Lyell Immunopharma Receives FDA Orphan Drug Designation for LYL845 for the Treatment of Melanoma
November 09 2023 - 4:05PM
Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical‑stage T-cell
reprogramming company advancing a diverse pipeline of cell
therapies for patients with solid tumors, today announced that the
U.S. Food and Drug Administration (FDA) granted Orphan Drug
Designation to LYL845, an investigational tumor infiltrating
lymphocyte (TIL) product candidate for the treatment of patients
with stage IIB-IV melanoma.
“There remains a high unmet medical need for patients with
advanced melanoma and we believe LYL845 has the potential for
differentiated potency and durability needed to deliver better
outcomes for patients with melanoma as well as other solid tumors
where TIL therapy has not yet been widely effective,” said Lynn
Seely, M.D., Lyell’s President and CEO. “We are pleased to have
received Orphan Drug Designation for LYL845 in advanced melanoma
and look forward to presenting initial clinical data from this
program next year.”
LYL845, an autologous TIL product candidate enhanced with
Lyell’s Epi-R™ manufacturing protocols, is currently being
investigated in a Phase 1 clinical trial in patients with relapsed
or refractory metastatic or locally advanced melanoma, non-small
cell lung cancer (NSCLC), and colorectal cancer (CRC). In
nonclinical studies, LYL845 TIL expanded with Epi-R exhibit
characteristics that have previously been associated with improved
clinical response rate, including a higher percentage of cytotoxic
T cells and stemness phenotypes. LYL845 TIL have also demonstrated
enhanced T-cell potency and maintenance of tumor-reactive
polyclonality in nonclinical experiments. Initial data from Lyell’s
ongoing Phase 1 clinical trial are expected in 2024.
The FDA's Orphan Drug Designation program provides
orphan status to drugs or biologics intended for the prevention,
diagnosis, or treatment of diseases that affect fewer than 200,000
people in the United States. Sponsors of medicines that are
granted Orphan Drug Designation are entitled to certain incentives,
including tax credits for qualified clinical trials, prescription
drug user-fee exemptions, and potential seven-year marketing
exclusivity upon FDA approval.
Phase 1 Trial Design (NCT05573035)
The Phase 1 clinical trial is an open-label, dose-escalation
trial for patients with relapsed and/or refractory metastatic or
locally advanced melanoma with expansion cohorts for patients with
melanoma, NSCLC, and CRC. The primary objective of the trial is to
determine safety, tolerability and a recommended phase 2 dose range
of LYL845. The secondary objective is to determine antitumor
activity as evaluated by response rates, duration of response,
progression-free survival and overall survival. Exploratory
biomarkers of T-cell stemness will also be assessed.
About Melanoma
Melanoma of the skin is among the most common cancers in the
United States. It is one of the most common cancers in young adults
and especially in young women. It is estimated there are over
100,000 new cases of melanoma diagnosed in the United States per
year. Melanoma arises due to genetic mutations in melanocytes, the
pigment producing cells, which can be found in the skin, eye, inner
ear and leptomeninges, and represents the most aggressive and the
deadliest form of skin cancer. Although melanoma accounts for only
~1% of all dermatologic cancers, it is responsible for ~80% of
deaths from skin cancer, with only ~14% of patients with advanced
melanoma surviving for five years.
About LYL845
LYL845 is an investigational autologous TIL product enhanced
with Epi-R manufacturing protocols for patients with relapsed
and/or refractory metastatic or locally advanced melanoma, NSCLC
and CRC. In nonclinical studies, Epi-R creates polyclonal
populations of T cells that demonstrate properties of durable
stemness and anti-tumor functionality. Durable stemness is the
ability of T cells to persist and self-renew to drive durable tumor
cytotoxicity.
