BioPharmCatalyst (A Scientist.com Company)

?? Weekly Watchlist: Biotech Regulatory Catalysts Before Year-End?? With just 20 PDUFAs left in 2024, this week’s spotlight shines on key regulatory decisions that could shape the biotech landscape. Here’s a look at significant milestones and their market implications: ?? Applied Therapeutics Inc. (NASDAQ: APLT) Focus: Rare metabolic diseases Catalyst: AT-007 (govorestat) PDUFA - November 28, 2024 ?? AT-007 targets Galactosemia, a genetic disorder with limited treatment options. Backed by Fast Track Designation and promising data, this therapy could establish a new standard in an underserved market. ?? Ionis Pharmaceuticals Inc. (NASDAQ: IONS) Focus: RNA-targeted therapeutics Catalyst: Olezarsen PDUFA - December 19, 2024 ?? With Priority Review and Orphan Drug Designation, olezarsen shows potential to transform Familial Chylomicronemia Syndrome (FCS) management. Approval could bolster Ionis’ leadership in RNA-based therapies. ?? Fortress Biotech Inc. (NASDAQ: FBIO) Focus: Immuno-oncology Catalyst: Cosibelimab PDUFA - December 28, 2024 ?? Cosibelimab offers a cost-effective alternative for cutaneous squamous cell carcinoma (CSCC). A positive decision could position Fortress as a competitive player in the immunotherapy space. ?? Lexicon Pharmaceuticals Inc. (NASDAQ: LXRX) Focus: Cardiovascular and metabolic diseases Catalyst: Zynquista (sotagliflozin) PDUFA - December 20, 2024 ?? Despite operational challenges and workforce reductions, Lexicon is laser-focused on Zynquista’s CKD review. Approval could address critical unmet needs for diabetic CKD patients. Stay tuned as we revisit these watchlists next week, following updates from earnings releases and developments on these pivotal catalysts. ?? What milestones are you watching this quarter? Let’s discuss below! #Biotech #Pharma #LifeSciences #DrugDevelopment #RegulatoryAffairs #PDUFA #FDACalendar #FasterScience #BeThereWhenItHappens #BioPharmCatalyst #BiotechInvesting #Catalysts

We are excited to officially announce the launch of our new chatbot, Aiden! Aiden is designed to make the navigation and usefulness of our site much easier and client friendly, and to assist with customer inquiries. You can test Aiden out by clicking the brain logo on the bottom right-hand corner of any of our site pages! www.biopharmcatalyst.com #biotech #biotechinvesting #ai #llm #artificialintelligence #fasterscience #betherewhenithappens https://lnkd.in/e4crce2i

?? Weekly Watchlist: Phase 1/2 Catalysts to Watch in Q4 2024 ?? This week, we turn our focus to pivotal Phase 1/2 catalysts driving innovation in biotech. With over 300 catalysts remaining this year and less than 50 featuring topline data, these programs stand out for their potential impact. Recursion Pharmaceuticals (NASDAQ: RXRX) is leveraging AI and automation to revolutionize drug discovery. Its lead asset, REC-2282, is targeting neurofibromatosis type 2 (NF2), a rare condition with no FDA-approved treatments. Interim data from the POPLAR-NF2 trial is due by December 31, 2024. With Orphan Drug and Fast Track designations, REC-2282 could be a game-changer for NF2 patients. Enanta Pharmaceuticals (NASDAQ: ENTA) is advancing zelicapavir (EDP-938) for RSV, the only N-inhibitor in clinical development for this indication. Phase 2b data from its adult and pediatric trials are expected by year-end, highlighting its potential to address severe RSV outcomes in vulnerable populations like the elderly and high-risk children. Trevi Therapeutics (NASDAQ: TRVI) is developing Haduvio for chronic cough in idiopathic pulmonary fibrosis (IPF). With no approved treatments available, the Phase 2b CORAL trial could position Haduvio as a first-in-class therapy. An important milestone is expected in December, with topline data coming in early 2025. Which of these biotech innovators are you keeping on your radar? Let’s discuss their potential!?? #Biotech #ClinicalTrials #DrugDevelopment #Innovation #RSV #RareDiseases #Investing #FDACalendar #BioPharmCatalyst #BeThereWhenItHappens #FasterScience

