As gene edit­ing ex­plodes, a new re­port from Gold­man says Chi­nese groups are seiz­ing the lead on CRISPR and CAR-T stud­ies

Con­trary to what you might be­lieve, bio­phar­ma com­pa­nies in the US are not the lead­ers in clin­i­cal tri­als us­ing gene edit­ing tech. While a slate of US/Eu­ro­pean pi­o­neers have been lin­ing up their first hu­man tri­als, in­ves­ti­ga­tors in Chi­na are al­ready well ahead in test­ing CRISPR-edit­ed cells in hu­mans, ac­cord­ing to a deep dive on gene ther­a­pies from Gold­man Sachs an­a­lyst Salveen Richter.

“As of the end of Feb­ru­ary 2018,” Richter and her team re­port­ed, “there were nine reg­is­tered clin­i­cal stud­ies test­ing CRISPR-edit­ed cells to treat var­i­ous can­cers and HIV in­fec­tion in Chi­na, vs. on­ly one study in the U.S. All of the stud­ies were ini­ti­at­ed / spon­sored by top-tier pub­lic hos­pi­tals across Chi­na, and >80 pa­tients were re­port­ed as be­ing treat­ed by these in­ves­ti­ga­tion­al genome med­i­cines.”

Last year alone, Richter adds, the Na­tion­al Nat­ur­al Sci­ence Foun­da­tion of Chi­na pro­vid­ed fund­ing for more than 90 CRISPR projects — more than 270 over the last 4 years. With no reg­u­la­tions on these gene edit­ing projects, hos­pi­tals in Chi­na in par­tic­u­lar have been quick to ac­cel­er­ate their CRISPR work, a sit­u­a­tion that might change soon if Chi­na’s main drug agency steps in and ap­plies the brakes, as Gold­man ex­pects will hap­pen.

And Chi­nese in­ves­ti­ga­tors are al­ready ri­val­ing the US in the to­tal num­ber of CAR-T stud­ies that are be­ing done. As of Feb­ru­ary, Gold­man Sachs count­ed 153 CAR-T tri­als in Chi­na, com­pared to 164 in the US, 73 in Eu­rope and 56 in all of the rest of the world com­bined.

The one com­pa­ny fur­thest out in front is Leg­end Biotech­nol­o­gy, which J&J paid $350 mil­lion to part­ner with as it en­tered the glob­al race to de­vel­op new ther­a­pies in the wake of his­toric ap­provals for Kym­ri­ah (No­var­tis) and Yescar­ta (Gilead/Kite). And if these com­pa­nies and tri­als hit pay-dirt da­ta, as they promise to, Gold­man be­lieves that Chi­na will reap a con­sid­er­able ben­e­fit by sell­ing these ther­a­pies at a much low­er rate than the pi­o­neers on the mar­ket, spurring a sig­nif­i­cant amount of med­ical tourism.

What is this kind of mar­ket worth for the ri­vals look­ing to com­pete in it?

That de­pends on the sup­ply of pa­tients.

If your new genome med­i­cine ei­ther promis­es to cure a dis­ease or de­lay any fur­ther ther­a­py for a lengthy pe­ri­od of time, notes the Gold­man re­port, then your mar­ket could play out quick­ly as you race through the pa­tient pool that is avail­able. That’s what Gilead ex­pe­ri­enced when it came up with a pain­less cure for he­pati­tis C.

Not on­ly did the drugs from Gilead shrink the pool, Gold­man re­ports in a cold analy­sis of the num­bers, they al­so re­duced the num­ber of car­ri­ers present to keep spread­ing the dis­ease, fur­ther shrink­ing the mar­ket. From the re­port:

The po­ten­tial to de­liv­er “one shot cures” is one of the most at­trac­tive as­pects of gene ther­a­py, ge­net­i­cal­ly-en­gi­neered cell ther­a­py and gene edit­ing. How­ev­er, such treat­ments of­fer a very dif­fer­ent out­look with re­gard to re­cur­ring rev­enue ver­sus chron­ic ther­a­pies, par­tic­u­lar­ly in cer­tain dis­eases where it is pos­si­ble to cure a large pro­por­tion of the preva­lent pa­tient pool (or at least pre­vent an ad­di­tion­al dose from be­ing re­quired for an ex­tend­ed pe­ri­od). While this propo­si­tion car­ries tremen­dous val­ue for pa­tients and so­ci­ety, it could rep­re­sent a chal­lenge for genome med­i­cine de­vel­op­ers look­ing for sus­tained cash flow. Gilead is a case in point, where the suc­cess of its he­pati­tis C fran­chise has grad­u­al­ly ex­haust­ed the avail­able pool of treat­able pa­tients. 

The most lu­cra­tive dis­eases for these new cu­ra­tive ther­a­pies, Richter adds, would be big fields like he­mo­phil­ia or ar­eas where there’s a large sup­ply of new pa­tients each year — like can­cer. Spinal mus­cu­lar at­ro­phy, which af­flicts a stan­dard set of in­fants each year, is al­so vi­able. 

That may not be the kind of math that bio­phar­ma ex­ecs like to dis­cuss in pub­lic, but it’s cer­tain­ly the kind of equa­tions they re­view care­ful­ly while de­cid­ing how to spend R&D bud­gets.

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.