Pro­tal­ix, Chiesi fire up ac­cel­er­at­ed ap­proval pitch for Fab­ry dis­ease treat­ment

When Pro­tal­ix Bio­Ther­a­peu­tics re­port­ed its 2018 re­sults this March, it spent much of its time talk­ing up the po­ten­tial of nab­bing an ac­cel­er­at­ed ap­proval for its Fab­ry dis­ease treat­ment pe­gu­ni­gal­si­dase al­fa.

Weeks lat­er, the Is­raeli biotech — which has an un­ap­pe­tiz­ing pen­ny stock val­u­a­tion $PLX — and part­ner Chiesi are go­ing for it, op­ti­mistic af­ter months of dis­cus­sion with the FDA. The BLA fil­ing is sched­uled for Q1 2020.

Like Shire’s Re­pla­gal, which it’s be­ing com­pared against in a pair of late-stage tri­als, pe­gu­ni­gal­si­dase al­fa (or PRX-102) is an en­zyme re­place­ment ther­a­py de­signed to break down the ex­cess fat mol­e­cules build­ing up in Fab­ry pa­tients. Syn­the­sized from plants, this par­tic­u­lar re­com­bi­nant al­pha-galac­tosi­dase A en­zyme can be in­fused every four weeks in­stead of Re­pla­gal’s two.

An ini­tial glimpse of the Phase III study showed that high lev­els of the drug cir­cu­lat­ed in the blood­stream af­ter 28 days, with a mean con­cen­tra­tion of 138 ng/mL among 15 evalu­able pa­tients. In con­trast, Fab­razyme — an­oth­er ERT de­vel­oped by Sanofi Gen­zyme — had his­tor­i­cal­ly shown a mean con­cen­tra­tion of 20 ng/mL at 10 hours post in­fu­sion, HC Wain­wright an­a­lysts added in a Feb­ru­ary note.

But those are not the on­ly com­peti­tors Pro­tal­ix will face. Am­i­cus has trav­eled down its own wind­ing path to an ap­proval for Galafold, an oral drug that works by ad­dress­ing some mis­fold­ed α-GalA en­zymes. New ther­a­pies to in­tro­duce healthy copies of the GLA gene, which en­codes for the en­zyme, are com­ing up in the pipeline, with a no­table ef­fort from Avro­bio.

“This reg­u­la­to­ry ap­proval path is a sig­nif­i­cant achieve­ment as it means that we can start the ap­pli­ca­tion process and po­ten­tial­ly at­tain mar­ket ap­proval sig­nif­i­cant­ly ear­li­er than the ini­tial plan of da­ta from our on­go­ing phase III BAL­ANCE clin­i­cal tri­al,” CEO Moshe Manor said in a state­ment. “We plan to con­tin­ue the BAL­ANCE study to fur­ther strength­en the pro­file of pe­gu­ni­gal­si­dase al­fa.”

Chiesi holds the US rights to the drug from a deal that looped in $760 mil­lion in reg­u­la­to­ry and com­mer­cial mile­stones. Pro­tal­ix cur­rent­ly has one ap­proved prod­uct for Gauch­er dis­ease, mar­ket­ed by Pfiz­er.

That 2012 ap­proval, though, has done lit­tle to lift Pro­tal­ix’s suf­fer­ing stock af­ter a con­found­ing de­ci­sion in 2007 to price a sec­ondary of­fer­ing at an 85% dis­count to its trad­ing price the day pri­or. Shares plum­met­ed from $35 to $5 overnight and has bare­ly gone over $10 since, trad­ing way be­low $1 these days.

So­cial im­age: Shut­ter­stock

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology

ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development

CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin and UBS in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.

UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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