Pfizer to ​Acquire Soleno therapeutics for $1.2 Billion, Bolstering Neurological⁣ Disease Pipeline

BOSTON – Pfizer announced today it will acquire Soleno⁣ Therapeutics, a biopharmaceutical ⁤company focused on developing therapies for ⁤rare neurological disorders, in ⁢an ‍all-cash transaction valued at $1.2 billion, or $14.50 per share. The deal, ⁢expected to close in the first quarter of 2024, considerably expands Pfizer’s pipeline in the often-overlooked ⁤field of central nervous system (CNS) diseases and ​provides access to Soleno’s lead ⁢investigational drug, dimebon, for the treatment of ⁤Duchenne ‍muscular dystrophy (DMD).

The acquisition arrives as ‌pharmaceutical companies increasingly target rare diseases with substantial unmet medical needs, offering both scientific challenges and potentially high returns. Dimebon, a novel, ⁣orally available small molecule, aims to restore dystrophin expression – a protein crucial for muscle function – in DMD patients irrespective of their‍ genetic mutation. Currently,⁣ treatment options for DMD are limited and‌ primarily focus on managing symptoms. This deal underscores pfizer’s commitment to innovation‍ in neurological conditions, building⁢ upon existing programs and potentially offering a disease-modifying therapy for a devastating condition affecting thousands⁤ of children and young ⁢adults globally.

Soleno’s dimebon ⁢is currently in a Phase 3 clinical trial, known as the DESTINY ‌trial, evaluating its efficacy in ⁤ambulatory DMD patients.Topline data ⁣from DESTINY⁣ are anticipated in the first half of 2024. Pfizer anticipates that, if⁤ approved, dimebon coudl address a notable unmet need for patients with DMD, regardless of ​their genetic ‍mutation.

“We are excited to welcome the talented team at soleno to Pfizer and to‍ advance dimebon’s development for Duchenne muscular dystrophy,” said Chris Boshoff, Pfizer’s⁣ Chief Development ⁢Officer, oncology and Rare Disease. “This‌ potential acquisition aligns with our strategic‌ focus on expanding our pipeline in​ areas with‌ significant ‍unmet medical need, and we believe dimebon has the potential to provide a meaningful new treatment option for patients with DMD.”

The transaction is subject to customary‌ closing conditions,‌ including ‌regulatory approvals and the completion of the DESTINY trial. Metsera,a subsidiary of enGene,is‍ providing⁤ manufacturing support ‌for ​dimebon. ⁣ Further details of the agreement can be found in Pfizer’s investor relations‌ release and the Readout Newsletter.

Biotech Sector⁤ Faces Scrutiny as FDA Controversy Deepens, Sarepta in Focus

Boston, MA ⁤ – The biotechnology industry is navigating a ⁤period of heightened scrutiny following recent⁢ controversies⁣ at the Food and Drug Management, even ​as major earnings reports ​roll in from pharmaceutical giants like Pfizer and ⁤upcoming ‍releases from⁣ Novo Nordisk. The unfolding situation is especially relevant for companies like sarepta Therapeutics,currently⁣ awaiting a critical decision on its Duchenne muscular dystrophy treatment,and is fueling broader concerns about the agency’s review processes.

The FDA’s credibility has​ been ⁣shaken​ by revelations of improper communications between ⁣agency officials and pharmaceutical executives, specifically⁤ involving Biogen’s Alzheimer’s drug, ⁤Aduhelm. This has‍ triggered investigations and calls for⁢ reform, casting a⁢ shadow over all drug⁢ approvals and prompting questions⁤ about potential bias in the evaluation ​of new therapies. The stakes are high, impacting investor confidence, patient access to possibly life-changing ‌treatments, and the future of drug development. The agency⁣ is now under pressure to demonstrate transparency and ⁣rigor in its decision-making,with the Sarepta case becoming a key test.

Sarepta’s Elevidys, a gene therapy for Duchenne ⁢muscular dystrophy, is currently under review with a PDUFA​ (Prescription ⁤Drug User Fee Act) date of May​ 26th. The controversy surrounding the‌ FDA’s⁢ accelerated approval ​of Aduhelm, and subsequent advisory​ commitee ‍reversal, has raised anxieties that Sarepta’s request‌ could face similar hurdles, despite promising early clinical data. Analysts are closely watching for any‌ indication of how the agency’s internal turmoil might influence ⁤its decision.

