Full Press Release Details
Verastem Oncology Announces Strategic Transition
Plan to Accelerate Next Phase of Growth
John Johnson, current board member, appointed
to chairman of the board
Michael Kauffman, MD, PhD, lead director since
2016, appointed to president of development
Commercial launch progresses as RAMP 301 Phase
3 confirmatory trial in recurrent LGSOC completes additional patient enrollment; topline data anticipated in mid-2027
BOSTON--(BUSINESS WIRE)-Dec. 15, 2025-- Verastem Oncology (Nasdaq:
VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced
strategic leadership changes to accelerate its next phase of growth. Michael Kauffman, M.D., Ph.D., currently lead director of the Board,
has been appointed as the president of development and will join the Company's executive leadership team, while John Johnson, a
board member since 2020, has been appointed as chairman of the board of directors succeeding Dr. Kauffman. Dr. Kauffman will remain on
the Board but will no longer serve as lead director, or on the audit committee, or compensation committee of the Board upon transition
to his new role. As part of these changes, Matthew Ros, chief operating officer, will be departing from the organization as the Company
streamlines its operational structure and transitions his responsibilities across the executive team.
"2025 has been a year of significant accomplishments where we
advanced key clinical trials and launched an important new treatment for people living with a specific type of LGSOC, a rare ovarian cancer
that is persistent and highly recurrent. We expect to enter 2026 from a position of strength and in that regard, I'm pleased to
announce that John will assume the position of Chairman of the Board and bring his decades of corporate strategy and oncology commercialization
to the role," said Dan Paterson, president and chief executive officer of Verastem Oncology. "The appointment of Michael as
President of Development not only brings in a depth of experience in advancing novel agents from early development all the way through
successful commercial launch, but also underscores our commitment to our R&D program and specifically the importance of VS-7375, and
the anticipated positive impact we believe this potential best-in-class treatment may have on patients around the world."
Mr. Paterson added, "We thank Matt for his contributions to the
initial success of the commercial launch of AVMAPKI FAKZYNJA CO-PACK and establishing a strong organizational foundation this year."
"I am honored to continue the leadership Michael has established
over the past decade as Lead Director. Verastem is at a pivotal moment with the initial successful launch of AVMAPKI FAKZYNJA CO-PACK,
which has provided a benefit to women where previously there were no FDA-approved treatments specifically for their disease. I look forward
to working closely with Dan, Michael, and the rest of the Board to support the Company's commercial and clinical development plans,"
said John Johnson, chairman of the board.
"After more than a decade on the Board, I am thrilled to join
the executive team and dedicate myself full time to what I believe is a once-in-a-lifetime opportunity with VS-7375," said Michael
Kauffman, M.D., Ph.D. "This potential best-in-class KRAS G12D dual ON/OFF inhibitor could transform outcomes for patients with currently
limited options, and I am excited to bring my scientific expertise and proven track record of successful drug development and commercialization
to the organization at this critical time."
The Company also announced today that it has completed the additional
patient enrollment for RAMP 301, its international Phase 3 confirmatory trial in recurrent LGSOC. Following a pre-planned interim analysis
(IA), the Independent Data Monitoring Committee recommended a modest one-time increase of 29 patients across KRAS mutation status, based
on the total enrollment achieved in October. The Company remains blinded to the IA results.
RAMP 301 is evaluating the combination of avutometinib plus defactinib
versus standard chemotherapy for patients with recurrent LGSOC with and without a KRAS mutation. The trial will serve as a confirmatory
study for the initial indication and has the potential to expand the indication regardless of KRAS mutation status. The Company expects
to report a topline read-out of the primary endpoint in mid-2027.
Biographies for John Johnson and Michael Kauffman, M.D., Ph.D.
John Johnson is a recognized biopharma executive leader in the industry
with more than three decades of experience across corporate strategy, operations, investing, clinical development, and oncology drug commercialization.
He most recently served as Executive Chairman at Applied Therapeutics, a company focused on developing transformative treatments for rare
disease. Mr. Johnson also previously was the Chief Executive Officer of Reaction Biology, a global Contract Research Organization. Prior
to that he was the Chief Executive Officer of Stonebridge Biopharma prior to its merger with Xeris Biopharma. Mr. Johnson has held executive
management roles at leading global corporations, including Johnson & Johnson, where he spent the majority of his career and served
as the Company Group Chairman of Biopharmaceuticals within Johnson & Johnson. He was responsible for Johnson & Johnson Biotechnology,
Immunology, and Oncology commercial businesses. Mr. Johnson also served as President of Eli Lilly & Company's Worldwide Oncology
unit, following the company's 2008 acquisition of Imclone Systems, Inc., where he served as Chief Executive Officer and a member
of Imclone's Board of Directors.
Mr. Johnson has served on 19 boards and presently serves on the boards
of Reaction Biology, Axogen (AXGN), Xeris Pharmaceuticals (XERS), and Verastem Oncology (VSTM). He served on two private equity backed
company boards through successful exits. He has also served as a member of the board of directors of Pharmaceutical Research and Manufacturers
of America (PhRMA) and as a member of the Health Section Governing Board of Biotechnology Industry Organization (BIO).
Michael Kauffman, M.D., Ph.D.
As a Lead Director of Verastem's Board of Directors since June
2016, Dr. Kauffman has a deep understanding of the Company's strategy, clinical development plans, and operations. Previously,
Dr. Kauffman served as the Chief Executive Officer of Nereid Therapeutics. He was co-founder and Chief Executive Officer of Karyopharm,
where he guided that company's transition from a discovery stage biotechnology company to a commercial stage organization and the
global approvals of XPOVIO . Prior to joining Karyopharm, Dr. Kauffman was Chief Medical Officer of Onyx Pharma, where
he led the development of Kyprolis following the Onyx acquisition of Proteolix , where he served as board member and
then Chief Medical Officer. Previously, Dr. Kauffman was President and Chief Executive Officer of EPIX Pharmaceuticals (previously Predix
Pharmaceuticals.). Before that, he was the leader of the Velcade development program at Millennium Pharmaceuticals. He
also held a number of senior positions at Millennium Predictive Medicine and Biogen.
Dr. Kauffman received his M.D. and Ph.D. from Johns Hopkins Medical
School, trained in Internal Medicine at Beth Israel (Deaconess) Medical Center and in Rheumatology at Massachusetts General Hospital,
and is board certified in Internal Medicine.
About AVMAPKI and FAKZYNJA Combination Therapy
AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking
the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking
RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib
and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance
of RAS/MAPK pathway-dependent tumors.
The U.S. Food and Drug Administration (FDA) approved AVMAPKI
FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC
who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification
and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK)
(NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy
or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is
also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first line for patients
with advanced pancreatic cancer (RAMP 205; NCT05669482) and advanced KRAS G12C mutant non-small cell lung cancer (RAMP 203; NCT05074810).
Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies,
for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on
a stand-alone basis for any use.
AVMAPKI FAKZYNJA CO-PACK U.S. Indication
AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients
with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.
This indication is approved under accelerated approval based on tumor
response rate and duration of response. Continued approval for this indication may be contingent upon verification and description
of clinical benefit in a confirmatory trial.
Important Safety Information
Warnings and Precautions
The most common ( 25%) adverse reactions, including laboratory
abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal
pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased