Months away from com­plet­ing a key sick­le cell tri­al, up­start Imara scores $63M in round co-led by Ar­ix, Or­bimed

A Cam­bridge, Mass­a­chu­setts-based sick­le cell dis­ease start­up that launched about three years ago has sparked the in­ter­est of UK life sci­ences com­pa­ny Ar­ix, which has agreed to co-lead a $63 mil­lion round of fi­nanc­ing for the drug de­vel­op­er Imara a few months ahead of the com­ple­tion of a key Phase II study.

Rahul Bal­lal

Imara burst in­to the biotech zeit­geist in June 2016 — as a col­lab­o­ra­tion be­tween or­phan drug ac­cel­er­a­tor Cy­dan De­vel­op­ment and Lund­beck — with $31 mil­lion in Se­ries A fund­ing from a slew of in­vestors, in­clud­ing New En­ter­prise As­so­ci­ates and Pfiz­er Ven­ture In­vest­ments. Since then it has en­list­ed the ser­vices of Rahul Bal­lal — who is tap­ping his busi­ness de­vel­op­ment ex­pe­ri­ence at North­ern Bi­o­log­ics and Flex­ion Ther­a­peu­tics and his VC chops from Ver­sant Ven­tures and No­var­tis Ven­ture Fund — in his role as CEO.

Imara is fo­cused on its sole ex­per­i­men­tal drug, IMR-687, for sick­le cell dis­ease (SCD) — a group of in­her­it­ed red blood cell dis­or­ders that large­ly af­flict those of African an­ces­try. SCD pa­tients have atyp­i­cal he­mo­glo­bin mol­e­cules, which can dis­tort red blood cells in­to a sick­le, or cres­cent, shape. Symp­toms such as ane­mia, re­peat­ed in­fec­tions and pe­ri­od­ic episodes of sear­ing pain be­gin to ap­pear in ear­ly child­hood. These episodes de­prive the body of oxy­gen-rich blood, which can cul­mi­nate in wide­spread tis­sue and or­gan dam­age, par­tic­u­lar­ly in the lungs, kid­neys, spleen, heart and brain, and dras­ti­cal­ly di­min­ish life ex­pectan­cy.

IMR-687 — an in­hibitor of phos­pho­di­esterase-9 (PDE9i) in blood cells — is de­signed to to treat the un­der­ly­ing caus­es of SCD by tar­get­ing the same bio­chem­i­cal path­way as stan­dard sick­le cell treat­ment (and chemother­a­peu­tic agent) hy­drox­yurea, but sans its safe­ty is­sues. Hy­drox­yurea makes the red blood cells big­ger, help­ing them stay rounder and more flex­i­ble — and makes them less like­ly to turn in­to a sick­le shape. In an­i­mal mod­els, IMR-687 has been shown to in­crease fe­tal glo­bin, sub­vert­ing the poly­mer­iza­tion of the sick­led he­mo­glo­bin — there­by re­duc­ing red blood cell sick­ling, red blood cell death and the oc­clu­sion of blood ves­sels. PDE9 in­hi­bi­tion al­so di­min­ish­es white blood cell “stick­i­ness” which fur­ther lessens the block­age of blood ves­sels, Imara said.

The drug is cur­rent­ly be­ing test­ed in a mid-stage study, which is ex­pect­ed to be con­clud­ed by June. Ini­tial da­ta is ex­pect­ed by the sec­ond half of this year, and the full read­out in the first quar­ter of 2020, an Ar­ix spokesper­son told End­points News.

Oth­er than hy­drox­yurea — which has been used for decades for sick­le cell — Em­maus Med­ical’s En­dari was ap­proved in 2017 for adult and pe­di­atric pa­tients to re­duce the acute com­pli­ca­tions of sick­le cell dis­ease. Oth­er de­vel­op­ers, in­clud­ing No­var­tis $NVS, Glob­al Blood Ther­a­peu­tics $GBT, CRISPR Ther­a­peu­tics $CR­SP/Ver­tex $VRTX and blue­bird bio $BLUE (whose gene ther­a­py-in-de­vel­op­ment gar­nered a flur­ry of at­ten­tion in a re­cent 60 min­utes episode) are al­so de­vel­op­ing drugs for the or­phan dis­ease that af­fects an es­ti­mat­ed 100,000 Amer­i­cans.

Mark Chin

The Se­ries B round for Imara was co-led by new in­vestors Ar­ix and Or­bimed Ad­vi­sors, and in­clud­ed the par­tic­i­pa­tion of RA Cap­i­tal and Rock Springs Cap­i­tal, as well as ex­ist­ing in­vestors NEA, Pfiz­er Ven­tures, Bay City Cap­i­tal, Lund­beck­fonden Ven­tures and Alexan­dria Ven­ture In­vest­ments. The in­jec­tion will be used for clin­i­cal de­vel­op­ment for the sick­le cell pro­gram, as well as broad­er ap­pli­ca­tions in be­ta tha­lassemia and oth­er haema­to­log­i­cal con­di­tions.

As part of the fi­nanc­ing, Ar­ix has com­mit­ted to in­vest $15 mil­lion for a 10% stake in Imara and Ar­ix’s in­vest­ment di­rec­tor Mark Chin will join Imara’s board, it said on Mon­day.

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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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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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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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

UP­DAT­ED: 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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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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