Flex sends out an SOS sig­nal, scrap­ping stud­ies and slash­ing staff as it hunts a sur­vival strat­e­gy — shares crater

Flex Phar­ma to­day said that its lead drug has a big prob­lem that is forc­ing the lit­tle biotech to scrap mid-stage stud­ies and hun­ker down — slash­ing 60% of its work­force as it search­es for a way out of the dilem­ma.

Christoph West­phal

The prob­lem, not dis­closed un­til now, is that the cramp­ing drug FLX-787 is not tol­er­a­ble to a sub­set of pa­tients at 30 mg. Now they need to go back and do some ba­sic for­mu­la­tion and dos­ing work to fix the is­sue — but with a beat­en down share price, the com­pa­ny doesn’t have the cash to do it alone.

It has even less room to move to­day, with its stock cra­ter­ing 75%, wip­ing out the bulk of the $75 mil­lion mar­ket cap it start­ed the day with. The stock is down to about $1, a far cry from the $16 it priced at in 2015, when Christoph West­phal took the com­pa­ny he found­ed pub­lic, tout­ing the in­volve­ment of No­bel Prize-win­ning neu­ro­sci­en­tist and ath­lete Rod MacK­in­non.

Bill McVicar

The com­pa­ny al­so de­vel­oped a con­sumer an­ti-cramp­ing prod­uct called Hot­Shot, an­oth­er strat­e­gy that West­phal has em­ployed in the past, with mixed suc­cess. West­phal — best known for sell­ing Sir­tris to GSK for $720 mil­lion — left the CEO job at Flex a year ago. He al­so briefly ran GSK’s ven­ture group, SR One, then set up Ve­rastem, which wound up hit­ting a brick wall of its own be­fore go­ing in a new di­rec­tion. 

Flex will now op­er­ate with a skele­ton crew while they go out in search of a deal, and they’d be hap­py to con­sid­er a buy­out of­fer.

Please.

“In the past few months we have re­port­ed pos­i­tive ef­fi­ca­cy da­ta in two se­ri­ous and dis­tinct­ly dif­fer­ent neu­ro­log­i­cal dis­eases: mul­ti­ple scle­ro­sis (MS) and ALS. We be­lieve that these clin­i­cal da­ta demon­strate the clear po­ten­tial of FLX-787 as a symp­to­matic ther­a­py to re­duce painful cramps and spasms in these pa­tient pop­u­la­tions,” stat­ed Bill McVicar, who took the helm af­ter West­phal left.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

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With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

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The Big Phar­ma axe: Mer­ck cuts chikun­gun­ya vax, Bris­tol My­ers drops Cy­tomX-part­nered pro­gram, and more

As fourth quarter earnings come in, Big Pharmas are disclosing changes to their pipelines during their investor calls, and sometimes more quietly in presentation appendices.

Merck dropped its chikungunya vaccine candidate, which completed a Phase II study. Merck acquired the vaccine through its purchase of Themis Bioscience in 2020. In developing a vaccine for chikungunya, a mosquito-borne virus, Valneva is the frontrunner, as it submitted its vaccine to the FDA at the end of December.

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