Beyond Sarepta,the fallout from⁤ the FDA’s​ issues is ⁣reverberating across the sector. ⁣ Hims & Hers Health, Inc. (Hims), a telehealth company offering prescription medications, is among those observing the‍ situation ‍closely, ⁣as regulatory changes ​could impact its business model. ⁢ The company recently reported first-quarter‍ results, highlighting continued growth​ in its subscription base, but also acknowledging the broader ‌uncertainties ⁣within the‌ pharmaceutical landscape.

Adding another layer to⁣ the industry’s‌ complexities, a bidding war for⁤ metsera, a company focused on cardiovascular disease, is‍ unfolding, with potential implications for consolidation within the space. The outcome of this competition ‍will be closely monitored alongside the FDA ‍developments.

STAT News continues to provide in-depth coverage ​of these critical issues, ​including exclusive reporting on the‌ FDA ‍controversies and their impact on‌ the‌ biotech industry.

Amgen’s Repatha Demonstrates Significant Heart Risk Reduction in Landmark Trial

A large-scale clinical‌ trial revealed that Amgen’s cholesterol-lowering injection,Repatha ⁢(evolocumab),significantly reduced the risk ‌of major cardiovascular events-including heart attack,stroke,and cardiovascular⁣ death-in a study of 12,000 patients. The findings, announced ⁤yesterday, offer a substantial boost to the prospects ​for PCSK9‍ inhibitors, ​a ⁣class of drugs that have faced hurdles ⁤in achieving widespread adoption despite their proven efficacy in lowering LDL cholesterol.

the positive results arrive at a‌ pivotal ⁤moment for cardiovascular health and pharmaceutical competition. While statins remain the first-line treatment⁣ for high cholesterol, Repatha’s⁢ demonstrated risk reduction provides a compelling option for​ patients unable to tolerate statins or who require additional cholesterol lowering. The trial’s success could reshape treatment paradigms and intensify⁣ the rivalry between Amgen and other⁤ pharmaceutical ​giants,‍ including Pfizer, in the competitive cholesterol management market.

The trial assessed a composite outcome of ⁣cardiovascular death, heart attack, and stroke. Detailed results are currently behind a paywall at STAT News,but Amgen’s announcement confirms​ a statistically significant benefit for patients ‌receiving Repatha. This outcome strengthens the argument for​ broader insurance ⁣coverage and increased utilization of PCSK9 inhibitors, ⁣which have historically been limited​ by high costs and restrictive access criteria.

Beyond cholesterol management, the pharmaceutical landscape continues to evolve.GSK recently released‌ it’s Readout Newsletter, providing updates on its research and development pipeline. Simultaneously, ongoing ⁤legal battles surrounding access to​ the abortion pill, mifepristone, continue to unfold, impacting reproductive healthcare access across the United States. These developments underscore⁣ the dynamic interplay between scientific advancement, ‌regulatory challenges, ​and ‌public health concerns within the pharmaceutical ‌industry.

FDA Halts Sarepta Gene Therapy, Leaving Duchenne Families in Uncertainty

Families of boys diagnosed with Duchenne muscular dystrophy are facing profound distress after the Food and Drug Governance (FDA) requested Sarepta Therapeutics to suspend shipments of it’s gene therapy, Elevidys. The agency cited safety concerns as the reason for this action, a request that Sarepta subsequently agreed to earlier this week. Parents who had invested notable effort over several months to gain access to the therapy are now in a state of limbo, with scheduled appointments abruptly canceled and limited dialog from regulatory bodies, according to reporting by STAT’s Jason Mast.

Duchenne muscular dystrophy is a rare,genetic disorder characterized by progressive muscle degeneration and weakness.Gene therapy offers a potential new avenue for treatment by aiming to correct or compensate for the genetic defect that causes the disease. Elevidys,developed by Sarepta Therapeutics,represents a significant advancement in this area,offering hope to families affected by this debilitating condition.

The FDA’s decision to halt shipments, even temporarily, raises critical questions about the safety profile and long-term efficacy of the gene therapy. While the agency’s primary mandate is to ensure the safety and effectiveness of medical treatments, such halts can have a devastating impact on patients and their families who are ofen facing a rapidly progressing disease with limited treatment options.

The lack of clear communication from regulators exacerbates the anxiety and uncertainty experienced by these families. The abrupt cancellation of appointments and the ensuing silence leave parents without crucial information regarding the future availability of the therapy and the specific safety concerns that led to the FDA’s intervention. This situation underscores the complex challenges in bringing novel and potentially life-changing therapies to market,notably in the realm of rare diseases.