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

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

UP­DAT­ED: New Kaiser analy­sis shows how lim­it­ing price ne­go­ti­a­tions to tar­get­ed drugs may bet­ter fo­cus up­com­ing leg­is­la­tion

As Congress considers whether to adopt sweeping new legislation to lower prescription drug prices across the board, the Kaiser Family Foundation is out with a new report on Monday showing how a more targeted approach on a subset of drugs might be a more efficient way to save government funds.

“This analysis shows that Medicare Part D and Part B spending is highly concentrated among a relatively small share of covered drugs, mainly those without generic or biosimilar competitors,” wrote Juliette Cubanski, deputy director of the program on Medicare policy at KFF, and Tricia Neuman, SVP of KFF. “Focusing drug price negotiation or reference pricing on a subset of drugs that account for a disproportionate share of spending would be an efficient use of administrative resources, though it would also leave some potential savings on the table.”

FDA lays the ham­mer on Emer­gen­t's Bal­ti­more plant af­ter J&J de­ba­cle, halt­ing all pro­duc­tion in un­usu­al move

Emergent BioSolutions has had a tough month: First, the CDMO ruined 15 million doses of J&J’s Covid-19 vaccine in March and then suffered the ignominy of the FDA seizing the reins. Now, as the agency receives a full accounting of the site’s problems, Emergent has slammed the brakes on all production at the FDA’s behest.

Emergent will cease manufacturing at its Baltimore plant until the FDA’s inspection and remediation of any findings is complete, the company said in a statement. Emergent will also quarantine existing materials that have already been manufactured. That stoppage started on Friday, four days after the initiation of the FDA’s inspection.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 107,200+ biopharma pros reading Endpoints daily — and it's free.

Jeremy Levin via YouTube

Ovid cuts its loss­es on con­tro­ver­sial An­gel­man syn­drome drug, mak­ing its crush­ing de­vel­op­ment halt per­ma­nent

After a turbulent couple of years, Ovid is officially doing away with a program it had once championed in the face of heavy analyst skepticism.

Ovid has discontinued development of OV101, or gaboxadol, in Angelman syndrome and will not pursue further clinical trials for Fragile X syndrome, the company announced Monday morning. The news comes after Ovid had previously paused development in Angelman when the compound flunked a Phase III trial in December.

FDA slaps a hold on Mof­fitt’s next-gen CAR-T as reg­u­la­tors de­mand an­oth­er de­lay on clin­i­cal work — shares crater

Close to a year-and-a-half after tapping the brakes on one of its preclinical programs to do some added genetic engineering work on their next-gen CAR-T, Anixa $ANIX Therapeutics says the regulatory light is flashing red on their IND.

The San Jose, CA-based biotech — which changed its name from ITUS in 2018 — explained in late 2019 that they were taking a knee for at least a year so that researchers could go back and amp up the expression of follicle stimulating hormone on T cells to improve targeting of the FSH receptor on a specific set of ovarian cells. That required new vector engineering work by their partners at Moffitt Cancer Center.

Tillman Gerngross (Adagio)

Till­man Gern­gross' Covid-19 an­ti­body moon­shot scores $336M with the help of new ace CFO. Is an IPO next?

Less than a year into its existence, serial biotech entrepreneur Tillman Gerngross’ antibody play Adagio has raced ahead into a pivotal trial for its lead drug for Covid-19 on the back of some very promising preclinical data. Now, crossover investors led by Peter Kolchinsky at RA are rolling up the Brinks truck — and that could spell an IPO in the offing for Adagio.

Adagio has bagged $336 million as part of a Series C round led by RA Capital to advance lead single-shot antibody ADG20 through a pivotal Phase I/II/III trial for the treatment of mild to moderate Covid-19 patients at high risk of infection, the biotech said Monday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 107,200+ biopharma pros reading Endpoints daily — and it's free.

When is a drug re­al­ly a de­vice? Court knocks down FDA ap­peal in try­ing to sort that grey area

It’s always a surprise when a court has to step in to tell the FDA that it erred in performing one of its main duties: classifying whether a medical product is drug or a device.

But that’s what the US Court of Appeals for the District of Columbia did on Friday, making clear to the world’s top drug regulator that Genus Medical Technologies’ contrast agent barium sulfate (also known as Vanilla SilQ) should not be considered a drug, as the FDA had said, but a medical device.

Q1: A flood of in­vestor cash drove biotech's num­bers to new record highs, and the tor­rent of cash is mov­ing up­stream fast

If you thought biotech was booming last year, wait until you get a load of the numbers from Q1 2021.

On virtually every level, with one exception, the money engine was working around the clock in the first 3 months of this year. Venture capital has reached such a fever peak that the average B round now weighs in at an average mega-weight value of $100 million. The money flow is also finding its way to the mouth of the R&D river, where discovery work now merits the big bucks instead of cautionary seed funds.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.