Ritter Pharmaceuticals Inc. is reeling from the phase III failure of its lead candidate, RP-G28, designed for patients with lactose intolerance. RP-G28 failed to demonstrate statistical significance in its primary endpoint as the top-line data showed it had no or little difference on patients compared to placebo. The data also show RP-G28 missed its secondary endpoints, all of which casts a shadow on the company and its pipeline. Read More
Glaxosmithkline plc has won FDA approval to market Nucala (mepolizumab) for use in children as young as 6 with severe eosinophilic asthma (EA). The therapy already had FDA approval as an add-on maintenance treatment for kids with the same condition ages 12 and older. Approval of the sBLA, submitted last November, catches the U.S. market up to the EU, where Nucala has been approved as an add-on treatment for children ages 6 to 17 since August 2018. Read More
BOSTON – Panelists at Biopharm America debated strategies for early stage financing as they weighed the importance of a prestigious board, the wisdom of small deals by startups with pharma firms, and the likelihood (or not) that angel investors will help make the case with other backers yet to come. Read More
HONG KONG – South Korean biopharmaceutical company Samsung Bioepis Co. Ltd., based in Incheon, will expand into the Brazilian market with Brenzys (etanercept), having recently signed a marketing agreement with Brazil's Ministry of Health. The drug is now available through the country's public health system, Sistema Único de Saùde (SUS). Read More
Royal Oak, Mich.-based liquid biopsy startup Oncocell MDx Inc. reeled in $22.2 million in a series B financing led by Savitr Capital, with participation from existing investors. The company, which has raised a total of $30 million to date, plans to use the proceeds to support ongoing development of its pan-disease immunogenomics platform and commercialization of its novel noninvasive blood-based tests. Read More
CTD Holdings Inc., of Alachua, Fla., said it received notice that an individual IND was cleared by the FDA for allowing investigational Trappsol Cyclo to a single pediatric patient diagnosed with Niemann-Pick disease type C. The company said it will provide the hydroxypropyl beta cyclodextrin drug to the patient under expanded access. Read More
The Highly Specialised Technology committee of the U.K.'s National Institute of Health and Care Excellence (NICE) said it is supporting a positive recommendation for Biomarin Pharmaceutical Inc.'s Brineura (cerliponase alfa), for children with the rare and fatal neurodegenerative condition ceroid lipofuscinosis type 2 (CLN2), affecting between one and six babies each year in the U.K. Read More
Springworks Therapeutics Inc., said it priced its IPO of 9 million shares of common stock at $18 each with gross proceeds expected to be $162 million. The underwriters have been granted a 30-day option to purchase up to 1.35 million additional shares of its common stock at the IPO price, less underwriting discounts and commissions. Shares (NASDAQ:SWTX) rocketed on their first day of trading Friday, ending the day at $22.63, up 25.7%. Read More
Aimmune Therapeutics Inc. won the qualified backing of members of the FDA's Allergenic Products Advisory Committee (adcom) during a Friday meeting held to discuss whether or not safety and efficacy data support licensure of Palforzia (AR-101), the company's oral immunotherapy (OIT) to reduce the incidence and severity of allergic reactions to peanut. Read More
Scientists at the University of Eastern Finland have investigated the effects of predisposing Alzheimer's disease (AD) mutations in induced pluripotent stem cell (iPSC)-derived microglia (iMGLs), and they found that APOE4 mutations had a much stronger effect that either presenilin (PSEN) or amyloid precursor protein (APP) in that cell type. Read More
