Pachyonychia congenita

Palvella Therapeutics Reports Topline Results from Pivotal Phase 3 VAPAUS Study of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Pachyonychia Congenita

Retrieved on: 
Thursday, July 20, 2023
Partnership, Infection, COVID-19, Quality of life, PC, Patient, Nature, Physician, Lower respiratory tract infection, Common, Pachyonychia congenita, FDA, Therapy, Pharmaceutical industry

WAYNE, Pa., July 20, 2023 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc., a clinical-stage biopharmaceutical company developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies, today announced topline results from VAPAUS, a 24-week, randomized, double-blind, placebo-controlled pivotal Phase 3 study of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of Pachyonychia Congenita (PC).

Key Points: 
  • WAYNE, Pa., July 20, 2023 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc., a clinical-stage biopharmaceutical company developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies, today announced topline results from VAPAUS, a 24-week, randomized, double-blind, placebo-controlled pivotal Phase 3 study of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of Pachyonychia Congenita (PC).
  • In the Intent-to-Treat population (n=87), QTORIN rapamycin did not show a treatment effect on the Patient Global Assessment of Activities Difficulty primary endpoint, a daily patient-reported outcome measure which assessed the difficulty of patients carrying out activities on their feet, when compared to placebo.
  • QTORIN rapamycin was well-tolerated in the study and no participants withdrew due to drug-related adverse events.
  • Based on these top-line results, Palvella does not plan to invest in additional clinical studies or commercial preparation activities for QTORIN rapamycin for the treatment of PC.

Ligand Reports Fourth Quarter and Full Year 2022 Financial Results

Retrieved on: 
Wednesday, February 22, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Health Technology, Pachyonychia congenita, Coronavirus, Nevoid basal-cell carcinoma syndrome, RNA-dependent RNA polymerase, Maintenance, IND, Immunoglobulin A, MC, Focal segmental glomerulosclerosis, NDA, COPD, Jazz Pharmaceuticals, Basal-cell carcinoma, COVID-19, NMPA, SB206, Infection, Therapy, Pelican, Retirement, Syndrome, Marketing, Patient, Pneumonia, File, SARS-CoV-2, Telecanthus, Travers Smith, Kidney, Research, GAAP, Investment, RASP, Growth, Income tax, MLM, Total, NASH, FSGS, RNA, Acute myeloid leukemia, Telephone, IM, Depreciation, Forecasting, Quality of life, Microcystic lymphatic malformation, Verona, Molluscum contagiosum, BLA, Risk, FDA, EPS, Element, EMA, Communication, IV, Cryptocurrency, Pharmaceutical industry, Video game, Ligand, IgA nephropathy

Total revenues for the fourth quarter of 2022 were $50.4 million, compared with $56.4 million for the same period in 2021.

Key Points: 
  • Total revenues for the fourth quarter of 2022 were $50.4 million, compared with $56.4 million for the same period in 2021.
  • Core Captisol sales were $3.3 million for the fourth quarter of 2022, compared with $7.1 million for the same period in 2021.
  • Captisol sales related to COVID-19 were $23.5 million for the fourth quarter of 2022, compared with $28.3 million for the same period in 2021.
  • Contract revenue was $1.5 million for the fourth quarter of 2022, compared with $3.5 million for the same period in 2021.

Palvella Therapeutics Announces Pipeline Update on QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ Rapamycin) for Serious, Rare Genetic Skin Diseases with No FDA-approved Therapies

Retrieved on: 
Tuesday, February 21, 2023
PC, Dermatology, Sirolimus, Standard of care, Blood, Nevoid basal-cell carcinoma syndrome, Yale University, Treatment, Systematic review, BCC, Pain, Safety, Pachyonychia congenita, Basal-cell carcinoma, Columbia University, Patient, Platform supply vessel, Infection, Face, EMA, Microcystic lymphatic malformation, Skin, Recurrence, Research, Neoplasm, Therapy, European Medicines Agency, Partnership, FDA, Bleeding, DSM-IV codes, Pharmacokinetics, Cellulitis, Pharmaceutical industry, Vaccine, Beauty salon, Microcystic adnexal carcinoma, GS

WAYNE, Pa., Feb. 21, 2023 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc., a late clinical-stage biopharmaceutical company whose vision is to become the leading rare disease company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases in indications for which there are no FDA-approved therapies, today reported progress on its three lead development programs for QTORIN™ rapamycin. The company announced that it has completed enrollment in clinical studies that are investigating QTORIN™ rapamycin for the treatment of individuals with Pachyonychia Congenita (PC) and Microcystic Lymphatic Malformations (Microcystic LM), as well as for the prevention of Basal Cell Carcinomas (BCCs) in individuals with Gorlin Syndrome (GS). There are currently no FDA-approved therapies indicated for the treatment of PC, Microcystic LM, or for the prevention of BCCs in GS.

Key Points: 
  • There are currently no FDA-approved therapies indicated for the treatment of PC, Microcystic LM, or for the prevention of BCCs in GS.
  • Palvella also announced preliminary timelines for release of top-line results from each of those studies.
  • “Individuals living with serious, functionally debilitating, rare genetic skin diseases deserve access to targeted therapies,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella.
  • “The rapid enrollment of our three ongoing clinical studies reflects the urgent unmet need in each of these diseases.

