PTC shares rock­et up as Eu­ro­pean reg­u­la­tors of­fer a thumbs-up on Duchenne drug mar­ket­ing

PTC Ther­a­peu­tics CEO Stu­art Peltz

A few days af­ter warn­ing in­vestors of the ris­ing un­cer­tain­ty it faced in the on­go­ing Eu­ro­pean re­view of its con­di­tion­al ap­proval for Translar­na, shares of PTC Ther­a­peu­tics $PTCT sky­rock­et­ed this morn­ing af­ter reg­u­la­tors gave it a thumbs up on keep­ing the con­tro­ver­sial Duchenne drug on the mar­ket.

There is a con­di­tion at­tached to the rec­om­men­da­tion from the Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use. The CHMP wants PTC to con­duct a place­bo-con­trolled, 18-month study of their drug fol­lowed by an 18-month ex­ten­sion study, with da­ta ex­pect­ed in ear­ly 2021. That would give them a clear mar­ket­ing run­way of close to 5 more years, if the EMA for­mer­ly ac­cepts the rec­om­men­da­tion.

PTC’s shares, beat­en down by the skep­ti­cism is faced, shot up more than 100% on the news.

PTC skep­tics abound. Its drug has re­peat­ed­ly failed clin­i­cal tri­als and a num­ber of an­a­lysts have spec­u­lat­ed that EU reg­u­la­tors would jerk the drug, which was giv­en a con­di­tion­al ap­proval back in 2014, ahead of a failed Phase III study. The FDA re­cent­ly re­fused to even con­sid­er its ap­pli­ca­tion, say­ing PTC had too weak a case to mer­it a thor­ough re­view.

PTC is ap­peal­ing that de­ci­sion, look­ing to get its drug and its pa­tient ad­vo­cates in front of the same reg­u­la­tors that gave Sarep­ta an ap­proval for Ex­ondys 51, even though in­sid­ers at the agency waged a hard fought cam­paign in­sist­ing that the drug had nev­er come close to meet­ing the FDA’s stan­dards for an ap­proval.

But Duchenne mus­cu­lar dy­s­tro­phy is a field un­to it­self, where the hard log­ic and high reg­u­la­to­ry bar that of­ten trig­gers drug re­jec­tions have been blunt­ed by pa­tient ad­vo­cates des­per­ate to have some drug avail­able that might slow a dis­ease that grad­u­al­ly kills its young vic­tims. That de­sire to have some­thing, any­thing, avail­able has been a boon to PTC and Sarep­ta, even as crit­ics shake their heads at the idea of ex­per­i­men­tal drugs with lit­tle ev­i­dence of ef­fi­ca­cy be­ing sold to pa­tients for six-fig­ure prices.

Bar­clays fol­lowed up with a dis­cus­sion with PTC ex­ecs and came away with this:

In speak­ing with man­age­ment, it is clear that there was sig­nif­i­cant dis­cus­sion and en­gage­ment with EMA over the past year. CHMP de­ter­mined that the drug does slow pro­gres­sion and has a fa­vor­able risk ben­e­fit pro­file. The com­pa­ny has not yet dis­closed oth­er as­pects of the tri­al, name­ly end­points and in­clu­sion/ ex­clu­sion cri­te­ria, but not­ed that the tri­al will be sim­i­lar in size to ACT DMD (230 en­roll­ment). Man­age­ment em­pha­sized that this up­date on­ly per­tains to EMA, as FDA dis­cus­sions are still on­go­ing. While the com­pa­ny would like to align tri­al ef­forts be­tween the EU and US, they not­ed that they hope that Ata­lau­ren would be avail­able in the US well be­fore the 2021 read out of this new con­fir­ma­to­ry tri­al. Giv­en the high lev­el of un­cer­tain­ty around nmD­MD in the US we con­tin­ue to as­sign no val­ue in our NPV analy­sis.

“We are pleased with this out­come which took in­to ac­count all avail­able da­ta for Translar­na,” said Stu­art W. Peltz, PhD, Chief Ex­ec­u­tive Of­fi­cer, PTC Ther­a­peu­tics, Inc. “This de­ci­sion re­flects the ben­e­fit that Translar­na is hav­ing for pa­tients suf­fer­ing from non­sense mu­ta­tion Duchenne mus­cu­lar dy­s­tro­phy.”

