Cell ther­a­py start­up rais­es $16 mil­lion to fund its quest for the Holy Grail in re­gen­er­a­tive med­i­cine

Shinya Ya­mana­ka No­bel Prize

In 2006, Shinya Ya­mana­ka shook stem cell re­search with his dis­cov­ery that ma­ture cells can be con­vert­ed in­to stem cells, re­liev­ing a long­stand­ing po­lit­i­cal-eth­i­cal block­age and throw­ing open med­ical re­search on every­thing from curb­ing eye de­gen­er­a­tion to or­gan print­ing.

But that process still has pit­falls, in­clud­ing in risk and scal­a­bil­i­ty, and some re­searchers are ex­plor­ing an­oth­er way first hint­ed at years ago: new tech­nol­o­gy to con­vert ma­ture cells di­rect­ly in­to oth­er ma­ture cells with­out the com­plex and time-con­sum­ing process of first mak­ing them in­to stem cells.

One of those com­pa­nies, Mo­gri­fy, just raised $16 mil­lion in Se­ries A fi­nanc­ing to bring its over­all fund­ing to over $20 mil­lion since its Feb­ru­ary launch. Led by CEO Dar­rin Dis­ley, the fund­ing will help ex­pand their new base in Cam­bridge to a 60-strong staff and push for­ward their di­rect-con­ver­sion ap­proach to cell ther­a­py through re­search and li­cens­ing. In­vestors in­clude Park­walk Ad­vi­sors and Ahren In­no­va­tion Cap­i­tal.

They list po­ten­tial ap­pli­ca­tions as treat­ments for mus­cu­loskele­tal and au­to-im­mune dis­or­ders, can­cer im­munother­a­py, and ther­a­pies for oc­u­lar and res­pi­ra­to­ry dis­eases. For ex­am­ple, you could use it re­gen­er­ate car­ti­lage in arthri­tis pa­tients.

“If you could take a cell from one part of the body and turn it in­to any oth­er cell at any oth­er stage of de­vel­op­ment for an­oth­er part of the body, you ef­fec­tive­ly have the Holy Grail of re­gen­er­a­tive med­i­cine,” Dis­ley told Labiotech.eu in April.

Mo­gri­fy’s ad­van­tage over the Ya­mana­ka method called in­duced pluripo­tent stem cells (iPS), is that in the­o­ry it can be more scal­able and avoid the prob­lems as­so­ci­at­ed with iPS. These in­clude in­sta­bil­i­ties aris­ing from the in­duced im­ma­ture state and an in­creased risk of can­cer if any pluripo­tent cells re­main in the body.

The con­cept be­hind Mo­gri­fy ac­tu­al­ly pre­dates, by near­ly 19 years, Ya­mana­ka’s dis­cov­ery, which fast won him the 2012 No­bel Prize in Med­i­cine. A 2017 Na­ture study on “trans­d­if­fer­en­ti­a­tion,” as the process is called, of fi­brob­lasts in­to car­diac tis­sue traced the idea to a 1987 find­ing that a mas­ter gene reg­u­la­tor could con­vert mice fi­brob­lasts in­to skele­tal mus­cle.

The prob­lem though, ac­cord­ing to Mo­gri­fy, is that most cur­rent ef­forts re­ly on an ex­haust­ing guess-and-check process. With hun­dreds of cell types and an even greater num­ber of tran­scrip­tion fac­tors — the pro­gram that re­codes the cell — find­ing the right fac­tor for the right cell can be like a cus­to­di­an with a jan­gling, un­marked key ring try­ing to get in­to a build­ing with thou­sands of locks.

Mo­gri­fy’s key tech is a com­put­er mod­el they say can pre­dict the right com­bi­na­tion. The sci­en­tists be­hind the plat­form pub­lished a 2016 study in Na­ture ap­ply­ing the mod­el to 173 hu­man cell types and 134 tis­sues.

Be­fore Mo­gri­fy, Dis­ley led the Cam­bridge-based gene-edit­ing com­pa­ny Hori­zon Dis­cov­ery.

The 20 un­der 40: In­side the next gen­er­a­tion of bio­phar­ma lead­ers

“Each generation needs a new music,” Francis Crick wrote in 1988, reflecting back on his landmark discovery. Crick was 35, then, in 1953, when he began working with a 23-year-old named James Watson, and 37 when the pair unveiled the double helix. Rosalind Franklin, whose diffraction work undergirded their metal model, was 32.

The model would become the score for a new era in biology, one devoted to cracking the basic structures turning inside life. Subsequent years would bring new conductors and new rhythms: Robert Swanson, 29 when he convinced a 39-year-old Herb Boyer to build a company off his work and call it Genentech; Phillip Sharp, 29 when he discovered RNA splicing and 34 when he co-founded Biogen; Frances Arnold, 36 when she pioneered directed evolution; Feng Zhang, 31 when he published his CRISPR paper.

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Chart-top­ping ven­ture cash? Strong deal flow? In the month Covid-19 ripped around the globe? Yup

It turns out that even sending everyone from the CEO to rank-and-file staffers home to work in the middle of a Category 5 pandemic wasn’t enough to put a crimp in the flow of venture cash into biopharma. And even dealmaking held its own against the howling winds of misfortune — largely because a group of savvy players was quick to adjust to the new reality.

