Pfizer tightens DMD patient criteria after serious adverse events in phase 3 gene therapy trial
Pfizer is tightening up the criteria for Duchenne muscular dystrophy patients to partake in phase 3 clinical trials of its experimental gene therapy after three severe events were cropping up in ongoing studies.
The New York-based Big Pharma declared the news with the DMD community in a letter for the Parent Project Muscular Dystrophy patient advocacy group. Pfizer said three cases of muscle weakness, including inflammation of the heart tissue called myocarditis, were observed in patients after fordadistrogene movaparvovec treatment. The adverse events were attributed to the study therapy.
An external data evaluating committee for the trial later concluded that specific mutations were associated with a higher risk for severe events.
Pfizer will now amend the study protocol to eliminate DMD patients with specific gene mutations. The selected mutations are observed in about 15% of the Duchenne muscular dystrophy population.
DMD is a rare, incurable genetic disease caused by mutations in the DMD gene, which decrease the production of a protein called dystrophin, leading to progressive muscle degeneration.
The trial will continue, enrolling up to 99 patients who fit within the new protocol.
Right now, the trial is being conducted outside the U.S. as Pfizer waits for the FDA clearance to open up study sites here. Dosing at the non-U.S. sites commenced late last year, and according to a May update, Pfizer did not anticipate the trial to come stateside in the first half of this year at least.
Kytopen raises USD 30 Million in Series A Funding
Kytopen Corp., a biotechnology company, spun out of MIT offering a scalable technology for engineered cell therapies, declared it has raised USD 30 million in an oversubscribed Series A funding. The new capital will be utilized to commercialize Kytopen’s Flowfect® Tx for cGMP-compliant cell therapy manufacturing and move towards treating the first human patient with Flowfect® engineered cells. Kytopen aims to allow efficient and straightforward non-viral manufacturing of cell therapies in days versus weeks to increase patient access to life-saving therapies.
The Series A investor syndicate was led by the experienced life sciences investment team at Northpond Ventures, a leading science-driven venture capital firm, with participation from current investors: The Engine, Horizon Ventures, and Mass Ventures, as well as Aldevron Co-Founders Michael Chambers and John Ballantyne and Alexandria Venture Investments. Adam Wieschhaus, Ph.D., CFA, Director at Northpond Ventures, will join the Kytopen board of directors and Kytopen Co-Founders Paulo Garcia and Cullen Buie and Theresa Tribble, Venture Partner at The Engine.
The new funding will also speed up the commercialization of Kytopen’s high-throughput automated platform, which seamlessly links discovery to manufacturing and has the potential to reveal personalized medicines. The company’s proprietary Flowfect® platform is a transformative solution that eradicates the complexity of gene editing and integrates discovery, development, and manufacturing in one flexible and scalable non-viral delivery solution.
Kytopen’s proprietary Flowfect® technology accelerates therapies from discovery to the clinic by enabling cell engineering without compromising functionality or viability. Flowfect® decreases risk and provides maximum control and flexibility to decrease time to market by months or even years while providing safer and cost-efficient engineered cell therapies.
Eli Lilly taps Reify Health’s Care Access to diversify trials of Verzenio
Eli Lilly is once again linking arms with decentralized research organization Care Access. The Indianapolis Big Pharma worked with Reify Health’s Care Access on decentralized trials of its COVID-19 treatment bamlanivimab last year. Now, the two hope to extend a phase 3 trial of Lilly’s approved drug Verzenio in HR-positive and HER2-positive, high-risk, early breast cancer patients.
The trial, which commenced in May, will enroll about 2,450 patients taking hormone therapy after surgery, and the study is scheduled to finish primary work in May 2025.
Verzenio, or abemaciclib, snagged its first approval in September 2017 for use in patients with HR-positive, HER2-negative metastatic breast cancer who had previously been treated. In 2018, Verzenio use expanded into the first-line setting, or patients who had not received any prior treatment.
The prescribing information for the drug details the three main trials used to secure the approvals and show the therapy was evaluated mainly in white patients in those trials. One trial was 58% white and 30% Asian, another was 56% white, and the third was 85% white. Other races or ethnicities were not broken out.
Care Access said that the goal of the new partnership is to grow the trial participation of African American, Hispanic and Latinx patients. The company said that Care Access will work with local health systems, physician groups, diversity-focused organizations, advocacy partners, and community centers.
Med-tech investment firm Vensana Capital raises USD 325 Million in the second fund
Vensana Capital, a med-tech-focused investment firm, announced that it raised a second fund worth USD 325 million.
The Minneapolis and Washington, D.C.-based firm invests in med-tech companies in their development and commercial stages.
Since closing its first fund of USD 225 million in 2019, Vensana has made ten investments, comprising Wayne, Pennsylvania-based Intact Vascular, which Phillips took over, and Minneapolis-based CVRx, which completed an IPO in June.
The firm recently hired Venture Partner Mike Kramer, Principal Amrinder Singh, and Chief Financial Officer Steve Schwen. Kramer joined from healthcare investment firm CRG, while Singh moved from Medtronic Ventures.
Managing Partner Kirk Nielsen said that they are proud of the progress that Vensana has made since its launch, especially their growing investment team and their portfolio of companies tackling vital unmet needs. They are grateful for the continued strong support of their limited partners. Their entire team is excited about the opportunity in front of them as they work hard to build a leading MedTech investment and company-building platform.