New­com­er Do­va hus­tles a phar­ma castoff through to an FDA ap­proval, shares sink any­way

Less than a year af­ter lit­tle Do­va Phar­ma­ceu­ti­cals popped up on Nas­daq to raise $75 mil­lion from an IPO, the biotech has raced ahead to an FDA ap­proval of its lead drug.

Durham-based Do­va picked up ava­trom­bopag from Ei­sai for a song — plus $5 mil­lion up front. It was al­ready Phase III ready at the time, al­low­ing Do­va to quick march to the mar­ket­place.

Richard Paz­dur

The FDA of­fered a green light to use the drug — to be mar­ket­ed as Doptelet — for a set of chron­ic liv­er dis­ease pa­tients suf­fer­ing from low blood platelet counts.

The news brought on­ly a tem­po­rary lift to its shares $DO­VA, though, which end­ed the day down 13% as a num­ber of biotech stocks went on a roller coast­er ride. Nev­er­the­less, at a lit­tle north of $24, the stock is still well up from its $17 launch price, which hit on the high end of the range.

Leerink’s Ge­of­frey Porges has been crunch­ing the num­bers and be­lieves that Do­va is look­ing at $350 mil­lion in an­nu­al sales by 2025, with a Eu­ro­pean ap­proval pos­si­ble next sum­mer along with some la­bel ex­ten­sions to help boost its rev­enue. He notes:

This ap­proval of Do­va’s first and on­ly drug is ex­act­ly on the FDA’s pri­or­i­ty re­view PDU­FA date and matched in­vestors’ gen­er­al­ly pos­i­tive views of the like­li­hood of the ap­proval. The la­bel re­flects the ef­fi­ca­cy and safe­ty of the two piv­otal tri­als, ADAPT-1 and ADAPT-2, does not in­clude a Black Box warn­ing for he­pa­to­tox­i­c­i­ty, and lists the clin­i­cal ex­pe­ri­ence of throm­bot­ic com­pli­ca­tions (0.2% of pa­tients) as a warn­ing for high-risk pa­tients. Do­va has hired all sales, mar­ket­ing, and re­im­burse­ment sup­port re­sources and is ready to launch in June, and the price will not be dis­closed un­til that time (we an­tic­i­pate $6,000).

“Doptelet was demon­strat­ed to safe­ly in­crease the platelet count,” said the FDA’s Richard Paz­dur, step­ping in as act­ing di­rec­tor of the Of­fice of Hema­tol­ogy and On­col­o­gy Prod­ucts. “This drug may de­crease or elim­i­nate the need for platelet trans­fu­sions, which are as­so­ci­at­ed with risk of in­fec­tion and oth­er ad­verse re­ac­tions.”

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

FDA pan­el large­ly op­pos­es ex­pand­ing use of Lil­ly, Boehringer's SGLT2 in­hibitor to type I di­a­bet­ics

Last week, Eli Lilly and partner Boehringer Ingelheim rejigged the terms of their 2011 diabetes pact that nurtured the development of their blockbuster Jardiance franchise. Akin to manufacturers of rival SGLT2 drugs, the companies are working to expanding the use of their type II diabetes drug to reach a broader group of patients. On Wednesday, an expert panel to the FDA resisted that effort by largely voting against their quest to market the drug in type I diabetics.

Amir Nashat, World Medical Innovation Forum via Youtube

Bay­er bets up to $100M on ex­plor­ing new bio­mol­e­c­u­lar con­den­sate ter­rain with a biotech up­start

In the Indiana Jones warehouse of genomic oddities, the millions of units of so-called “junk DNA” that create nothing but play a hand in tons of things have grabbed most of the attention. But there are other arks and Templar crosses out there.

Among them: the code for intrinsically disordered regions. Floating like boundless clumps of boiling spaghetti throughout the cell, these regions first appeared in scientific sketches at the turn of the century before vanishing from most cell diagrams, such as those in a high school textbook. Most organelles were neatly bound in membranes. These loose molecules resisted characterization. Scientists largely ignored them.

In­vestors could emerge from Neil Wood­ford de­ba­cle with £1B loss, in­ter­nal analy­sis re­veals

When Link Fund Solutions announced that it is closing Woodford Equity Income Fund permanently and kicking out Neil Woodford, it was implied that investors probably won’t get back everything they entrusted to the fund manager. But nobody knew just how much they would lose.

An internal analysis commissioned by Link suggested that the collective loss could amount to £1 billion — out of a fund last valued at £3.1 billion — Citywire has revealed.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.