Beam partnership up to $ 1.35 billion; Alliances with Acuity, Codex DNA and BioNTech

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Pfizer has signaled its intention to expand its already large presence in messenger RNA (mRNA) by launching collaborations with four companies to develop more therapies and vaccines based on this technology.

The four collaborations include a partnership of up to $ 1.35 billion with core edition developer Beam Therapeutics, alliances with Codex DNA and Acuitas Therapeutics, all announced on Monday; and a third mRNA vaccine partnership with BioNTech, its partner in the development of the successful COVID-19 vaccine COMIRNATY®, announced last week.

Pfizer and Beam have entered into a four-year research collaboration, with a possible one-year extension, designed to apply Beam’s in vivo delivery technologies to core editing programs to develop three undisclosed targets for rare genetic diseases of the liver, muscles and central nervous system. system. The technologies are intended to deliver basic editors to target organs using mRNA and lipid nanoparticles (LNP).

“At Pfizer, we believe in the powerful potential of mRNA and LNP technologies to address the greater unmet patient needs, as evidenced by the beneficial impact of our mRNA / LNP-based COVID-19 vaccine on the pandemic, ”Mikael Dolsten, MD, PhD, CSO of Pfizer and president of global research, development and medicine, said in a statement.

“We have a strong history in the development of 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 – with the potential to lead to transformation for people living with rare genetic diseases. diseases, ”Dolsten added.

Under the terms of their collaboration agreement, Beam will conduct all research activities through the selection of development candidates for the three targets, which are not included in Beam’s existing programs.

Pfizer maintains an exclusive global licensing option for each development candidate. For each option Pfizer exercises, it will oversee all development activities, as well as potential regulatory approvals and commercialization, for that program.

Upon completion of Phase I / II studies, by paying an unspecified option exercise fee, Beam has the right to opt into a global co-development and co-commercialization agreement focused on a licensed program. as part of the collaboration. Under the deal, Pfizer would assume 65% of development and marketing costs and receive 65% of net profits, with the remaining 35% going to Beam.

“This collaboration will provide a unique opportunity to create potentially transformative core editing programs for indications with critical unmet needs, leveraging our proprietary core editing technology and expanding delivery capabilities,” said said Beam CEO John Evans. “We look forward to working with Pfizer to advance these technologies and potentially extend our impact for people with serious illnesses. “

$ 300 million in advance

Pfizer agreed to pay Beam $ 300 million upfront. If Pfizer exercised its opt-in license rights for all three targets, the pharmaceutical giant would pay Beam up to $ 1.05 billion in payments related to the achievement of development, regulatory and commercial milestones. Beam is also eligible to receive worldwide net sales royalties for each licensed program.

“Our first impression is that the terms of the collaboration are favorable for Beam,” wrote today Rick Bienkowski, PhD, vice president of equity research at SVB Leerink focused on genetic drugs, in a research note. . “The membership of a large pharmaceutical partner reflects positively on Beam’s core publishing technology, and the large upfront cash payment will strengthen Beam’s balance sheet with non-dilutive capital (to approximately $ 1.2 billion pro forma cash and cash equivalents) while potential milestones and royalties can add longer-term value.

“We believe this collaboration supports our foundational thesis that Beam’s core editing technology has enormous scarcity value and will continue to enable partnerships on business-friendly terms,” added Bienkowski.

Jefferies analyst Michael Yee and four colleagues observed in a research note that Pfizer became the first “big pharmaceutical company” to partner with a drug developer using a genome editing approach.

“We understand [Pfizer] came directly to Beam and started discussions to partner with unique base pair editing programs, “wrote Yee and colleagues, adding that the Pfizer collaboration” adds early validation to preclinical data, to the technology and the potential of Beam over the next few years ”.

They noted that the partnership can build on strengths, including:

  • Pfizer’s expertise in in vivo delivery through its work on the COVID-19 vaccine and significant manufacturing capacity for LNP
  • Beam’s cash balance, which, thanks to the collaboration with Pfizer, has grown to approximately $ 1.2 billion.
  • Beam’s proprietary LNP and new LNPs acquired with its $ 440 million purchase of Guide Therapeutics last year.

“Beam is working on novel tissue-targeted LNPs for the CNS and muscle and can leverage investments from the [Pfizer] working in more programs Beam is working on the descent, ”wrote Yee and his colleagues.

Investors, however, seemed less enticed by collaborations with Pfizer, as the company’s stock price rose about 1% on Monday, to $ 56.24 from Friday’s close of $ 55.72. . Beam shares fell 2.7% to $ 68.46 from $ 70.36 on Friday.