TIL products are created by expanding T cells taken from the
patient’s own tumor. Previous clinical experiences suggest that the
efficacy of adoptive transfer of ex vivo expanded TILs is largely
driven by specific recognition of mutated tumor neoantigens
specific to each patient. To date, broad efficacy of TIL therapies
has been limited by variable and often poor product quality, lack
of stemness or potential durability of expanded TILs, failure to
maintain polyclonality of TILs during production, and failure to
enrich the TIL product with tumor-reactive T cells. TIL products
manufactured using Lyell’s Epi-R manufacturing protocol aim to
overcome these challenges. Nonclinical studies supporting the
development of LYL845 suggest Epi-R technology improves TIL
products by maintaining properties of durable stemness, which leads
to superior ex vivo cell expansion and product qualities,
maintenance of tumor reactive clones, and enhanced
polyclonality.
About Lyell
Lyell is a clinical-stage T-cell reprogramming company advancing
a diverse pipeline of cell therapies for patients with solid
tumors. Lyell is currently enrolling a Phase 1 clinical trial
evaluating a ROR1-targeted CAR T-cell therapy in patients with
relapsed refractory triple-negative breast cancer and non-small
cell lung cancer (NSCLC) and a second Phase 1 clinical trial
evaluating reprogrammed tumor infiltrating lymphocytes (TIL) in
patients with advanced melanoma, NSCLC and colorectal cancer. The
technologies powering its product candidates are designed to
address barriers that limit consistent and long-lasting responses
to cell therapy for solid tumors: T-cell exhaustion and lack of
durable stemness, which includes the ability to persist and
self-renew to drive durable tumor cytotoxicity. Lyell is applying
its proprietary ex vivo genetic and epigenetic reprogramming
technologies to address these barriers in order to develop new
medicines with improved durable clinical outcomes. Lyell is based
in South San Francisco, California with facilities in Seattle and
Bothell, Washington. To learn more, please visit www.lyell.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Forward-looking statements expressed or implied in this press
release include, but are not limited to, statements regarding:
Lyell’s anticipated progress, business plans, business strategy and
clinical trials; the potential for LYL845 to have differentiated
potency and durability needed to deliver better outcomes for
patients with melanoma as well as other solid tumors where TIL
therapy has not yet been widely effective; the nonclinical data
associated with LYL845 and the potential for this data to be
replicated in human clinical trials; the benefits associated with
the FDA's Orphan Drug Designation, including tax credits for
qualified clinical trials, prescription drug user-fee exemptions,
and potential seven-year marketing exclusivity upon FDA approval;
and other statements that are not historical fact. These statements
are based on Lyell’s current plans, objectives, estimates,
expectations and intentions, are not guarantees of future
performance and inherently involve significant risks and
uncertainties. 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, but are not limited to, risks and uncertainties related
to: the effects of the COVID-19 pandemic; geopolitical instability;
macroeconomic conditions; Lyell’s ability to submit planned INDs or
initiate or progress clinical trials on the anticipated timelines,
if at all; our lack of experience as a company in enrolling,
conducting or completing clinical trials; Lyell’s ability to
manufacture and supply its product candidates for its clinical
trials; the preclinical profiles of Lyell’s product candidates not
translating in clinical trials; the potential for results from
clinical trials to differ from preclinical, early clinical,
preliminary or expected results; significant adverse events,
toxicities or other undesirable side effects associated with
Lyell’s product candidates; the significant uncertainty associated
with Lyell’s product candidates ever receiving any regulatory
approvals; Lyell’s ability to obtain, maintain or protect
intellectual property rights related to its product candidates;
implementation of Lyell’s strategic plans for its business and
product candidates; the sufficiency of Lyell’s capital resources
and need for additional capital to achieve its goals; and other
risks, including those described under the heading “Risk Factors”
in Lyell’s most recently filed annual report on Form 10-K and
subsequent filings with the SEC. Forward-looking statements
contained in this press release are made as of this date, and Lyell
undertakes no duty to update such information except as required
under applicable law.
Contact:Ellen RoseSenior Vice President,
Communications and Investor Relationserose@lyell.com
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