R&D channel feed

CMO Carmen Bozic (Vertex)

Ver­tex shares take a hit as liv­er tox forces the re­search team to scrap a PhII drug for AAT pa­tients. Will we see new deals now?

Vertex is taking the ax to one of their rare disease drugs after evidence of liver damage emerged in a key Phase II study that blocks any uses of the higher doses that would be needed to treat genetic cases of alpha-1 antitrypsin deficiency. And with that program down and out, one top analyst says that the big biotech has no choice but to start lining up deals. Now.

The big biotech had highlighted a similar approach to mid-stage work that got them through major R&D work in cystic fibrosis, which created the foundation the company is built on. But in the trial of some 50 patients, 4 taking different doses of the drug demonstrated elevated liver enzymes “greater than 8 times the upper limit of normal.”

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Peter Hecht spun Ironwood into two different companies after an investor attack, but the results so far leave much to be desired. (Business Wire)

Cy­cle­ri­on ax­es an­oth­er pro­gram as strug­gles grow for Pe­ter Hecht's Iron­wood spin­out

Things have not been going well in the old Ironwood pipeline.

Last month, the Boston-based pharmaceutical company announced that the second of just two experimental drugs they had left in the pipeline failed a Phase III trial, triggering significant layoffs. And today, their Peter Hecht-led R&D-focused spinoff, Cyclerion, announced that it had also failed their second major study in a year, forcing them to abandon the program.

Pair of Lancet stud­ies give fi­nal word on a promis­ing Sh­iono­gi an­tibi­ot­ic that turned out to be 'as good' as the oth­er 'sub­op­ti­mal' op­tions

Last October, the FDA OK’d a semi-controversial new antibiotic from Shionogi.

In one of two large studies for the drug, known chemically as cefiderocol and commercially as Fetroja, more patients died in the treatment arm than the control arm. But it had already cleared another randomized controlled trial and the agency determined the benefits at a time of growing drug resistance outweighed the risk.

Sheila Gujrathi (Gossamer Bio)

Gos­samer shares take an­oth­er beat­ing as its lead drug fails 2 key clin­i­cal stud­ies, high­light­ing harsh re­views for DP2

Ever since a key DP2 drug at Novartis called fevipiprant failed a pivotal lineup of clinical studies by the end of last year, a number of analysts have been sitting back waiting to see how the same kind of drug would fare at Gossamer.

Tuesday morning they found out, and it wasn’t pretty.

The biotech says their lead drug GB001 failed the primary endpoint in their dose-ranging asthma study as well as all the endpoints in a parallel trial they ran for chronic rhinosinusitis.

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Rid­ing a PhII suc­cess, a small French biotech lands a rare 'break­through' sta­tus in NASH

For all the hype NASH drugs have received, the FDA has not been quick to roll out the red carpet for the most talked-about compounds. The agency gave breakthrough designation to Intercept’s obeticholic acid in 2015 but, since then, have declined to offer that seal to other late-stage drugs, including those from Gilead or Genfit.

On Monday afternoon, though, the French biotech Inventiva announced that they had won breakthrough status, becoming — by their count — the first company to receive the designation for a NASH drug since Intercept did 5 years ago. It was enough to send the company’s stock up 6%, from $13.65 to $14.50 pre-market.

Bob Langer-backed spin­out shoots for $75M-plus IPO while biotech SPACs con­tin­ue to rule on Wall Street

SQZ Biotech is looking to go public, squeezing its way onto a long list of biotechs to hit Wall Street this year.

The Watertown, MA-based biotech filed on Friday for a $75 million IPO to develop its cell therapies, now the main focus following a shift in priorities to pipeline building. Number 1 on its list of priorities is its Roche-partnered antigen presenting cell (APC) tech, which is currently in Phase I for HPV and solid tumors.

Lil­ly shows more pos­i­tive Crohn's da­ta for its IL-23 in­hibitor as it con­tin­ues to chase down ri­vals

About a year and a half after presenting positive topline results in Crohn’s disease, Eli Lilly gave its experimental IL-23 anti-inflammatory mirikizumab a boost Monday with new data.

The fresh info comes from the Phase II study and is a 52-week follow-up of the original trial. Mirikizumab showed that, after the initial 12-week period, patients who continued treatment for another 40 weeks saw further improvement in endoscopic response and Patient-Reported Outcomes remission.

No­var­tis vets get $35M to pluck one of the phar­ma gi­ant’s aban­doned drugs off the shelf and put it back in­to a clin­i­cal tri­al

Back in 2006, Novartis execs scooped up rights to Kyorin’s immunosuppressant drug program for KRP-203, an S1P drug that the pharma giant initially thought had some real potential.

But Mayzent (siponimod) ultimately stole the S1P show at Novartis, which would go on to win a first-in-class FDA OK in multiple sclerosis, with KRP-203 abandoned along the way still in early-stage development.

Until today.

Pfiz­er's last bid to re­verse Lil­ly's CDK4/6 ear­ly-stage takeover fails, as a sec­ond Ibrance study fal­ters

Eli Lilly staged a coup over the summer, showing their CDK4/6 drug Verzenio could help prevent recurrence in early-stage breast cancer patients just 3 weeks after Ibrance, the Pfizer CDK4/6 that had long been the market leader, failed on a similar study.

The study added $18 billion in market cap to the large pharma, but it came with a wrinkle. Pfizer had another early-stage study set to roll out later this year, one that, if successful, could tip the scales back towards a balance.

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It's not just the an­ti­bod­ies: Eli Lil­ly bur­nish­es its sec­ond EUA in play with new Covid-19 da­ta on Olu­mi­ant plus remde­sivir

Eli Lilly is on a roll.

Days after reporting positive data for its Covid-19 neutralizing antibodies, the pharma giant said adding its JAK inhibitor Olumiant to Gilead’s remdesivir cut the risk of death — although the result wasn’t statistically significant.

New data from ACTT-2, an adoptive treatment trial conducted by the NIH, are in line with what investigators disclosed a month ago regarding improvements in time to recovery.