Alnylam Pharmaceuticals

Brain Cancer Canada Funds $68,000 for Innovative DIPG Vaccine Clinical Trial

5 days ago

• Brain Cancer Canada has awarded $68,000 to researchers at the University of Calgary and Alberta Children's Hospital for a clinical trial of a neoantigen-based anti-cancer vaccine targeting DIPG, the most aggressive form of pediatric brain cancer. • The research, led by Dr. Aru Narendran and Dr. Patrick Sipila, aims to train patients' immune systems to recognize and eliminate tumor cells, potentially offering new hope for a disease with fewer than 10% of children surviving two years post-diagnosis. • The announcement coincides with DIPG Awareness Day, with landmarks across Canada illuminated in yellow to raise awareness for this devastating pediatric brain cancer that has seen little improvement in survival rates despite decades of cancer research advances.

Alnylam Concedes Defeat in COVID-19 Vaccine Patent Dispute with Pfizer and BioNTech

22 days ago

• Alnylam Pharmaceuticals has requested a Delaware federal court to end its patent infringement case against Pfizer and BioNTech following an unfavorable patent interpretation ruling. • The lawsuit, filed in 2022, alleged that Pfizer and BioNTech's COVID-19 vaccines infringed on Alnylam's patents covering lipid nanoparticle technology used to deliver genetic material. • This concession follows a similar withdrawal in Alnylam's case against Moderna last year, though the company is evaluating options for a potential appeal.

Scholar Rock Appoints David L. Hallal as CEO, Strengthens Leadership Team Ahead of Apitegromab Launch for SMA

24 days ago

• Scholar Rock has appointed David L. Hallal as CEO, transitioning from his role as Chairman of the Board, as the company prepares for the global launch of apitegromab for spinal muscular atrophy. • The company has bolstered its leadership team with three key appointments: Akshay Vaishnaw as President of R&D, R. Keith Woods as Chief Operating Officer, and Vikas Sinha as Chief Financial Officer. • Apitegromab, a first-in-class muscle-targeted treatment for SMA, has received FDA priority review following successful Phase 3 trials, with potential approval and launch expected later this year.

Vir Biotechnology to Present Key Hepatitis B and Delta Clinical Data at EASL Congress 2025

a month ago

• Vir Biotechnology will present 24-week subgroup analysis data from its Phase 2 SOLSTICE trial in chronic hepatitis delta at the EASL Congress 2025 in Amsterdam, examining how baseline viral parameters and cirrhosis status affect treatment responses. • The company will share 24-week post-treatment follow-up data from the MARCH Phase 2 study evaluating combinations of tobevibart and elebsiran in chronic hepatitis B patients, building on promising earlier results showing hepatitis B surface antigen clearance. • Previous end-of-treatment data from the MARCH study demonstrated promising rates of HBsAg loss in participants with low baseline HBsAg levels (<1000 IU/mL) when treated with combination regimens including tobevibart and elebsiran.

Medtronic's Evolut TAVR System Demonstrates Superior Durability and Performance at Five-Year Mark

2 months ago

• Medtronic's Evolut TAVR system showed comparable rates of all-cause mortality or disabling stroke to surgical valve replacement at five years (15.5% vs 16.4%), with numerically lower cardiovascular mortality rates. • The self-expanding Evolut valve demonstrated significantly larger effective orifice areas and lower mean gradients compared to surgical valves, reinforcing its superior hemodynamic performance in low-risk aortic stenosis patients. • These five-year outcomes, presented at the American College of Cardiology's Annual Scientific Session, support Evolut TAVR as a durable alternative to surgery regardless of surgical risk, potentially shifting treatment paradigms for younger patients.

Linus Health to Showcase Digital Cognitive Assessment Tools at AD/PD 2025 Conference

2 months ago

• Linus Health will present findings from two clinical studies at AD/PD 2025, demonstrating how their digital tools effectively detect cognitive and motor impairments in Parkinson's disease patients. • The company's DCTclock technology accurately quantifies drawing tremors in Parkinson's patients, while their 7-minute Digital Assessment of Cognition achieves comparable results to traditional 3-hour neuropsychological evaluations. • Following its 2024 acquisition of Aural Analytics, Linus Health has expanded its life sciences footprint, now supporting clinical trials for neurodegenerative diseases and mental health conditions at major pharmaceutical companies.

