FDA puts Solid Bio’s lead gene therapy program on hold — again — after another patient is hurt by SGT-001

November 12, 2019 07:15 AM ESTUpdated 07:35 AM

John Carroll

FDA puts Solid Bio’s lead gene therapy program on hold — again — after another patient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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March 30, 2020 05:42 PM EDTUpdated 05:53 PM

John Carroll

R&D

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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April 1, 2020 07:26 AM EDTUpdated 09:47 AM

John Carroll

Coronavirus

Regulatory

UPDATED: Have a new drug that promises to fight Covid-19? The FDA promises fast action but some developers aren't happy

After providing an emergency approval to use malaria drugs against coronavirus with little actual evidence of their efficacy or safety in that setting, the FDA has already proven that it has set aside the gold standard when it comes to the pandemic. And now regulators have spelled out a new approach to speeding development that promises immediate responses in no uncertain terms — promising a program offering the ultimate high-speed pathway to Covid-19 drug approvals.

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March 31, 2020 10:15 AM EDTUpdated 11:38 AM

John Carroll

Regulatory

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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Principal - Alexandria LaunchLabs

Alexandria Real Estate Equities

San Francisco, CA

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March 31, 2020 07:21 AM EDTUpdated 8 hours ago

Endpoints Staff

Coronavirus

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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April 1, 2020 11:07 AM EDTUpdated 11:27 AM

Natalie Grover

Coronavirus

The race to develop Covid-19 drugs and vaccines is on — here’s what’s happening in the UK

Weeks away from the results of ongoing US and China trials testing its experimental antiviral remdesivir, Gilead is going to trial the failed Ebola drug in a small group of coronavirus patients in England and Scotland. The United Kingdom is also home to a range of other therapeutic efforts, as the pandemic rages on across the globe.

On Tuesday, Southampton, UK-based startup Synairgen kicked off a mid-stage placebo-controlled study testing its experimental drug, SNG001 — an inhaled formulation of interferon-beta-1a — that has previously shown to be safe and effective in improving lung function in asthma patients with a respiratory viral infection in a pair of Phase II trials.

April 1, 2020 11:00 AM EDT

Jason Mast

Venture

‘There was a growing weariness’: Rushing against a pandemic clock, Aspen Neurosciences secures $70M Series A

Just before Christmastime, Howard Federoff got a tip from Washington: There was a new virus in China. And this one could be bad.

News report of the virus had not yet appeared. Federoff, a neuroscientist, was briefed because years before, he was vetted as part of a group — he didn’t give a name for the group — to consult for the US government on emerging scientific issues. His day job, though, was CEO of Aspen Neurosciences, a Parkinson’s cell therapy startup that days before had come out of stealth mode and gave word to investors they were hoping to raise $70 million. That, Federoff realized, would be difficult if a pandemic shut down the global economy.

April 1, 2020 10:34 AM EDT

Amber Tong

Cell/Gene Tx

Regulatory

FDA puts pediatric aGVHD drug on priority review lane — will they go virtual with the adcomm?

Despite worries about regulatory delays due to new work arrangements under Covid-19, the FDA appears intent to go full speed ahead with its everyday work, not only granting priority review to a stem cell therapy for acute graft versus host disease but also plotting an advisory committee meeting for it.

With a PDUFA date of September 30, the journey of the drug — remestemcel-L, or Ryoncil — could shed light on the agency’s capacity to facilitate drug development unrelated to Covid-19.

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Executive Director/Vice President of Business Development

Caribou Biosciences, Inc.

Berkeley, CA

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April 1, 2020 10:03 AM EDTUpdated 11:08 AM

Endpoints Staff

Coronavirus

Covid-19 roundup: Trump pushes his new favorite, untested drug; CRISPR outlines crippling impact of Covid-19

President Trump has a new favorite Covid-19 drug.

After a conversation with Japanese Prime Minister Shinzo Abe, Politico reports, the president is pressuring the FDA to issue emergency use authorization for favipiravir, a flu drug that showed glimpses of success in China but remains unproven and carries a list of worrying side effects. The push comes after a week-plus in which the White House touted a potentially effective but unproven malaria medication despite the concerns of scientific advisors such as NIAID director Anthony Fauci. And Trump ally Rudy Giuliani has been talking up unproven cell therapy efforts on Twitter.

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Mene Pangalos via YouTube

March 30, 2020 07:10 AM EDTUpdated 10:35 AM

John Carroll

Pharma

R&D

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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