Following positive data from competitors earlier this year, United Therapeutics Corp. rolled out impressive and unexpected phase III results of nebulized Tyvaso (treprostinil) for the notoriously difficult indication of idiopathic pulmonary fibrosis (IPF), setting the stage for an sNDA filing with the U.S. FDA next year. If approved for the indication, United could tap into what analysts say is a $4 billion-plus market. Its shares (NASDAQ:UTHR) climbed 37.2% to $418.10 in early trading Sept. 2, while shares of Mannkind Corp. (NASDAQ:MNKD) rose 24% to $5.70 on the news. United’s dry powder formulation of treprostinil uses technology licensed from Mannkind. In the Teton-2 phase III study, inhaled Tyvaso changed the absolute forced vital capacity by 95.6 mL from baseline to week 52, meeting the primary endpoint. The benefits were observed across all subgroups, including among those receiving either of the two approved therapies for IPF, Ofev (nintedanib) or Esbriet (pirfenidone). Tyvaso also met key secondary endpoints, was well-tolerated, and its safety was consistent with other studies.

Developers in sHTG Ionis, Arrowhead make separate news with phase III data, Novartis deal

Shares of Ionis Pharmaceuticals Inc. (NASDAQ:IONS) were trading at $56.10, up $13.46, or 31%, on positive top-line results from the pivotal phase III Core and Core2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG). The antisense oligonucleotide targeting mRNA for apolipoprotein C3 was approved by the U.S. FDA under the brand name Tryngolza in December 2024 for use as an adjunct to diet as a way of reducing triglycerides in adults with familial chylomicronemia syndrome, a rare form of severe hypertriglyceridemia that can lead to life-threatening acute pancreatitis. Another player in sHTG, Arrowhead Pharmaceuticals Inc., had news as well, signing a potential $2 billion global licensing and collaboration deal with Novartis AG for ARO-SNCA, Arrowhead’s preclinical siRNA therapy against alpha-synuclein for the treatment of synucleinopathies such as Parkinson’s disease and other targets. Shares of Arrowhead (NASDAQ:ARWR) were trading at $24.49, up $2.46, or 11%.

Enlaza takes the helm in a $2B+ Vertex deal

Enlaza Therapeutics Inc. will take charge of research through nominating candidates in its more than $2 billion deal with Vertex Pharmaceuticals Inc. For its efforts, Enlaza is getting $45 million in an up-front payment and equity investment, plus the opportunity to bring in more than $2 billion in milestones and royalties on net sales. Enlaza is tasked with developing small format drug conjugates and T-cell engagers for undisclosed autoimmune diseases and improved conditioning in sickle cell disease and beta thalassemia. Vertex is footing the bill for R&D costs in the four-year partnership. Once the candidates are nominated, Vertex will handle R&D, manufacturing and commercialization.

South Korea clears Moderna’s Spikevax LP.8.1 vaccine for COVID-19

South Korea’s Ministry of Food and Drug Safety (MFDS) approved Moderna Inc.’s Spikevax LP.8.1 vaccine as an updated shot for COVID-19 targeting the LP.8.1 variant, according to Moderna Korea. The regulatory clearance comes days after the U.S. FDA accepted, on Aug. 27, Moderna’s supplemental BLAs for two of its COVID-19 vaccines, Spikevax and Mnexspike. Moderna Korea confirmed that both are mRNA-based vaccines targeting the LP.8.1 variant but differ in formulation. A single-dose vial of Spikevax contains 0.5 mL; a dose of Mnexspike has 0.2 mL.

Charm raises $80M series B to develop better menin inhibitors using AI

Charm Therapeutics Ltd. is heading to the clinic after closing an oversubscribed series B, which will fund initial development of a menin inhibitor that is AI-designed to circumvent shortcomings of first-generation molecules. Although menin inhibition is clinically validated as a treatment for acute leukemias, somatic mutations in the menin (multiple endocrine neoplasia) gene have sparked resistance, reducing efficacy. There also have been safety problems with QTc prolongation and with differentiation syndrome, a potentially fatal event that occurs when leukemic cells rapidly release cytokines. Using its Dragonfold platform to predict how a protein and a small-molecule ligand will fold together, Charm claims to have overcome these and other shortcomings.

Chinese biopharma financings up in Hong Kong

Financings are on the rise in Hong Kong as a number of Chinese pharmaceutical companies announced capital raisings, including Simcere Pharmaceutical Group Ltd., Qyuns Therapeutics Co., Ltd., Harbour Biomed Ltd. and Cutia Therapeutics.

Letting lab mice run wild improves preclinical translatability

Only a third of translational medicine animal trials that are published in highly cited journals ever form the basis for a human trial. And the numbers get worse from there, Steven Austad told the audience at the 12th Aging Research & Drug Discovery Meeting in Copenhagen last week. He argued that experiments looking for translationally relevant biology need to be conducted differently than those teasing out mechanisms, where “we try to get rid of every variable except for the one specific thing that we’re looking at,” and applied it to translation.

Phase III wins in July include Apnimed, Livzon, Takeda

In July 2025, BioWorld recorded 140 clinical trial updates spanning phases I through III, with 16 trials reporting successful phase III results. Of note, Apnimed Inc. reported a second set of positive phase III results for AD-109 for obstructive sleep apnea, Livzon Mabpharm Inc. reported positive phase III results for LZM-012 in moderate to severe plaque psoriasis and Takeda Pharmaceutical Co. Ltd. scored dual phase III wins with oveporexton (TAK-861) in narcolepsy type 1.

Holiday notice

BioWorld's offices were closed in observance of Labor Day in the U.S. No issue was published Monday, Sept. 1.

Also in the news

Accro, Beone, BMS, Cascade Prodrug, Eagle, Esperion, Exelixis, Fosun, GSK, Hemostemix, Ikris, Johnson & Johnson, Santhera, Zydus