?? Biotech Watchlist: Phase 3 Catalysts to Watch in Q4 2024 ?? As we near the year's end, fewer than 100 Phase 3 catalysts remain on the horizon, with about 30 expected to release topline data. This week, we spotlight some pivotal programs that could reshape their fields. Cassava Sciences (NASDAQ: SAVA) is advancing simufilam for Alzheimer’s disease. With two Phase 3 trials underway and data expected by Q4 2024, this small molecule therapy could transform care for a population with significant unmet needs. Early data hinted at cognitive benefits, and the latest studies aim to confirm efficacy and safety over a 52-week period. BioCardia (NASDAQ: BCDA) is developing CardiAMP, a breakthrough cell therapy for ischemic heart failure. Phase 3 data due Q4 2024 could validate early findings, including a 37% reduction in heart failure-related mortality. This milestone could pave the way for approval of a therapy addressing critical cardiovascular needs. NovoCure (NASDAQ: NVCR) is exploring Tumor Treating Fields (TTFields) for pancreatic cancer in its PANOVA 3 Phase 3 trial. Results expected in Q4 2024 could expand the utility of TTFields, already approved for other cancer types, offering hope for patients with this difficult-to-treat malignancy. Let’s hear from you! Which of these programs are you watching most closely? What do you think could have the biggest impact? Share your thoughts!?? #Biotech #Phase3 #DrugDevelopment #Investing #Innovation #Healthcare #FDACalendar #BeThereWhenItHappens #FasterScience #BioPharmCatalyst

?? Biotech Watch: Key Catalysts on the Horizon for April 2025 This week, we’re spotlighting upcoming milestones that could be transformative for companies in the biotech sector. With over 600 catalysts expected by April 2025—100+ of which are crucial topline data releases—there’s a lot to track! Here’s a quick dive into some high-impact events we’re watching. ?? Immuron Limited (NASDAQ: IMRN) ?? Focus: Polyclonal antibody treatments, addressing digestive and immune health ?? Key Catalyst: April 2025 topline data from a Phase 2 trial of Travelan, targeting gastrointestinal infection prevention ?? What’s at Stake: Travelan’s success could pave the way for Phase 3 trials later in 2025, potentially positioning it as a leading preventive treatment for traveler’s diarrhea. With 85% of participants recruited, Immuron’s momentum is building toward a potentially breakthrough data readout. ?? Gyre Therapeutics Inc. (NASDAQ: GYRE) ?? Focus: Protease therapeutics for complement and coagulation disorders ?? Key Catalyst: Early 2025 topline data from a Phase 3 trial on hydronidone (F351) for chronic hepatitis B (CHB)-induced liver fibrosis ?? What’s at Stake: As the trial in China concludes, hydronidone could emerge as the first approved therapy for hepatitis B-induced liver fibrosis—a critical unmet need. A U.S. Phase 2 trial for MASH-associated fibrosis is on the horizon, expanding its potential reach. ?? NewAmsterdam Pharma Company N.V. (NASDAQ: NAMS) ?? Focus: Oral, non-statin cardiovascular treatments ?? Key Catalyst: Q1 2025 topline data from the Phase 3 TANDEM trial on a fixed-dose combination of obicetrapib and ezetimibe ?? What’s at Stake: With CVD as a leading cause of death, a new LDL-lowering treatment could significantly impact patients with hyperlipidemia and ASCVD. This milestone could accelerate NewAmsterdam’s path to market with a potent therapeutic option for patients who struggle with conventional treatments. Next week, stay tuned as we break down updates from earnings releases, including developments in Phase 3 trials. The next few months are shaping up to be pivotal for these companies and the biotech landscape! #Biotech #PharmaUpdates #ClinicalTrials #InvestmentWatch #Biopharma #BioPharmCatalyst #FDACalendar #BiotechInvesting #FasterScience #BeThereWhenItHappens

?? Biotech Catalysts to Watch in Q4 2024 ?? As we head into November and December, our weekly focus is on pivotal milestones in the biotech world—each with the potential to drive significant clinical and market impact. Here are some of the key companies and their upcoming catalysts: ?? Puma Biotechnology, Inc. (NASDAQ: PBYI) Focused on oncology with a primary emphasis on small-cell lung cancer (SCLC), Puma’s lead drug, Alisertib, holds Orphan Drug Designation and is showing promising results. In its ongoing Phase 2 ALISCA-Lung1 trial, Alisertib demonstrated an 81% disease control rate, and more data expected this quarter could further highlight its potential to address high-unmet needs in SCLC. ?? Autolus Therapeutics plc (NASDAQ: AUTL) With a $1.19B market cap, this London-based biotech aims to fill a crucial treatment gap in B-cell Acute Lymphoblastic Leukemia (B-ALL) with its CAR T-cell therapy, Obe-cel (AUTO1). Having received RMAT designation and priority review, the FDA’s PDUFA decision on November 16, 2024, is a major milestone that could shape the future of B-ALL treatment. ??? Trevi Therapeutics, Inc. (NASDAQ: TRVI) Developing therapies for neurologically mediated conditions, Trevi’s Haduvio is currently in Phase 2b for chronic cough in idiopathic pulmonary fibrosis (IPF). With interim results anticipated in December, this trial could support a symptom-focused therapy in IPF, where options are limited. ?? Cardiff Oncology Inc. (NASDAQ: CRDF) Valued at $135.7M, Cardiff’s CRDF-004 is tackling metastatic colorectal cancer (mCRC) through an innovative combination with Onvansertib. With interim data expected in H2 2024, Cardiff’s approach could change the landscape for this challenging cancer type. ?? Quince Therapeutics, Inc. (NASDAQ: QNCX) Developing EryDex for Ataxia-Telangiectasia (A-T), a rare neurodegenerative disease, Quince’s ongoing Phase 3 NEAT trial represents hope for a condition with no approved treatments. With a data readout expected in Q4 2025, the company is positioned to be a pioneer in this high-unmet-need area. Stay tuned as we continue to monitor and share updates on these exciting developments across the biotech sector! #Biotech #Pharma #ClinicalTrials #Oncology #RareDiseases #LifeSciences #Biotechnology #Newsletter #BiotechInvesting #FasterScience #BeThereWhenItHappens #BioPharmCatalyst