Beam was founded in 2017 by Harvard University chemist David Liu, PhD, who developed the underlying technology behind Core Editing with research conducted by post-docs. Alexis Komor, PhD, and Nicole Gaudelli, PhD.

Along with his friends and colleagues Feng Zhang, PhD, and Keith Joung, MD, Liu co-founded Beam Therapeutics to market the Core Edition. Beam went public in 2020 through an initial public offering that ultimately raised $ 188.3 million in net proceeds.

One and done approach

Speaking on GEN Close to the Edge “series of video interviews last summer, Evans noted that the basic editing’s potential lay in its” one-and-done “approach to precision medicine. Last year, the company published promising results in CRISPR review on a redesigned family of base editors to modify the single mutated base in sickle cell disease.

Beam Therapeutics is one of three companies whose partnerships Pfizer announced on Monday, coinciding with the start of the JP Morgan 40th Healthcare Conference.

Pfizer has also agreed to access Codex DNA’s new enzymatic DNA synthesis (EDS) technology and further develop it in its mRNA-based vaccines and other biopharmaceuticals. As part of a strategic collaboration and licensing agreement, Pfizer agreed to make an undisclosed upfront payment to Codex DNA, as well as potentially more than $ 100 million in payments related to the completion of short technical milestones. term ”.

By partnering with Codex DNA, Pfizer believes that Codex technology can reduce production time from weeks to days by eliminating several initial steps in the mRNA production process, including going from DNA assembly biological to a synthetic DNA assembly.

San Diego-based Codex DNA is a synthetic biology company that went public in June 2021, raising net proceeds of $ 112.5 million from its IPO. The company’s products aim to enable researchers to quickly, accurately and reproducibly construct or “write” high-quality synthetic DNA and mRNA, ready for use in numerous downstream markets. . Codex DNA markets the BioXp ™ System, a fully automated end-to-end automated workstation designed to provide an end-to-end turnkey solution to generate synthetic DNA and mRNA from DNA sequence.

Last year, Codex DNA was among the “Up & Comers” highlighted in GEN A list of the top 10 synthetic biology companies.

Pfizer has also entered into a development and option agreement with Acuitas Therapeutics granting the pharmaceutical giant an option to license, on a non-exclusive basis, Acuitas’ LNP technology for up to 10 targets for vaccine or therapeutic development. . The value of the deal and other financial terms were not disclosed.

Overcome two challenges

Through their collaboration, Pfizer aims to overcome two challenges related to the use of mRNA in drugs and vaccines. One is the difficulty for mRNA to cross the cell wall. Another is the vulnerability of “naked” mRNA to degradation or severe attack by the immune system before reaching the desired cells. Pfizer and Acuitas claim that modified LNPs allow more efficient intracellular delivery of mRNA vaccines and drugs into the cytoplasm.

Acuitas, based in Vancouver, British Columbia, is developing LNPs designed to protect mRNA and transport it into patients’ cells.

The collaborations with Beam, as well as the alliances with Codex DNA and Acuitas, come about a year after Pfizer joined BioNTech to launch COMIRNATY (also known as BNT162b2), which uses Acuitas’ LNP technology.

COMIRNATY generated combined revenue of $ 24.277 billion for Pfizer in direct sales and revenue from its alliance with BioNTech in the first three quarters of 2021. BioNTech reported an additional € 13.303 billion ($ 15.067 billion ) revenue from COVID-19 vaccines.

Pfizer has forecast COMIRNATY’s revenue to be approximately $ 36 billion in 2021 and approximately $ 29 billion this year. BioNTech has forecasted between 16 and 17 billion euros in revenue this year (18 to 19 billion dollars).

Last week, Pfizer and BioNTech signed a third collaboration on a mRNA vaccine by agreeing to accelerate the development of a first mRNA-based vaccine for the prevention of shingles (herpes zoster virus, or HZV) —Two years after joining forces to develop COMIRNATY and four years after partnering on an mRNA influenza vaccine.

The companies have agreed to apply proprietary antigen technology identified by scientists at Pfizer and BioNTech’s proprietary mRNA platform technology used in the companies’ COVID-19 vaccine. Pfizer and BioNTech plan to launch clinical trials in the second half of this year and have agreed to share the development costs, as well as the gross profits from commercialization.

Pfizer agreed to pay BioNTech $ 225 million upfront, including $ 75 million in cash and a $ 150 million equity investment, while BioNTech agreed to pay Pfizer $ 25 million for the use. of its exclusive antigenic technology. BioNTech is eligible to receive up to $ 200 million in regulatory and commercial milestone payments.

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