Ligand Earns $3 Million Milestone Payment from Palvella Therapeutics
May 28 2020 - 4:22PM
Business Wire
Ligand Pharmaceuticals Incorporated (NASDAQ: LGND)
announces it has earned a $3 million milestone payment from
Palvella Therapeutics as a result of Palvella raising $45 million
in an oversubscribed Series C financing to leading biotechnology
crossover and venture investors. The milestone payment will consist
of $2 million in cash and $1 million in Palvella Series C Preferred
Stock.
Palvella is a privately-held rare disease biopharmaceutical
company focused on developing and commercializing pathogenetically
targeted therapies for serious, rare genetic diseases with no
approved treatments. In December 2018 Ligand entered into a
development funding and royalties agreement with Palvella to
advance their lead product candidate PTX-022 (QTORIN™ 3.9%
rapamycin anhydrous gel) for the treatment of pachyonychia
congenita (PC), a rare, chronically debilitating and lifelong
genetic disease estimated to affect more than 9,000 individuals in
the U.S. In addition, Palvella’s second product candidate, PTX-367,
will enter into a late-stage clinical study for individuals with
Gorlin syndrome. Ligand paid $10 million to Palvella and in return
will receive a tiered royalty on net sales of certain Palvella
products (including PTX-022 and PTX-367) in the mid-to-upper single
digits, as well as regulatory and financing milestones.
“We are proud of our history of partnering with innovative
biopharmaceutical companies to develop important medicines,
including our relationship with Palvella. The PTX-022 program is
currently being assessed in a Phase 2/3 pivotal study and is led by
a team of distinguished and capable scientists who have identified
a novel way to treat PC,” said John Higgins, Chief Executive
Officer of Ligand Pharmaceuticals. “Palvella intends to deploy the
Series C proceeds to support the advancement of PTX-022 for the
treatment of adults with PC and ultimately prepare for
commercialization. We are also pleased to see the progress Palvella
has made advancing PTX-367 for people afflicted with Gorlin
syndrome, a disease where patients can develop hundreds of basal
cell carcinomas, oftentimes beginning in adolescence.”
In March 2020 Palvella announced completion of enrollment in its
Phase 2/3 pivotal VALO Study of PTX-022 in adults with PC. Palvella
expects topline results from this trial to be available in the
fourth quarter of 2020.
VALO is a multicenter, four-part, Phase 2/3 study evaluating the
safety and effectiveness of PTX-022 in adults with PC. In November
2019 Palvella began treating participants in the Phase 3
double-blind, placebo-controlled, randomized withdrawal portion of
the study where those who met the pre-specified clinical response
criteria during the Phase 2 portion were assigned to one of three
arms: placebo, twice-daily PTX-022 or once-daily PTX‑022. Following
completion of Phase 3, Palvella intends to initiate an open-label
extension program where patients will have the option to continue
to receive study drug.
About PTX-022
PTX-022 is a novel, topical formulation of the mTOR inhibitor
rapamycin that leverages Palvella’s proprietary and patent-pending
QTORIN™ formulation and delivery technology. The potential for
rapamycin to treat patients with PC was discovered by leading
scientists in the field who elucidated a direct mechanism of action
of rapamycin on the mutant keratin genes, which are believed to be
the root cause of PC. QTORIN™ employs a highly-specific composition
of excipients that enables distribution of mTOR inhibitors into the
basal keratinocytes, which harbor the mutant keratin genes. PTX-022
is supported by multiple issued method-of-use patents in the U.S.
that broadly cover the use of mTOR inhibitors in PC through 2032.
PTX-022 has received FDA Fast Track Designation and Orphan Drug
Designation for the treatment of PC.