FDA Approves Alnylam's Oxlumo as First Treatment for Ultra-Rare Kidney Disease PH1

2 months ago

• The FDA has approved Oxlumo (lumasiran), the first-ever treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare genetic disorder that causes kidney damage through excessive oxalate production. • Oxlumo, Alnylam's third RNA interference therapeutic to reach market, works by silencing genes that lead to oxalate production, with clinical trials showing significant reductions in urinary oxalate levels. • The approval gives Alnylam a competitive advantage in this small but potentially profitable market, with pricing expected to be substantial given the rarity of PH1 and the company's previous rare disease therapies costing $450,000-575,000 annually.

Moderna and Pfizer/BioNTech Locked in Escalating mRNA Patent Battle with Billions at Stake

2 months ago

• Moderna has filed patent infringement lawsuits against Pfizer/BioNTech in multiple countries, claiming their Comirnaty vaccine infringes on mRNA technology patents that Moderna pioneered and invested billions in developing. • The legal battle has expanded globally with Moderna scoring a recent victory in Germany, while simultaneously suffering defeats at the USPTO's Patent Trial and Appeal Board where some of its patents were found unpatentable. • With combined COVID-19 vaccine sales exceeding $100 billion, the financial stakes are enormous, as evidenced by significant settlements already reached with NIH—$400 million from Moderna and $791.5 million from BioNTech.

Alnylam's Vutrisiran Shows Strong Efficacy in ATTR Cardiomyopathy, Setting Stage for New Treatment Standard

3 months ago

• Alnylam's RNAi drug vutrisiran demonstrated a 28% reduction in all-cause mortality and recurrent cardiovascular events in ATTR cardiomyopathy patients, with efficacy increasing to 33% in patients not taking Pfizer's tafamidis. • The HELIOS-B trial showed vutrisiran's benefits increased over time, with a 36% reduction in the primary endpoint at 42 months, positioning the quarterly-administered injection as a potential new standard of care. • Vutrisiran, already approved as Amvuttra for ATTR polyneuropathy, could reach multibillion-dollar sales if approved for cardiomyopathy, though it faces competition from BridgeBio's acoramidis and AstraZeneca/Ionis' eplontersen.

FDA Approves Genentech's TNKase for Acute Ischemic Stroke with Simplified Administration

3 months ago

• The FDA has approved Genentech's TNKase, a clot-dissolving agent, for treating acute ischemic stroke in adults, marking the company's second stroke treatment approval. • TNKase offers a streamlined five-second intravenous bolus administration, presenting a significant advantage over the current standard treatment Activase's 60-minute infusion protocol. • The approval is supported by a multi-center non-inferiority study across 22 Canadian stroke centers, demonstrating comparable safety and efficacy to Activase in patients with disabling neurological deficits.

Scholar Rock's Phase III Trial Advances Apitegromab Development for Spinal Muscular Atrophy

3 months ago

• Scholar Rock's Phase III clinical trial demonstrates promising results for apitegromab in treating spinal muscular atrophy, marking a significant advancement in neuromuscular disease therapeutics. • The company's strategic initiatives and positive trial outcomes have attracted favorable analyst attention, leading to optimistic market projections and strengthened investor confidence. • The development represents a potential breakthrough for SMA patients, offering a novel therapeutic approach that complements existing treatment options.

UK Faces Growing Challenges in Access to Rare Disease Medicines Despite Recent Approvals

3 months ago

• Recent data reveals a concerning trend as UK drops from 2nd to 10th place in European rankings for orphan medicine availability, signaling declining access to rare disease treatments. • Analysis shows over 100% increase in terminated NICE appraisals over the past decade, with rates rising from 9.8% (2016-2019) to 20.1% (2020-2023), limiting patient access to licensed medicines. • Industry survey indicates 11 out of 18 pharmaceutical companies expect to launch less than 75% of their rare disease pipeline in the UK, citing unfavorable reimbursement environment and high payment rates.

Fitusiran Shows Promise in Reducing Bleeding Episodes in Hemophilia A and B Patients

4 months ago

• Fitusiran, an RNA interference therapy, is highlighted as a potential breakthrough for hemophilia A and B treatment, regardless of inhibitor status. • Phase 3 trials (NCT03417245) demonstrated that fitusiran significantly reduced annualized bleeding rates in hemophilia A and B patients compared to on-demand factor concentrates. • Another study (NCT03417102) showed a 90.8% reduction in annualized bleeding rate in patients with inhibitors treated with fitusiran compared to on-demand treatment. • Fitusiran's efficacy and convenient subcutaneous administration position it as a drug to watch in hematology, offering a more personalized approach to hemophilia management.

