Step right up: A biotech may soon sell an ex­per­i­men­tal med to dy­ing ALS pa­tients un­der 'Right to Try'

Dur­ing the de­bate over the new Right to Try leg­is­la­tion, a num­ber of crit­ics raised the ar­gu­ment that some com­pa­nies would try to cash in by sell­ing ex­per­i­men­tal drugs with an un­de­fined risk pro­file to des­per­ate, dy­ing pa­tients.

Brain­storm Cell Ther­a­peu­tics $BCLI ap­pears to be on track to be one of the first to test the let­ter of the law on that score.

The Is­raeli biotech has been slow­ly work­ing on a stem cell ther­a­py for ALS, or Lou Gehrig’s dis­ease. For months now, com­pa­ny ex­ecs have been hint­ing at the move, not­ing the num­ber of ALS pa­tients who have been clam­or­ing for a ther­a­py they dubbed NurOwn. And in an in­ter­view with Bloomberg, CEO Chaim Lebovits said that they’re think­ing of a price in the re­gion of the pi­o­neer­ing per­son­al­ized cell ther­a­pies that have been ap­proved.

Say $300,000. That might be the right “in­cen­tive.”

I went hunt­ing for the da­ta they have so far, and it’s not much. The com­pa­ny points out that 75 peo­ple have been treat­ed with the ther­a­py, de­liv­er­ing a “clin­i­cal­ly mean­ing­ful ben­e­fit.” But that’s ar­guable.

If you look at the 24-week chart the com­pa­ny of­fered on the 48 pa­tients in the small, ex­plorato­ry study, the drug out­per­formed the place­bo arm but any rel­a­tive treat­ment ef­fect they were track­ing quick­ly evap­o­rat­ed for most of the pa­tients suf­fer­ing from this slow, lethal ail­ment. The p-val­ues were a mess. The in­ves­ti­ga­tors in­volved said that if you ex­clud­ed the slow pro­gres­sors, the num­bers looked bet­ter — but the same tran­sient ef­fect is ev­i­dent, with neg­a­tive p-val­ues.

That’s why it’s in Phase III, which is a big­ger study — backed by the Cal­i­for­nia In­sti­tute of Re­gen­er­a­tive Med­i­cine — that will show whether the drug can help a sig­nif­i­cant num­ber of pa­tients, or if it’s a waste of time and mon­ey.

Un­til Right to Try was passed re­cent­ly, the drug would on­ly be avail­able to peo­ple who made a plea to get the drug at cost, but the CEO ev­i­dent­ly be­lieves the new law gives him a right to mar­ket it. Health in­sur­ers are un­like­ly to even con­sid­er a re­im­burse­ment for an ex­per­i­men­tal ther­a­py like this.

I asked the three key in­ves­ti­ga­tors in the Phase II study — Mer­it Cud­kow­icz, Mass­a­chu­setts Gen­er­al Hos­pi­tal; prin­ci­pal in­ves­ti­ga­tor Robert Brown, UMass Med­ical School; and An­tho­ny Winde­bank from the Mayo Clin­ic — what they thought about the com­pa­ny’s plans. I haven’t heard back yet.

If they do, I’ll add their re­spons­es.

At best the com­pa­ny has an ul­tra high-risk Phase III un­der­way that in­vestors are re­luc­tant to back, es­pe­cial­ly af­ter the era of stem cell tri­al fail­ures we’ve been see­ing over the past decade. At worst there’s a good chance that the pa­tients who do raise the cash for this ther­a­py will find that the law would have been bet­ter called Right to Bank­rupt­cy.

And we’re not even in­to the first round of pure snake oil sales­men. 

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

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Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.