Microcystic lymphatic malformation

Palvella Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations

Retrieved on: 
Thursday, November 16, 2023
Disease, Natural history, Food, European Medicines Agency, Therapy, Cellulitis, Patient, Breakthrough therapy, Infection, FDA, Breakthrough, DSM-IV codes, Skin, Bleeding, Cyst, Orphan, Life, Patent, Pharmaceutical industry, Microcystic lymphatic malformation

WAYNE, Pa., Nov. 16, 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 that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to QTORIN rapamycin™ for the treatment of Microcystic Lymphatic Malformations (Microcystic LMs). QTORIN rapamycin is a novel, 3.9% rapamycin anhydrous gel currently under development by Palvella for the treatment of Microcystic LMs and other serious, functionally debilitating skin diseases driven by the overactivation of the mammalian target of rapamycin (mTOR) pathway.

Key Points: 
  • QTORIN rapamycin is a novel, 3.9% rapamycin anhydrous gel currently under development by Palvella for the treatment of Microcystic LMs and other serious, functionally debilitating skin diseases driven by the overactivation of the mammalian target of rapamycin (mTOR) pathway.
  • Microcystic LMs is a rare, chronically debilitating genetic disease caused by dysregulation of the PI3K/mTOR pathway.
  • In addition to Breakthrough Therapy Designation, the FDA previously granted Fast Track Designation and Orphan Drug Designation to QTORIN rapamycin for the treatment of Microcystic LMs.
  • The European Medicines Agency has also granted Orphan Drug Designation to QTORIN rapamycin for the treatment of Microcystic LMs.

Protara Therapeutics Announces Third Quarter 2023 Financial Results and Business Update

Retrieved on: 
Friday, November 3, 2023
CIS, Research, LM, LMS, Food, Rare, Injection, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Carcinoma in situ, Microcystic lymphatic malformation, Protaras, Cancer, Investment, BCG, Patient, Therapy, Macrocyst, Pharmaceutical industry, NMIBC

The Company remains on track to report preliminary results in the first half of 2024.

Key Points: 
  • The Company remains on track to report preliminary results in the first half of 2024.
  • General and administrative expenses for both the third quarter of 2023 and 2022 were $4.5 million.
  • For the third quarter of 2023, Protara reported a net loss of $9.9 million, or $0.87 per share, compared with a net loss of $7.7 million, or $0.68 per share, for the same period in 2022.
  • Net loss for the third quarter of 2023 included approximately $1.4 million of stock-based compensation expenses.

Protara Therapeutics Announces Dosing of First Patient in Phase 2 STARBORN-1 Trial of TARA-002 in Pediatric Patients with Lymphatic Malformations

Retrieved on: 
Monday, October 23, 2023
FDA, City of Hope National Medical Center, LM, LMS, Child, Food, Research institute, Rare, MD, Injection, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Microcystic lymphatic malformation, Marketing, Safety, Cancer, Therapy, University of Iowa, Patient, Macrocyst, Pharmaceutical industry, Health

“There is a pressing need for an effective FDA-approved therapy to treat LMs, a rare condition mainly affecting children.

Key Points: 
  • “There is a pressing need for an effective FDA-approved therapy to treat LMs, a rare condition mainly affecting children.
  • Including an age de-escalation safety lead-in, the trial will enroll approximately 30 patients who will receive up to four injections of TARA-002 spaced approximately six weeks apart.
  • This cell therapy is currently approved in Japan and Taiwan for LMs and has been used to successfully treat thousands of pediatric patients with this rare condition.
  • TARA-002 has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of LMs.

Palvella Therapeutics Announces Planned Pivotal Phase 3 Study Design of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations and Topline Results from Phase 2b CODY Study in Gorlin Syndrome

Retrieved on: 
Thursday, July 20, 2023
European Medicines Agency, Partnership, Microcystic adnexal carcinoma, Microcystic lymphatic malformation, Face, Pediatrics, George Washington University School of Medicine & Health Sciences, Dermatology, Skin cancer, BCC, Associate, Șaeș, SAE, Stanford University School of Medicine, Patient, Biopsy, Nature, Itch, Outline of health sciences, Cancer, GS, Clutch (eggs), Adolescence, Orphan, Toxic epidermal necrolysis, FDA, Fast track (FDA), Skin, Safety, Dietary supplement, Pharmaceutical industry, Medicine, Nevoid basal-cell carcinoma syndrome

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 the planned pivotal Phase 3 study design of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), a topical mTOR inhibitor and the lead product candidate from the QTORIN™ platform, in Microcystic Lymphatic Malformations (Microcystic LMs). Microcystic LMs are a serious, rare, chronically debilitating genetic disease for which there are no FDA-approved therapies. The company also announced topline results from the Phase 2b CODY study that evaluated QTORIN rapamycin in individuals with Basal Cell Carcinomas (BCCs) in Gorlin Syndrome (GS).

