Apellis and Beam Therapeutics Enter Exclusive Research Collaboration to Apply Base Editing to Discover Novel Therapies for Complement-Driven Diseases

Rhea-AI Summary

Apellis Pharmaceuticals (Nasdaq: APLS) and Beam Therapeutics (Nasdaq: BEAM) announced a five-year exclusive collaboration focused on base editing technology to develop treatments for complement-driven diseases. The partnership will target six research programs directed at the eye, liver, and brain, utilizing Beam's technology for precision gene editing. Apellis will lead the development of licensed programs and has committed $75 million, with further milestone payments possible. This strategic alliance aims to enhance Apellis' leadership in complement therapeutics and expand treatment options for patients.

Positive

• Exclusive five-year collaboration with Beam Therapeutics to develop new treatments.
• Focus on six research programs targeting complement-driven diseases in key organs.
• Investment of $75 million from Apellis, enhancing its financial commitment toward innovation.

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• None.

Collaboration combines Apellis’ expertise in complement, a complex biological system, with Beam’s proprietary base editing platform

Companies will collaborate on six research programs directed to tissues modulated by the complement system, including the eye, liver, and brain

WALTHAM, Mass. and CAMBRIDGE, Mass., June 30, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Beam Therapeutics Inc. (Nasdaq: BEAM) today announced an exclusive five-year research collaboration focused on the use of Beam’s proprietary base editing technology to discover new treatments for complement-driven diseases. The companies will collaborate on six research programs focused on C3 and other complement targets in the eye, liver, and brain.

“Beam has pioneered base editing, which holds significant promise as a best-in-class technology for precision gene editing. This collaboration builds on our deep scientific expertise in complement and, together with our growing pipeline, positions Apellis for long-term leadership in the complement field,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. “Apellis and Beam share a vision for advancing transformative medicines for patients, which is critically important as we embark on a highly innovative effort to modulate complement and discover new treatments across a wide range of debilitating diseases.”

Base editing represents a potential new class of precision genetic medicine that uses a chemical reaction designed to create precise, predictable, and efficient single base changes at targeted genomic sequences without making double-stranded breaks in the DNA. Editing key elements of the complement pathway in target organs has the potential to alter the complement cascade and durably address diseases driven by abnormal complement activity.

“Apellis has established itself as a leader in complement with the advancement of compelling targeted C3 therapies,” said John Evans, chief executive officer, Beam. “This collaboration allows us to combine our proprietary technologies and capabilities in base editing with Apellis’ expertise in targeting the complement pathway to develop new medicines for diseases driven by complement biology. This also represents an important strategic initiative to explore opportunities that expand the application of base editing to address more biologically complex diseases for patients in need of new treatment options.”

Under the terms of the collaboration agreement, Beam will apply its base editing technology and conduct preclinical research on up to six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis will have exclusive rights to license each of the six programs and will assume responsibility for subsequent development. Beam may elect to enter a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to one program licensed under the collaboration.

As part of the collaboration, Beam will receive a total of $75 million in upfront and near-term milestones from Apellis – $50 million upon signing and an additional $25 million payment on the one-year anniversary of the contract execution date. After exercise of the opt-in license rights for each of the up to six programs, Beam will be eligible to receive development, regulatory, and sales milestones from Apellis, as well as royalty payments on sales. The collaboration has an initial term of five years and may be extended up to two years on a per year and program-by-program basis.

About Apellis 
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit https://www.apellis.com.

About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that enables precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.

Apellis Forward-Looking Statement 
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements in respect of the expected closing of the exchanges. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether the research collaboration will result in programs that are licensed by Apellis; whether any product candidates that arise from these programs or are otherwise developed by Apellis will advance into clinical trials or through the clinical trial process on a timely basis or at all; whether the results of clinical trials of the company’s product candidates will warrant submissions for regulatory approval or regulatory approval; whether any products that receive regulatory approval will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q with the Securities and Exchange Commission on April 28, 2021 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Beam Forward-Looking Statement 
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the therapeutic applications and potential of our technology, including our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our platform technology; the potential impact of the COVID-19 pandemic; risks related to competitive products; and the other risks and uncertainties identified under the heading “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2020, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

Contacts:

Apellis:

Media:
Lissa Pavluk
media@apellis.com
617.977.6764

Investors: 
Argot Partners 
apellis@argotpartners.com
212.600.1902

Beam Therapeutics:

Media:
Dan Budwick
1AB
dan@1abmedia.com

Investors:
Chelcie Lister
THRUST Strategic Communications
chelcie@thrustsc.com

FAQ

What is the collaboration between Apellis and Beam about?

Apellis and Beam are collaborating on a five-year research program focused on using Beam's base editing technology to develop treatments for complement-driven diseases.

How much is Apellis investing in the collaboration with Beam?

Apellis is investing a total of $75 million in the collaboration, with $50 million paid upon signing and an additional $25 million after one year.

What organs are targeted in the Apellis and Beam collaboration?

The collaboration targets the eye, liver, and brain for complement-driven diseases.

What is the duration of the collaboration between APLS and BEAM?

The collaboration lasts for five years, with the possibility of extending up to two additional years.

What potential outcomes does the collaboration aim for?

The collaboration aims to discover new treatments targeting complement pathways, with an emphasis on precision genetic medicine.