Nu­clear scare to fu­el an­ti-ra­di­a­tion drug sales? PTx buys ex-Sanofi drug just in case

Part­ner Ther­a­peu­tics (PTx), a Boston start­up formed last week by a pair of Mer­ri­mack and Ser­agon ex­ec­u­tives, has made its first move: it’s ac­quired an ex-Sanofi can­cer drug with hopes that the FDA will OK it for ra­di­a­tion poi­son­ing. Like… the kind from an atom­ic bomb or a nu­clear plant dis­as­ter.

Bob Mul­roy

Seems like a rare and un­com­mon event to tar­get, but founder and CEO Bob Mul­roy says it’s best to be pre­pared.

“You would hope you’d nev­er need a drug like this for this kind of in­ci­dent,” Mul­roy told The Boston Globe. “But I think that, should an in­ci­dent like that oc­cur, be­ing pre­pared for it is a very im­por­tant thing.”

The Globe and oth­ers are posit­ing that in­sults slung be­tween Pres­i­dent Trump and North Ko­re­an leader Kim Jong Un are fu­el­ing fears of nu­clear war, lead­ing to de­mand from peo­ple and gov­ern­ments to stock­pile ra­di­a­tion sick­ness drugs.

The med­i­cine is called Leukine, and it’s cur­rent­ly ap­proved to treat acute myeloid leukemia pa­tients fight­ing in­fec­tions af­ter un­der­go­ing bone mar­row trans­plants. But da­ta pre­sent­ed at the 2016 Amer­i­can So­ci­ety of Hema­tol­ogy meet­ing showed Leukine’s abil­i­ty to boost sur­vival in mon­keys ex­posed to ra­di­a­tion if in­ject­ed 48 hours af­ter ex­po­sure. That’s in­ter­est­ing, con­sid­er­ing Am­gen’s ra­di­a­tion sick­ness drugs Ne­u­pogen and Neu­las­ta must be in­ject­ed al­most im­me­di­ate­ly af­ter ex­po­sure to ra­di­a­tion for it to work.

De­ba­sish Roy­chowd­hury

As part of the as­set ac­qui­si­tion deal, PTx al­so got a ded­i­cat­ed bi­o­log­ics man­u­fac­tur­ing fa­cil­i­ty in Lyn­nwood, Wash­ing­ton. The lo­ca­tion will serve as the core man­u­fac­tur­ing and sup­ply chain cen­ter for PTx’s op­er­a­tions, the com­pa­ny said in a state­ment.

An sBLA for the drug was filed in Sep­tem­ber 2017. The ap­pli­ca­tion WAS grant­ed pri­or­i­ty re­view and has a PDU­FA da­ta of March 29, 2018.

While ra­di­a­tion poi­son­ing in the con­text of nu­clear war is cer­tain­ly the most eye-pop­ping ap­pli­ca­tion, PTx has plen­ty of oth­er plans for Leukine’s de­vel­op­ment.

Leukine has shown promise in com­bo stud­ies with Yer­voy (ip­il­i­mum­ab) against melanoma. A 250-pa­tient, ran­dom­ized Phase II study in re­frac­to­ry melanoma in com­bi­na­tion with Yer­voy demon­strat­ed an im­prove­ment in sur­vival over Yer­voy alone, Part­ner says. Leukine is cur­rent­ly in a Phase III tri­al in front-line melanoma in com­bi­na­tion with Yer­voy and Op­di­vo (nivolum­ab).

Leukine is even a hope­ful for Alzheimer’s dis­ease.

“In con­trast to oth­er ap­proved growth fac­tors that stim­u­late one cell type, Leukine’s abil­i­ty to stim­u­late a broad­er va­ri­ety of cells, en­dows it with unique clin­i­cal po­ten­tial to ad­dress se­ri­ous med­ical needs across hema­to­log­ic dis­eases and can­cer as well as in­fec­tious, neu­ro­log­i­cal and pul­monary dis­or­ders,” said De­ba­sish Roy­chowd­hury, CMO at PTx and for­mer Ser­agon CMO, 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.