#ASCO19: Struggling Nektar braves a tough crowd to offer up 4 new CRs for skin cancer drug

June 1, 2019 06:05 PM EDTUpdated June 2, 11:16 AM

John Carroll

#ASCO19: Struggling Nektar braves a tough crowd to offer up 4 new CRs for skin cancer drug

CHICAGO — Nektar Therapeutics $NKTR came back to ASCO looking for some redemption after their pratfall on their NKTR-214 presentation here last year -- causing damage to the share price that has yet to be repaired. And while they are a long way from being back in the good graces of the analysts, its top researchers are making a determined march ahead with what they still believe is a game-changing cancer therapy well worth Bristol-Myers’ blockbuster buy-in.

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Nick Leschly via Getty

June 14, 2019 06:59 AM EDTUpdated 02:30 PM

John Carroll

R&D

UPDATED: Bluebird shares sink as analysts puzzle out $1.8M sticker shock and an unexpected delay

Bluebird bio $BLUE has unveiled its price for the newly approved gene therapy Zynteglo (LentiGlobin), which came as a big surprise. And it wasn’t the only unexpected twist in today’s story.

With some analysts betting on a $900,000 price for the β-thalassemia treatment in Europe, where regulators provided a conditional early OK, bluebird CEO Nick Leschly said Friday morning that the patients who are successfully treated with their drug over 5 years will be charged twice that — $1.8 million — on the continent. That makes this drug the second most expensive therapy on the planet, just behind Novartis’ newly approved Zolgensma at $2.1 million, with analysts still waiting to see what kind of premium can be had in the US.

Ted Love. HAVERFORD COLLEGE

June 14, 2019 07:16 AM EDTUpdated 08:25 AM

Natalie Grover

R&D

Global Blood Therapeutics poised to submit application for accelerated approval, with new pivotal data on its sickle cell disease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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June 14, 2019 10:55 AM EDTUpdated 12:38 PM

Endpoints Staff

R&D

Newsmakers at #EHA19: Regeneron, ArQule track progress on response rates

Regeneron’s closely-watched bispecific continues to ring up high response rates

Regeneron’s high-profile bispecific REGN1979 is back in the spotlight at the European Hematology Association scientific confab. And while the stellar numbers we saw at ASH have eroded somewhat as more blood cancer patients are evaluated, the response rates for this CD3/CD20 drug remain high.

A total of 13 out of 14 follicular lymphomas responded to the drug, a 93% ORR, down from 100% at the last readout. In 10 out of 14, there was a complete response. In diffuse large B-cell lymphoma the response rate was 57% among patients treated at the 80 mg to 160 mg dose range. They were all complete responses. And 2 of these Cars were for patients who had failed CAR-T therapy.

June 11, 2019 11:21 AM EDTUpdated 12:12 PM

Amber Tong

R&D

Searching for the next blockbuster to follow Darzalex, J&J finds a $150M anti-CD38 drug from partner Genmab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.

Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.

Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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June 13, 2019 08:36 AM EDT

John Carroll

Cell/Gene Tx

Pharma

Gene therapies seize the top of the list of the most expensive drugs on the planet — and that trend has just begun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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June 13, 2019 06:58 AM EDT

John Carroll

R&D

Savara shares are crushed as PhIII trial flunks primary, key secondaries — but they can’t stop believing

Investors are in no mood to hear biotechs tout the success of a “key” secondary endpoint when the pivotal Phase III flunks the primary goal. Just ask Savara.

The Texas biotech $SVRA went looking for a silver lining as company execs bluntly conceded that Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), failed to spur significantly improved treatment outcomes for patients with a rare respiratory disease called autoimmune pulmonary alveolar proteinosis, or aPAP.

June 13, 2019 06:44 AM EDTUpdated 08:11 AM

John Carroll

R&D

As another antibiotics biotech sinks into a crisis, warnings of a sector ‘collapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

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June 10, 2019 07:34 AM EDTUpdated June 11, 04:32 AM

Amber Tong

R&D

'We kept at it': Jeffrey Bluestone plots late-stage comeback after teplizumab shown to delay type 1 diabetes

Late-stage data presented at the American Diabetes Association annual meeting in 2010 pushed Eli Lilly to put a crimp on teplizumab as the pharma giant found it unable to reset the clock on newly diagnosed type 1 diabetes. At the same conference but in different hands nine years later, the drug is making a critical comeback by scoring success in another niche: delaying the onset of the disease.

In a Phase II trial with 76 high-risk individuals — relatives of patients with type 1 diabetes who have diabetes-related autoantibodies in their bodies — teplizumab almost doubled the median time of diagnosis compared to placebo (48.4 months versus 24.4 months). The hazard ratio for diagnosis was 0.41 (p=0.006).

June 12, 2019 07:00 AM EDTUpdated 11:39 AM

John Carroll

Venture

Bain’s biotech team has created a $1B-plus fund — with an eye to more Big Pharma spinouts

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

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