Cit­ing sig­nif­i­cant progress on a cure for di­a­betes, No­vo Nordisk beefs up its stem cell pipeline with new col­lab­o­ra­tions

A grow­ing group of biotech star­tups look­ing to ad­vance a line­up of po­ten­tial stem cell cures for Type 1 di­a­betes can look to a big and very ef­fec­tive ri­val mov­ing for­ward in the field.

Mads Krogs­gaard Thom­sen

No­vo Nordisk says it’s been mak­ing ma­jor progress on de­vel­op­ing the em­bry­on­ic stem cell lines it needs for a world-class man­u­fac­tur­ing op­er­a­tion. And No­vo adds that they’ve al­so been mov­ing for­ward in de­vel­op­ing a new en­cap­su­la­tion de­vice to guard their stem cell ther­a­py from a de­struc­tive im­mune re­sponse — all part of the Holy Grail for de­vel­op­ing a cure for the dis­ease.

No­vo is still some years away from de­fin­i­tive hu­man stud­ies, but their pre­clin­i­cal progress will be­come a stan­dard bear­er for one of sev­er­al se­ri­ous ef­forts to find a cure for di­a­betes. Eli Lil­ly re­cent­ly part­nered with Sig­ilon on its pur­suit of a stem cell cure. Then there’s Sem­ma, out of the lab of Har­vard’s Doug Melton, which raised $114 mil­lion for its ap­proach to cre­at­ing in­sulin pro­duc­ing be­ta cells. 

Stem cell ther­a­pies have been up — and they’ve been down. But this new ac­tiv­i­ty among big and lit­tle com­pa­nies in re­gen­er­a­tive med­i­cine un­der­scores the grow­ing be­lief that new tech­nol­o­gy re­lat­ed to man­u­fac­tur­ing and de­liv­ery can over­come some big chal­lenges that sep­a­rat­ed the ear­ly hype from the re­al­i­ty of de­vel­op­ing a cure. As they move for­ward, you can bet that Sanofi and oth­er gi­ants will pay close at­ten­tion.

No­vo, one of the most fo­cused R&D play­ers in the world, says its progress on di­a­betes in­spired the com­pa­ny to broad­en its stem cell pipeline to in­clude pro­grams for Parkin­son’s, work­ing with the Swedish biotech com­pa­ny Bi­o­lam­i­na and Lund Uni­ver­si­ty. And Bi­o­lam­i­na is al­so col­lab­o­rat­ing with Duke (Sin­ga­pore) and No­vo on chron­ic heart fail­ure and age-re­lat­ed mac­u­lar de­gen­er­a­tion. More are on the way.

“Our col­lab­o­ra­tion with UCSF is al­so ex­pect­ed to ac­cel­er­ate cur­rent and fu­ture part­ner­ships to de­vel­op stem cell-based ther­a­pies for treat­ment of oth­er se­ri­ous chron­ic dis­eases,” said Mads Krogs­gaard Thom­sen, ex­ec­u­tive vice pres­i­dent and chief sci­ence of­fi­cer of No­vo Nordisk.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA buck­les un­der the strain of a pan­dem­ic work­load, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Pal­la­dio bags $20 mil­lion Se­ries B to top­ple a prob­lem­at­ic kid­ney dis­ease drug

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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