Our deal expert Chris Dokomajilar ran the numbers for us on a month-to-month basis and found that not only was venture money flowing during the panicky month of March, but it was also hitting home in record sums compared to the last 26 months of deal flow.

Say what?

As you can see in the top chart below, Dokomajilar outlined how the industry racked up $2.41 billion in total for March, just barely ahead of one other topper during the heady days of August 2018.

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FDA Commissioner Stephen Hahn and President Donald Trump at a press briefing on March 19, 2020. (AP Images)

Biotech ex­ecs warn that the FDA is fum­bling their re­sponse to the Covid-19 open-door promise, de­lay­ing progress

A few days ago the FDA touted a procedure for Covid-19 meds that committed the agency to immediate action for developers, formalizing a high-speed response that’s been promised for weeks.

Bioregnum Opinion Column by John Carroll

Decisions that once required months would be measured in hours under the Coronavirus Treatment Acceleration Program. “In many cases” trial protocols could be hammered out in less than a single day. If you had a potential solution to the crisis, the appropriate staffer would be in touch “to get studies underway quickly.”

It would be the ultimate high-speed regulatory pathway from Phase I to approval. Red tape was banished.

But it’s clear that for some — and quite likely many — biopharma execs, the actual agency response has not measured up to the promise. Beyond the front ranks of advanced companies in the field, like Gilead, or for drugs endorsed by President Trump, it may not even come close.

“The first response is this form letter everyone gets,” says one biotech CEO who’s reached out to the FDA on Covid-19. And when you try to cut through that, the ball gets dropped as it is passed from top officials to the frontline staff actually charged with getting things done.

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GSK's Hal Bar­ron buys a $250M stake in George Scan­gos' Vir and makes a bee­line to the clin­ic with Covid-19 an­ti­bod­ies

GlaxoSmithKline is diving straight into the swirling waters of Covid-19 R&D work, and investing $250 million to grab a chunk of equity in one of the emerging stars in infectious disease research to make it official.

GSK put out word this morning that it is partnering with Vir Biotechnology $VIR, the infectious disease startup founded in the Bay Area by former Biogen CEO George Scangos. They’re planning a leap into Phase II studies for 2 preclinical antibody candidates — VIR-7831 and VIR-7832 — that have been engineered to target the SARS-CoV-2 spike protein.

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UP­DAT­ED: A small, ob­scure biotech just won big with their IPO. In this mar­ket. Are you kid­ding me?

How could a small, largely unknown biotech that emerged from stealth mode just months ago with early-stage cancer programs jump onto Wall Street in the middle of a Category 6 financial hurricane and sail through with a $165 million IPO?

And what does that mean for the rest of the industry waiting to see just how much damage global lockdowns will wreak on clinical development?

The biotech is a company called Zentalis. The crew there nabbed an $85 million crossover round late last year — notably waiting 5 years before waving the numbers around to attract attention, according to my read of a FierceBiotech story. Perceptive joined in, but the syndicate was not in general the kind of marquee affair that gets tongues wagging.

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Ready to de­clare a de­fin­i­tive come­back in two months, Im­munomedics stops PhI­II ear­ly, re­cruits new CEO

More than a year ago, hit by a surprise complete response letter from the FDA, Immunomedics bid its then-CEO, Michael Pehl, adieu and began a 15-month quest to resolve the manufacturing issues cited in the CRL and seek a new leader — all the while moving forward with a Phase III study on its lead drug for metastatic triple-negative breast cancer.

Today the biotech said their stars are finally aligning. Not only is Novartis Oncology vet Harout Semerjian coming on board as CEO to steer what they believe will be a smooth sail to a new PDUFA date in June, Immunomedics has also been informed that their late-stage trial can be stopped early due to “compelling evidence of efficacy.”

An­oth­er day, an­oth­er boat­load for biotech. Deer­field adds $840M to rush of ven­ture dol­lars

The biotech dollars just keep rolling in.

Even as the world economy faces an economic contraction unprecedented in nature, biotech venture capital firms are announcing huge new investment pots. The latest? Deerfield Management Co.

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Small mol­e­cules, bi­o­log­ics and now gene ther­a­pies: Ger­many's Evotec adds an­oth­er feath­er to its R&D cap

German drug discovery company Evotec — which has a thriving rolodex of biopharma partners such as Bayer, Boehringer Ingelheim, Novartis, Novo Nordisk, Pfizer, Sanofi, and Takeda — is now venturing into gene therapies.

The company swallowed Seattle-based Just Biotherapeutics, a company focused on reducing the cost of manufacturing protein therapies last year. It is now setting up a dedicated R&D site for gene therapies in Austria, in an effort to achieve a “modality-agnostic” repertoire — small molecules, biologics and now gene therapies.

A pair of PhI­II fail­ures spells last rites for Men­lo’s once-promis­ing Mer­ck drug

Four months after an intercontinental merger, Menlo Therapeutics is counting yet another pair of trial failures — ones with significant consequences for the companies, their shareholders and the drug.

In two pivotal Phase III trials, Menlo’s lead drug serlopitant failed to treat pruritus associated with prurigo nodularis — basically itchiness from a particular skin disease that causes red lesions on a person’s arms or legs. Serlopitant has long been the company’s only drug and as recently as 2018, it looked promising enough to support a stock price of $37. In April of that year, a Phase II failure demolished the stock price overnight: $35 to $9. Other subsequent stumbles trickled the ticker down to just above $2.