Dutch rare dis­ease biotech Pro­QR carves out new com­pa­ny ded­i­cat­ed to epi­der­mol­y­sis bul­losa

Nine months af­ter kick­start­ing hu­man test­ing for its ex­on skip­ping drug for a rare skin dis­or­der called dy­s­troph­ic epi­der­mol­y­sis bul­losa, Dutch biotech Pro­QR Ther­a­peu­tics is spin­ning off the pro­gram in­to a new com­pa­ny — Wings Ther­a­peu­tics — formed and fi­nanced by a non­prof­it found­ed by a group of par­ents, in­clud­ing Pearl Jam’s Ed­die Ved­der.

Mark de Souza

The on­go­ing Phase I/II study is test­ing the drug — QR-313 — in pa­tients with re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa (DEB), a se­vere form of an in­her­it­ed skin blis­ter­ing dis­ease that is char­ac­ter­ized by frag­ile skin, chron­ic pain and a high risk of in­fec­tion and mal­nu­tri­tion.

There are no treat­ments ap­proved to tar­get the un­der­ly­ing cause of DEB.

In­fants with EB are born with skin so frag­ile they are of­ten called ‘but­ter­fly chil­dren’ – with skin is as vul­ner­a­ble as the wing of a but­ter­fly. Some type of EB oc­curs in an es­ti­mat­ed 1 out of every 50,000 live births, ac­cord­ing to the Na­tion­al Or­ga­ni­za­tion for Rare Dis­or­ders.

Mu­ta­tions in ex­on 73 of the COL7A1 gene can cause the de­pri­va­tion of a func­tion­al col­la­gen pro­tein, which cul­mi­nates in the loss of an­chor­ing fib­rils that nor­mal­ly link the der­mal and epi­der­mal lay­ers of the skin. QR-313, which has won or­phan drug sta­tus in the US and Eu­rope, is de­signed to tar­get this un­der­ly­ing cause of DEB by ex­clud­ing ex­on 73 to re­store the func­tion­al­i­ty of the an­chor­ing fib­rils.

The 8-pa­tient Phase I/II tri­al is ex­pect­ed to be com­plet­ed by mid-April.

Wings Ther­a­peu­tics will shep­herd QR-313 through clin­i­cal de­vel­op­ment, as well as ad­di­tion­al RNA mol­e­cules de­signed to tar­get oth­er mu­ta­tions that cause DEB. Pro­QR will hold a mi­nor­i­ty stake in the new com­pa­ny and will be el­i­gi­ble for mile­stone and roy­al­ty pay­ments on any com­mer­cial prod­ucts. Fi­nan­cial de­tails of the deal were not dis­closed.

Wings Ther­a­peu­tics will be led by in­ter­im CEO Mark de Souza, the for­mer CEO of Lo­tus Tis­sue Re­pair, and Hal Landy, for­mer med­ical ad­vi­sor to Lo­tus Tis­sue Re­pair and CMO of Eno­bia.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

Daphne Koller, insitro CEO (insitro)

Daphne Koller’s AI start­up gets $143M in new cash from a16z, oth­ers

Biotech is becoming saturated with machine learning companies promising to reinvent and hasten drug development, but few, if any, have amassed the war chest Daphne Koller has.

Entering Tuesday, the former Stanford professor, MacArthur Fellowship recipient, Coursera founder and chief computing officer of Google’s secretive anti-aging biotech Calico had raised $100 million for her AI startup insitro. Now she’s raised $143 million more.

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