MeiraGTx update after Q3

 

• Received 3 Rare Pediatric Disease Designations (RPDD) from FDA for each of 3 potential therapies for 3 different rare inherited retinopathies including AAV-AIPL1

• Agreed on pathway with MHRA for Marketing Authorization Application (MAA) under exceptional circumstances for AAV-AIPL1 for the treatment of Leber congenital amaurosis (LCA4) retinal dystrophy without further clinical studies

• Announced positive data from randomized, sham-controlled clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease

https://www.globenewswire.com/news-release/2024/11/13/2980218/0/en/MeiraGTx-Announces-Third-Quarter-2024-Financial-and-Operational-Results-and-Recent-Business-Updates.html

Adaptimmune's Lete-cel Achieves Primary Endpoint in Pivotal Trial

 42% of people with advanced or metastatic synovial sarcoma or MRCLS had clinical responses with lete-cel

Results include six complete responses (6/64); twenty-one partial responses (21/64)

Data to be presented at Connective Tissue Oncology Society 2024 Annual Meeting

Company plans to initiate a rolling BLA submission for lete-cel for the treatment of advanced or metastatic synovial sarcoma and MRCLS by end of 2025

Company to host virtual KOL event Monday, November 18, 2024; 2:30 PM EST featuring
Dr. Sandra D'Angelo, M.D. (Memorial Sloan Kettering Cancer Center) - register here

CTOS presentation details:

• Title: Planned Analysis of the Pivotal IGNYTE-ESO Trial of Lete-Cel in Patients with Synovial Sarcoma or Myxoid/Round Cell Liposarcoma (Paper 84)
• Session 12: Immunology: Podium presentation
• Presenter: Sandra D'Angelo, MD, Sarcoma Medical Oncologist and Cell Therapist, Memorial Sloan Kettering Cancer Center
• Date/Time: Saturday, November 16, 10:30 AM - 12:00 PM PT / 1:30 - 3:00 PM ET

Adaptimmune virtual KOL event November 18th
Adaptimmune will host a virtual event to discuss and review the IGNYTE-ESO dataset and the impact of engineered cell therapies on the treatment landscape in sarcoma. The event will feature Sandra D'Angelo, M.D., Sarcoma Medical Oncology, Memorial Sloan Kettering Cancer Center, an investigative clinician in both the SPEARHEAD and IGNYTE-ESO clinical trials, and author and presenter of the IGNYTE-ESO data update at CTOS. A live question and answer session will follow the formal presentation. The virtual event will take place on Monday, November 18, 2024 from 2:30 PM ET to 3:30 PM ET. To register, click here.

https://www.newsfilecorp.com/release/229711/Adaptimmunes-Letecel-Achieves-Primary-Endpoint-in-Pivotal-Trial

BioNTech to Acquire Biotheus to Boost Oncology Strategy

 

• Acquisition to support the global execution of BioNTech’s oncology strategy and provide full global rights to BNT327/PM8002, an investigational PD-L1 x VEGF-A bispecific antibody, with potential to replace current checkpoint inhibitor standard of care treatments for solid tumors
• With the acquisition of Biotheus, BioNTech aims to further strengthen its capabilities to develop, manufacture and commercialize next-generation bispecific antibodies and novel treatment combinations
• BioNTech and Biotheus plan to initiate multiple registrational trials with BNT327/PM8002 in late 2024 and 2025; further clinical trials evaluating BNT327/PM8002 as combination therapies are planned to start in 2024 and 2025
• BioNTech to pay $800 million to acquire 100 percent of the issued share capital and up to $150 million in potential milestone payments
• Additional details will be shared at BioNTech’s Innovation Series R&D Day event on 14 November 2024

BioNTech’s Innovation Series R&D Day
BioNTech leadership will present additional details on the Biotheus transaction, as well as updates on the corporate strategy, commercial strategy and clinical progress across its pipeline during an edition of the company’s Innovation Series R&D Day on 14 November. The live webcast of the event will be available via this link and will begin at 4:30 pm CET (3:30 pm GMT, 10:30 am EST). A replay of the webcast will be available shortly after the event’s conclusion and archived on BioNTech’s website for one year.

https://www.globenewswire.com/news-release/2024/11/13/2980068/0/en/BioNTech-to-Acquire-Biotheus-to-Boost-Oncology-Strategy.html