Key Points: 
  • “Patients are looking forward to an FDA-approved topical therapy that targets the underlying pathogenesis via direct inhibition of the mTOR pathway.
  • The FDA has granted Orphan Drug and Fast Track Designations to QTORIN rapamycin for the treatment of Microcystic LMs.
  • The European Medicines Agency has also granted Orphan Drug Designation to QTORIN rapamycin for the treatment of Microcystic LMs.
  • QTORIN rapamycin has received Fast Track Designation for the prevention of BCCs in Gorlin Syndrome from the FDA.

Renowned NYC Otolaryngologist Dr. Gregory Levitin Receives Castle Connolly's 2023 Top Doctor® Award

Retrieved on: 
Monday, May 8, 2023
New York Eye and Ear Infirmary, Ear, Birthmark, American Academy of Otolaryngology–Head and Neck Surgery, Glomus tumor, Castle Connolly Medical, American Academy, Hemangioma, Microcystic lymphatic malformation, Snoring, Nose, Suite, Sinusitis, MD, Patient, Physician, Cavernous venous malformation, Allergy, FACS, Columbus Circle, Medical device, Dentistry, Nightclub, Otorhinolaryngology

NEW YORK, May 8, 2023 /PRNewswire-PRWeb/ -- Dr. Gregory Levitin has been selected as a 2023 Top Doctor® by Castle Connolly. First selected in 2018, he has continued to earn this impressive award for his expertise in Otolaryngology. For patients seeking the best care, Castle Connolly publishes its list of Top Doctors at http://www.castleconnolly.com, as well as in a wide variety of partner publications, magazines and affiliate websites. After a thorough and rigorous vetting process, around 5% of the nation's licensed physicians are selected as Castle Connolly Top Doctor for their specialty.

Key Points: 
  • NEW YORK, May 8, 2023 /PRNewswire-PRWeb/ -- Dr. Gregory Levitin has been selected as a 2023 Top Doctor® by Castle Connolly.
  • First selected in 2018, he has continued to earn this impressive award for his expertise in Otolaryngology.
  • "It is always such an honor to be recognized by my peers for this award, and I consider it a significant professional accomplishment," says Dr. Gregory Levitin.
  • Dr. Gregory Levitin, MD, FACS is a board-certified otolaryngologist-head and neck surgeon and fellow of the American Academy of Otolaryngology (AAO-HNS).

Ligand Reports First Quarter 2023 Financial Results

Retrieved on: 
Thursday, May 4, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Health Technology, Telephone, Fulvestrant, GAAP, Congress, Investment, Focal segmental glomerulosclerosis, Patient, NYSE, PROTECT, Novartis, LGG, NASH, Mutation, Element, Forecasting, EMA, Proteinuria, Molluscum contagiosum, PDUFA, Growth, SB206, FDA, Lancet, THC, Total, Safety, Pelican, CDK, Microcystic lymphatic malformation, COVID-19, FSGS, BRAF, Immunoglobulin A, Research, Therapy, NVS, ESR1, NDA, Income tax, Woman, Sale, Trial of the century, Nephrology, Physician, EPS, Breast cancer, Cryptocurrency, Pharmaceutical industry, Travers Smith, Ligand, IgA nephropathy

Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) today reported financial results for the three months ended March 31, 2023, and provided an operating forecast and business updates.

Key Points: 
  • Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) today reported financial results for the three months ended March 31, 2023, and provided an operating forecast and business updates.
  • Core Captisol sales were $10.6 million for the first quarter of 2023, compared with $6.2 million for the same period in 2022.
  • Contract revenue was $16.2 million for the first quarter of 2023, compared with $11.0 million for the same period in 2022.
  • Ligand is increasing 2023 revenue and EPS guidance provided on its fourth quarter earnings call held on February 22, 2023.

