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.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.


Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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In a boost to Rit­ux­an fran­chise, Roche nabs quick ap­proval for po­latuzum­ab ve­dotin

Roche’s lat­est an­ti­body-drug con­ju­gate has crossed the FDA fin­ish line, gain­ing an ac­cel­er­at­ed ap­proval a full two months ahead of sched­ule.

Po­livy, or po­latuzum­ab ve­dotin, is a first-in-class drug tar­get­ing CD79b — a pro­tein promi­nent in B-cell non-Hodgkin lym­phoma. It will now be mar­ket­ed for dif­fuse large B-cell lym­phoma as part of a reg­i­men that al­so in­cludes the chemother­a­py ben­damus­tine and a ver­sion of rit­ux­imab (Rit­ux­an).

J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

An in­censed Cat­a­lyst Phar­ma sues the FDA, ac­cus­ing agency of bow­ing to po­lit­i­cal pres­sure and break­ing fed­er­al law

Af­ter hint­ing it was ex­plor­ing the le­gal­i­ty of the FDA’s ap­proval of a ri­val drug from fam­i­ly-run com­pa­ny Ja­cobus Phar­ma­ceu­ti­cals, Cat­a­lyst Phar­ma­ceu­ti­cals on Wednes­day filed a law­suit against the health reg­u­la­tor — ef­fec­tive­ly ac­cus­ing the agency of bow­ing to po­lit­i­cal pres­sure sur­round­ing sky­rock­et­ing drug prices.

Be­fore Cat­a­lyst’s Fir­dapse (which car­ries an av­er­age an­nu­al list price of $375,000) was sanc­tioned for use in Lam­bert-Eaton myas­thenic syn­drome (LEMS) by the FDA, hun­dreds of pa­tients had been able to ac­cess a sim­i­lar drug from com­pound­ing phar­ma­cies for a frac­tion of the cost, or Ja­cobus’ for free, as part of an FDA-rat­i­fied com­pas­sion­ate use pro­gram. But the ap­proval of the Cat­a­lyst drug — ac­com­pa­nied by mar­ket ex­clu­siv­i­ty span­ning sev­en years — ef­fec­tive­ly pre­clud­ed Ja­cobus and com­pound­ing phar­ma­cies from sell­ing their ver­sions.

Plagued by de­lays, As­traZeneca HQ costs soar to £750M as it edges to­ward 2020 com­ple­tion

In the lat­est up­date on As­traZeneca’s de­lay-prone HQ project, the phar­ma gi­ant re­vealed that the cost of con­struc­tion has swelled to £750 mil­lion ($956 mil­lion) — more than dou­ble the orig­i­nal es­ti­mate in 2013.

The move-in date is still in 2020, a spokesper­son con­firmed, af­ter As­traZeneca pushed pro­ject­ed com­ple­tion from 2016 to 2017, and then to the spring of 2019. While the ini­tial plan called for a £330 mil­lion (then $500 mil­lion) in­vest­ment, the cost bal­looned to £500 mil­lion ($650 mil­lion), and more in the most re­cent up­date.