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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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FDA re­view­ers en­dorse J&J sin­gle-dose shot, lay­ing path for 3rd US vac­cine

J&J’s single-dose vaccine may not have produced quite the stellar numbers of the mRNA shots, but it still won a ringing endorsement from FDA reviewers, who argued in briefing documents that it could provide robust protection against the still-raging virus.

The FDA confirmed that across just under 40,000 volunteers, the vaccine proved 66% effective at preventing symptomatic Covid-19, including 72% effective in the United States. Although that’s short of the 95% figures put up by Moderna and Pfizer-BioNTech, the shot was still 85% effective at stopping severe disease 28 days after administration. There were seven deaths in the placebo group — zero in the vaccine group.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Biden is look­ing be­yond Wood­cock or Sharf­stein for FDA com­mis­sion­er — re­port

Neither Janet Woodcock nor Joshua Sharfstein is likely to be nominated as the permanent FDA commissioner, Steve Usdin at BioCentury reports.

The White House is looking for alternatives to Woodcock, the acting chief and longtime CDER director, after opposition from several Democratic senators who are calling on others to block her nomination if her name is put forth, according to Usdin. Sharfstein, the former principal deputy FDA commissioner and current Johns Hopkins professor, is out of the running altogether.