely monitor patients. The letter aims to enhance awareness and promote safety reporting. Gavreto was transferred from Blueprint Medicines to Rigel in June 2024.
🔹 Novo Nordisk has asked the FDA to ban compounding pharmacies from producing copycat versions of its weight-loss and diabetes drugs, Wegovy and Ozempic, citing safety concerns. These drugs, both containing semaglutide, have been in short supply in the U.S., prompting compounders to create alternatives. Novo argues that semaglutide is too complex to be safely copied and claims that unauthorized versions have led to nearly 400 adverse events since 2018. The FDA is reviewing Novo’s petition. This move is part of Novo’s broader effort, alongside rival Eli Lilly, to stop the sale of cheaper, unapproved versions of their obesity drugs.
🔹 Merck has acquired Modifi Biosciences, a Yale University spinout formed in 2021, for $30M upfront, with potential milestone payments up to $1.3B. Modifi Biosciences specializes in developing small molecules that target cancer cells with DNA repair defects, particularly those lacking the MGMT protein. This innovative approach aims to address resistance mechanisms in challenging cancers, such as glioblastoma. The acquisition allows Merck to advance these preclinical compounds into clinical trials and potential commercialization.
🔹 On Oct. 23, Eccogene received a $60M milestone payment from AstraZeneca after dosing the first patient in a global phase 2b trial for ECC5004/AZD5004, an investigational oral GLP-1 receptor agonist aimed at treating obesity and type 2 diabetes. ECC5004/AZD5004, which was licensed to AstraZeneca in 2023, is being evaluated in two trials, VISTA and SOLSTICE, that focus on patients with obesity and weight-related comorbidities or type 2 diabetes, respectively. This milestone highlights the ongoing collaboration between the two companies, with Eccogene retaining co-development rights in China. The deal with AstraZeneca could bring Eccogene up to $1.825B in future payments.
🔹 DBV Technologies announced positive regulatory updates for its Viaskin Peanut patch, a skin patch designed to treat peanut allergies using epicutaneous immunotherapy. The company will pursue an accelerated approval pathway for toddlers aged 1-3 in the U.S., contingent on the completion of a six-month safety study set to begin in Q2 2025. Additionally, DBV’s phase 3 VITESSE study for children aged 4-7 exceeded enrollment targets, with topline results expected by Q4 2025. In Europe, the EMA confirmed a path for a marketing authorization application for children aged 1-7. Following the news, DBV’s stock surged by 45% from €0.73 to €1.40 but has since returned down to €0.78. The Viaskin Peanut patch works by delivering small amounts of peanut protein through the skin, gradually desensitizing the immune system and reducing the risk of severe allergic reactions. Unlike oral therapies, the patch’s design allows peanut proteins to be absorbed without entering the bloodstream, offering a less invasive treatment for children and toddlers with peanut allergies.
🔹 The FDA has approved Pfizer’s RSV vaccine, Abrysvo, for adults aged 18 to 59 who are at high risk of developing lower respiratory tract disease caused by respiratory syncytial virus (RSV). Abrysvo had previously been approved for adults aged 60+ and pregnant women to protect newborns up to six months of age. The decision is based on results from the phase 3 MONeT study, which demonstrated the vaccine’s safety and efficacy.
CLINICAL TRIAL UPDATES
🔹 On Oct. 23, Aprea Therapeutics presented preliminary findings on its oral WEE1 inhibitor, APR-1051, at the EORTC-NCI-AACR International Conference. The ongoing phase 1 ACESOT-1051 trial is evaluating APR-1051 as a monotherapy for advanced solid tumors with cancer-associated gene alterations. Early results show that APR-1051 is safe and well-tolerated with no hematologic toxicity, as patients experienced no significant adverse effects. Active enrollment is ongoing across three U.S. sites. Aprea believes APR-1051 has the potential to address unmet needs in cancer therapy by selectively targeting WEE1 without off-target effects.
🔹 On Oct. 22, Alto Neuroscience announced that its phase 2b trial of ALTO-100 for major depressive disorder (MDD) did not achieve its primary endpoint, showing no significant improvement in depressive symptoms compared to placebo, despite a favorable safety and tolerability profile. The study, which included 301 adults and utilized a memory-based cognitive biomarker, did not replicate earlier positive results from a phase 2a trial. The company is committed to analyzing the data further and continues to support its research with a strong cash position expected to fund operations through 2027 and upcoming clinical milestones for other treatments in its pipeline.
SCIENCE SPOTLIGHT
🔹 A study published on Oct. 24 in Nature reveals a concerning link between rifaximin, an antibiotic commonly prescribed for liver disease, and the emergence of resistance to daptomycin, a last-resort antibiotic used to treat vancomycin-resistant Enterococcus faecium (VREfm).
This discovery marks the first documented case of one antibiotic causing resistance to another from a different class, challenging the long-held belief that rifaximin posed a low risk for developing antibiotic resistance. The research, led by an international team including scientists from the University of Melbourne, found that rifaximin exposure triggers specific changes in the bacterial RNA polymerase. These alterations lead to the upregulation of a previously unknown gene cluster (prdRAB), resulting in cell membrane remodeling and cross-resistance to daptomycin. Alarmingly, VREfm strains with these mutations have spread globally, potentially compromising the clinical efficacy of daptomycin, a critical antibiotic for treating multidrug-resistant infections.
The findings underscore the need for more cautious antibiotic use and improved monitoring of resistance patterns, particularly in hospital settings where these resistant strains might be transmitted between patients.
ON THE HORIZON
🔹 Obesity Week 2024 conference will be taking place in San Antonio, TX, from Nov. 3 to 6 , 2024. This annual event, hosted by The Obesity Society, will feature over 800 presentations covering a wide array of topics related to obesity science, including metabolic health, clinical interventions, and health policy.
Viking Therapeutics will present results from two studies related to their lead candidate, VK2735.
Rhythm Pharmaceuticals is set to present five posters related to their lead asset, setmelanotide, which is an MC4R agonist designed for treating hyperphagia and severe obesity.
🔹 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.
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.
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 HERE.
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.
Girl you ought to be on Twitter. Your content is fire 🔥 not only bc if that though. We/I would enjoy discussions about biotech space. Resurrect one of the handles that you have.