Do­va Phar­ma's bet on Ei­sai castoff spells gold as So­bi spends mil­lions to ac­quire it

In 2016, Do­va Phar­ma­ceu­ti­cals paid Japan’s Ei­sai a pal­try $5 mil­lion up­front for the rights to the Phase III-ready ava­trom­bopag. The fol­low­ing year, the Durham, North Car­oli­na-based com­pa­ny made its pub­lic de­but with a $75 mil­lion IPO, and by 2018 the drug — brand­ed as Doptelet — se­cured FDA ap­proval. Do­va’s bet paid off hand­some­ly — on Mon­day, the com­pa­ny un­veiled Swedish Or­phan Biovit­rum AB was ac­quir­ing its shares in a deal worth up to a ro­bust $915 mil­lion.

At the cen­ter of the buy­out is the drug, Doptelet, which was cleared last year as a treat­ment for cer­tain pa­tients chron­ic liv­er dis­ease pa­tients suf­fer­ing from low blood platelet counts. Months ago, that ap­proval was ex­pand­ed to in­clude pa­tients with chron­ic im­mune throm­bo­cy­tope­nia (ITP), who have not ben­e­fit­ed from ini­tial treat­ment.

The ther­a­py is an oral throm­bopoi­etin (TPO) re­cep­tor ag­o­nist — throm­bopoi­etin is a hor­mone pro­duced by the liv­er and kid­ney that reg­u­lates platelet pro­duc­tion.

The on­ly oth­er oral TPO ap­proved for ITP is No­var­tis’ $NVS Pro­mac­ta, which is an­nu­al­iz­ing at $600 mil­lion-plus, main­ly in ITP pa­tients. There is al­so an in­jectable TPO — Am­gen’s Nplate —which brings in rough­ly $450 mil­lion in US sales, not­ed Ever­core ISI’s Umer Raf­fat ear­li­er this year.

Un­like Pro­mac­ta, Doptelet does not car­ry a black box warn­ing for he­pa­tox­i­c­i­ty, nor does is it re­quired to be tak­en one hour be­fore, or two hours post a meal — and both prod­ucts are priced at par­i­ty at the most rec­om­mend­ed dose, he said.

“No­var­tis re­mains the in­cum­bent and a huge play­er.  We’re not sug­gest­ing by any means that Do­va’s Doptelet takes over the TPO class.  How­ev­er, it’s not un­re­al­is­tic to ex­pect a path to­wards ~$200M ITP peak … es­pe­cial­ly al­so giv­en how ITP pts cy­cle off NVS Pro­mac­ta.”

Doptelet — which is sold at $9,000 (40 mg) and $13,500 (60 mg) whole­sale price — gen­er­at­ed $3.5 mil­lion in sec­ond-quar­ter sales. Do­va is on the hook, how­ev­er, to pay Ei­sai $135 mil­lion in net sales-based mile­stone pay­ments.

So­bi has agreed to pay $27.50 per Do­va share, which rep­re­sents a pre­mi­um of 36% to Do­va’s Fri­day clos­ing. The trans­ac­tion — which al­so in­cludes an ad­di­tion­al $1.50 per share in cash, once Doptelet is sanc­tioned for use in chemother­a­py-in­duced throm­bo­cy­tope­nia (CIT) — is ex­pect­ed to close in the fourth quar­ter of 2019.

Do­va’s shares $DO­VA jumped 27.4% to $27.75 in Mon­day pre­mar­ket trad­ing.

Top-line da­ta from a late-stage tri­al in CIT is ex­pect­ed in the first half of next year.

“Cur­rent­ly, there are no ap­proved phar­ma­co­log­i­cal op­tions to treat CIT, and on­ly a frac­tion of pa­tients, re­ferred to high risk of bleed­ing, may un­der­go a platelet trans­fu­sion or SoC pro­ce­dure in the hos­pi­tal,” HC Wain­wright’s Joseph Pant­gi­nis wrote in a note on Mon­day.

“How­ev­er, SoC comes with sev­er­al short­com­ings, which in­clude: (1) pa­tients need to be hos­pi­tal­ized; (2) dif­fi­cul­ties in find­ing the cor­rect units; and (3) the oc­cur­rence of lung and sys­temic in­flam­ma­to­ry episodes, fever, hy­poten­sion, and dif­fuse in­travas­cu­lar co­ag­u­la­tion.”

There are rough­ly 765,000 pa­tients an­nu­al­ly who re­ceive chemother­a­py, of which 10% may de­vel­op se­vere CIT — un­der the cur­rent pric­ing ap­proach, man­age­ment es­ti­mates a $2 bil­lion mar­ket in the Unit­ed States alone, he added. “We think these fig­ures could be con­ser­v­a­tive. Our es­ti­mates and due dili­gence found that 21.8% of pa­tients un­der chemo de­vel­oped se­vere CIT and 15% had re­ceived SoC (stan­dard of care).”

Jef­feries an­a­lysts, in a note last month, fore­cast US peak sales in the in­di­ca­tion (if ap­proved) to hit $600 mil­lion.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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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.

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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Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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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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Wuhan virus out­break trig­gers in­evitable small-biotech ral­ly

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.