Syndax’s revumenib meets primary results in AML trial

 Syndax (SNDX) announced positive topline results from the relapsed or refractory mutant NPM1 – mNPM1 – acute myeloid leukemia, or AML, cohort in the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib, an oral, small molecule menin inhibitor. The primary endpoint was met with a complete remission, or CR, plus CR with partial hematological recovery rate of 23% among the efficacy evaluable adults with R/R mNPM1 AML in the Phase 2 portion of the AUGMENT-101 trial. Among the patients who achieved CR/CRh, 12 patients had a CR and three had a CRh. The observed median duration of CR/CRh responses was 4.7 months at data cutoff with three patients remaining in response. Overall response rate was 47%. 17% of patients who achieved an overall response underwent hematopoietic stem cell transplant following treatment with revumenib, with three resuming revumenib therapy post-transplant. The safety profile observed with revumenib in this population was consistent with previously reported data. Treatment-related adverse events leading to treatment discontinuations were 5%. Grade 3 treatment-related DS was observed in 11% of patients while 2% experienced Grade 4 DS and no patients experienced Grade 5.

https://finance.yahoo.com/news/syndax-revumenib-meets-primary-results-122135013.html

Syros Crashes Over 90% on Late-Stage MDS Fail, Loan Default

 

With the Phase III failure, Syros will discontinue the study of tamibarotene for myelodysplastic syndrome and will default on its loan from Oxford Finance LLC.

Syros Pharmaceuticals on Tuesday reported that its oral drug candidate tamibarotene failed the Phase III SELECT-MDS-1 trial, as it did not significantly improve complete response rates in certain patients with myelodysplastic syndrome compared to the approved drug azacitidine plus placebo. The failure “constitutes an event of default under its secured loan facility with Oxford Finance LLC,” the biotech said. Syros’ stock price fell precipitously after the news was announced.

Topline data from the Phase III study showed that tamibarotene combined with azacitidine hit a complete response (CR) rate of 23.8% in newly diagnosed patients with higher-risk myelodysplastic syndrome (HR-MDS) with an overexpression of the RARA gene. In comparison, patients given placebo plus azacitidine hit an 18.8% CR rate.

The treatment effect of tamibarotene was not statistically significant, with a p-value of 0.2084, according to Syros’ news release. The biotech’s shares fell as much as 92% in post-market trading Tuesday, according to SeekingAlpha.

CEO Conley Chee in a statement said that the biotech is “deeply disappointed by this outcome.” Syros will discontinue SELECT-MDS-1 and “evaluate the next steps” as it completes its review of the late-stage data.

Designed to be orally available, tamibarotene is a selective agonist of the retinoic acid receptor alpha (RARA), which when overexpressed can prevent the differentiation of bone marrow cells into healthy myeloid cells. Around half of MDS patients show RARA overexpression, according to Syros’ website. By binding to excess RARA, tamibarotene helps restore myeloid differentiation.

In December 2023, Syros announced encouraging Phase II data that seemed to validate tamibarotene’s mechanism of action. When combined with azacitidine and venetoclax, tamibarotene achieved 100% complete response or complete response with incomplete hematologic recovery in all nine patients who were evaluable at the time.

Syros’ late-stage stumble on Tuesday illustrates the difficulty of developing an effective therapy for MDS, an indication that has tripped up several other biopharma companies.

In April 2024, for instance, Gilead announced that it would no longer invest in development of the anti-CD47 antibody magrolimab, which it was trialing for MDS and acute myeloid leukemia, following several clinical and regulatory setbacks. A few months later, in June 2024, the pharma completed its analysis of the Phase III ENHANCE study—which had been discontinued a year earlier—showing that magrolimab aggravated the risk of death by 20% in HR-MDS patients.

In February 2024, Roivant shuttered its subsidiary Hemavant after a disappointing Phase I/II readout for the MDS candidate RVT-2001.

Not all the news is bleak for MDS patients, though. In June 2024, the FDA signed off on Geron’s first-in-class telomerase blocker Rytelo (imetelstat) for the treatment of patients with lower- to intermediate-risk MDS. In October 2023, the regulator expanded the label of Servier’s Tibsovo (ivosidenib tablets) to include relapsed or refractory MDS with susceptible IDH1 mutations.

https://www.biospace.com/drug-development/syros-crashes-over-90-on-late-stage-mds-fail-loan-default

Amgen dismisses bone density concerns related to its new weight-loss drug

 Amgen said on Wednesday there was no link between its experimental weight-loss drug and changes in bone density, a day after those concerns wiped off more than $12 billion from the company's market value.

