Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology
company focused on developing precision therapies for genetically
defined diseases, today reported positive updated data from Part 1
of the Company’s ongoing Phase 2 APEX clinical trial evaluating
bezuclastinib in patients with advanced systemic mastocytosis
(AdvSM) at the 66th American Society of Hematology (ASH 2024)
Annual Meeting & Exposition taking place December 7-10, 2024 in
San Diego, CA.
“Bezuclastinib has the potential to transform the treatment
landscape for people living with advanced systemic mastocytosis,”
said Daniel J. DeAngelo, M.D., Ph.D., Chief of the Division of
Leukemia at the Dana-Farber Cancer Institute and Professor of
Medicine, Harvard Medical School. “The impressive clinical data
presented today from APEX Part 1 demonstrates a combination of
rapid and deep clinical responses, with a safety profile that
avoids several of the most concerning side effects for AdvSM
patients today.”
“We are excited to share today the updated clinical data from
APEX Part 1 studying bezuclastinib in patients with advanced
systemic mastocytosis,” said Andrew Robbins, Cogent’s President and
Chief Executive Officer. “These results show the enormous promise
that a highly potent, highly selective, non-brain penetrant KIT
inhibitor may provide to this patient population. We look forward
to completing enrollment in APEX Part 2 and sharing the results
from that study in mid-2025.”
Patient DemographicsAPEX is a global,
open-label, multi-center, two-part Phase 2 clinical trial in
patients with AdvSM evaluating the safety, efficacy,
pharmacokinetic, and pharmacodynamic profiles of bezuclastinib.
Thirty-two patients were treated in Part 1 at one of four dose
levels (50 mg BID, 100 mg BID, 200 mg BID or 400 mg QD). Earlier
this year, Cogent announced APEX Part 2 would be conducted at the
optimized 150mg QD dose, which closely matches the exposure from
100 mg BID dose in APEX Part 1. The median age of patients at study
entry was 68 years (ranging from 33-87 years). Patients were
enrolled with the following sub-types: seven patients with
aggressive systemic mastocytosis (ASM), 23 patients with systemic
mastocytosis with associated hematologic neoplasm (SM-AHN), and two
patients with mast cell leukemia (MCL). Five patients had received
prior avapritinib and 10 patients had received prior midostaurin
treatment.
Clinical Activity DataAs of the data cutoff
date of October 11, 2024, 32 patients enrolled were evaluated for
signs of clinical activity, 27 of whom were mIWG-MRT-ECNM
evaluable. Clinical activity analyzed across dose levels and
focused on 100 mg BID cohort showed:
- 52% ORR (CR+CRh+PR+CI) per mIWG-MRT-ECNM criteria, including
61% ORR for TKI-treatment-naïve patients
- 83% ORR for patients treated at 100 mg BID dose cohort
- 88% ORR (CR+PR) per pure pathological response (PPR) criteria
- 100% ORR for patients treated at 100 mg BID dose cohort
- Median time to achieve response was 2.2 months and median
duration of response has not yet been reached
- Median PFS was not yet reached at median follow-up of 20
months; PFS rate at 24 months was 82%
Pharmacodynamic DataNearly all patients
demonstrated a significant improvement in biomarkers associated
with disease burden. Patients without post baseline biomarker data
were excluded from relevant analyses.
- 94% of patients achieved ≥50% reduction in serum tryptase
levels
- 100% of patients receiving ≥2 cycles achieved ≥50%
reduction
- 66% of patients achieved reduction of serum tryptase below 20
ng/mL
- 93% of KITD816V-positive patients achieved ≥50% reduction in
KIT D816V variant allele fraction (VAF)
- 100% of evaluable patients achieved a ≥50% reduction in bone
marrow mast cell burden
- 83% achieved complete clearance of mast cell aggregates by
central review
Safety DataAs of the data cutoff date of
October 11, 2024, bezuclastinib continues to demonstrate a
differentiated safety and tolerability profile across doses. The
majority of hematological adverse events were low grade and
reversible. There have been no new treatment related serious
adverse events or discontinuations reported since ASH 2023. Due to
confounding medical issues, one patient previously reported with
DILI has been reassessed and reported as a Grade 4 gamma-glutamyl
transferase (GGT) elevation case. Twelve patients required dose
reduction, eight of whom were treated at a 400 mg daily dose.
