J&J sees positive data from pair of multiple myeloma approaches at ASH

 

• At the American Society of Hematology annual meeting over the weekend, Johnson & Johnson (JNJ -0.5%) posted attention-getting data on a pair of approaches to multiple myeloma.
• One study confirmed the company's cilta-cel as the most efficacious CAR-T therapy, with more than three-quarters of patients treated with the experimental therapy alive at least a year without their cancer worsening.
• The results have been stronger than those of a rival CAR-T therapy from Bristol Myers Squibb (BMY -1.1%) and Bluebird Bio (BLUE -0.4%), who might now face some long-run pressure to come up with a better approach.
• Toxicity is still an issue for cilta-cel, with six subjects dying, leaving 97 evaluable subjects. But overall remission rate is at 97% (vs. 73% overall, or 82% at highest dose, for the BMY/Bluebird idle-cel).
• Meanwhile, in an indication that multiple myeloma approaches vary beyond targeting the BCMA antigen, JNJ's talquetamab impressed with data from its dose-escalation trial.
• Talquetamab is a biospecific against the GPRC5D antigen that has been used to treat 157 patients.
• The lowest doses have turned out inactive, but subjects given over 20µg/kg are seeing a response rate of 66%, while 13 patients who received 405µg/kg subcutaneously (the Phase 2 dose) saw ORR of 69%.
• https://seekingalpha.com/news/3642175-johnson-johnson-sees-positive-data-from-pair-of-multiple-myeloma-approaches-ash

Sarepta Therapeutics defended after gene therapy data sends shares lower

 

• Wall Street firms update on Sarepta Therapeutics (SRPT -6.0%) after the company's initial data from its SRP-5051 gene therapy showed an improvement in exon skipping over the company's first generation technology, but is still seen falling short of some expectations.
• Credit Suisse: "Overall, we consider this to be an encouraging start for the drug and note that optionality for the program is preserved given the clean safety profile to date; at this time, we cannot conclude there is a meaningful advance beyond Exondys, though, and it is unclear whether dose levels can be achieved that are likely to confer a meaningful functional benefit. While the drug appears safe so far, we await 30mg/kg results expected in Q2 2021 to begin to address these questions."
• Mizuho called today's share price drop unwarranted and SVB Leerink says investor eyes remain fixated on 102’s readout in Q1, despite the early data that were presented this morning.
• Read Sarepta's full clinical trial update.
• https://seekingalpha.com/news/3642199-sarepta-therapeutics-defended-after-gene-therapy-data-sends-shares-lower

Lyra Therapeutics Phase 2 Study of LYR-210 Misses Primary Endpoint

 

• While a strong treatment effect was observed at week 4, the LANTERN study’s primary endpoint of change from baseline in a composite of 4CS scores at week 4 was not met at either dose (7500 mcg: (-0.36) (p=0.306); 2500 mcg: (0.04) (p=0.525)), we believe primarily due to curtailed enrollment resulting from COVID-19.
  

• Given the comparable safety profile of LYR-210 at both 2500 mcg and 7500 mcg doses, Lyra anticipates progressing the LYR-210 program at the 7500 mcg dose level.
  

• Full results from the LANTERN study will be submitted for future presentation at an upcoming scientific meeting.

• Based on these results Lyra plans to initiate a pivotal Phase 3 study for LYR-210 in chronic rhinosinusitis for both non-polyp and polyp patients.

• https://finance.yahoo.com/news/lyra-therapeutics-announces-positive-topline-130000705.html

Russia approves clinical trials for Chinese COVID-19 vaccine Ad5-Ncov

 Russia has granted approval for clinical trials to be held for the Chinese COVID-19 vaccine Ad5-Ncov involving 8,000 volunteers, the Interfax news agency reported on Monday.

