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.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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News brief­ing: Ve­rastem CMO ex­its two weeks af­ter join­ing com­pa­ny; Ther­mo Fish­er inks $550M M&A deal

Two weeks after joining Verastem Oncology as chief medical officer, Frank Neumann is leaving the company for another job.

Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

FDA hits the brakes on His­to­gen's knee car­ti­lage ther­a­py, ask­ing for more in­fo on man­u­fac­tur­ing process

A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Andrew Allen, Gritstone CEO (Gritstone via website)

Grit­stone con­tin­ues Covid-19 push with deal to de­vel­op 'self-am­pli­fy­ing RNA' vac­cines, as shares con­tin­ue bal­loon­ing

Gritstone Oncology has had a big week, and it’s only Wednesday.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.

Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Bris­tol My­ers Squibb gets re­view date for Op­di­vo com­bo in gas­tric can­cer, look­ing to over­turn Keytru­da's 3-year lead

The past two months have been tough for Bristol Myers Squibb and its checkpoint inhibitor Opdivo after setbacks in lung and brain cancers. But in the battle against Merck’s Keytruda, any success matters — and now Bristol could be looking at a quick approval for Opdivo in an unmatched indication.

The FDA will launch a speedy review of a combination of Bristol Myers Squibb’s Opdivo and chemotherapy to treat first-line patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer or esophageal adenocarcinoma, the drugmaker said Wednesday. The agency set an action date of May 25 for the application.

Covid-19 claims an­oth­er PDU­FA vic­tim as Glax­o­SmithK­line push­es back planned PD-1 roll­out

Bristol Myers Squibb isn’t the only pharma giant that’s been standing in the FDA’s waiting line for site inspections.

GlaxoSmithKline is telling us today that their H2 2020 PDUFA deadline for the PD-1 drug dostarlimab — picked up in its Tesaro buyout — was pushed back due to a delay in the manufacturing site inspection needed for a regulatory decision. And that is forcing the company to revise its timeline.

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