- First clinical program focuses on
fibrodysplasia ossificans progressiva (FOP), a rare genetic
disorder causing extra-skeletal bone formation -
- Experienced leadership team poised to
accelerate pipeline development with lead asset based on novel
research -
āshibio, a privately held biotechnology company developing novel
therapeutics for the treatment of bone and connective tissue
disorders, exited stealth mode today with $40 million in seed and
Series A financing. The company was founded in 2022 by Chief
Executive Officer Pankaj Bhargava, M.D., and the team at MPM
BioImpact, where Dr. Bhargava is also an entrepreneur partner. MPM
BioImpact led the Series A round, with contributing funds from
Agent Capital, YK Bioventures, and Mirae Asset Venture Investment.
In conjunction with the financing, Agent Capital Partner and
Co-Founder Preston Noon has joined the Board of Directors.
The funding will allow āshibio to continue to advance
development of several investigative therapies including a
potential treatment for fibrodysplasia ossificans progressiva
(FOP), a rare genetic disease characterized by severe and
progressive heterotopic ossification (HO), a pathological condition
that causes abnormal bone formation in muscles, tendons, ligaments
and other soft tissues. FOP typically starts in early childhood, is
characterized by episodic and painful flare-ups, and leads to
severe debilitation and shortened life span.
“We are building a pipeline of innovative therapeutics that
leverage emerging biology and novel mechanisms to impact bone and
connective tissue disorders that have no approved treatments or
where current treatment options are inadequate,” said Dr. Bhargava.
“We are grateful to our investors for recognizing the promise of
our approach and for supporting our development programs.”
The company’s lead program is based on the discovery that the
matrix metalloproteinase-9 (MMP-9) enzyme could serve as a novel
target for FOP, as published in Journal of Bone and Mineral
Research in February 2024.
The research described the case of a unique 35-year-old patient
who carries the classic genetic mutation of FOP (ACVR1 R206H,
present in over 95% of patients), yet has extreme lack of
heterotopic ossification (HO) and near normal mobility. The authors
found that the patient has an MMP-9 mutation in addition to the FOP
mutation that appears to protect him from flare-ups and the
abnormal bone formation typically seen in individuals with FOP.
Further studies with MMP-9 gene knockout and pharmacological
experiments confirmed that MMP-9 may serve as a novel target in FOP
and other more common forms of HO.
āshibio licensed andecaliximab, a humanized antibody that
specifically inhibits MMP-9, from Gilead Sciences, Inc.
Andecaliximab has been administered to approximately 1,000 patients
in previous clinical trials.
āshibio plans to initiate a Phase 2/3 trial of andecaliximab in
the second half of 2024 in patients with FOP. The U.S. Food and
Drug Administration (FDA) designated andecaliximab an Orphan Drug
for the treatment of FOP in March 2024, one month after the
European Medicines Agency (EMA) issued its own Orphan Drug
Designation for the therapy. The FDA also cleared āshibio's
Investigational New Drug (IND) application for andecaliximab in
March 2024.
Experienced senior leadership team announced Prior to
joining MPM BioImpact, Dr. Bhargava was the Oncology Therapeutic
Area Head at Gilead Sciences. He has also held executive positions
at multiple biotech companies during his career, including Sanofi
and Dicerna Pharmaceuticals, where he led development programs for
oncology and rare genetic diseases.
In addition to completion of the Series A funding, āshibio also
announced the following appointments to its senior leadership
team:
- Victoria Smith, Ph.D., joins āshibio as Chief Scientific
Officer. She has worked across several therapeutic areas including
oncology, and autoimmune and inflammatory diseases. She has
previously held executive or senior positions at Amphivena
Therapeutics, Gilead Sciences, and Arresto Biosciences, where she
was a co-inventor of andecaliximab.
- Deborah Wenkert, M.D., joins āshibio as Chief Medical Officer.
She is an experienced pediatric rheumatologist, specializing in
rare bone and connective tissue disorders, with extensive
experience in both academic and industry settings. She has
previously held executive or clinical development positions at
Inozyme Pharma, PreciThera, and Amgen.
“At āshibio, we are rapidly advancing novel therapeutics for
severe and debilitating bone and connective tissue disorders, an
area of great clinical need,” commented Dr. Smith. “It is extremely
gratifying to be a part of such an impressive team of seasoned
industry executives, clinicians, and researchers who are devoted to
improving the lives of patients who have not been adequately served
by currently available treatment options.”
“āshibio is clearly at the forefront of drug development in
heterotopic ossification and other bone and connective tissue
pathologies,” added Todd Foley, managing partner at MPM BioImpact
and Chair of the āshibio Board of Directors. “The company is now on
a clinical path for its lead asset and is poised to advance a
pipeline of programs that address unmet needs.”
āshibio researchers will present an update on the andecaliximab
FOP program in an oral and poster presentation at the FOP Drug
Development Forum in Stockholm, Sweden on June 27-28, 2024. Company
leadership will also present a poster at the International
Conference on Children’s Bone Health in Salzburg, Austria from June
22-25, 2024.
About āshibio āshibio is a privately held biotechnology
company developing a pipeline of novel therapeutics for the
treatment of bone and connective tissue disorders. Founded in 2022
by company CEO Pankaj Bhargava, M.D., and the team at MPM
BioImpact, āshibio exited stealth mode in June 2024 with $40
million in Seed and Series A financing. The company plans to
initiate a Phase 2/3 trial of its lead asset, andecaliximab, in the
second half of 2024 in patients with fibrodysplasia ossificans
progressiva (FOP), a rare genetic disorder characterized by
progressive heterotopic ossification (HO), a pathological condition
characterized by abnormal bone formation in muscle and soft
tissues. For more information, visit www.ashibio.com.
About MPM BioImpact MPM BioImpact is a world-leading
biotechnology investment firm with over 30 years of expertise
creating and investing in innovative companies to deliver
transformative therapies to patients. Its experienced and dedicated
team of investment professionals, entrepreneurs, advisors and
leading scientists translate scientific discoveries into
breakthrough medicines and potential cures. For more information,
visit www.mpmbioimpact.com.
About Agent Capital Agent Capital is an international
life sciences investment firm that supports disruptive healthcare
companies focusing on novel, differentiated therapeutics and
treatments that address unmet patient needs. Agent Capital aligns
with scientists, entrepreneurs, and other investors to develop the
next generation of healthcare innovations, leverages their industry
expertise and successful track record to source premier deals,
accelerate value, and drive successful exits. Since 2017, Agent
Capital has invested in over 25 companies, the majority of which
have executed collaborations with major pharmaceutical companies or
successfully raised additional capital in the private or public
markets. For more information, visit www.agentcapital.com.
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Media: SmithSolve, LLC Matt Pera (219) 628-0258