Mer­ck goes in deep­er with Keytru­da/Lyn­parza com­bo; PureTech un­veils stem cell ther­a­py start­up

→ Mer­ck is open­ing up a new wing of its Keytru­da pipeline, de­vot­ing it to a slate of new Phase III com­bi­na­tion stud­ies with Lyn­parza in treat­ing prostate can­cer. Re­searchers spot­ted an­ti-tu­mor ac­tiv­i­ty in 3 co­horts, send­ing them the sig­nal they were look­ing for to pile on piv­otal stud­ies in search of a new mar­ket. “These promis­ing da­ta pre­sent­ed at AS­CO GU cou­pled with the sig­nif­i­cant un­met med­ical need in pa­tients with metasta­t­ic cas­tra­tion-re­sis­tant prostate can­cer, pro­pelled us to ini­ti­ate three new Phase 3 tri­als to fur­ther eval­u­ate these KEYTRU­DA com­bi­na­tion reg­i­mens,” not­ed Mer­ck’s Roy Baynes.

PureTech Health is help­ing chart a path for Sid­dhartha Mukher­jee to bring some of his re­search from Co­lum­bia Uni­ver­si­ty to the clin­ic with a new start­up. De­but­ing with a $42 mil­lion round led by 5AM Ven­tures and RA Cap­i­tal Man­age­ment, Vor Bio­phar­ma is look­ing to treat hema­to­log­i­cal ma­lig­nan­cies with hematopoi­et­ic stem cell ther­a­pies, start­ing with acute myeloid leukemia. 5AM man­ag­ing part­ner Kush Par­mar is tak­ing the ex­ec­u­tive chair­man role while Joshua Resnick of RA Cap­i­tal al­so joins the board. Oth­er in­vestors in­clude John­son & John­son In­no­va­tion – JJDC, No­var­tis In­sti­tutes for Bio­Med­ical Re­search and Os­age Uni­ver­si­ty Part­ners.

Tesaro‘s PARP drug Ze­ju­la, which in 2017 saw its la­bel ex­pand­ed by the FDA, has shown ear­ly promise in a mid-stage study in cer­tain metasta­t­ic cas­tra­tion-re­sis­tant prostate can­cer pa­tients. J&J $JNJ and Tesaro – which is now a part of GSK $GSK  – agreed to col­lab­o­rate on test­ing the drug, known chem­i­cal­ly as ni­ra­parib, in 2016. Pre­lim­i­nary da­ta from the 120-pa­tient study showed 40% of pa­tients with path­way de­fects – BR­CA1 or BR­CA2 – who re­ceived treat­ment with ni­ra­parib demon­strat­ed an ob­jec­tive re­sponse rate as mea­sured by a de­creased tu­mor size in soft tis­sue. 

→ It’s on. Sarep­ta $SRPT to­day said that the FDA has ac­cept­ed its NDA for golodirsen (SRP-4053) as it hunts an­oth­er ac­cel­er­at­ed ap­proval for a new drug to treat Duchenne mus­cu­lar dy­s­tro­phy. The agency set the PDU­FA date at Au­gust 19.

→ A gener­ic ver­sion of Cel­gene’s best-sell­ing can­cer drug Revlim­id will soon be avail­able around the world. Alvo­gen an­nounced that it has launched the gener­ic in Ro­ma­nia, Croa­t­ia, Bul­gar­ia and the Baltic states, with a glob­al roll­out to fol­low.

→ TCR2 Ther­a­peu­tics has priced its IPO at $15 a share. The Cam­bridge, MA-based TCR biotech raised $75 mil­lion from the of­fer­ing, pric­ing at the mid­point of the range it set for it­self. The stock will be­gin trad­ing to­day as $TCRR.

→ Ver­tex $VRTX says it came up with pos­i­tive da­ta from their Phase III study of teza­caftor in com­bi­na­tion with iva­caftor, hit­ting the pri­ma­ry end­point of ab­solute change in lung clear­ance in­dex (LCI2.5) through 8 weeks of treat­ment. Re­searchers say that there was “a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in LCI2.5 among pa­tients treat­ed” with the com­bo. An ap­pli­ca­tion is due in H2.

→ Cal­adrius Bio­sciences says its Phase IIa study us­ing a Treg strat­e­gy for au­toim­mune dis­eases failed. Re­searchers test­ed CLBS03 in new­ly di­ag­nosed Type 1 di­a­betes pa­tients, hop­ing that the well-known ef­fect that Tregs have in blunt­ing an im­mune sys­tem at­tack would work for this dis­ease. Its shares $CLBS were down 15% in mid-morn­ing trad­ing.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — and they're prep­ping a pitch to the FDA

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company says that a new analysis of an old dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and spread the chill to a high-pro­file biotech

Back in July, during an investor call where Novartis execs ran through an upbeat assessment of their Q2 performance, CEO Vas Narasimhan and development chief John Tsai were pressed to predict which of the two looming Phase III readouts — involving cardio drug Entresto and asthma therapy fevipiprant, respectively — had a higher likelihood of success. Tsai gave the PARAGON-HF study with Entresto minimally better odds, but Narasimhan emphasized that their strategy of giving fevipiprant to more severe patients gave them confidence.

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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.

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Take­da tees up $420M deal for celi­ac an­ti­dote, con­tin­u­ing R&D re­fo­cus

Sometime in the 1st century AD, a patient presented to Arataeus looking like a varicose ghost. He was “emaciated and atrophied, pale, feeble and incapable of performing any of his accustomed works,” the Greek physician wrote, with hollow temples and huge veins running all over his body.

A dysfunctional digestive system, Arataeus concluded – an imbalance he attributed to a “heat” deficiency in a system he and other Greeks regarded as functioning similarly to an oven – and coined a term: coeliac disease, after the Greek word for abdomen.

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.

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Photo credit: Jacquelyn Martin

Where are the in­ter­change­able biosim­i­lars?

In June 2017, Leah Christl, former biosimilar lead at FDA, told a conference in Chicago that interchangeable biosimilars were likely coming to the US market within two years.

And although no interchangeable biosimilar has been approved by FDA yet, and Christl has since moved on to Amgen, progress on interchangeable biosimilars has been slow in the intervening years.

Most recently, Boehringer Ingelheim announced that it has completed, as of last April, a switching study necessary for launching an interchangeable biosimilar for Humira (adalimumab), although the company did not offer any further details on the timing of its submission to FDA or whether there will be an advisory committee to review the data. Boehringer already has an adalimumab biosimilar approved by FDA, which it will launch in the US on 1 July 2023.

FDA re­buffs lit­tle As­ser­tio Ther­a­peu­tic­s' long-act­ing ACTH for­mu­la­tion, shares sink

Tiny Assertio Therapeutics’ shares plunged pre-market on Tuesday, after the FDA has spurned its man-made version of the hormone ACTH, which was being reviewed as a diagnostic for patients presumed to have adrenocortical insufficiency.

The Lake Forest, Illinois-based drugmaker said its development partner West Therapeutic Development had received a complete response letter from the US regulator, which indicated that that certain “pharmacodynamic parameters were not adequately achieved” for the product.

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:

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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.