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Bris­tol My­ers de­tails PhI­II win in first-line test of Re­blozyl for re­duc­ing trans­fu­sions in ear­ly blood can­cer pa­tients

Reblozyl, a drug that spurs bone marrow to make more blood cells, helped nearly twice as many anemic patients with an early form of blood cancer prolong the need for transfusions compared to those who got an older therapy.

The results, which Bristol Myers Squibb announced Thursday evening, had been previously teased from a Phase III study called COMMANDS, which tested Reblozyl as a first-line treatment in about 350 adults with very low to intermediate risk myelodysplastic syndrome, or MDS, a blood cancer that prevents red blood cells from maturing and can progress into leukemia.

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BioX­cel looks to ex­pand la­bel to at-home use for lead psych drug. Ini­tial da­ta are mixed

BioXcel reported Thursday morning that Igalmi, a drug approved to treat agitation in patients with certain psychiatric disorders, had mixed results in the first part of a Phase III study.

The biotech hopes to use the study to expand Igalmi’s label so the drug can be used at home — and would not have to be administered with a healthcare provider present.

At two hours post-treatment, people who received the drug did not perform significantly differently on a scale that measures agitation from those who received placebo — but the p-value just barely missed the cut at 0.077. But at four hours post-treatment, there was more separation between the two groups, and with a p-value of 0.049, the difference was statistically significant.

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Apel­lis re­ports PhII ALS fail af­ter stop­ping open-la­bel ex­ten­sion in April

Apellis and Sobi will discontinue development in ALS after their approved rare blood and eye disease drug failed a Phase II study.

The pair was trying to see if pegcetacoplan, greenlighted in two different indications since 2021, could also help patients with the fatal neurodegenerative disease. But pegcetacoplan missed its primary endpoint of the “Combined Assessment of Function and Survival” rank score after a year, and also missed several secondary goals.

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Douglas Love, Annexon CEO

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

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Eloxx aims to take lead can­di­date in­to PhI­II for rare dis­ease

Eloxx Pharmaceuticals said it’s hoping to take its lead candidate into a Phase III clinical trial for an ultra-rare disease after the same drug failed a Phase II study in cystic fibrosis.

The RNA biotech announced Wednesday that it is planning on taking small molecule candidate ELX-02 into a pivotal trial for patients with Alport syndrome with nonsense mutations, a rare genetic condition characterized by progressive loss of kidney function. The company says there are about 9,400 to 13,000 patients who have this form of Alport syndrome, for which there are no approved treatments.

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FDA de­lays Sarep­ta's Duchenne gene ther­a­py de­ci­sion by a month

The FDA delayed its decision whether to grant accelerated approval to Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, pushing it back about one month to June 22.

Sarepta announced the delay in a news release on Wednesday morning, saying the FDA needs “modest additional time to complete the review, including final label negotiations and post-marketing commitment discussions.”

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En­ta­sis lands first an­tibi­ot­ic ap­proval eight years af­ter spin­ning out of As­traZeneca

Almost eight years after spinning out AstraZeneca’s antibiotics pipeline, Entasis Therapeutics has landed its first approval.

The FDA approved its Xacduro, made up of sulbactam and durlobactam injections, as a treatment for pneumonia caused by difficult-to-treat bacteria strains known as Acinetobacter baumannii-calcoaceticus complex (A. baumannii) among adult patients. Particularly among already vulnerable hospitalized patients, these infections can be deadly.

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Terrie Curran, Phathom Pharmaceuticals CEO (Arcutis)

Phath­om Phar­ma­ceu­ti­cals re­sub­mits NDA for acid block­er, bags $130M+ from of­fer­ing

Phathom Pharmaceuticals is making another run at getting a green light to add two new indications for its drug vonoprazan while pricing a public offering to bring in $130.7 million, the company announced today.

In February, the FDA sent the company complete response letters for its erosive esophagitis NDA and H. pylori post-approval supplement to “address specifications and controls for a nitrosamine drug substance related impurity, N-nitroso-vonoprazan.”

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PTC cuts gene ther­a­py pro­grams, dis­clos­es PhI­II fail in Friedre­ich's atax­ia and CFO ex­it

PTC Therapeutics disclosed a string of disappointing news late Tuesday, revealing a Phase III flop, a pipeline reorg to cut gene therapy programs and its CFO’s sudden departure.

The dramatic shakeup — which will lead to layoffs of an unknown number of employees — comes just two months after Matthew Klein took over as CEO from longtime leader Stuart Peltz. Shares of the biotech $PTCT sank 21% to $46 in pre-market trading Wednesday.

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Weight loss field ex­pands as Pfiz­er's oral GLP-1 gath­ers com­par­isons to Novo's We­govy in PhII da­ta

Pfizer’s attempt to catch up in the weight loss market picked up steam on Monday with research showing the company’s GLP-1 agonist helped people with type 2 diabetes shed weight.

With data published in the JAMA network, led by Pfizer VP Aditi Saxena, the Big Pharma took a step closer to staking its spot in the burgeoning R&D field for obesity drugs, many of which started as diabetes meds, and are touted as becoming the biggest drug class in history with more than $100 billion on the line.

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