Mixed da­ta does­n't stop this biotech uni­corn from plot­ting a loom­ing PhI­II show­down

Sa­mumed has of­ten found it eas­i­er to come up with lots more in­vestor cash than con­sis­tent­ly pos­i­tive clin­i­cal da­ta. And that odd jux­ta­po­si­tion has nev­er been more ap­par­ent than in its lat­est read­out for their lead drug in the pipeline.

Two months af­ter rais­ing a jaw-drop­ping $438 mil­lion at a $12 bil­lion val­u­a­tion, the biotech has come up with a dis­tinct­ly mixed batch of Phase IIb da­ta on SM04690. But that won’t stop this uni­corn from bar­rel­ing in­to a late-stage study in search of an FDA OK.

The drug — a Wnt path­way in­hibitor — is de­signed for os­teoarthri­tis, fit­ting eas­i­ly in­to a broad pipeline that in­cludes ther­a­pies of every­thing from hair loss to pso­ri­a­sis and Alzheimer’s.

In this lat­est study, in­vestors in­ject­ed the drug di­rect­ly in­to the knees of pa­tients feel­ing con­sid­er­able pain from their con­di­tion — with the in­ves­ti­ga­tors look­ing close­ly at pa­tient re­port­ed out­comes.

One of their dos­es — 0.23 mg — hit for pain and func­tion in sta­tis­ti­cal­ly sig­nif­i­cant PRO rat­ings. One low­er dose hit on one mea­sure for pain while two dos­es didn’t hit any­thing. And all 4 dos­es failed to score on a key pri­ma­ry out­come: an im­prove­ment in me­di­al joint space width over 24 weeks, though they hit it at 52 weeks in an ear­li­er study.

Com­pa­ny ex­ecs told Busi­ness In­sid­er that the 24-week study was just too short a pe­ri­od to ex­pect a sig­nif­i­cant im­prove­ment, which they plan to fac­tor in­to the Phase III.

If it was a pub­lic com­pa­ny, that mixed da­ta would like­ly trig­ger some nau­sea in in­vestors. But Sa­mumed is still no­tably pri­vate, so they don’t have to man­age any mar­ket symp­toms for now.

“The safe­ty pro­file, the im­prove­ments in signs and symp­toms, and the dis­ease-mod­i­fy­ing ben­e­fits of SM04690 that we have ob­served in this Phase IIb as well as ear­li­er stud­ies, are quite promis­ing,” said Os­man Kibar, CEO of Sa­mumed. “We look for­ward to work­ing with the FDA to take SM04690 in­to Phase III piv­otal tri­als, tar­get­ing a start date in ear­ly 2019.”

Im­age: Os­man Kibar

An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Mer­ck Serono’s Se­nior Vice Pres­i­dent and Glob­al Head of On­col­o­gy
EL­LIOTT LEVY — Am­gen’s Se­nior Vice Pres­i­dent of Glob­al De­vel­op­ment
CHRIS BOSHOFF — Pfiz­er On­col­o­gy’s Chief De­vel­op­ment Of­fi­cer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.

Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future. 

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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