Con­tend­ing for CD47 throne, Arch On­col­o­gy scores $50M as it ramps up PhI stud­ies

Arch On­col­o­gy, one of the old­er star­tups in a crop of im­muno-on­col­o­gy play­ers chas­ing af­ter the CD47 tar­get, has bagged $50 mil­lion to fu­el a long dri­ve in the clin­ic that re­cent­ly be­gan.

Julie Cher­ring­ton

Some might re­mem­ber the com­pa­ny as Tioma Ther­a­peu­tics, break­ing out in 2016 with some years of pre­clin­i­cal work and in­vest­ments from Glax­o­SmithK­line, Roche and No­vo. A year lat­er, it re­named it­self to Arch On­col­o­gy and brought in new CEO Julie Cher­ring­ton to re­place John Dono­van in pur­suit of a “more high­ly dif­fer­en­ti­at­ed” com­pound.

In the process, River­Vest Ven­ture Part­ners, Roche Ven­ture Fund and 3×5 Part­ners be­came the main back­ers, all of whom par­tic­i­pat­ed in the Se­ries B, led by new in­vestor Lightchain. Lightchain is the fam­i­ly of­fice of Rodger Riney, a bil­lion­aire, mul­ti­ple myelo­ma sur­vivor and St. Louis res­i­dent who’s pre­vi­ous­ly do­nat­ed to the Wash­ing­ton Uni­ver­si­ty School of Med­i­cine — where Bris­bane, CA-based Arch had its sci­en­tif­ic roots.

Both the cor­po­rate/clin­i­cal de­vel­op­ment team in the Bay Area and the re­search group at St. Louis will be ex­pand­ing in the com­ing year, bring­ing the to­tal head­count from 20 to 30, Cher­ring­ton tells me.

The big idea at Arch On­col­o­gy — pop­u­lar­ized by the likes of Forty Sev­en and Boehringer In­gel­heim — is to block the “don’t eat me” sig­nal emit­ted by CD47 and there­by help im­mune cells spot and de­stroy can­cer.

What makes its lead pro­gram dif­fer­ent, the biotech says, is that in ad­di­tion to tear­ing down the bar­ri­er for macrophages and in­cit­ing a T cell re­sponse, AO-176 al­so works by di­rect­ly killing tu­mor cells, fur­ther ac­ti­vat­ing the adap­tive im­mune re­sponse.

The hy­poth­e­sis is now be­ing test­ed in a Phase I tri­al, with a slate of dis­cov­ery-stage can­di­dates lin­ing up to fol­low. Pro­grams range from SIR­Pα (a pro­tein on the sur­face of macrophages that’s key to CD47 sig­nalling) to oth­er tar­gets har­ness­ing the pow­er of in­nate im­mu­ni­ty, ac­cord­ing to Cher­ring­ton.

“We be­lieve AO-176 has a best-in-class pro­file among agents in the an­ti-CD47 space and we are ex­cit­ed to see the progress ad­vanc­ing the pipeline,” John McK­earn, man­ag­ing di­rec­tor of River­Vest and chair­man of Arch On­col­o­gy, said in a state­ment.

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

UP­DAT­ED: In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

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UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Stephen Hahn (via Senate HELP Committee)

Stephen Hahn gets through Sen­ate’s soft­ball job in­ter­view — but most­ly plays dodge­ball on the is­sues fac­ing the FDA

Anyone looking for fresh insights on what kind of FDA commissioner Stephen Hahn will be got precious few clues during Wednesday’s Senate hearing on the nomination.

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Op­di­vo/Yer­voy com­bo for melanoma fails in key pa­tient pop­u­la­tion

Bristol-Myers Squibb’s efforts to expand their checkpoint inhibitor combination have run into another recalcitrant cancer.

The NJ-based pharma announced that a combination of Yervoy and Opdivo didn’t beat out Opdivo alone in patients with resected high-risk melanoma who had very low levels of PD-L1. The drug combo couldn’t improve recurrence-free survival in these post-surgery patients.

Ver­tex's stel­lar quar­ter car­ries on with French re­im­burse­ment deal

Vertex’s golden quarter just got brighter. About a month after the US drugmaker finally clinched a deal with UK authorities to cover its slate of cystic fibrosis (CF) drugs following years of protracted negotiations, the company on Wednesday secured a deal with France for its CF therapy, Orkambi.

After the UK, France has one of the largest CF populations outside the United States. Achieving French reimbursement unlocks an ~7000-patient CF population, around ~2500-3000 of which will likely be eligible to receive (and be reimbursed for) Orkambi, Stifel’s Paul Matteis wrote in a note.

Nello Mainolfi, Kymera via Youtube

Kymera hands the helm to No­var­tis vet — and found­ing CSO — Nel­lo Main­olfi

Kymera Therapeutics is turning to a co-founder to run the company.
The protein degradation specialist with a deep-pocket syndicate behind them has opted to give the helm officially to Nello Mainolfi. The new CEO is a veteran of the Novartis Institutes for Biomedical Research. He joined Atlas Venture in their entrepreneur-in-residence program and helped launch Kymera as the CSO three years ago with Atlas’ Bruce Booth.
The boast at Kymera is that they’re angling to create a new class of protein degraders, a popular field where there’s been a variety of startups. One of its chief advocates is NIBR head Jay Bradner, who launched C4 just ahead of joining Novartis, where he’s also been doing new work in the field.