About Pachyonychia Congenita
Pachyonychia congenita is a rare inherited, severe and
chronically debilitating skin disorder caused by mutations in
certain keratin genes. The disorder is manifested by the
overproduction of keratin, which are proteins that give shape and
strength to skin cells. In patients with PC, the keratin structure
does not form properly, leading to painful conditions including
blisters and calluses on the feet that impact mobility, as well as
thickened nails, cysts and sores. It is a lifelong disorder that
significantly impacts quality-of-life and often necessitates the
use of aids to assist movement or alternative forms of mobility
such as crawling on ones hands and knees. PC affects more than
9,000 people in the U.S. and no FDA-approved therapies exist to
treat the disorder.
About Ligand Pharmaceuticals
Ligand is a biopharmaceutical company focused on developing or
acquiring technologies that help pharmaceutical companies discover
and develop medicines. Our business model creates value for
stockholders by providing a diversified portfolio of biotech and
pharmaceutical product revenue streams that are supported by an
efficient and low corporate cost structure. Our goal is to offer
investors an opportunity to participate in the promise of the
biotech industry in a profitable, diversified and lower-risk
business than a typical biotech company. Our business model is
based on doing what we do best: drug discovery, early-stage drug
development, product reformulation and partnering. We partner with
other pharmaceutical companies to leverage what they do best
(late-stage development, regulatory management and
commercialization) to ultimately generate our revenue. Ligand’s
OmniAb technology platform is a patent-protected transgenic animal
platform used in the discovery of fully human mono- and bispecific
therapeutic antibodies. The Captisol platform technology is a
patent-protected, chemically modified cyclodextrin with a structure
designed to optimize the solubility and stability of drugs. The
Vernalis Design Platform (VDP) integrates protein structure
determination and engineering, fragment screening and molecular
modeling, with medicinal chemistry, to help enable success in novel
drug discovery programs against highly-challenging targets. Ab
Initio™ technology and services for the design and preparation of
customized antigens enable the successful discovery of therapeutic
antibodies against difficult-to-access cellular targets. Ligand has
established multiple alliances, licenses and other business
relationships with the world’s leading pharmaceutical companies
including Amgen, Merck, Pfizer, Sanofi, Janssen, Takeda, Gilead
Sciences and Baxter International.
For more information, please visit www.ligand.com. Follow Ligand
on Twitter @Ligand_LGND.
Forward-Looking Statements
This news release contains forward-looking statements by Ligand
that involve risks and uncertainties and reflect Ligand’s judgment
as of the date of this release. These include statements regarding:
the potential for future regulatory and financing milestones as
well as royalties from net sales of PTX-022 if approved; Palvella’s
plans to initiate an open-label extension program; Palvella’s
expectation of topline results in the fourth quarter of 2020; the
size of the PC patient population; and Palvella’s expectations
regarding the length and scope of patents covering PTX-022. Actual
events or results may differ from Ligand’s expectations due to
risks and uncertainties, including; VALO could fail to reach its
primary endpoints or show sufficient safety or efficacy to continue
development or submit a new drug application (NDA) to the FDA; the
FDA could require additional clinical trials in addition to the
VALO study; the FDA could rescind Fast Track or Orphan Drug
designations previously granted to PTX-022; even if approved,
Palvella may not successfully launch PTX-022; and patents covering
PTX-022 could be challenged or may not provide the expected scope
of coverage to exclude other products used to treat PC. The failure
to meet expectations with respect to any of the foregoing matters
may reduce Ligand’s stock price. Additional information concerning
these and other important risk factors affecting Ligand can be
found in Ligand’s prior press releases as well as in Ligand’s
public periodic filings with the Securities and Exchange
Commission. Ligand disclaims any intent or obligation to update
these forward-looking statements beyond the date of this press
release, except as required by law. This caution is made under the
safe harbor provisions of the Private Securities Litigation Reform
Act of 1995.
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version on businesswire.com: https://www.businesswire.com/news/home/20200528005815/en/
Ligand Pharmaceuticals Incorporated Patrick O’Brien
investors@ligand.com (858) 550-7768 @Ligand_LGND
LHA Investor Relations Bruce Voss bvoss@lhai.com (310)
691-7100
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