Protara Therapeutics Announces First Quarter 2023 Financial Results and Business Update

Retrieved on: 
Thursday, May 4, 2023
Parenteral nutrition, Microcystic lymphatic malformation, LMS, NMIBC, Cancer, CIS, BCG, Incidence, Macrocyst, Investment, Patient, Protaras, Bacillus, Diagnosis, R, Rare, Research, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, Life insurance

Protara’s prospective study to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition is ongoing, with results expected in the third quarter of 2023.

Key Points: 
  • Protara’s prospective study to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition is ongoing, with results expected in the third quarter of 2023.
  • Research and development (R&D) expenses for the first quarter of 2023 decreased to $5.1 million from $5.3 million during the first quarter of 2022.
  • For the first quarter of 2023, Protara reported a net loss of $9.0 million, or $0.80 per share, compared with a net loss of $10.8 million, or $0.96 per share, for the same period in 2022.
  • Net loss for the first quarter of 2023 included approximately $1.6 million of stock-based compensation expenses.

Protara Therapeutics Receives Regulatory Clearance from FDA to Commence Phase 2 STARBORN-1 Trial of TARA-002 in Pediatric Patients with Lymphatic Malformations

Retrieved on: 
Tuesday, May 2, 2023
Injection, University, Microcystic lymphatic malformation, LMS, Marketing, Cancer, Human genetics, Genetics, Macrocyst, Patient, LM, Birth, Rare, FDA, Therapy, Recurrence, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Safety, Pharmaceutical industry, Health, Otorhinolaryngology

Trial initiation is expected in the fourth quarter of 2023.

Key Points: 
  • Trial initiation is expected in the fourth quarter of 2023.
  • “There are currently no FDA-approved treatments for LMs, a rare, serious condition mostly affecting children,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics.
  • This cell therapy is currently approved in Japan and Taiwan for LMs and has been used to successfully treat thousands of pediatric patients.
  • TARA-002 has been granted Rare Pediatric Disease designation by the FDA for the treatment of LMs.

Palvella Therapeutics Announces Positive Topline Results from Phase 2 Study of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations, a Serious, Rare Genetic Skin Disease with No FDA-approved

Retrieved on: 
Thursday, March 9, 2023
Therapy, Food and Drug Administration, FDA, Disease, Bleeding, Erythema, DSM-IV codes, MD, Infection, Cellulitis, Pharmaceutical industry, Microcystic lymphatic malformation

Microcystic LM is a rare, chronically debilitating genetic disease caused by dysregulation of the PI3K/mTOR pathway.

Key Points: 
  • Microcystic LM is a rare, chronically debilitating genetic disease caused by dysregulation of the PI3K/mTOR pathway.
  • The disease is characterized by localized masses of malformed lymphatic vessels that protrude through the skin barrier and persistently leak lymph fluid (lymphorrhea) and bleed, often leading to recurrent serious infections and cellulitis.
  • “The encouraging results from the Phase 2 study of QTORIN™ rapamycin build upon the large, growing evidence base supporting targeted therapeutic intervention of Microcystic LMs via the mTOR pathway.
  • Twelve individuals (n=12) with Microcystic LMs received QTORIN™ rapamycin once-daily for 12 weeks.

Protara Therapeutics Announces Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 8, 2023
Investment, CIS, Microcystic lymphatic malformation, NYSE, Therapy, Safety, Society, Parenteral nutrition, Rare, Carcinoma in situ, Annual general meeting, Cancer, NMIBC, Research, Macrocyst, Incidence, Patient, LMS, Pharmaceutical industry, Cryptocurrency

NEW YORK, March 08, 2023 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced financial results for the full year and fourth quarter ended December 31, 2022, and provided a business update.

Key Points: 
  • The fourth quarter increase was primarily due to an increase in non-clinical studies performed in the quarter versus the comparable period.
  • The fourth quarter and full year decreases were primarily due to a reduction in stock based compensation expense and market development activities.
  • Net loss for the fourth quarter included approximately $1.4 million of stock-based compensation expenses.
  • Net loss for the year ended December 31, 2022 included approximately $6.7 million of stock-based compensation expenses.