While Jiankui He gets all the at­ten­tion, don’t over­look the gi­ant step that Ed­i­tas is tak­ing to­day on the CRISPR front

Bioreg­num Opin­ion Col­umn by John Car­roll

As health of­fi­cials the world over — and es­pe­cial­ly in Chi­na — breathe fire over Jiankui He’s ap­par­ent­ly cred­i­ble claims that he skat­ed clear of reg­u­la­tors and ge­net­i­cal­ly al­tered the em­bryos of two new­borns to guard them against HIV, Ed­i­tas $ED­IT to­day is cel­e­brat­ing a crit­i­cal mile­stone in the long, hard jour­ney to launch­ing its first hu­man tri­al with ma­jor im­pli­ca­tions for the CRISPR field.

The FDA has ac­cept­ed Ed­i­tas’ IND for their first gene-edit­ing tri­al in a hand­ful of pa­tients suf­fer­ing from an in­her­it­ed reti­nal de­gen­er­a­tive dis­ease called Leber Con­gen­i­tal Amau­ro­sis type 10.

Nor­mal­ly, that’s not some­thing that gets much at­ten­tion in the bio­phar­ma world these days. As mon­ey has pumped in­to the sys­tem, cre­at­ing a wave of new biotechs, INDs have be­come rou­tine — even if, as in Ed­i­tas’ case, it comes with a $25 mil­lion mile­stone pay­ment.

Ka­trine Bosley, Ed­i­tas

Ed­i­tas, though, set out ful­ly 5 years ago to wide­spread ac­claim, one of sev­er­al star­tups that were in­spired by 3 sci­en­tists: Feng Zhang, Jen­nifer Doud­na and Em­manuelle Marie Char­p­en­tier. Now, af­ter the ap­plause has died down, Ed­i­tas and its part­ners at Al­ler­gan will fi­nal­ly have their shot to see whether gene edit­ing tech — in this case CRISPR/Cas9 and the new­er CRISPR/Cpf1 — can play a big role in cor­rect­ing se­vere dis­eases. 

To get here Ed­i­tas has had to fight off a stiff patent chal­lenge, keep the mon­ey pump­ing in as a pub­lic com­pa­ny which nec­es­sar­i­ly had to spend years in pre­clin­i­cal de­vel­op­ment, bat back per­sis­tent ques­tions about off-tar­get ef­fects and con­vince reg­u­la­tors that it had every­thing lined up prop­er­ly be­fore it shift­ed from an­i­mal stud­ies to hu­mans.

“The FDA’s ac­cep­tance of our IND for ED­IT-101 is a sig­nif­i­cant mo­ment in the field of genome edit­ing,” not­ed CEO Ka­trine Bosley, “and im­por­tant­ly, a crit­i­cal mile­stone for pa­tients, as we are now one step clos­er to a treat­ment for LCA10.”

Jiankui He will end the week as one of the most no­to­ri­ous rogue sci­en­tists in the world. Ed­i­tas will be bet­ter off fi­nan­cial­ly and one big step clos­er to a dra­mat­ic test case to gauge the near-term po­ten­tial of what is still a rad­i­cal­ly new tech­nol­o­gy. It won’t get as much at­ten­tion, but in the long run it’s much more im­por­tant than the head­lines com­ing out of Hong Kong.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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Sanofi takes a $260M hit to ex­tri­cate it­self from a dis­as­trous al­liance with Lex­i­con

Sanofi spent $300 million in cash to get into a $1.7 billion alliance with Lexicon on their SGLT1/2 diabetes drug sotagliflozin. And now that the drug has been spurned by the FDA after burning through a program that provided mixed late-stage data and a late shot at a last-place finish, the French pharma giant is forking over another $260 million to get out of the deal.

Sanofi’s unhappiness was already apparent when the company — now under new CEO Paul Hudson — posted a statement back in July that they were dropping the deal. But it wasn’t that simple. 

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.