// Biotech and Pharma Therapeutics
Roche wades deeper into RNA waters, paying Ascidian $42M to develop exon editing drugs
June 18, 2024 / Alchem / Price Fixing
“EU antitrust regulators have accused Alchem International of participating in a cartel to fix prices of N-Butylbromide Scopolamine, used in abdominal antispasmodic drugs. The charges could lead to significant fines for the company.
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Healthcare Execs’ Urgent Calls To Action
June 17, 2024 / AHIP 2024 Panel / AI / Healthcare
During AHIP 2024, healthcare executives highlighted the urgent need for industry changes, focusing on enhancing primary care, leveraging AI, addressing systemic issues, and rebuilding trust among consumers.
Syncona melds two gene therapy biotechs for better shot at new nervous system treatments
June 17, 2024 / Syncona / Gene Therapy / Spur Therapeutics / CNS Disease Treatments / Gene Therapy / Parkinson’s
Syncona is merging Freeline Therapeutics and SwanBio Therapeutics to form Spur Therapeutics, focusing on gene therapy for central nervous system diseases, including Gaucher disease and AMN. Syncona is investing an additional $50 million to support this initiative.
Alchem accused of pharma price fixing by EU watchdog
June 14, 2024 / Roche Ascidian / Partnership / RNA exon editing / Neurological Disease Treatment / mRNA
Roche invests $42 million in Ascidian Therapeutics to develop RNA exon editing drugs for neurological diseases, with potential milestone payments up to $1.8 billion. This partnership aims to treat diseases by rewriting mRNA instead of editing DNA.
AstraZeneca, Amgen and BMS Add New Indications for Cancer Drugs
June 17, 2024 / FDA / Drug Approval / Cancer Drug / AstraZeneca / Amgen / Cell Therapies / Gene Therapies
AstraZeneca, Amgen, and BMS received FDA approval for new cancer drug indications in 2023, a year marked by 55 new drug approvals and 34 cell and gene therapies, alongside several Complete Response Letters.
// 4th Industrial Revolution
Medidata Launches New AI Solution to Modernize the Data Experience in Clinical Trials
June 18, 2024 / Medidata Clinical Data Studio / AI / Clinical Trials
Medidata’s new AI-powered Clinical Data Studio enhances clinical trials by integrating data from various sources, improving decision-making, and accelerating data review by up to 80%. It aims to provide comprehensive data management and risk strategies.
Color Health uses OpenAI to develop cancer screening copilot for doctors
June 18, 2024 / Color Health / AI / Cancer Screening / OpenAI
Color Health is utilizing OpenAI’s language model to develop a cancer screening copilot for doctors, aiming to expedite prior authorization and improve patient treatment timelines. The tool is being tested in collaboration with UCSF to enhance early detection and diagnostics.
How to attain and sustain transparency in medical devices outfitted with AI
June 12, 2024 / FDA / AI / Medical Devices / Patient Safety / Machine Learning
New guidance from the FDA, Health Canada, and the UK’s MHRA emphasizes the importance of transparency in machine learning-enabled medical devices (MLMDs). It outlines how to communicate relevant information to ensure patient safety, foster trust, and enhance device usability.
7 ways artificial intelligence can boost the pharmaceutical industry
June 18, 2024 / AI / Drug Discovery
AI can revolutionize the pharmaceutical industry by improving supply chain logistics, quality control, demand forecasting, and data accuracy. AI’s capabilities enhance efficiency, reduce costs, and accelerate drug discovery and clinical trials, benefiting patient safety and treatment timelines.
DIA: Pharma expert offers advice on AI best practices for industry
June 8, 2024 / AI / Risk Management / San Diego
Rose Purcell of Takeda suggests that AI best practices in pharma can build on existing guidelines from organizations like NIST and ISO. Key considerations include data governance, patient privacy, and a risk management framework.
// Business & Markets
Cancer Biotech Day One Biopharma Expands to ADCs, Paying $55M to Nab a Phase 1-Ready Drug
June 18, 2024 / Day One Biopharma / Cancer Treatment / PTK7 Targeting
Day One Biopharma has acquired global rights, excluding Greater China, to MabCare Therapeutics’ ADC, DAY301, for $55 million upfront. DAY301, targeting PTK7, will enter Phase 1 trials and aims to treat various cancers, including pediatric and adult solid tumors.
