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.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Mer­ck KGaA spin­out gets first fund­ing to bring dual-act­ing can­cer mol­e­cules in­to the clin­ic

Two and a half years after launch, Merck KGaA spinout iOnctura is getting its first major round of funding.

The oncology startup raised €15 million ($16.6 million) to put its lead drug into the clinic and get its second drug past IND-enabling tests. INKEF Capital and VI Partners co-led the round and were joined by the biotech’s longtime backer M Ventures, an arm of Merck KGaA, and Schroder Adveq.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Am­gen aug­ments Asia foothold by tak­ing over Astel­las joint ven­ture in Japan

California-based Amgen, which does the bulk of its business in the United States, made its ambition to reinvigorate its growth prospects by expanding its presence in Asia clear at the sidelines of the JP Morgan healthcare conference in San Francisco earlier this month.

The Thousand Oaks-based company on Thursday executed its plan to dissolve the joint venture with Astellas — created in 2013 — to operate the unit independently in Japan. With its rapidly aging population, the region represents an appealing market for Amgen’s osteoporosis treatments Prolia and Evenity as well as a cholesterol-lowering injection Repatha.

Daphne Zohar (PureTech)

PureTech bags $200M from sale of Karuna shares — still siz­zling from promis­ing schiz­o­phre­nia da­ta

Cashing in on the exuberance around Karuna Therapeutics and its potential blockbuster CNS drug, PureTech has sold a chunk of the biotech’s shares to Goldman Sachs for $200 million.

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