Bezuclastinib in Systemic
Mastocytosis Cogent is actively enrolling patients
into APEX Part 2 which is anticipated to complete enrollment in Q1
2025 with top-line results expected in mid-2025.
Cogent will present 24-week follow-up data from patients who
participated in the Open Label Extension portion of the ongoing
SUMMIT trial on Monday, December 9, 2024 at ASH. SUMMIT is a
randomized, double-blind, placebo-controlled, global, multicenter
Phase 2 trial evaluating bezuclastinib in patients with Nonadvanced
Systemic Mastocytosis (NonAdvSM).
Webcast Information and ASH PostersCogent will
host a webcast on Monday, December 9, 2024 at 8:00 a.m. ET to
discuss updated clinical results from both the APEX and SUMMIT ASH
presentations. The live event will be available on the Investors
& Media page of Cogent’s website
at investors.cogentbio.com. A replay of the webcast will be
available approximately two hours after the completion of the event
and will be archived for up to 30 days. The ASH posters will
be available to registered conference attendees and will also be in
the Posters and Publications section of Cogent’s website at
www.cogentbio.com/research.
About Cogent Biosciences, Inc. Cogent
Biosciences is a biotechnology company focused on developing
precision therapies for genetically defined diseases. The most
advanced clinical program, bezuclastinib, is a selective tyrosine
kinase inhibitor that is designed to potently inhibit the KIT D816V
mutation as well as other mutations in KIT exon 17. KIT D816V is
responsible for driving systemic mastocytosis, a serious disease
caused by unchecked proliferation of mast cells. Exon 17 mutations
are also found in patients with advanced gastrointestinal stromal
tumors (GIST), a type of cancer with strong dependence on oncogenic
KIT signaling. In addition to bezuclastinib, the Cogent Research
Team is developing a portfolio of novel targeted therapies to help
patients fighting serious, genetically driven diseases initially
targeting mutations in FGFR2, ErbB2, PI3Kα and KRAS. Cogent
Biosciences is based in Waltham, MA and Boulder, CO. Visit our
website for more information at www.cogentbio.com. Follow Cogent
Biosciences on social media: X (formerly
known as Twitter) and LinkedIn. Information that
may be important to investors will be routinely posted on our
website and X.
Forward Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995, including, but
not limited to, statements regarding: the expectation for the
company to complete enrollment for APEX Part 2 in Q1 2025 and to
have top-line data in mid-2025; the potential for bezuclastinib to
transform the treatment landscape for people living with AdvSM; and
the potential benefit that a highly potent, highly selective,
non-brain penetrant KIT inhibitor may provide to patients with
AdvSM. The use of words such as, but not limited to, "anticipate,"
"believe," "continue," "could," "estimate," "expect," "intend,"
"may," "might," "plan," "potential," "predict," "project,"
"should," "target," "will," or "would" and similar words
expressions are intended to identify forward-looking statements.
Forward-looking statements are neither historical facts nor
assurances of future performance. Instead, they are based on our
current beliefs, expectations and assumptions regarding the future
of our business, future plans and strategies, our clinical results,
the rate of enrollment in our clinical trials and other future
conditions. New risks and uncertainties may emerge from time to
time, and it is not possible to predict all risks and
uncertainties. No representations or warranties (expressed or
implied) are made about the accuracy of any such forward-looking
statements. We may not actually achieve the forecasts or milestones
disclosed in our forward-looking statements, and you should not
place undue reliance on our forward-looking statements. Such
forward-looking statements are subject to a number of material
risks and uncertainties including but not limited to those set
forth under the caption "Risk Factors" in Cogent's most recent
Quarterly Report on Form 10-Q filed with the SEC. Any
forward-looking statement speaks only as of the date on which it
was made. Neither we, nor our affiliates, advisors or
representatives, undertake any obligation to publicly update or
revise any forward-looking statement, whether as result of new
information, future events or otherwise, except as required by law.
These forward-looking statements should not be relied upon as
representing our views as of any date subsequent to the date
hereof.
Contact:Christi WaarichSenior Director,
Investor Relationschristi.waarich@cogentbio.com617-830-1653
Cogent Biosciences (NASDAQ:COGT)
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