Kailera's HRS9531 Shows Strong Phase II Weight Loss, Pfizer Cautious on Danuglipron

4 months ago

• Kailera Therapeutics' HRS9531, a dual GLP-1/GIP receptor agonist, demonstrated a 21.1% placebo-adjusted weight reduction in Phase II trials over 36 weeks. • Pfizer CEO Albert Bourla expressed caution regarding their oral obesity candidate, danuglipron, despite plans for late-stage studies of the once-daily formulation. • Kailera reported that 59% of patients on HRS9531 achieved at least 20% body weight loss, with no plateau observed, suggesting potential for greater efficacy with longer use. • Pfizer abandoned a twice-daily danuglipron formulation due to toxicities, focusing on a once-daily version, but analysts remain divided on its prospects in the competitive obesity market.

Verve Therapeutics Advances Pipeline with Focus on Gene Editing for Cardiovascular Disease

4 months ago

• Verve Therapeutics anticipates initial data from the Heart-2 Phase 1b trial of VERVE-102, targeting PCSK9, in Q2 2025, including safety and efficacy data. • VERVE-301 has been nominated as the development candidate targeting the LPA gene, triggering a milestone payment from Eli Lilly, who will fund Phase 1 development. • The Pulse-1 Phase 1b trial for VERVE-201, which targets ANGPTL3, is progressing, with an update expected in the second half of 2025. • Verve's strong financial position, boosted by the Lilly milestone payment, extends the company's cash runway into mid-2027.

J&J's Nipocalimab Receives Priority Review for Generalized Myasthenia Gravis

4 months ago

• The FDA granted priority review to Johnson & Johnson's nipocalimab for treating generalized myasthenia gravis (gMG) in antibody-positive patients, based on Phase III Vivacity-MG3 study data. • J&J's posdinemab, a phosphorylated tau-directed monoclonal antibody, received Fast Track designation for early Alzheimer's disease, joining JNJ-2056 in Phase IIb studies. • Rybrevant plus Lazcluze demonstrated statistically significant improvement in overall survival versus osimertinib in EGFR-mutated non-small cell lung cancer (NSCLC).

Scholar Rock Submits Apitegromab BLA to FDA for Spinal Muscular Atrophy Treatment

4 months ago

• Scholar Rock has submitted a Biologics License Application (BLA) to the FDA for apitegromab to treat spinal muscular atrophy (SMA). • The BLA is supported by positive data from the Phase 3 SAPPHIRE trial, showing statistically significant motor function improvement. • Apitegromab has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA for SMA treatment. • Scholar Rock plans to initiate a Phase 2 trial (OPAL) in SMA patients under two years old in mid-2025.

Roche Partners with Dicerna for Hepatitis B Gene-Silencing Drug

5 months ago

Roche has entered into a $200 million upfront deal with Dicerna Pharmaceuticals for a gene-silencing drug aimed at treating hepatitis B virus (HBV) infections, with potential payments reaching up to $1.47 billion based on the drug's success. The drug, DCR-HBVS, currently in phase 1 testing, targets genes used by the virus to enter liver cells, offering hope for a functional cure by suppressing the virus below detectable levels.

FDA Approves BridgeBio's Attruby, Setting Up Competition with Pfizer's Vyndamax in ATTR-CM Market

6 months ago

• The FDA has approved BridgeBio Pharma's Attruby (acoramidis) for transthyretin amyloidosis cardiomyopathy (ATTR-CM), a heart condition leading to heart failure and death. • Attruby demonstrated a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalizations compared to placebo in the ATTRibute-CM trial. • BridgeBio is launching Attruby at a list price of $18,759 for a 28-day supply, positioning it to compete with Pfizer's Vyndamax, which dominates the ATTR-CM market. • Several other companies, including Alnylam and AstraZeneca/Ionis, are also developing therapies for ATTR, potentially disrupting the current treatment landscape.

Acoramidis Gains Global Momentum: FDA Approval, EU Recommendation, and Promising Clinical Data

6 months ago

• Acoramidis (Attruby), developed by Stanford Medicine and BridgeBio, receives FDA approval for transthyretin amyloid cardiomyopathy (ATTR-CM) treatment, marking a significant milestone. • The European Medicines Agency's CHMP recommends acoramidis for EU marketing authorization based on positive Phase 3 ATTRibute-CM trial results. • Clinical trials demonstrate acoramidis' efficacy in reducing cardiovascular-related hospitalizations and improving survival rates for ATTR-CM patients. • Bayer and BridgeBio collaborate to commercialize acoramidis, with Bayer holding EU rights and plans for a launch in Europe in early 2025.