- Registrational Phase 2 data demonstrate olutasidenib's
potential as a market-leading, oral, mutant isocitrate
dehydrogenase 1 (mIDH1) inhibitor for the treatment of relapsed or
refractory acute myeloid leukemia
- FDA has accepted Forma's NDA for olutasidenib, with a PDUFA
target action date of February 15,
2023
- Forma to receive an upfront payment of $2.0 million and is eligible to receive an
additional $17.5 million upon the
achievement of certain near-term regulatory, approval, and first
commercial sale milestones, as well as potential future development
and commercial milestone payments of $215.5
million and tiered royalties in the low-teens to
mid-thirties
- If approved, olutasidenib would be Rigel's second commercial
product in hematology-oncology and highly synergistic with Rigel's
existing commercial and medical affairs infrastructure
- Rigel to host conference call today to discuss transaction
details at 4:30 p.m. Eastern Time and
will be joined by Key Opinion Leader and olutasidenib Phase 2 trial
investigator, Jorge E. Cortes,
M.D.
SOUTH
SAN FRANCISCO, Calif. and WATERTOWN, Mass., Aug. 2, 2022
/PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) and
Forma Therapeutics, Inc. (Nasdaq: FMTX) today announced that they
have entered into an exclusive, worldwide license agreement to
develop, manufacture and commercialize olutasidenib, an oral, small
molecule inhibitor of mIDH1 being investigated for the treatment of
relapsed/refractory acute myeloid leukemia (R/R AML) and other
malignancies.
In a Phase 2 registrational study of olutasidenib in patients
with mIDH1 R/R AML, olutasidenib demonstrated a robust composite
complete remission rate and duration of response and was
well-tolerated. The U.S. Food and Drug Administration (FDA) has
accepted Forma's New Drug Application (NDA) for olutasidenib. The
Prescription Drug User Fee Act (PDUFA) target action date is
February 15, 2023.
"Olutasidenib is a potential market-leading treatment that we
believe, based on the registrational Phase 2 data, can improve
outcomes in patients with mIDH1+ relapsed or refractory acute
myeloid leukemia, and is a strategic fit for our business," said
Raul Rodriguez, Rigel's president
and CEO. "This transaction expands our hematology-oncology
portfolio and enables us to leverage our strong commercial
capabilities to provide a potential new therapy for these patients
who remain underserved despite currently available therapies."
"The compelling efficacy and safety data generated to date
highlight the potential for olutasidenib to transform the treatment
of mIDH1+ R/R AML. The development and approval of olutasidenib,
pending a favorable FDA decision, would represent an important
milestone for Forma that highlights our R&D capabilities," said
Frank Lee, Forma's president and
CEO. "Given Rigel's focus on hematologic diseases and cancers and
the strength of their commercial infrastructure, we believe they
are well-positioned to execute on our shared objective of
delivering olutasidenib to patients in need."
The registrational cohort of the open-label Phase 2 study
evaluated olutasidenib as monotherapy in 153 mIDH1+ R/R AML
patients. The primary efficacy-evaluable population of the cohort
was comprised of 123 R/R AML patients, who received olutasidenib
150 mg twice daily at least six months prior to the interim
analysis cutoff date of June 18, 2020
and had a centrally confirmed IDH1 mutation. The primary endpoint
was a composite of a complete remission (CR) plus a complete
remission with partial hematological recovery (CRh), defined as
less than 5% blasts in the bone marrow, no evidence of disease, and
partial recovery of peripheral blood counts (platelets
>50,000/microliter and ANC >500/microliter).
Results from the interim analysis of the trial1
demonstrated a 33% CR+CRh in mIDH1+ R/R AML patients. Among those
with CR+CRh, the estimated 18-month survival was 87% and the median
duration of CR+CRh was not yet reached, with a more conservative
sensitivity analysis indicating a median duration of 13.8 months.