Curious how presidential elections in the U.S. impact the biotech industry? Check out our article on "Election Volatility & Biotech", where we dive into how previous elections have impacted the industry as we await the results of the 2024 presidential election. #biotech #biotechinvesting #betherewhenithappens #fasterscience https://lnkd.in/gvD6FY4p

?? Key Catalysts to Watch in Q4 2024: Corcept Therapeutics, Omega Therapeutics, & Marinus Pharmaceuticals ?? As we move through the fourth quarter, several biotech companies are set to announce pivotal clinical trial results that could reshape their future—and the landscape of treatments for severe conditions. Here's a snapshot of the top catalysts to keep on your radar: 1?? Corcept Therapeutics (NASDAQ: CORT) The highly anticipated Phase 3 GRADIENT trial results for relacorilant in treating Cushing’s syndrome are expected in Q4 2024. This investigational therapy has already secured orphan drug status in the U.S. and EU, offering hope to patients affected by adrenal adenomas. Success here could drive a major step forward for Corcept. 2?? Omega Therapeutics (NASDAQ: OMGA) Omega is exploring the frontier of mRNA therapeutics with its lead program OTX-2002, targeting liver cancer (HCC) via MYC downregulation. With positive early data from the MYCHELANGELO I trial, upcoming updates in Q4 2024 could open doors for further development, potentially in combination with Roche’s atezolizumab. 3?? Marinus Pharmaceuticals (NASDAQ: MRNS) In the race to treat tuberous sclerosis complex (TSC)-related epilepsy, Marinus is nearing the finish line. The Phase 3 TrustTSC trial for ganaxolone is set to deliver topline data in Q4 2024. This could be a game-changer for patients with refractory epilepsy and a critical milestone for the company as it preps for an NDA submission in 2025. Stay tuned for these updates—they could have a profound impact on therapeutic options and shape the future of these biotech companies. ???? #Biotech #Pharmaceuticals #LifeSciences #ClinicalTrials #CushingsSyndrome #LiverCancer #Epilepsy #MRNA #BiotechInnovation #DrugDevelopment #CatalystWatch #FDACalendar #BiotechInvesting #FasterScience #BeThereWhenItHappens

We are always happy and eager to help out investment communities and anyone interested in learning more about the biotech market. Thanks Bristol Investment Fund! #biopharmcatalyst #betherewhenithappens #fasterscience #biotechinvesting #fdacalendar #investmentcommunity

?? Key FDA PDUFA Catalysts to Watch in Early 2025 ?? As we head into 2025, the biotech industry has several major regulatory milestones on the horizon! From innovative cell therapies to breakthrough treatments, these PDUFA dates could reshape the landscape for key players in the sector. Here’s a quick look at some companies with major FDA decisions early next year: ?? Mesoblast Limited (NASDAQ: MESO) Focused on regenerative medicine, Mesoblast is advancing RYONCIL (remestemcel-L) for treating pediatric acute graft versus host disease (aGVHD). With a PDUFA date set for January 7, 2025, this decision could address a critical unmet need and boost the company’s position in the market. ?? Atara Biotherapeutics Inc. (NASDAQ: ATRA) Developing T-cell therapies, Atara's lead candidate tabelecleucel (tab-cel) is targeting Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease. With a January 15, 2025 PDUFA, approval could further solidify Atara in the cell therapy space. ?? SpringWorks Therapeutics Inc. (NASDAQ: SWTX) With a focus on targeted oncology, SpringWorks is gearing up for the February 28, 2025 PDUFA for mirdametinib, aimed at treating neurofibromatosis type 1-associated plexiform neurofibromas. ?? Axsome Therapeutics Inc. (NASDAQ: AXSM) Already making strides with Auvelity for depression, Axsome is awaiting the January 31, 2025 PDUFA for AXS-07, a migraine treatment. A positive decision could significantly expand Axsome’s CNS portfolio and drive future growth. These milestones could shape the biotech landscape and bring transformative therapies to patients. Keep an eye on these companies in the months ahead! #Biotech #FDA #PDUFA #CellTherapy #Oncology #RareDiseases #Pharmaceuticals #RegenerativeMedicine #ClinicalTrials #FasterScience #BioPharmCatalyst #BeThereWhenItHappens