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.

Cy­cle­ri­on said to­day that a Phase II study for a drug meant to ame­lio­rate symp­toms in pa­tients with sick­le cell dis­ease “did not demon­strate ad­e­quate ac­tiv­i­ty to sup­port fur­ther clin­i­cal de­vel­op­ment.” The com­pa­ny did not re­lease any da­ta but said they would pub­lish the re­sults in a fu­ture fo­rum. They in­di­cat­ed, as they did with their pre­vi­ous tri­al miss, that they saw po­ten­tial for the com­pound, though they would not bring it for­ward.

“While we are dis­ap­point­ed that we won’t be con­tribut­ing a much-need­ed new treat­ment op­tion for SCD, we are con­tin­u­ing to an­a­lyze the da­ta to un­der­stand sev­er­al po­ten­tial bio­mark­er sig­nals, in­clud­ing in­flam­ma­tion, as we ex­plore part­ner­ship op­tions for this pro­gram,” Hecht said in a state­ment.

The sick­le cell pro­gram will be the sec­ond to bite the dust in Cy­cle­ri­on’s two-year his­to­ry. Last Oc­to­ber, a drug known as prali­ciguat that the biotech pushed in­to mid-stage tri­als failed back-to-back stud­ies on heart fail­ure and di­a­bet­ic neu­ropa­thy, cleav­ing a 68% chunk out of the stock price and forc­ing 30 lay­offs.

The com­pa­ny’s stock fell an­oth­er 44% Mon­day morn­ing, from $7.12 to $3.97.

The re­peat­ed stum­bles have dealt a sub­stan­tial blow to both Cy­cle­ri­on and Iron­wood since the two part­ed ways in the wake of an ac­tivist at­tack from in­vestor Alex Den­ner. Iron­wood was meant to fo­cus on com­mer­cial­iza­tion of an ap­proved drug and two com­pounds in Phase III. Cy­cle­ri­on, ini­tial­ly la­beled sim­ply “R&D Co.,” was meant to de­vel­op their ear­ly-stage line of cyclic guano­sine monophos­phates.

Now, the two monophos­phates that are fur­thest along have failed and Iron­wood has no drugs in its pipeline. Cy­cle­ri­on is worth about a third of its val­ue when it launched, though Iron­wood has main­tained its mar­ket cap on the back of its com­mer­cial drug Linzess.

Hecht’s fo­cus will now piv­ot to cen­tral ner­vous sys­tem dis­or­ders. The com­pa­ny an­nounced to­day re­sults from a Phase I phar­ma­col­o­gy study for a monophos­phate that can cross the blood-brain bar­ri­er. They have plans to start tri­als in two dif­fer­ent dis­or­ders: mi­to­chon­dr­i­al en­cephalomy­opa­thy, lac­tic aci­do­sis and stroke-like episodes (a sin­gle con­di­tion of­ten short-hand­ed as MELAS) and Alzheimer’s dis­ease with vas­cu­lar pathol­o­gy.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

FDA ad­comm nar­row­ly votes in fa­vor of Mer­ck­'s an­tivi­ral for out­pa­tient Covid-19

With little explanation for why Merck’s potential Covid-19 antiviral was less effective in reducing Covid hospitalizations and deaths in a full analysis of a Phase III trial versus an interim look, the FDA’s antimicrobial drugs advisory committee on Tuesday voted 13-10 in favor of the pill’s benefits outweighing the risks for adults within 5 days of developing Covid symptoms.

Molnupiravir will likely be authorized by FDA in the coming days for adults with mild or moderate Covid-19. While Pfizer’s antiviral may prove to be more effective, Merck’s pill will be another weapon in the armamentarium of Covid-19 treatments for countries around the world, adding to the mAb treatments already in use in the outpatient space from Regeneron, Eli Lilly and Vir/GlaxoSmithKline.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

How to use reg­istry da­ta to sup­port FDA de­ci­sion mak­ing: Agency ex­plains in new guid­ance

Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.