Samir Ounzain, Haya CEO

A new RNA start­up looks to reimag­ine heart treat­ments, with eyes set on En­tresto

When Samir Oun­z­ian left Lon­don a decade ago to study a lit­tle-un­der­stood branch of the genome at a Swiss lab, he wasn’t sure what he would find. Known as long-non-cod­ing RNA, these sec­tions were trans­lat­ed in­to loop­ing rings of RNA — hence the name — but didn’t ac­tu­al­ly trans­late in­to pro­teins, and no one was quite sure how many there were or pre­cise­ly what they were do­ing.

“We didn’t ex­pect any­thing at the time,” Oun­z­ian told End­points News, “be­cause we didn’t re­al­ly un­der­stand how fre­quent they were in the genome.”

Over the years, though, Oun­zain and his col­lab­o­ra­tors found these strands ex­pressed every­where, in­clud­ing in one area they found par­tic­u­lar­ly promis­ing: the heart. In 2017, they showed in Sci­ence Trans­la­tion Med­i­cine that one such loop, called Wis­per, con­trols fi­bro­sis — scar­ring — in the heart af­ter in­jury, and set about find­ing a way to drug it.

On Tues­day, the com­pa­ny Oun­zain built around the ap­proach, Haya Ther­a­peu­tics, an­nounced $20 mil­lion in seed fund­ing led by Broad­view Ven­tures, a car­dio­vas­cu­lar-fo­cused VC. They’ll use the pro­ceeds to try to push an an­ti­sense ther­a­py to neu­tral­ize Wis­per strands in­to the clin­ic in 2 to 3 years, while al­so iden­ti­fy­ing oth­er long-cod­ing RNAs to drug through­out the body.

Long-non-cod­ing RNAs are im­por­tant, re­searchers now be­lieve, in part be­cause they gov­ern how the same genes are ex­pressed in dif­fer­ent tis­sues, in­clud­ing how those genes re­spond to the en­vi­ron­ment. In the heart, Wis­per turns on fi­bro­sis in re­sponse to some type of trig­ger. Evo­lu­tion­ary, that was like­ly an ad­van­tage, help­ing pro­tect the tis­sue from in­jury, Oun­zain said. But now peo­ple are ex­posed to all sorts of dam­age — di­a­betes, high blood pres­sure, clogged ar­ter­ies — that fi­bro­sis on­ly wors­ens.

“These new stress­es of our species make the heart think it’s con­stant­ly stressed, and then the fi­bro­sis just con­tin­ues over time,” he said.

Al­though a se­ries of heart fail­ure drugs have hit the mar­ket over the last decade, most no­tably No­var­tis’s En­tresto and Mer­ck’s Verqu­vo, but none di­rect­ly treat fi­bro­sis. By shut­ting down Wis­per, Oun­zain be­lieves they can not on­ly stop fi­bro­sis but re­verse ex­ist­ing dam­age; scar tis­sue in the heart turns over quick­ly, he said, so if you stop the scar­ring process, even­tu­al­ly the tis­sue will re­store it­self.

They plan to first test the ther­a­py in a rare form of heart fail­ure called non-ob­struc­tive hy­per­trophic car­diomy­opa­thy, where fi­bro­sis lev­els are linked to symp­tom sever­i­ty. If they get proof-of-con­cept there, they’ll move to heart fail­ure with pre­served ejec­tion frac­tion — a com­mon con­di­tion in­volv­ing scar­ring in the heart and where there are so few op­tions, the FDA ap­proved En­tresto for it, de­spite a failed piv­otal tri­al.

Oun­zain, though, doesn’t want Haya thought of as a heart com­pa­ny. They can do much more go­ing af­ter long-cod­ing RNAs, he said.

“We be­lieve this par­a­digm is more than the heart,” he said. “And we ac­tu­al­ly think it could be a very pow­er­ful par­a­digm for the fu­ture of an­ti-fi­brot­ic drug de­vel­op­ment.”

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

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(AP Photo/Gemunu Amarasinghe)

Some phar­ma com­pa­nies promise to cov­er abor­tion-re­lat­ed trav­el costs — while oth­ers won't go that far yet

As the US Department of Health and Human Services promises to support the millions of women who would now need to cross state lines to receive a legal abortion, a handful of pharma companies have said they will pick up employees’ travel expenses.

GSK, Sanofi, Johnson & Johnson, BeiGene, Alnylam and Gilead have all committed to covering abortion-related travel expenses just four days after the Supreme Court overturned Roe v. Wade and revoked women’s constitutional right to an abortion.

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Aurobindo Pharma co-founders P. V. Ram Prasad Reddy (L) and K. Nityananda Reddy

Au­robindo Phar­ma re­ceives warn­ing let­ter from In­di­a's SEC fol­low­ing more FDA ques­tion marks

Indian-based generics manufacturer Aurobindo Pharma has been in the crosshairs of the FDA for several years now, but the company is also attracting attention from regulators within the subcontinent.

According to the Indian business news site Business Standard, a warning letter was sent to the company from the Securities Exchange Board of India, or SEBI.

The letter is related to disclosures made by the company on an ongoing FDA audit of the company’s Unit-1 API facility in Hyderabad, India as well as observations made by the US regulator between 2019 and 2022.

New Charles River Laboratories High Quality (HQ) Plasmid DNA Centre of Excellence at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. (Charles River)

Charles Riv­er Lab­o­ra­to­ries to start cell and gene ther­a­py man­u­fac­tur­ing at UK site in Sep­tem­ber

While Massachusetts-based Charles River Laboratories has been on an acquisition spree, they are not against planting their flag. The latest move by the company sees them crossing the pond to establish a manufacturing site in the UK.

The company on Tuesday opened its cell and gene therapy manufacturing center at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. The expansion follows Charles River’s acquisition of Cognate BioServices and Cobra Biologics in 2021 for $875 million. Cognate is a plasmid DNA, viral vector and cell therapy CDMO.

Bristol Myers Squibb (Alamy)

CVS re­sumes cov­er­age of block­buster blood thin­ner af­ter price drop fol­lows Jan­u­ary ex­clu­sion

Following some backlash from the American College of Cardiology and patients, Bristol Myers Squibb and Pfizer lowered the price of their blockbuster blood thinner Eliquis, thus ensuring that CVS Caremark would cover the drug after 6 months of it being off the major PBM’s formulary.

“Because we secured lower net costs for patients from negotiations with the drug manufacturer, Eliquis will be added back to our template formularies for the commercial segment effective July 1, 2022, and patient choices will be expanded,” CVS Health said in an emailed statement. “Anti-coagulant therapies are among the non-specialty products where we are seeing the fastest cost increases from drug manufacturers and we will continue to push back on unwarranted price increases.”