The company's stock slumped 7% on Tuesday after analysts at Cantor Fitzgerald said their review of early-stage data on Amgen's MariTide showed the drug had led to a drop in bone mineral density.

"The Phase 1 study results do not suggest any bone safety concern or change our conviction in the promise of MariTide," Amgen said, adding that it looks forward to data from its mid-stage study later this year.

Shares of Amgen rose 2% in premarket trade after the company's statement.

Cantor analysts said they found the bone mineral density changes when reviewing supplemental data that was published along with the results in February.

At least four analysts said the concerns were overblown, especially considering the company was conducting a mid-stage study and planned to invest in a larger late-stage trial as well.

"While a new safety signal would certainly be (a) cause for alarm on any drug, the truth is, (Amgen) knows a lot more about this molecule than the Street," Piper Sandler analyst Christopher Raymond said in a note.

https://www.msn.com/en-ca/health/other/amgen-dismisses-bone-density-concerns-related-to-its-new-weight-loss-drug/ar-AA1u0g9J

Musk, Ramaswamy to lead Trump's Department of Government Efficiency

 President-elect Trump announced that billionaire Elon Musk and former GOP presidential candidate Vivek Ramaswamy will lead the Department of Government Efficiency.

Trump said that the pair will work together to "dismantle Government Bureaucracy, slash excess regulations, cut wasteful expenditures, and restructure Federal Agencies."

"It will become, potentially, ‘The Manhattan Project’ of our time," the announcement on Tuesday evening said. "Republican politicians have dreamed about the objectives of ‘DOGE’ for a very long time."

The president-elect said that Musk and Ramaswamy will provide "advice and guidance from outside of Government, and will partner with the White House and Office of Management & Budget to drive large scale structural reform, and create an entrepreneurial approach to Government never seen before."

Vivek Ramaswamy and Elon Musk. The pair have been tapped to lead Trump's Department of Government Efficiency. (Getty Images/AP Images)

Trump said that the agency will be focused on creating a more efficient U.S. government that looks to make "life better for all Americans."

"Importantly, we will drive out the massive waste and fraud which exists throughout our annual $6.5 Trillion Dollars of Government Spending. They will work together to liberate our Economy, and make the U.S. Government accountable to 'WE THE PEOPLE.'" Trump said. 

"Their work will conclude no later than July 4, 2026 - A smaller Government, with more efficiency and less bureaucracy, will be the perfect gift to America on the 250th Anniversary of The Declaration of Independence. I am confident they will succeed!"

Republican presidential candidate former President Trump greets Vivek Ramaswamy while speaking during a campaign rally in New Hampshire.  (Brandon Bell/Getty Images)

In a X post, Ramaswamy reacted to his appointment.

"We will not go gently, Elon Musk," he wrote in the post.

Ramaswamy has been a vocal supporter of Trump after he suspended his presidential campaign in Jan. 24.

In a statement on X, the founder Roivant Sciences, a pharmaceutical company, said that he would be dropping out of the Ohio Senate appointment.

"And yes, this means I’m withdrawing myself from consideration for the pending Senate appointment in Ohio," he wrote. "Whoever Governor DeWine appoints to JD’s seat has some big shoes to fill. I will help them however I can."

Elon Musk jumps on the stage as Republican presidential nominee former President Trump speaks at a campaign rally at the Butler Farm Show, Oct. 5, in Butler, Pa.  (AP Photo/Evan Vucci)

Similarly, Musk has been a key component to Trump's campaign – with the tech entrepreneur crisscrossing key battleground states leading up to the 2024 election.

In response to his position in Trump's White House, Musk wrote: "Threat to democracy? Nope, threat to BUREAUCRACY!!!"

The world's richest man, who said he voted for former Democratic presidential candidates including President Biden in the past, endorsed Trump this summer following the first assassination attempt on the 45th president on July 13. 

The slew of Trump Cabinet positions came quickly after the president-elect's landslide victory against Vice President Kamala Harris.

Trump has selected top Republicans, with the president-elect expected to select Florida Sen. Marco Rubio to serve as his Secretary of State and South Dakota Gov. Kristi Noem as secretary of Homeland Security. 

In addition, Rep. Elise Stefanik, R-N.Y., has been tapped for United Nations ambassador and former Arkansas Gov. Mike Huckabee as ambassador to Israel.

https://www.foxnews.com/politics/elon-musk-vivek-ramaswamy-lead-trumps-department-government-efficiency