Ad5-nCoV is a vaccine candidate co-developed by CanSino Biologics and a Chinese military-backed research unit.

https://www.reuters.com/article/us-health-coronavirus-china-russia/russia-approves-clinical-trials-for-chinese-covid-19-vaccine-ad5-ncov-ifax-idUSKBN28H0RN

Bayer accelerates cell and gene therapy drive with Atara deal

 Bayer AG has struck a deal with Atara Biotherapeutics to jointly work on Atara’s CAR-T cell anti-tumour treatments, as the German group firms up its commitment to build a specialist cell and gene therapy development platform.

The deal to develop experimental mesothelioma treatments comes after Bayer said last week it was setting up a unit within its healthcare division to speed up and support cell and gene therapy development after recent takeovers.

With the agreement, the German drugmaker will get access to Atara’s CAR T-cell therapy for solid tumours, ATA3271, which had shown anti-tumour activity in a pre-clinical study, as well as another treatment called ATA2271.

Bayer said it would make an upfront payment of $60 million to Atara and may also pay up to $610 million upon achievement of certain milestones, as well as tiered royalties up to a low double-digit percentage of net sales.

Bayer is eager to brush up its drug development pipeline after its agriculture business was hit by 9.25 billion euros ($10.8 billion) in impairment charges and following an outline agreement costing more than an $11 billion to settle U.S. claims that its Roundup weed killer causes cancer.

Wolfram Carius, head of its newly formed Cell and Gene Therapy Unit, said the Atara deal demonstrated the German group’s commitment to grow in the area.

“It’s really a defining moment for Bayer Pharmaceuticals”, showing the “clear commitment by the entire company” to advance cell and gene therapy, he said in an interview.

Among previous steps, Bayer acquired cell and gene therapy companies BlueRock Therapeutics and Asklepios Biopharmaceutical in 2019 and 2020 respectively.

So far, the focus of cell therapies in oncology has been on blood-based cancers, where malignant cells do not lump together.

But companies including GlaxoSmithKline and partner Lyell Immunopharma have started to tackle solid tumours that often build barriers of defence.

Bayer’s Carius said researchers were getting a better understanding of how tumours evade an immune system response.

https://www.reuters.com/article/us-bayer-atara-cancer/bayer-accelerates-cell-and-gene-therapy-drive-with-atara-deal-idUSKBN28H0A5

FDA places clinical hold on Bellicum's cancer therapy

 Bellicum Pharmaceuticals said on Monday the U.S. Food and Drug Administration had placed a clinical hold on patient enrollment and dosing in an early-stage trial of its cancer treatment, after the death of a patient.

Shares fell 12.62% in premarket trade.

The company said the death was unrelated to the treatment, BPX-601, but that it would work with the FDA to resume the trial.

Bellicum was testing the therapy in patients that were previously treated for metastatic pancreatic or prostate cancer.

The company said the clinical hold will not affect its plans to start enrolling patients in the trial of a related drug.

https://www.reuters.com/article/bellicum-fda/fda-places-clinical-hold-on-bellicums-cancer-therapy-idUSKBN28H1KS

Inovio doses first volunteer as part of its COVID-19 vaccine mid-stage trial

 Inovio Pharmaceuticals Inc said on Monday it has dosed the first participant in a mid-stage clinical trial testing its COVID-19 vaccine candidate, INO-4800.

The Phase 2 portion of the Phase 2/3 study will enroll about 400 participants who are 18 years or older, to assess the vaccine’s ability to produce immune response and to determine the appropriate doses for a later study, the company said.

Inovio said it plans to fully enroll the Phase 2 segment of the trial, which is being funded by the U.S. Department of Defense, by the end of this month.

The U.S. Food and Drug Administration in September put Phase 3 of the mid-to-late stage trial on hold, as it sought more information, including details on a delivery device called Cellectra used to inject INO-4800 into skin cells.

That portion of the trial remains on hold until Inovio resolves the agency’s questions, the company said.

Inovio said it plans to address the remaining device-related questions during the Phase 2 part and prior to the start of the Phase 3 segment of the trial.

https://www.reuters.com/article/health-coronavirus-inovio-pharma/inovio-doses-first-volunteer-as-part-of-its-covid-19-vaccine-mid-stage-trial-idUSKBN28H1L3