Linux Foundation seeks collaborators for new interoperability open standard
June 18, 2024 / Linux Foundation / Margo Initiative / Digital Transformation
The Linux Foundation’s Joint Development Foundation, including companies like Microsoft and Siemens, is launching the Margo initiative to create an interoperability standard for edge computing. This aims to enhance digital transformation by facilitating multi-vendor data exchange and innovation.
Roche Inks Potential $1.8B Deal With Ascidian to Develop RNA Exon Therapies
June 18, 2024 / Roche Ascidian / Partnership / RNA Exon / Editing Therapies / Neurological Disease
Roche has partnered with Ascidian Therapeutics in a deal worth up to $1.8 billion, paying $42 million upfront to develop RNA exon editing therapies for neurological diseases. Ascidian will handle discovery and preclinical activities, while Roche will manage clinical development and commercialization.
Decheng Capital Eyes $700M Raise for Fifth Life Sciences Fund as VC Heats Up
June 18, 2024 / Eli Lilly / Regeneron / Roche / Early-stage Biotech / Healthcare / Acquisitions
Decheng Capital is targeting a $700 million raise for its fifth life sciences fund, focusing on early-stage and growth-stage healthcare companies. This follows significant investments and successful exits, including acquisitions by Eli Lilly, Regeneron, and Roche.
San Diego biotech making melanoma skin patch files for Chapter 11 bankruptcy
June 18, 2024 / San Diego Biotech / Dermtech Bankruptcy / Skin Cancer
DermTech, a San Diego biotech company developing a noninvasive melanoma detection patch, filed for Chapter 11 bankruptcy to restructure debt and sell assets. Despite some insurance coverage, it faces financial challenges and the need for additional capital.
// Legal & Regulatory
Kansas AG accuses Pfizer of misrepresenting COVID vaccine, hiding safety risks in lawsuit
June 18, 2024 / Pfizer / Lawsuit / Vaccine
Kansas Attorney General Kris Kobach has filed a lawsuit against Pfizer, accusing the company of misrepresenting the safety and efficacy of its COVID-19 vaccine. The lawsuit claims Pfizer hid adverse event data, including risks of myocarditis and strokes, violating the state’s Consumer Protection Act.
Merck wins FDA OK for vaccine rival to Pfizer’s pneumococcal shot
June 18, 2024 / Merck Capvaxive Approval / FDA / Vaccines
Merck’s new pneumococcal vaccine, Capvaxive, has received FDA approval. Targeting 21 serotypes, it surpasses Pfizer’s Prevnar 20, which covers 20 serotypes. Capvaxive aims to protect against serotypes responsible for 84% of invasive pneumococcal disease in adults 50+.
Zentalis’ shares plunge 30% after FDA places WEE1 drug on partial hold over sepsis deaths
June 18, 2024 / FDA / Partial Clinical Hold / Ovarian Cancer / Stock Drop
Zentalis’ shares dropped 30% after the FDA placed a partial hold on trials of its WEE1 inhibitor, azenosertib, due to two sepsis-related deaths in the DENALI study. The hold affects trials in ovarian, uterine serous carcinoma, and solid tumors.
Woes mount for Marinus with investor lawsuit, trial failure
June 17, 2024 / Marinus Pharma / Lawsuit / Share Price Drop
Marinus Pharmaceuticals faces challenges after the phase 3 RAISE trial for ganaxolone in refractory status epilepticus (RSE) met only one of two primary endpoints, and a shareholder lawsuit was filed. The company’s stock has dropped 81% since April.
FDA halts trial of BioNTech-MediLink ADC over ‘significant risk of illness’
June 17, 2024 / FDA / BioNTech-MediLink
The FDA has partially halted a phase 1 trial of BioNTech-MediLink’s antibody-drug conjugate BNT326/YL202 due to significant risk of illness or injuries, including three fatalities. MediLink must review and address safety concerns to resume patient enrollment.
