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.

George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Josh Cohen, Justin Klee

Armed with pos­i­tive ALS da­ta, Amy­lyx scores $30M in fresh fund­ing to com­plete Alzheimer's PhII

Four years after announcing themselves to the biotech world with a new idea for drugging neurodegeneration, backing by the late Henri Termeer and $5 million from Morningside Venture, the young entrepreneurs at Amylyx are back for round 2.

Morningside continued to lead the $30 million Series B, with participation from unnamed investors. Having celebrated a topline Phase II win for its lead program in amyotrophic lateral sclerosis, Amylyx expects the cash to fund talks with regulators as well as a separate trial for the same drug in Alzheimer’s — for which they had just finished enrolling.

An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Anthony Johnson, Goldfinch Bio CEO (Goldfinch via YouTube)

Gilead-part­nered Goldfinch Bio lands $100M as it piv­ots to clin­i­cal stage biotech

A year after landing $109 million in cash for an early-stage discovery deal with Gilead, Goldfinch Bio has scored a similar bounty from investors, raising $100 million in the biotech’s first financing round since its 2016 launch.

The new round, led by Eventide Asset Management and joined by 8 other firms, confirms that last year’s Gilead deal began a new, busier, more vocal era for the company. After launching out of Third Rock with $55 million and a plan to tackle kidney disease, they focused on preclinical research and made few material announcements. But the latest press release is full of plans, including their first Phase II study and a new therapy scheduled to enter the clinic next year.