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,600+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: The FDA sets a reg­u­la­to­ry speed record, pro­vid­ing a snap OK for Ver­tex's break­through triplet for cys­tic fi­bro­sis

The FDA has approved Vertex’s new triplet for cystic fibrosis at a record-setting speed.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,600+ biopharma pros reading Endpoints daily — and it's free.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,600+ biopharma pros reading Endpoints daily — and it's free.

That $335M JV Bay­er set up on CRISPR/Cas9? They’re let­ting the biotech part­ner car­ry on

Bayer committed $300 million to set up a joint venture on CRISPR/Cas9 tech with CRISPR Therapeutics $CRSP. But they’re handing off control now to the smaller biotech while retaining a couple of opt-ins for programs nearing an IND.

Bayer $BAY made much of the fact that they were going all-in on gene editing when they did their deal 3 years ago with CRISPR Therapeutics, which pitched $35 million in on their end. This was the cornerstone of their plan to set up new JVs that could make some serious leap forwards in hot new R&D spaces. Now CRISPR will have full management control of Casebia as they pursue programs in hemophilia, ophthalmology and autoimmune diseases.
Samarth Kulkarni, the CEO at CRISPR, made it sound like a natural progression.

J&J's block­buster Ste­lara wins US ap­proval for ul­cer­a­tive col­i­tis

J&J’s Stelara, which is set to be in the top ten list of blockbusters come 2025, is now cleared by the FDA for use in ulcerative colitis (UC), an inflammatory disease of the large intestine.

The biologic targets interleukin (IL)-12 and IL-23 cytokines, which are known to play a key role in inflammatory and immune responses. Stelara, which generated about $4.7 billion in the first nine months of 2019, is a key player in the crowded marketplace of drugs to treat autoimmune disorders such as psoriasis, rheumatoid arthritis and Crohn’s disease. AbbVie’s star therapy, Humira, continues to dominate, despite its looming patent cliff in the United States, while others including J&J’s $JNJ own anti-IL23 Tremfya, Lilly’s $LLY anti-IL-17 Taltz and AbbVie’s $ABBV recently approved anti-IL-23 antibody Skyrizi carve out a slice of market share.

Drug com­pa­nies reach $260M set­tle­ment just ahead of opi­oid tri­al; Oys­ter Point set terms for $85M IPO

→ Hours before the first federal opioid trial was set to begin, three drug distributors and an opioid manufacturer agreed to a $260 million agreement settlement, the Wall Street Journal was the first to report. The deal — which will see McKesson, Cardinal Health and AmerisourceBergen pay $215 million to Summit and Cuyahoga counties, and Teva deal out $35 million in cash and addiction treatments — does not resolve the pending, nationwide litigation that may result in a settlement worth upwards of $40 billion. Negotiators in that case, brought by 2,300 tribes, counties and cities nationwide and led by several states’ attorneys general, worked through much of Friday without success. Josh Stein, the attorney general for North Carolina, said they were trying to put together a $48 billion deal.

GSK of­floads two vac­cines in $1.1B deal as it works to re­vive the pipeline

GlaxoSmithKline is leaving the deep dark woods and its viruses behind.

GSK has agreed to divest its vaccines for rabies, RabAvert, and tick-born encephalitis vaccine, Encepur, to Bavarian Nordic, part of the company’s broader efforts to narrow its pipeline and focus on oncology and immunology.

The deal is worth up to nearly $1.1 billion, with a $336 million upfront payment. GSK acquired the vaccines from Novartis as part of an exchange for their late-stage oncology programs in 2015 under former chief Sir Andrew Witty.

Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrate their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of. The market leader here is Norditropin, a growth hormone from Novo Nordisk that uses the same basic ingredient as Genotropin, which the Danish company sells with a kid-friendly self-injectable pen. That would also present some big competition if the new therapy from Opko/Pfizer makes it to the market.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,600+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.