An up­start jumps al­most overnight from pre­clin­i­cal to PhI with a $35M Take­da deal and a buy­out op­tion

In­grid Swan­son Pultz

Here’s a non­tra­di­tion­al way to set up a biotech com­pa­ny and shoot straight at a buy­out.

About six years ago, In­grid Swan­son Pultz ad­vised a group of stu­dents at the Uni­ver­si­ty of Wash­ing­ton who took on the chal­lenge of us­ing 3D struc­ture pro­tein de­sign tech to de­vel­op a new ther­a­py for celi­ac dis­ease, marked by a se­vere re­ac­tion to gluten.

Start­ing out with an en­zyme that could with­stand the harsh, acidic en­vi­ron­ment of the stom­ach, they en­gi­neered it specif­i­cal­ly to go af­ter frag­ments of gluten that trig­ger a gut re­ac­tion. And they did well, win­ning a pres­ti­gious com­pe­ti­tion.

But Pultz didn’t stop there.

She con­tin­ued to work on the en­zyme dur­ing her post­doc days and then as a fac­ul­ty mem­ber at the In­sti­tute for Pro­tein De­sign at the Uni­ver­si­ty of Wash­ing­ton. Grant mon­ey was avail­able to go far be­yond the usu­al bound­aries that can lim­it an aca­d­e­m­ic project, she tells me, tak­ing it right up to the clin­ic. A cou­ple of months ago her ther­a­py — now called Ku­ma­Max — was spun out in a start­up dubbed PvP Bi­o­log­ics. And to­day Take­da is an­nounc­ing that it is putting up $35 mil­lion to cov­er a Phase I proof of con­cept study, with an op­tion to buy the new­ly cre­at­ed com­pa­ny for an undis­closed pack­age of fees and mile­stones.

PvP is a sin­gle-as­set play with a small team, five peo­ple in San Diego, where Adam Simp­son is pres­i­dent and CEO. And Swan­son re­mains in Seat­tle with a team of three.

Guid­ing them through a lot of this has been their chair­man, Tadata­ka “Tachi” Ya­ma­da, a part­ner at Fra­zier Health­care and the for­mer chief med­ical and sci­en­tif­ic of­fi­cer at Take­da, which has been un­der­go­ing a top-to-bot­tom over­haul over the past year.

Pultz’s work here is just the lat­est of sev­er­al ex­am­ples of aca­d­e­mics who are bust­ing through some old bar­ri­ers and mov­ing new ther­a­pies in­to or close to the clin­ic. Re­cent­ly a Van­der­bilt team took an Alzheimer’s drug in­to Phase I. Stan­ford’s Irv Weiss­man, us­ing CIRM grants, al­so went in­to ear­ly stage work with grant mon­ey and lat­er start­ed Forty Sev­en, a can­cer drug com­pa­ny. And Har­vard’s Blavat­nik Bio­med­ical Ac­cel­er­a­tor took a promis­ing pre­clin­i­cal AML pro­gram from the lab of Matthew Shair and sold it to Mer­ck for $20 mil­lion up­front last spring.

Pultz may do bet­ter than any of them, in less time. The Take­da deal puts her new com­pa­ny on a short track to a po­ten­tial ac­qui­si­tion, and the PvP team will get through Phase I with­out rais­ing a tra­di­tion­al round from a ven­ture group.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

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Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

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KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.

UP­DAT­ED: Ob­sE­va makes case for best-in-class hor­mone sup­pres­sive ther­a­py in pos­i­tive uter­ine fi­broid study

About a month after the Swiss biotech disclosed a failed late-stage study in its IVF program, ObsEva on Monday unveiled positive pivotal data on its experimental treatment for heavy menstrual bleeding triggered by uterine fibroids.

ObsEva in-licensed the drug, linzagolix, from Japan’s Kissei Pharmaceutical in 2015. Two doses of the drug (100 mg and 200 mg) were tested against a placebo in the 535-patient Phase III study, dubbed PRIMROSE 2, in patients who were both on and off hormonal add-back therapy (ABT).

Samit Hirawat. Bristol-Myers Squibb

Bris­tol-My­ers is mak­ing a bee-line to the FDA with pos­i­tive liso-cel da­ta — but is it too late in the CAR-T game?

Bristol-Myers Squibb came to ASH this past weekend with a variety of messages on the new cancer drugs they had acquired in the big Celgene buyout, including liso-cel, the lead CAR-T program picked up in the $9 billion Juno acquisition. And one of the most important was that they had the pivotal efficacy and safety data needed to snag an approval from the FDA next year, with the BLA on track for a filing this month.