FibroGen Announces Topline Results from LELANTOS-2, a Phase 3 Clinical Study of Pamrevlumab in Ambulatory Duchenne Muscular Dystrophy
August 29 2023 - 4:15PM
FibroGen, Inc. (NASDAQ: FGEN) today announced topline data
from the Phase 3 LELANTOS-2 trial of pamrevlumab for the treatment
of ambulatory patients with Duchenne muscular dystrophy (DMD) on
background systemic corticosteroids. The study did not meet the
primary endpoint of change in the North Star Ambulatory Assessment
(NSAA) total score from baseline to week 52 (placebo-corrected mean
difference -0.528 points; 95% CI -2.308 to 1.251; p=0.5553).
Secondary endpoints measured by change from baseline at week 52 in
4-stair climb velocity, 10-meter walk/run test, time to stand, time
to loss of ambulation, and proportion of patients with greater than
10 seconds in the 10-meter walk/run test were also not met.
“We are deeply disappointed that the LELANTOS-2
study did not meet its primary endpoint,” said Thane Wettig,
Interim Chief Executive Officer, FibroGen. “We are grateful for the
courageous efforts of patients, their caregivers, the advocacy
community, and the trial investigators who have contributed to this
important clinical study. We are committed to sharing all learnings
from this trial with the Duchenne community and hope that there are
insights that may help future efforts to develop treatments for
this devastating disease.”
Preliminary safety data showed that pamrevlumab
was generally safe and well tolerated. The majority of treatment
emergent adverse events were mild or moderate. Treatment-emergent
serious adverse events were observed in 8.3% of patients in the
pamrevlumab group and 2.8% of patients in the placebo group.
FibroGen is in the process of evaluating the
totality of the data, including other pre-specified endpoints, to
determine the next steps for the program. The Company plans to
communicate the full results of the LELANTOS-2 study at an upcoming
medical forum.
About LELANTOS-2A total of 73
boys with ambulatory DMD ages 6 to <12 years were enrolled in
LELANTOS-2, a global, Phase 3, randomized, double-blind trial of
pamrevlumab or placebo in combination with systemic
corticosteroids. The primary endpoint of the study was ambulatory
function measured by change in the North Star Ambulatory Assessment
(NSAA) total score from baseline to Week 52. Secondary endpoints
assessed from baseline to Week 52 included changes in 4-stair climb
velocity, 10-meter walk/run test, time to stand, time to loss of
ambulation, and proportion of patients with greater than 10 seconds
in the 10-meter walk/run test. In LELANTOS-2, patients were dosed
with pamrevlumab (35 mg/kg IV on Day 1 and every two weeks
thereafter with last dose at Week 52) or placebo.
About Duchenne Muscular Dystrophy Duchenne
muscular dystrophy (DMD) is a rare and debilitating neuromuscular
disease that affects approximately 1 in every 5,000 newborn boys.
About 20,000 children are diagnosed with DMD globally each year.
The fatal disease is caused by a genetic mutation leading to the
absence or defect of dystrophin, a protein necessary for normal
muscle function. The absence of dystrophin results in muscle
weakness, muscle loss, fibrosis, and inflammation. Patients with
DMD are often wheelchair-bound before the age of 12, and their
progressive muscle weakness may lead to serious medical problems
relating to respiratory and cardiac muscle.
About PamrevlumabPamrevlumab is
a potential first-in-class antibody being developed
by FibroGen to inhibit the activity of connective tissue
growth factor (CTGF), a common factor in fibrotic and proliferative
disorders characterized by persistent and excessive scarring that
can lead to organ dysfunction and failure. Pamrevlumab is in Phase
3 clinical development for the treatment of ambulatory Duchenne
muscular dystrophy (DMD), and locally advanced unresectable
pancreatic cancer (LAPC), and in Phase 2/3 for the treatment of
metastatic pancreatic cancer. The U.S. Food and Drug
Administration has granted Orphan Drug Designation to
pamrevlumab for treatment of patients with DMD and pancreatic
cancer, and Fast Track designation to pamrevlumab for the treatment
of patients with DMD and LAPC. The U.S. Food and Drug
Administration has also granted Rare Pediatric Disease
Designation to pamrevlumab for the treatment of patients with DMD.
Pamrevlumab has demonstrated a safety and tolerability profile that
has supported ongoing clinical investigation in DMD, LAPC, and
metastatic pancreatic cancer. Pamrevlumab is an investigational
drug and not approved for marketing by any regulatory authority.
For information about our pamrevlumab studies please
visit www.clinicaltrials.gov.
About
FibroGen FibroGen, Inc. is a
biopharmaceutical company committed to discovering, developing, and
commercializing a pipeline of first-in-class therapeutics. The
Company applies its pioneering expertise in connective tissue
growth factor (CTGF) biology and hypoxia-inducible factor (HIF) to
advance innovative medicines for the treatment of unmet needs.
Pamrevlumab, a fully human anti-CTGF monoclonal antibody, is in
clinical development for the treatment of locally advanced
unresectable pancreatic cancer (LAPC), metastatic pancreatic
cancer, and ambulatory Duchenne muscular dystrophy (DMD).
Roxadustat (爱瑞卓®, EVRENZO™) is currently approved
in China, Europe, Japan, and numerous other
countries for the treatment of anemia in CKD patients on dialysis
and not on dialysis. Roxadustat is in clinical development for
chemotherapy-induced anemia (CIA) in China.
FibroGen recently expanded its research and development
portfolio to include product candidates in the immuno-oncology
space along with an exclusive license for FG-3246. For more
information, please visit www.fibrogen.com.
Forward-Looking Statements This
release contains forward-looking statements regarding FibroGen’s
strategy, future plans and prospects, the development and
commercialization of the company’s product candidates, the
potential safety and efficacy profile of its product candidates,
and the potential impact of clinical data. These forward-looking
statements include, but are not limited to, statements about
FibroGen’s plans and objectives and typically are identified by use
of terms such as “may,” “will”, “should,” “on track,” “could,”
“expect,” “plan,” “anticipate,” “believe,” “estimate,” “predict,”
“potential,” “continue” and similar words, although some
forward-looking statements are expressed differently. FibroGen’s
actual results may differ materially from those indicated in these
forward-looking statements due to risks and uncertainties related
to the continued progress and timing of its various programs,
including the enrollment and results from ongoing and potential
future clinical trials, and other matters that are described in our
Annual Report on Form 10-K for the fiscal year ended December 31,
2022 and our Quarterly Report on Form 10-Q for the quarter ended
June 30, 2023, each as filed with the Securities and Exchange
Commission (SEC), including the risk factors set forth therein.
Investors are cautioned not to place undue reliance on these
forward-looking statements, which speak only as of the date of this
release, and FibroGen undertakes no obligation to update any
forward-looking statement in this press release, except as required
by law.
Contacts:FibroGen,
Inc.
Investors:David DeLucia,
CFAVice President, Corporate FP&A and Investor
Relationsddelucia@fibrogen.com
Media:Michael SzumeraExternal
Communicationsmszumera@fibrogen.com
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