Palvella Therapeutics Announces Series D Financing of Up to $37.7 Million to Accelerate Late-Stage Development and Support Commercialization of Novel Therapies for Serious, Rare Genetic Skin Diseases

Retrieved on: 
Thursday, January 5, 2023
Pachyonychia congenita, Standard of care, Basal-cell carcinoma, Therapy, GS, Nevoid basal-cell carcinoma syndrome, Patient, Business Council of Canada (BCC), FDA, Fast track (FDA), Microcystic lymphatic malformation, Pharmaceutical industry, Microcystic adnexal carcinoma

WAYNE, Pa., Jan. 05, 2023 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc., a late clinical-stage biopharmaceutical company whose vision is to become the leading rare disease company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases in indications for which there are no FDA-approved therapies, today announced the initial closing of its Series D financing of up to $37.7 million. The financing was led by Petrichor and included new investor Gore Range Capital. Existing investors Samsara BioCapital, BVF Partners L.P., Agent Capital, Nolan Capital, and BioAdvance also participated in the financing. Tadd Wessel, Founder and Managing Partner of Petrichor, has joined the Palvella Board of Directors.

Key Points: 
  • The financing was led by Petrichor and included new investor Gore Range Capital.
  • Existing investors Samsara BioCapital, BVF Partners L.P., Agent Capital, Nolan Capital, and BioAdvance also participated in the financing.
  • Tadd Wessel, Founder and Managing Partner of Petrichor, has joined the Palvella Board of Directors.
  • QTORIN™ rapamycin has received FDA Fast Track Designation for PC, Microcystic LM, and for the prevention of BCCs in GS.

Ligand Provides Highlights from its Investor and Analyst Day Event

Retrieved on: 
Tuesday, December 13, 2022
Professional Services, Health, FDA, Finance, Clinical Trials, Pharmaceutical, Biotechnology, Water, Baxter International, Gilead Sciences, Security (finance), Molluscum contagiosum, NDA, Immunoglobulin A, EPS, Drug discovery, Partnership, EMA, ESG, Amgen, Element, Economics, FDA, Solubility, Pachyonychia congenita, Investment, Gilead, LBL, COVID-19, GAAP, Nevoid basal-cell carcinoma syndrome, News, Vaccine, IgA nephropathy, COPD, Social Security Government Pension Offset, MLM, Merck, Marketing, Forecasting, Social, Risk management, Fine chemical, Renewable energy, Pharmaceutical industry, Pfizer, COVID, Takeda, Ligand

Highlights of todays event held in-person in New York City and virtually included the following:

Key Points: 
  • Highlights of todays event held in-person in New York City and virtually included the following:
    Management reviewed Ligands business model and the ongoing diversification of its partnership portfolio.
  • Ligand shares in the promise of the biopharmaceutical industry through royalty economics earned from providing development capital and access to its platform technologies.
  • Today Ligand has economic rights to more than 100 programs being developed or commercialized by nearly 100 different partners.
  • Ligand disclaims any intent or obligation to update these forward-looking statements beyond the date of this release.

Palvella Therapeutics Completes $45 Million Series C Financing

Retrieved on: 
Thursday, May 28, 2020
Genodermatoses, Organ systems, Pachyonychia congenita, Medical specialties, PTX, Sirolimus, PC, Medicine

Proceeds to Accelerate Pipeline of Rare Disease Therapies, Including Late-Stage Programs in Pachyonychia Congenita and Gorlin Syndrome

Key Points: 
  • Proceeds to Accelerate Pipeline of Rare Disease Therapies, Including Late-Stage Programs in Pachyonychia Congenita and Gorlin Syndrome
    WAYNE, Pa., May 28, 2020 (GLOBE NEWSWIRE) -- 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.
  • 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.
  • 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.
  • In partnership with Pachyonychia Congenita Project, Palvella completed enrollment in March 2020 of the Phase 2/3 pivotal VALO Study for PTX-022.

Palvella Therapeutics Completes Enrollment in Phase 2/3 Pivotal Study of PTX-022 for Treatment of Pachyonychia Congenita

Retrieved on: 
Friday, March 6, 2020
Branches of biology, Medicine, Signal transduction, Clinical medicine, Genodermatoses, Pachyonychia congenita, mTOR inhibitors, Sirolimus, MTOR, PC, PTX

VALO is a multi-center, four-part, Phase 2/3 study evaluating the safety and effectiveness of PTX-022 in adults with PC.

Key Points: 
  • VALO is a multi-center, four-part, Phase 2/3 study evaluating the safety and effectiveness of PTX-022 in adults with PC.
  • Palvella also intends to initiate an open-label extension program where patients will have the option to continue to receive study drug.
  • PTX-022 is supported by multiple issued method-of-use patents in the U.S. broadly covering the use of mTOR inhibitors in pachyonychia congenita.
  • Palvellas lead program, PTX-022 (QTORIN 3.9% rapamycin anhydrous gel), is in Phase 2/3 development for pachyonychia congenita, a rare, chronically debilitating and lifelong genetic disease.

Palvella Therapeutics Commences Phase 3 Portion of Phase 2/3 Pivotal Study of PTX-022 in Pachyonychia Congenita

Retrieved on: 
Wednesday, November 13, 2019
Genodermatoses, Autosomal dominant disorders, Organ systems, Pachyonychia congenita, Medical specialties, Sirolimus, PC, Medicine

The Phase 2/3 VALO study is the largest clinical study ever conducted of a therapy for PC and is currently enrolling patients at nine sites across the US.

Key Points: 
  • The Phase 2/3 VALO study is the largest clinical study ever conducted of a therapy for PC and is currently enrolling patients at nine sites across the US.
  • 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.
  • Commencing the Phase 3 portion of this study represents a significant milestone for all who have been involved in accelerating the development of PTX-022 for pachyonychia congenita, noted Palvella president and CEO, Wes Kaupinen.
  • Palvellas lead program, PTX-022 (QTORIN 3.9% rapamycin anhydrous gel), is in Phase 2/3 development for pachyonychia congenita, a rare, chronically debilitating and lifelong monogenic disease.