GH Research announced that the FDA has placed a clinical hold on an IND that the company submitted for GH001 inhaled mebufotenin (5-MeO-DMT) for the treatment of treatment-resistant depression, citing "Insufficient information to assess risks to human subjects." The company said that it is waiting to learn more details from the FDA about the reasons for the clinical … [Read more...] about FDA places clinical hold on GH Research IND for GH001 inhaled 5-MeO-DMTFDA
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Avalyn Pharma raises $175 million for continued development of AP01 inhaled pirfenidone and AP02 inhaled nintedanib
Avalyn Pharma announced that an oversubscribed Series C financing brought in $175 million that the company will use for continued development of AP01 inhaled pirfenidone and AP02 inhaled nintedanib for the treatment of pulmonary fibrosis. Earlier this year, the company reported data from a Phase 1b trial of nebulized AP01 in patients with idiopathic pulmonary fibrosis … [Read more...] about Avalyn Pharma raises $175 million for continued development of AP01 inhaled pirfenidone and AP02 inhaled nintedanib
CF Foundation to provide an additional $9 million for development of Arcturus’ ARCT-032 inhaled mRNA therapy
The Cystic Fibrosis Foundation announced that it has agreed to provide an additional $9 million in funding for development of Arcturus Therapeutics' ARCT-032 inhaled mRNA therapy for the treatment of CF. The CF Foundation said that it has already provided $15.6 million for development of ARCT-032, which is based on Arcturus' LUNAR lipid nanoparticle delivery platform. … [Read more...] about CF Foundation to provide an additional $9 million for development of Arcturus’ ARCT-032 inhaled mRNA therapy
FDA accepts Defender’s NDA for DPI-386 scopolamine nasal gel for priority review
According to Defender Pharmaceuticals, the FDA has accepted the company’s NDA for DPI-386 scopolamine nasal gel for the treatment of motion sickness for priority review, with a PDUFA goal date of January 26, 2024. Defender submitted the NDA in July 2023. The company said that the FDA granted priority review at the request of the US Navy. Defender President and CEO … [Read more...] about FDA accepts Defender’s NDA for DPI-386 scopolamine nasal gel for priority review
FDA sets PDUFA date for Liquidia’s sNDA to add PH-ILD indication for Yutrepia treprostinil DPI
Liquidia Corporation said that the FDA has accepted its sNDA to add pulmonary hypertension associated with interstitial lung disease (PH-ILD) as an indication for Yutrepia treprostinil inhalation powder without requiring any additional studies and has set the PDUFA goal date as January 24, 2024. The FDA tentatively approved Yutrepia for the treatment of … [Read more...] about FDA sets PDUFA date for Liquidia’s sNDA to add PH-ILD indication for Yutrepia treprostinil DPI
Krystal Biotech announces plans for Phase 1 trial of KB408 inhaled gene therapy for the treatment of AAT
Krystal Biotech said that the FDA has cleared an IND for KB408 nebulized gene therapy for the treatment of alpha-1 antitrypsin deficiency, and the company said that it plans to initiate a Phase 1 trial in early 2024. According to Krystal, the study will evaluate 3 ascending doses of KB408 in adult patients with AAT deficiency who have a PI*ZZ genotype. KB408 is … [Read more...] about Krystal Biotech announces plans for Phase 1 trial of KB408 inhaled gene therapy for the treatment of AAT
Seelos says 2nd part of Phase 2 study of SLS-002 intranasal racemic ketamine for suicidal ideation and behavior demonstrated improvement in symptoms
Seelos Therapeutics asserts that the second part of its Phase 2 trial of SLS-002 intranasal racemic ketamine for the treatment of acute suicidal ideation and behavior in major depressive disorder would have met its primary endpoint if the study had been able to reach full enrollment. According to the company, the trial enrolled only 147 out of an anticipated 220 … [Read more...] about Seelos says 2nd part of Phase 2 study of SLS-002 intranasal racemic ketamine for suicidal ideation and behavior demonstrated improvement in symptoms
ISAM Congress 2023
The International Society for Aerosols in Medicine (ISAM) welcomed more than 260 attendees to its 2023 biannual congress August 26-30 in Saarbrücken, Germany, reports organizing committee member Claus-Michael Lehr of the Helmholtz Institute for Pharmaceutical Research Saarland (HIPS). In addition to the scientific program, the meeting included 6 workshops, 21 … [Read more...] about ISAM Congress 2023
ReCode Therapeutics announces additional funding for development of its inhaled mRNA therapies
ReCode Therapeutics announced that it has raised $50 million as an extension to a Series B financing, with funds going toward clinical development of its inhaled mRNA therapies for the treatment of primary ciliary dyskinesia and cystic fibrosis. The company says that it has now raised a total of $260 million in Series B funding. ReCode CEO Shehnaaz Suliman … [Read more...] about ReCode Therapeutics announces additional funding for development of its inhaled mRNA therapies
FDA issues CRL to ARS’ NDA for Neffy intranasal epinephrine for the treatment of allergic reactions
According to ARS Pharmaceuticals, the FDA has issued a complete response letter to the company's NDA for Neffy epinephrine nasal spray for the treatment of allergic reactions, including anaphylaxis, and the agency has requested the completion of a PK/PD repeated-dose study prior to approval. The CRL also asked for more information on nitrosamine impurities, which the … [Read more...] about FDA issues CRL to ARS’ NDA for Neffy intranasal epinephrine for the treatment of allergic reactions