Palvella Therapeutics Completes $45 Million Series C Financing
May 28 2020 - 4:08PM
Palvella Therapeutics, Inc., a rare disease biopharmaceutical
company focused on developing and commercializing pathogenetically
targeted therapies for serious genetic diseases with no approved
treatments, today announced the closing of an oversubscribed $45
million Series C financing. Leading biotech investors participating
in this round include CAM Capital, Samsara BioCapital, BVF Partners
L.P., Adams Street Partners, Opaleye Management, Ligand
Pharmaceuticals (Nasdaq: LGND), Agent Capital, BioAdvance and Nolan
Capital (the investment fund of former AveXis CEO Sean Nolan).
Concurrent with the close of the Series C financing, Palvella
announced that two of the lead investors, Scott Morenstein,
Managing Director of CAM Capital, and Cory Freedland, Principal of
Samsara BioCapital, have been nominated to join the Palvella Board
of Directors.
“Palvella was founded on fundamental beliefs that every
individual with a rare disease deserves a treatment and that
significant value creation occurs upon the introduction of the
first approved therapy for a serious rare genetic disease,” stated
Wes Kaupinen, President and Chief Executive Officer of Palvella.
“The capital invested from this highly regarded syndicate of public
market and venture investors strengthens our team’s unwavering
commitment to develop and commercialize targeted therapies to
individuals suffering from serious, life-altering rare genetic
diseases such as pachyonychia congenita (PC) and Gorlin
syndrome.”
Proceeds from the Series C financing will support the
advancement of PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel) for
the treatment of adults with PC, a rare, chronically debilitating
and lifelong genetic disease. Individuals with PC experience
extreme pain and difficulty with ambulation, frequently
necessitating the use of either ambulatory aids or alternative
forms of mobility such as crawling on hands and knees. There are
currently no FDA-approved therapies for the over 9,000 individuals
estimated to be living with PC in the U.S. (Gallagher et al,
2019).
In partnership with Pachyonychia Congenita Project, Palvella
completed enrollment in March 2020 of the Phase 2/3 pivotal VALO
Study for PTX-022. PTX-022 is a novel formulation of rapamycin,
which was shown by geneticist Dr. Roger Kaspar to have a direct
mechanism of action on mutant keratin genes which are the root
cause in PC. PTX-022 leverages Palvella’s QTORIN™ technology to
enable localized distribution of rapamycin into the suprabasal
keratinocytes which express the mutant keratin genes that are the
primary defect in PC. The company expects top-line results from
VALO to be available in the fourth quarter of 2020.
In partnership with the Gorlin Syndrome Alliance, Palvella’s
second candidate, PTX-367 (QTORIN™ rapamycin) will enter into a
late-stage clinical study for individuals with Gorlin syndrome, a
genetic disease caused by a mutation in PTCH1, a tumor suppressor
gene. Individuals afflicted with Gorlin syndrome can develop
hundreds of basal cell carcinomas (BCCs), oftentimes beginning in
adolescence. BCCs are a malignant skin cancer requiring repeated
and potentially disfiguring surgical removal for individuals with
Gorlin syndrome. Palvella initiated internal research efforts on
the potential for QTORIN™ rapamycin in Gorlin syndrome in 2017, and
in 2018 the role of the mTOR pathway in BCC tumorigenesis in Gorlin
syndrome was further elucidated (Kim et al, 2018). Gorlin syndrome
affects an estimated 10,000 people in the U.S. and there are no
FDA-approved therapies.
PTX-022 is protected by multiple issued method-of-use patents in
the U.S. broadly covering the use of rapamycin and derivatives
thereof in treating PC that expire as late as 2032 and an allowed
patent application in the U.S. covering the use of anhydrous gel
formulations of rapamycin for treating PC and Gorlin syndrome that
will expire in 2038. PTX-022 has received FDA Fast Track
Designation, FDA Orphan Drug Designation and EMA Orphan Drug
Designation.
About Palvella TherapeuticsFounded and led by
rare disease veterans, Palvella Therapeutics is a rare disease
biopharmaceutical company focused on developing and commercializing
pathogenetically targeted therapies for serious genetic diseases
with no approved treatments. Palvella’s development model involves
partnering with patient advocacy organizations and their patient
registries to design fit-for-purpose, accelerated clinical
development programs aimed at expediting the introduction of
targeted therapies to patients who currently lack any approved
treatment options. Palvella’s lead program, PTX-022 (QTORIN™ 3.9%
rapamycin anhydrous gel), is in a Phase 2/3 pivotal study for
pachyonychia congenita (PC), a rare, chronically debilitating and
lifelong genetic disease estimated to affect more than 9,000
individuals in the U.S.
More information on the company and its pipeline may be found on
the company’s website at www.palvellatx.com.
Forward-Looking StatementsThis press release
contains forward-looking statements concerning the development and
commercialization of Palvella’s products, the potential benefits
and attributes of such products, and the company’s expectations
regarding its prospects. Forward-looking statements are subject to
risks, assumptions and uncertainties that could cause actual future
events or results to differ materially from such statements. These
statements are made as of the date of this press release. Actual
results may vary. Palvella undertakes no obligation to update any
forward-looking statements for any reason.
Contact information:Investors:Wesley H.
KaupinenPresident and CEO, Palvella
Therapeuticswes.kaupinen@palvellatx.com
Media: Aline SherwoodPublic Relations, Palvella
Therapeuticsaline.sherwood@palvellatx.com
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