Step­ping on Bio­Mar­in's turf, PTC Ther­a­peu­tics picks up PKU drug for a song

While in­dus­try-shak­ing M&A deals are still nowhere in sight, small­er deals by bar­gain shop­pers con­tin­ue to trick­le in. PTC Ther­a­peu­tics has joined the club, bag­ging Cen­sa Phar­ma­ceu­ti­cals and its rare meta­bol­ic dis­ease drug.

Stu­art Peltz

The up­front cash comes in at just $10 mil­lion, though Cen­sa in­vestors might find more val­ue in the eq­ui­ty por­tion of the deal: 850,000 shares of PTC $PTCT, worth a lit­tle over $40 mil­lion if you take the clos­ing price on Wednes­day. An ad­di­tion­al $217.5 mil­lion is re­served for de­vel­op­ment and sales mile­stones for the two lead in­di­ca­tions of CN­SA-001.

With its oral spinal mus­cu­lar at­ro­phy ther­a­py — joint­ly de­vel­oped by Roche and the SMA Foun­da­tion — wide­ly ex­pect­ed to be ap­proved lat­er this year, PTC now gets to launch a new Phase III pro­gram in phenylke­tonuria.

Caused by mu­ta­tions in the pheny­lala­nine hy­drox­y­lase gene, the dis­ease is char­ac­ter­ized by tox­ic buildup of the amino acid pheny­lala­nine (Phe) in the brain.

As with SMA, even if it all goes well, PTC will be step­ping on the turf of a much larg­er com­peti­tor. Cowen an­a­lyst Joseph Thome sum­ma­rized it this way:

The PKU mar­ket is rel­a­tive­ly com­pet­i­tive head­lined by Bio­Marin’s Ku­van and Pa­lynz­iq, with gene ther­a­py ap­proach­es in ear­ly clin­i­cal de­vel­op­ment. In ad­di­tion, gener­ic ver­sions of Ku­van are set to launch lat­er this year, which may yield ad­di­tion­al re­im­burse­ment hur­dles. How­ev­er, non-re­spon­ders to gener­ic Ku­van should be iden­ti­fied fair­ly quick­ly should treat­ment fail­ure be re­quired ahead of treat­ment with a brand­ed agent.

Giv­en that on­ly rough­ly 30% to 40% of pa­tients re­spond to Ku­van, there is room for a new op­tion, he added.

The Bio­Marin drug re­places BH4, the PAH en­zyme co­fac­tor that’s de­fi­cient among pa­tients with PKU. Cen­sa’s ap­proach goes a lit­tle up­stream of the path­way, giv­ing pa­tients an oral for­mu­la­tion of syn­thet­ic sepi­apterin, which is a pre­cur­sor to BH4 in the cell.

In a Phase II tri­al where it was com­pared to Ku­van, CN­SA-001 hit the sta­tis­ti­cal thresh­old for re­duc­ing blood Phe con­cen­tra­tion.

PTC is now tak­ing over the late-stage work from Cen­sa, which was backed by Is­rael’s Arkin Bio Ven­tures, though it not­ed there is no oblig­a­tion to hire any of their staffers.

“We be­lieve that CN­SA-001 has the po­ten­tial to ad­dress the ma­jor­i­ty of PKU pa­tients whose con­di­tion is not ad­e­quate­ly man­aged by cur­rent treat­ments,” CEO Stu­art Peltz said in a state­ment.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Bris­tol-My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Caught in a stand­off with its con­tract man­u­fac­tur­er over Covid-19 vac­cine, In­ovio files suit in an at­tempt to break free while ri­vals race ahead

Inovio was one of the first vaccine developers to snag attention for a jab that their execs said promised to end the Covid-19 pandemic. Using their own unique DNA tech, CEO Joseph Kim said it took just 3 hours to work it out.

But while rivals are racing to the finish line with ambitious plans to make vast quantities of their vaccines with billions of dollars of deals, Inovio is still stuck at the starting line on manufacturing.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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