Importantly, these data provide compelling evidence of clinical
efficacy with a durable response and a favorable tolerability
profile, both of which we believe differentiates olutasidenib from
other currently available treatment options for mIDH1+ R/R AML
patients.
Olutasidenib was well-tolerated, with adverse events (AEs) being
consistent with the late stage of disease and the heavily
pre-treated population. A safety analysis for all 153 patients
enrolled in the registrational Phase 2 study found the most common
grade 3/4 (≥ 10%) treatment-emergent adverse events (TEAEs) were
febrile neutropenia (20%), anemia (19%), thrombocytopenia (16%),
and neutropenia (13%).
Updated data from the registrational study will be presented at
an upcoming medical congress.
"The data from the Phase 2 registrational trial of olutasidenib
demonstrated encouraging results, particularly on durability and
survival, with median duration of response that appears to
be longer than currently available treatment options and
an 18-month survival rate among those with CR+CRh of 87%," said
Jorge E. Cortes, M.D., Director,
Georgia Cancer Center, Cecil F. Whitaker
Jr., GRA Eminent Scholar Chair in Cancer, and Phase 2 trial
investigator. "Given the trial's compelling efficacy data in
duration of response, the favorable tolerability profile, and the
still limited treatment options of patients with mIDH1+ R/R AML,
olutasidenib has the potential to be an important new treatment
option for patients."
Under the terms of the agreement, Forma will receive an upfront
payment of $2.0 million, and is
eligible to receive an additional $17.5
million upon the achievement of certain near-term
regulatory, approval, and first commercial sale milestones.
In addition, Forma is eligible to receive a total of up to an
additional $215.5 million in
connection with the achievement of certain development and
commercial milestones. Forma is also eligible to receive tiered
royalties in the low-teens to mid-thirties. Moving forward, Rigel
will be responsible for the potential launch and commercialization
of olutasidenib in the U.S., and intends to work with potential
partners to further develop and commercialize olutasidenib outside
the U.S.
Conference Call and Webcast Today at 4:30 p.m. Eastern Time, with KOL and Olutasidenib
Phase 2 trial investigator, Jorge E.
Cortes, M.D.
Rigel will host a live conference call and webcast today at
4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss financial
results, provide an update on the business, including the licensing
agreement for olutasidenib. The conference call will also feature a
presentation of the olutasidenib Phase 2 interim results by
Jorge E. Cortes, M.D., Director,
Georgia Cancer Center, Cecil F. Whitaker
Jr., GRA Eminent Scholar Chair in Cancer, and Phase 2 trial
investigator.
Participants can access the live conference call by dialing
(877) 407-3088 (domestic) or (201) 389-0927 (international). The
conference call will also be webcast live and can be accessed from
the Investor Relations section of the company's website at
www.rigel.com. The webcast will be archived and available for
replay after the call via the Rigel website.
About Olutasidenib and AML
Olutasidenib is an oral,
small molecule investigational agent designed to selectively bind
to and inhibit mutated IDH1 enzymes. This targeted treatment has
the potential to provide therapeutic benefit by reducing 2-HG
levels and restoring normal cellular differentiation. IDH1 is a
natural enzyme that is part of the normal metabolism of all cells.
When mutated, IDH1 activity can promote blood malignancies and
solid tumors. IDH1 mutations are present in 6 to 9 percent of
patients with AML2. AML is a rapidly progressing cancer
of the bone marrow and blood3. AML occurs
primarily in adults and accounts for about 1 percent of all adult
cancers. The American Cancer Society estimates that about
20,940 new cases, most in adults, arose in 2021 in the United
States alone.4 Quality of life declines for
patients with each successive line of treatment for AML, and
well-tolerated treatments in relapsed or refractory disease remain
an unmet need.
