View all newsletters
Receive our newsletter - data, insights and analysis delivered to you
  1. Comment
January 24, 2022

Pfizer DMD fatality may clear the path for Sarepta’s gene therapy

The death of a participant in Pfizer's Phase lb study on PF-06939926 creates uncertainty around its future and may help boost Sarepta's gene therapy.

By GlobalData Healthcare

On December 20, 2021, Pfizer announced the unexpected death of a participant in the company’s Phase Ib open-label study (NCT03362502) on PF-06939926 (fordadistrogene movaparvovec), a gene therapy aiming to treat Duchenne muscular dystrophy (DMD). The FDA issued a clinical pause on the trial and Pfizer is in the process of investigating the causes of the death. The patient was part of the non-ambulatory arm of the study. Pfizer also has an active Phase III study (NCT04281485) of the same product, currently being developed globally. PF-06939926 is one of two gene therapies in late-stage development for DMD, with its main rival being Sarepta Therapeutics’s SRP-9001. The uncertainty surrounding PF-06939926’s future following this major safety event could potentially clear the way for Sarepta’s continued dominance in the field.

Free Case Study
img

Direct-to-Patient Trials: How IRT Plays an Important Role in Bellerophon's Direct-to-Patient Trials

As the industry strengthens its focus on patient centricity, Direct-to-Patient clinical trials have emerged as a popular trial design that have the potential to increase patient recruitment and retention. IRT plays a crucial role in the success of a Direct-to-Patient trial. Because drug supplies are being managed and shipped from distribution facilities directly to patients’ homes, a sponsor must have a high-quality system in place to accurately track the chain of custody, ensure patient-blinding and handle other logistical challenges. What You Will Learn Benefits and challenges associated with the Direct-to-Patient model Bellerophon's top considerations when implementing this trial design How IRT can equip study teams to successfully track chain of custody, ensure patient blinding, and handle logistical challenges
by Suvoda
Enter your details here to receive your free Case Study.

Despite both therapies having the same mechanism of action, the Pfizer and Sarepta products differ in composition. PF-06939926 delivers a mini-dystrophin encoding gene to patients, enabling them to produce mini-dystrophin, a shorter but still functional version of the protein dystrophin missing in DMD patients’ muscles. Pfizer’s gene therapy has an adeno-associated virus 9 (AAV-9) vector, while Sarepta’s candidate contains an AAVrh74 vector. A group of key opinion leaders (KOLs) interviewed by GlobalData were slightly in favour of Sarepta’s product, as they believed that Pfizer’s vector might more easily induce immune responses among patients, in addition to a group of certain mutation types having been excluded from Pfizer’s trial due to heart-related side effects. However, Pfizer’s AAV-9 vector is able to carry longer genes compared to Sarepta’s AAVrh74, which explains why PF-06939926 and SRP-9001 deliver mini-dystrophin and micro-dystrophin encoding genes, respectively, leading the rest of the KOLs to favour Pfizer’s product. Physicians are waiting for further efficacy and safety data to decide which product they would prefer to administer to their patients.

Gene therapies are the most anticipated pipeline candidates for the DMD space by patients, influential advocacy groups, and physicians. The latest development on Pfizer’s candidate, along with past efficacy and safety setbacks on both PF-06939926 and SRP-9001, might increase scrutiny by the FDA. This would likely delay its market launch until after 2025. Sarepta is one of the leading players in this space, with three out of five marketed products targeting DMD in the US market. Sarepta is also conducting trials on late-stage gene therapy. Currently, in Phase III (NCT05096221) of development, SRP-9001 (delandistrogene moxeparvovec) could see a potential advantage after PF-06939926’s latest development. Additionally, Sarepta holds three more pipeline drugs in the earlier stages of development, two of which are gene therapies. In contrast to Sarepta, Pfizer lacks further candidates that could penetrate this space and gain market share if its gene therapy fails to receive approval, which indicates the higher stakes for the latter. Even if both gene therapies make it to market, PF-06939926 would likely see a delay because of the recent death during its Phase Ib trial. This would provide SRP-9001 with a first-to-market advantage. Regardless, Pfizer will need to take the time to analyse the situation and gather insight that will allow for the continuation of its Phase Ib trial and modifications to future trials such as the exclusion of specific mutation types. Despite the risks above leading to a potentially lower uptake compared to Sarepta’s product, Pfizer could still capture a considerable market share and see a return on its investment before more gene therapies enter the market.

Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

Free Case Study
img

Direct-to-Patient Trials: How IRT Plays an Important Role in Bellerophon's Direct-to-Patient Trials

As the industry strengthens its focus on patient centricity, Direct-to-Patient clinical trials have emerged as a popular trial design that have the potential to increase patient recruitment and retention. IRT plays a crucial role in the success of a Direct-to-Patient trial. Because drug supplies are being managed and shipped from distribution facilities directly to patients’ homes, a sponsor must have a high-quality system in place to accurately track the chain of custody, ensure patient-blinding and handle other logistical challenges. What You Will Learn Benefits and challenges associated with the Direct-to-Patient model Bellerophon's top considerations when implementing this trial design How IRT can equip study teams to successfully track chain of custody, ensure patient blinding, and handle logistical challenges
by Suvoda
Enter your details here to receive your free Case Study.

Related Report
img
Healthcare
NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy
SUBSCRIBED

THANK YOU

Thank you for subscribing to Clinical Trials Arena