Take­da fore­casts $1.7B loss af­ter Shire in­te­gra­tion; Flag­ship's pre­clin­i­cal biotech Ax­cel­la clos­es IPO with $71M+ haul

Take­da’s $TAK first set of fi­nan­cial re­sults since con­sum­mat­ing its $62 bil­lion ac­qui­si­tion of Shire is in, and it’s giv­ing an­a­lysts a neg­a­tive sur­prise. Cit­ing costs for in­te­grat­ing the Ire­land-based Shire, the Japan­ese drug­mak­er fore­casts an op­er­at­ing loss of $1.7 bil­lion (193 bil­lion yen) for the year through March 2019 — where an­a­lysts sur­veyed by Re­fini­tiv es­ti­mat­ed a prof­it of $2.08 bil­lion (227.5 bil­lion yen), ac­cord­ing to Reuters. The pre­dic­tions come as Take­da at­tempts to re­lieve its mas­sive debt bur­den by di­vest­ing its non-core as­sets, in­clud­ing a dry eye drug and a sur­gi­cal patch sold to No­var­tis and J&J last week, re­spec­tive­ly.

→ About a year af­ter lur­ing No­var­tis ex­ec­u­tive Bill Hin­shaw to run its op­er­a­tions, Flag­ship Pi­o­neer­ing-backed Ax­cel­la Health closed its IPO on Mon­day, rais­ing $71.4 mil­lion to fund the de­vel­op­ment of its pre­clin­i­cal pipeline of drugs de­signed to mod­u­late meta­bol­ic ac­tiv­i­ty.

Bill Hin­shaw

Ax­cel­la has worked on pre­clin­i­cal mod­els to de­ter­mine which com­bi­na­tion of amino acids can re­pair dys­reg­u­lat­ed meta­bol­ic path­ways — and the plat­form has de­liv­ered pro­grams for liv­er, meta­bol­ic, CNS, and or­phan dis­eases. The com­pa­ny’s lead ex­per­i­men­tal drug is be­ing de­vel­oped for he­pat­ic en­cephalopa­thy, which are neu­ropsy­chi­atric ab­nor­mal­i­ties as­so­ci­at­ed with pa­tients suf­fer­ing from liv­er dys­func­tion, typ­i­cal­ly chron­ic liv­er dis­ease.

The Cam­bridge, Mass­a­chu­setts-based biotech — that com­menced trad­ing un­der the sym­bol $AXLA on May 9 — sold about 3.6 mil­lion shares at $20, the low end of the range of $20 to $22. Apart from Flag­ship, which owns a meaty 42.5% of Ax­cel­la, Fi­deli­ty In­vest­ments has a 13.1% share and Nestlé Health Sci­ences US Hold­ings has a 10.4% stake.

Er­ic Os­tertag

→ Fresh off a $142 mil­lion ven­ture raise — in lieu of an IPO — Po­sei­da Ther­a­peu­tics is now armed with an or­phan drug des­ig­na­tion as it heads in­to the BC­MA fren­zy. The San Diego-based biotech, backed by No­var­tis and the Cal­i­for­nia In­sti­tute for Re­gen­er­a­tive Med­i­cine, is de­vel­op­ing an au­tol­o­gous CAR-T made with stem cell mem­o­ry T cells. “P-BC­MA-101 has demon­strat­ed out­stand­ing po­ten­cy, with strik­ing­ly low rates of tox­i­c­i­ty in our phase 1 clin­i­cal tri­al,” said CEO Er­ic Os­tertag in a state­ment. “In fact, the FDA has ap­proved ful­ly out­pa­tient dos­ing in our Phase II tri­al start­ing in the sec­ond quar­ter of 2019.”

On­cono­va has picked up a few mil­lion in cash to fund the Phase III pro­gram for its lead drug, rigosert­ib, by li­cens­ing the myelodys­plas­tic syn­dromes treat­ment to its part­ner in Chi­na. HanX is dish­ing out $2 mil­lion in cash and com­mit­ting $2 mil­lion to buy On­cono­va eq­ui­ty at a pre­mi­um, in ex­change for de­vel­op­ment and com­mer­cial­iza­tion rights in the re­gion. The duo first teamed up in 2017 over a pre­clin­i­cal CDK 4/6 in­hibitor.

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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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.

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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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.