About Rigel
Rigel Pharmaceuticals, Inc., is a biotechnology company dedicated
to discovering, developing, and providing novel small molecule
drugs that significantly improve the lives of patients with
hematologic disorders, cancer, and rare immune diseases. Rigel's
pioneering research focuses on signaling pathways that are critical
to disease mechanisms. The company's first FDA-approved product is
TAVALISSE® (fostamatinib disodium hexahydrate)
tablets, the only oral spleen tyrosine kinase (SYK) inhibitor for
the treatment of adult patients with chronic immune
thrombocytopenia who have had an insufficient response to a
previous treatment. The product is also commercially available in
Europe (TAVLESSE), the
United Kingdom (TAVLESSE) and
Canada (TAVALISSE) for the
treatment of chronic immune thrombocytopenia in adult
patients.
Fostamatinib is currently being studied in a Phase 3 clinical
trial (NCT03764618) for the treatment of warm autoimmune hemolytic
anemia (wAIHA)5; a Phase 3 clinical trial (NCT04629703)
for the treatment of hospitalized high-risk patients with
COVID-195 and an NIH/NHLBI-sponsored Phase 3 clinical
trial (ACTIV-4 Host Tissue Trial, NCT04924660) for the treatment of
COVID-19 in hospitalized patients.
Rigel's other clinical programs include its interleukin
receptor-associated kinase (IRAK) inhibitor program, and a
receptor-interacting serine/threonine-protein kinase (RIPK)
inhibitor program in clinical development with partner Eli Lilly
and Company. In addition, Rigel has product candidates in
development with partners BerGenBio ASA and Daiichi Sankyo.
For further information, visit www.rigel.com or follow us on
Twitter or LinkedIn.
Please see www.TAVALISSE.com for full Prescribing
Information.
About Forma Therapeutics
Forma Therapeutics is a
clinical-stage biopharmaceutical company focused on the research,
development, and commercialization of novel therapeutics to
transform the lives of patients with rare hematologic diseases and
cancers. Our pipeline is led by etavopivat, an investigational,
once-daily, selective pyruvate kinase-R (PKR) activator designed to
be a disease-modifying therapy with the potential to improve red
blood cell (RBC) health and transform the lives of people living
with sickle cell disease, thalassemia, and lower risk MDS.
Our R&D engine combines deep biology insight, chemistry
expertise and clinical development capabilities to create drug
candidates with differentiated mechanisms of action focused on
indications with high unmet need. Our work has generated a broad
proprietary portfolio of programs with the potential to provide
profound patient benefit.
For more information, please visit www.FormaTherapeutics.com or
follow us on Twitter @FORMAInc and LinkedIn.
- De Botton, S., et al. Journal of Clinical Oncology 39,
no. 15_suppl (May 20, 2021)
7006-7006.
- NCCN Clinical Practice Guidelines in Oncology, Acute Myeloid
Leukemia. Version 2.2022 – June 14,
2022.
https://www.nccn.org/guidelines/guidelines-detail?category=1&id=1411
- Leukemia & Lymphoma Society. Accessed July 25, 2022.
https://www.lls.org/leukemia/acute-myeloid-leukemia
- The American Cancer Society. Key statistics for acute myeloid
leukemia (AML). Revised January 12, 2021. Accessed Dec.
2,
2021 at https://www.cancer.org/cancer/acute-myeloid-leukemia/about/key-statistics.html.
- The product for this use or indication is investigational and
has not been proven safe or effective by any regulatory
authority.
Rigel Forward Looking Statements
This press release contains forward-looking statements relating
to, among other things, that olutasidenib may provide a meaningful
benefit to people with relapsed or/ refractory acute myeloid
leukemia, our ability to commercialize olutasidenib in the U.S. and
identify potential partners outside of the U.S., and our
expectations related to the potential and market opportunity of
olutasidenib as therapeutics for R/R AML and other conditions. Any
statements contained in this press release that are not statements
of historical fact may be deemed to be forward-looking statements.