// Research & Development
Takeda’s Epilepsy Drug Stumbles in Back-to-Back Phase III Rare Disease Failures
June 17, 2024 / Epilepsy Drug Trial / Takeda
Takeda’s epilepsy drug soticlestat failed to meet primary endpoints in Phase III trials for Dravet syndrome (SKYLINE) and Lennox-Gastaut syndrome (SKYWAY). Despite some secondary benefits, the drug narrowly missed statistical significance in reducing seizures.
To treat obesity in children, task force favors behavioral therapy over drugs like Wegovy
June 18, 2024 / Obesity Treatment / Wegovy for Children
The United States Preventive Services Task Force recommends comprehensive behavioral interventions over obesity medications like Wegovy for children aged 6 and older with high BMI. This contrasts with the American Academy of Pediatrics’ advice to consider obesity drugs for children 12 and older.
GSK Reignites Blenrep Blockbuster Aspirations With Data from Phase III Studies
June 18, 2024 / GSK / Blenrep Revival
GSK is reviving its multiple myeloma treatment Blenrep (belantamab mafodotin) after promising Phase III trial results. Data from DREAMM-7 and DREAMM-8 studies showed significant improvements in progression-free survival and response rates, suggesting multi-blockbuster potential.
Google creates Tx-LLM for drug discovery and therapeutic development
June 17, 2024 /Drug Discovery / AI / Pharma Research
Google Research and DeepMind have introduced Tx-LLM, a large language model (LLM) fine-tuned from PaLM-2 for drug discovery and therapeutic development. Tx-LLM, trained on 709 datasets for 66 drug discovery tasks, outperformed state-of-the-art models in many tasks.
Takeda drug for rare types of epilepsy misses goal in late-stage trial
June 17, 2024 / Takeda / Dravet Syndrome Trial / Partnership
Takeda’s epilepsy drug soticlestat failed in a late-stage trial for Dravet and Lennox-Gastaut syndromes, casting doubt on milestone payouts from its $200 million deal with Ovid. Takeda is restructuring, cutting drugs from its pipeline but retaining interest in neuroscience.
// Politics
With sweeping NIH reform on the table, GOP previews new era of research scrutiny
June 18, 2024 / NIH / Disease Research
House Republicans propose significant reforms to the NIH, including reducing its institutes from 27 to 15, establishing director term limits, and tightening infectious disease research and grant reporting. These changes could gain traction if President Trump is re-elected.
WuXi buoyed as BIOSECURE Act is left out of US spending bill
June 13, 2024 / Biosecure Act / US Spending Bill / NDAA
WuXi’s stock rose after the BIOSECURE Act, which would ban US federal funding for biotech equipment from certain Chinese companies, was omitted from the US defense spending bill. This reduces immediate pressure on WuXi but concerns remain.
US Drug Price Negotiations May Not Cut Costs Significantly, Say Experts: ‘Sizzle Is Bigger Than The Steak’
June 17, 2024 / US Drug Price Negotiations / Inflation Reduction Act / Medicare Drug Prices / Healthcare
Experts suggest that the U.S. government’s new power to negotiate drug prices under the Inflation Reduction Act may not significantly lower medication costs. The law, affecting only Medicare and a few expensive drugs starting in 2026, might not threaten pharmaceutical profits.
BIOSECURE Act could affect half of US biopharmaceutical development
June 19, 2024 / Biosecure Act / Partnerships / WuXi AppTec / China / Samsung
The BIOSECURE Act could impact over 120 US biopharmaceutical drugs partnered with Chinese companies, with about half in clinical trials. This legislation, targeting companies like WuXi AppTec and BGI, may lead to increased costs and delays, prompting a shift to CDMOs outside China, such as Samsung Biologics.
Billions at stake as national battle over drug pricing plays out in Missouri
June 14, 2024 / Missouri / Drug Pricing / Biosecure Act
Missouri’s General Assembly passed a bill mandating that pharmaceutical manufacturers provide discounted drugs to qualifying hospitals and clinics, addressing gaps in federal law. This legislation, part of a national trend, aims to enhance access to affordable medications amid escalating drug prices.
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