Pfizer, Beam sign multi-target research collaboration for rare diseases

The collaboration will see the base editing programmes to be evaluated leverage Beam’s in vivo delivery technologies.

These technologies use messenger RNA (mRNA) and lipid nanoparticles (LNP) to deliver base editors to target organs

This collaboration aims to advance potentially transformative therapies for patients with rare diseases.

Beam’s base editing technologies are intended to allow a precision genetic medicines that target only a single base in the genome without actually causing a double-stranded break in the DNA.

This mechanism aims to offer a more accurate and efficient edit as against traditional gene editing methods that operate by creating targeted double-stranded breaks in the DNA. These breaks in the DNA come with potential challenges linked with unwanted DNA modifications.

Pfizer chief scientific officer and president of Worldwide Research, Development and Medical Mikael Dolsten said: “At Pfizer, we believe in the powerful potential of mRNA and LNP technologies to address the greatest unmet needs for patients, as evidenced by the beneficial impact our mRNA/LNP-based COVID-19 vaccine is having on the pandemic.

“We have a strong history in developing gene replacement therapies for rare diseases, and we see this collaboration with Beam as an opportunity to advance the next generation of gene editing therapies – an exciting scientific frontier – potentially leading to transformation for people living with rare genetic diseases.”

Beam CEO John Evans said: “We are thrilled to partner with Pfizer, a global leader in the design, development, and commercialization of novel medicines. Our leading platform for precision genetic medicine has greatly evolved over the last few years, and we are committed to ensuring the broadest reach of these potentially life-changing technologies.

“This collaboration will provide a unique opportunity to create potentially transformative base editing programs for indications with critical unmet needs, leveraging our proprietary base editing technology and expanding delivery capabilities. We look forward to working together with Pfizer to advance these technologies and potentially expand our impact for people suffering from serious diseases.”

As per the terms of the collaboration deal, Beam will undertake all research activities through development candidate selection for three undisclosed targets that are not part of Beam’s existing list of programmes.

Pfizer may choose to exclusive, worldwide licenses to each development candidate, following which it will be responsible to undertake all development activities besides seeking potential regulatory approvals and commercialisation, for each such candidate.

This agreement also gives Beam the right to opt in, at the end of Phase 1/2 studies, following the payment of an option exercise fee, to a global co-development and co-commercialisation agreement with regard to one programme licensed under the deal.

Following this deal, Pfizer and Beam would share net profits as well as development and commercialisation costs in a 65%/35% ratio, respectively.

Beam will secure an upfront payment of $300m. If Pfizer chooses to exercise its opt-in license rights for all three targets, Beam is eligible to receive development, regulatory and commercial milestone payments for potential total deal consideration of up to $1.35bn.

Beam is also eligible to get royalties on global net sales for each licensed programme.

This collaboration has an initial term of four years and may be extended up to one more year.