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.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Paul Hudson, Sanofi CEO (Photographer: Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi, Re­gen­eron’s Dupix­ent scores an­oth­er in­di­ca­tion with first-ever ap­proval for nodu­lar skin dis­or­der

Sanofi chief executive Paul Hudson told investors earlier this year that the Big Pharma was going to emphasize its sales kingpin Dupixent moving forward.

He wasn’t joking — the megablockbuster drug and sales king, recording just shy of $2 billion in sales this past quarter, has now officially secured its fifth indication from the FDA.

Sanofi and Regeneron, who jointly work on Dupixent development and commercialization, announced the new development on Thursday, saying that the FDA gave the all-clear to Dupixent to treat patients with prurigo nodularis, a rare autoimmune disorder characterized by a persistent, severe itch — and also visualized by hard, extremely itchy bumps known as nodules that form on the skin. The FDA noted in its announcement that it is the agency’s first approval for the disease.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) (Olivier Douliery/Sipa USA (Sipa via AP Images))

Sen­ate pass­es bill to re­work an­i­mal test­ing re­quire­ments for drug de­vel­op­ers

The US Senate passed via unanimous consent on Thursday afternoon a bipartisan bill that would eliminate a federal mandate for animal testing for new drugs.

Touted as a much-needed modernization of FDA’s rules, co-sponsor Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) have said the bill will stop lots of needless suffering of animals.

Pa­tient re­port finds con­sti­pa­tion con­di­tion not well man­aged, open­ing door for bet­ter ed­u­ca­tion from phar­ma

Advertising for constipation treatments often uses light-hearted humor in an effort to spur open discussions about the sometimes stigmatized topic. However, that may not be enough to get people to take the condition seriously, a new patient report from Phreesia finds.

Fewer than one-fifth (17%) of patients with constipation surveyed understand the longer-term health risks of constipation such as hemorrhoids and bowel incontinence. Many are trying to manage their condition with over-the-counter medicines, but often for much longer than recommended. An equal 68% say they use home remedies or OTC meds to manage constipation. But while 90% understand that OTCs are not intended for long-term use, 50% have used an OTC constipation medicine for more than a year.

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Cell and gene ther­a­pies from acad­e­mia: EMA to help 5 projects go­ing af­ter un­met clin­i­cal needs

The European Medicines Agency said Thursday that it’s launching a new pilot program to help academic and other nonprofit researchers developing advanced therapy medicinal products, which includes cell and gene therapies.

Academics have proven to be enormously useful in feeding new products, like chimeric antigen receptor (CAR)-T cell therapies first developed by Memorial Sloan Kettering, and ushered to the market by biopharma companies. Jean Bennett, formerly with the University of Pennsylvania, also saw her research lead to the approval of gene therapy Luxturna, which Roche now owns.