Forward-looking statements can be identified by words such as
"plan", "potential", "may", "expects", "will" and similar
expressions in reference to future periods. Forward-looking
statements are neither historical facts nor assurances of future
performance. Instead, they are based on Rigel's current beliefs,
expectations, and assumptions regarding the future of our business,
future plans and strategies, projections, anticipated events and
trends, the economy and other future conditions, and hence they
inherently involve significant risks, uncertainties and
changes in circumstances that are difficult to predict and many of
which are outside of our control. Therefore, you should not
rely on any of these forward-looking statements. Actual results and
the timing of events could differ materially from those anticipated
in such forward looking statements as a result of these risks and
uncertainties, which include, without limitation, risks that the
FDA, EMA or other regulatory authorities may make adverse decisions
regarding olutasidenib; risks that clinical trials may not be
predictive of real-world results or of results in subsequent
clinical trials; risks that olutasidenib may have unintended side
effects, adverse reactions or incidents of misuses; the
availability of resources to develop Rigel's product candidates;
market competition; as well as other risks detailed from time to
time in Rigel's reports filed with the Securities and Exchange
Commission, including its Quarterly Report on Form 10-Q for the
quarter ended March 31, 2022 and
subsequent filings. Any forward-looking statement made by us in
this press release is based only on information
currently available to us and speaks only as of the date on which
it is made. Rigel does not undertake any obligation to update
forward-looking statements, whether written or oral, that may be
made from time to time, whether as a result of new information,
future developments or otherwise, and expressly disclaims any
obligation or undertaking to release publicly any updates or
revisions to any forward-looking statements contained herein,
except as required by law.
Forma Forward Looking Statements
This press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995, as amended,
including, without limitation, express or implied statements
regarding the company's beliefs and expectations regarding:
therapeutic potential, clinical benefits, mechanisms of action,
efficacy, and safety of olutasidenib; the potential commercial and
collaboration opportunities, including potential future
collaborators, as well as the potential value and market for
olutasidenib; potential milestone payments; and presentation of
additional data at upcoming scientific conferences. The words
"may," "will," "could," "would," "should," "expect," "plan,"
"anticipate," "intend," "believe," "estimate," "predict,"
"project," "potential," "continue," "target" and similar
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words.
Any forward-looking statements in this press release are
based on management's current expectations and beliefs and are
subject to a number of risks, uncertainties and important factors
that may cause actual events or results to differ materially from
those expressed or implied by any forward-looking statements
contained in this press release, including, without limitation,
those risks and uncertainties associated with the following:
positive results from a clinical study may not necessarily be
predictive of the results of future or ongoing clinical studies;
adverse regulatory decisions relating to olutasidenib; Rigel's
ability to successfully develop and commercialize
olutasidenib and achieve milestones, including identifying
successful collaboration opportunities as well as those risks and
uncertainties set forth more fully under the caption "Risk Factors"
in our Quarterly Report on Form 10-Q for the quarter ended
May 6, 2022, filed with the
United States Securities and Exchange Commission (SEC) and
subsequent filings with the SEC. Forma disclaims any obligation to
publicly update or revise any such statements to reflect any change
in expectations or in events, conditions or circumstances on which
any such statements may be based, or that may affect the likelihood
that actual results will differ from those set forth in the
forward-looking statements. Any forward-looking statements
contained in this press release represent Forma's views only as of
the date hereof and should not be relied upon as representing its
views as of any subsequent date. Forma explicitly disclaims any
obligation to update any forward-looking statements.
Rigel Contacts
Media:
David Rosen, Argot Partners
Phone: 212.600.1902
Email: david.rosen@argotpartners.com
Investors:
Jodi Sievers, Rigel
Pharmaceuticals
Phone: 650.624.1232
Email: ir@rigel.com
Forma Contacts
Media:
Caitlin Hunt, Porter Novelli
Phone: 781.985.5967
Email: caitlin.hunt@porternovelli.com
Investors:
Adam Bero, Ph.D., Kendall Investor
Relations
Email: abero@kendallir.com
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SOURCE Rigel Pharmaceuticals, Inc.; Forma Therapeutics, Inc.