The di­a­betes 'cure' Ver­tex ac­quired in its 2019 Sem­ma buy­out? It's head­ed to the clin­ic

The stem cell-de­rived ther­a­py at the cen­ter of Ver­tex’s $950 mil­lion buy­out of Sem­ma Ther­a­peu­tics in 2019, one that fea­tures promi­nent­ly in the com­pa­ny’s over­all cell and gene ther­a­py push, is one step clos­er to reach­ing the clin­ic.

Ver­tex is ex­pect­ed to launch a Phase I/II tri­al in the first half of this year af­ter the type 1 di­a­betes treat­ment re­ceived IND clear­ance from the FDA on Thurs­day, the com­pa­ny an­nounced. The pro­gram, dubbed VX-880, will be eval­u­at­ed in type 1 di­a­betes pa­tients with se­vere hy­po­glycemia and those who strug­gle to per­ceive the on­set of hy­po­glycemia, like young chil­dren.

Re­searchers will try to de­ter­mine the safe­ty and prop­er dosage in the study, which will be sin­gle-arm and open-la­bel. Ver­tex plans to en­roll about 17 pa­tients. VX-880 it­self us­es stem-cell de­rived pan­cre­at­ic islet cells to try to re­store the body’s abil­i­ty to pro­duce glu­cose in com­bi­na­tion with im­muno­sup­pres­sive ther­a­py to pro­tect the cells.

The pro­gram has been de­scribed as a po­ten­tial “cure” for type 1 di­a­betes and comes from re­search done by Har­vard’s Doug Melton. In 2014, Melton pub­lished a study show­ing the po­ten­tial for us­ing stem cells to cre­ate in­sulin-pro­duc­ing pan­cre­at­ic be­ta cells, in­sert­ed in bulk in­to mice, that were suc­cess­ful­ly pro­tect­ed from an im­mune re­sponse.

That led him to launch Sem­ma the next year with a $44 mil­lion Se­ries A round, fol­lowed by a $114 mil­lion Se­ries B in 2017 af­ter his team fig­ured out how to up­scale the tech­nol­o­gy for hu­mans. It in­volved cre­at­ing an im­plantable, cred­it card-sized de­vice con­tain­ing the be­ta cells that func­tion like a healthy pan­creas.

Melton said at the time that it proved chal­leng­ing to de­vel­op such a mem­brane with pores large enough for mol­e­cules to pass through, but small enough to be pro­tect­ed from im­mune cells. He com­pared the mem­brane to a tea bag that couldn’t be over­loaded, giv­en that they would need some 150 mil­lion cells in or­der to pro­vide the nat­ur­al in­sulin.

All that work led up to two stud­ies that pre­sent­ed promis­ing re­sults in June 2019. In the first study, Sem­ma’s stem cell-de­rived islets per­formed as hoped for, pro­duc­ing in­sulin, in a study in­volv­ing non-hu­man pri­mates whose im­mune sys­tems had been flat­tened to pre­vent a re­jec­tion. Then in a study in two pigs, a pack­age of en­gi­neered islets con­tained in one of the im­plantable mem­branes suc­cess­ful­ly gen­er­at­ed in­sulin with­out need­ing an im­muno­sup­pres­sant.

About two months lat­er, Ver­tex swooped in with their buy­out of­fer to gam­ble on the cu­ra­tive po­ten­tial on the treat­ment. It came as part of a string of flashy deals, one that saw the big cap biotech snap up Ex­on­ics’ gene edit­ing ap­proach to Duchenne and Ri­bometrix’s RNA drug­ging ca­pa­bil­i­ties in 2019 col­lab­o­ra­tions.

Stacked on top of a long­time part­ner­ship with CRISPR Ther­a­peu­tics, Ver­tex’s deal­mak­ing con­tin­ued in­to 2020, lead­ing to a pact with Sky­hawk Ther­a­peu­tics last month. The for­mer had demon­strat­ed land­mark da­ta in its sick­le cell dis­ease pro­gram, while the lat­ter is an­oth­er RNA drug­ging out­fit.

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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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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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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Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Ben Zimmer, Priovant CEO

Roivant un­veils lat­est spin­out as Pfiz­er en­trusts JAK1/TYK2 to Pri­o­vant

In November, Pfizer disclosed it’s spun out the Phase II dual JAK1/TYK2 inhibitor to a startup formed in collaboration with an unnamed, experienced partner.

We now know who the partner is. And as Pfizer and Roivant officially take the wraps off Priovant Therapeutics, the companies reveal that they have started two registrational trials of the drug, brepocitinib, as part of a broader plan to develop a big, first-in-class franchise spanning multiple orphan and specialty autoimmune diseases.

No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP Images)

Up­dat­ing the Covid-19 vac­cine: FDA of­fers a qual­i­fied thumbs-up ahead of ad­comm

The FDA’s adcomm of outside vaccine experts will meet tomorrow to discuss how to protect the US from a likely coming wave of Omicron cases in the fall and winter, and whether to deploy vaccines that specifically target the Omicron variant.

While the data so far are limited, the FDA sounded an upbeat tone in the briefing documents on Pfizer/BioNTech’s candidates, released this weekend ahead of the VRBPAC meeting.

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.