CRISPR pi­o­neer Feng Zhang's up­start biotech seeks $100M-plus IPO for pre­clin­i­cal base edit­ing work

The CRISPR pi­o­neers’ new, gen­tler-touch com­pa­ny is aim­ing for a $100 mil­lion ini­tial pub­lic of­fer­ing.

Beam Ther­a­peu­tics, head­ed by for­mer Ed­i­tas founders and gene-edit­ing trail­blaz­ers David Liu, Feng Zhang, and J Kei­th Joung, de­buted last year, push­ing a soft­er, more flex­i­ble ver­sion of gene edit­ing, re­flec­tive of a larg­er trend in the biotech in­dus­try. The promise was that in­stead of hack­ing at base pairs like first-gen CRISPR does, Beam could in­stead mod­i­fy them in a far less dis­rup­tive way.

Un­sur­pris­ing for a com­pa­ny found­ed by sci­en­tists who helped re­shape ge­net­ic dis­ease med­i­cine, Beam has been well-fund­ed since its de­but. It emerged out of stealth mode with $87 mil­lion, pri­mar­i­ly from ARCH and F-Prime Cap­i­tal Part­ners, and then a $135 mil­lion Se­ries B from A round in­vestors plus Red­mile Group, Cor­morant As­set Man­age­ment, Al­ti­tude Life Sci­ence Ven­tures. It’s built in part off tech from Ed­i­tas and Liu’s lab at MIT.

ARCH and F-Prime are the biggest stock­hold­ers at 23.0% and 19.4%, re­spec­tive­ly, while sub­sidiaries of Hill­house Cap­i­tal and Temasek claim around 7% each. No­tably, founders Liu and Zhang kept 9.1% and 7.2% for them­selves. CEO John Evans owns 3.6% of the com­pa­ny.

CRISPR was rev­o­lu­tion­ary six years ago. It made widescale gene edit­ing pos­si­ble for the first time, of­fer­ing un­prece­dent­ed pre­ci­sion that al­lowed sci­en­tists to knock out one gene with­out hit­ting the rest.

The in­creas­ing­ly clear prob­lem, though, is that it func­tions as a very price nail re­mover. It can pull out the rusty or oth­er­wise prob­lem­at­ic nail you need gone, open­ing a slew of long-un­der­stood ge­net­ic dis­eases to po­ten­tial cures, but leaves ques­tions about the fall­out to the house those nails held up. And once out, you can’t put a nail back in.

Part of Beam’s an­swer to that ques­tion is the in­tu­itive one: It re­places the old nails with new ones; rather than just cut­ting out base pairs, it can splice in new ones, re­plac­ing in­di­vid­ual mu­tant nu­cleotides that give rise to dis­ease. It ac­com­plish­es this large­ly with ver­sions of base-edit­ing en­zyme called deam­i­nase.

Con­trast­ing with the con­ven­tion­al metaphor of CRISPR as “mol­e­c­u­lar scis­sors,” Beam likes to com­pare it to a pen­cil that can erase and rewrite genes. The cru­cial point is that the process doesn’t cre­ate the dou­ble-strand breaks in DNA that the first ver­sions of CRISPR tech­nol­o­gy do.

Beam is just one of a hand­ful of new biotechs now aim­ing to ac­com­plish CRISPR’s ini­tial goals with­out caus­ing these dou­ble-strand dis­rup­tions. An­oth­er Feng Zhang com­pa­ny, Ar­bor, is pi­o­neer­ing RNA — as op­posed to DNA — edit­ing, Flag­ship Pi­o­neer­ing’s new Omega Ther­a­peu­tics is work­ing on an in­no­v­a­tive epi­ge­net­ic mod­el, and Tru­Code is us­ing a syn­thet­ic com­pound called PNA to nat­u­ral­ly in­duce gene edit­ing.

Though still pre­clin­i­cal, Beam lists 10 dis­eases it’s aim­ing for, in­clud­ing long­time gene-edit­ing tar­gets be­ta tha­lassemia and sick­le cell dis­ease.

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.

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.

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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Ear­ly-stage can­cer biotech nails $85M C round; Flem­ming Orn­skov's Gal­der­ma scores 'break­through' sta­tus

→ Zentalis Pharmaceuticals just nabbed an $85 million round from a syndicate that includes Matrix Capital, Viking Global Investors, Redmile Group, Farallon Capital, Perceptive Advisors, Surveyor Capital and Eventide Asset Management. Their lead drug is ZN-c5, which is currently in Phase I/II trials. The biotech describes that drug as a “potential best-in-class oral Selective Estrogen Receptor Degrader for estrogen receptor-positive, HER2-negative (ER+/ HER2-) breast cancer.”

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).