Regulatory Hurdle For Innate Pharma's Blood Cancer Studies

 The FDA has placed a clinical hold on Innate Pharma SA's IPHA lacutamab Investigational New Drug (IND), leading to a pause in new patient enrollment in ongoing lacutamab trials IPH4102-201 (Phase 2 TELLOMAK) and 102 (Phase 1b PTCL). 

The partial clinical hold follows one fatal hemophagocytic lymphohistiocytosis (HLH), a rare hematologic (blood) disorder. 

Patients already on study treatment who are deriving clinical benefits may continue treatment after being reconsented.

TELLOMAK, Innate Pharma's ongoing Phase 2 trial of lacutamab in cutaneous T-cell lymphoma (CTCL), completed enrollment in Q2 2023 (n=170 patients). 

Enrollment is also completed in the initial cohort (n=20 patients) of the Phase 1b PTCL trial and is awaiting a futility interim analysis to progress to the next stage. 

Innate Pharma is on track for final data from the Phase 2 TELLOMAK trial and preliminary data on PTCL in Q4 2023.

"...Additionally, with all patients recruited into the Phase 2 TELLOMAK study, we do not currently anticipate any delay for the TELLOMAK Phase 2 final data due shortly," commented Mondher Mahjoubi, Chief Executive Officer of Innate Pharma. 

https://www.benzinga.com/general/biotech/23/10/35102760/regulatory-hurdle-for-innate-pharmas-blood-cancer-studies

Longeveron Positive Top-Line Results for Lomecel-B in Phase 2a Alzheimer's trial

 Company to Hold Conference Call & Webcast Today, October 5 at 8:00am ET

• Primary Endpoint of Safety Met Across all Study Groups
• Statistical Significance Met for Secondary Endpoint Composite Alzheimer’s Disease Score (CADS) for Lomecel-B™ Low-Dose and for the Pooled Lomecel-B™ Treatment Group Relative to Placebo
• Other Doses Demonstrated Numerical Slowing/Prevention of Disease Worsening Relative to Placebo
• Additional Biomarker Data Expected to be Announced in the Coming Weeks

https://www.globenewswire.com/news-release/2023/10/05/2755238/0/en/Longeveron-Announces-Positive-Top-Line-Results-for-Lomecel-B-in-its-CLEAR-MIND-Phase-2a-Clinical-Trial-in-the-Treatment-of-Mild-Alzheimer-s-Disease.html

Conference Call and Webcast Details
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Kyowa Kirin to Acquire Orchard Therapeutics

 -Orchard Therapeutics is a global gene therapy leader with experience successfully developing and commercializing medicines for rare diseases-

-Acquisition enriches Kyowa Kirin’s portfolio, enables the development of numerous promising candidates with a clinically differentiated platform, and helps resource the ongoing and future launches of Libmeldy® (atidarsagene autotemcel)-
-Acquisition price of $16.00 per ADS in cash plus an additional contingent value right of $1.00 per ADS, representing a total maximum equity value of approximately $477.6 million-
-Kyowa Kirin to host investor conference call on Oct 5 at 18:30 p.m. JST-

https://www.globenewswire.com/news-release/2023/10/05/2755103/0/en/Kyowa-Kirin-to-Acquire-Orchard-Therapeutics.html

Apellis Prelim Revenues $74 M for SYFOVRE® (pegcetacoplan injection) in Q3

 

• Continued strong demand, with more than 100,000 vials (commercial and sample) distributed to date; growth in week-over-week demand returned starting in August
  
• Permanent J-code effective as of October 1, and more than 95% of all Medicare payers now covering SYFOVRE
  
• Up to 45% reduction of nonsubfoveal GA lesion growth observed between Months 24-30 compared to projected sham in the GALE extension study
  
• Estimated rate of retinal vasculitis continues to be rare at 0.01% per injection

https://www.globenewswire.com/news-release/2023/10/05/2755234/0/en/Apellis-Announces-Preliminary-U-S-Net-Revenues-of-Approximately-74-Million-for-SYFOVRE-pegcetacoplan-injection-in-the-Third-Quarter-of-2023.html

J&J started at Outperform by RBC

 Target $178

https://finviz.com/quote.ashx?t=JNJ&p=d

uniQure reorg to cut projects, jobs

 Dutch gene therapy maker uniQure said on Thursday it would cut about 20% of its total workforce, or about 114 jobs, and end investments in more than half of its research and technology projects to reduce costs.

UniQure expects to generate savings of about $180 million over the next three years through the restructuring.

The company had cash, cash equivalents and investment securities of $628.6 million as of June 30, to fund operations into the second quarter of 2027, it said on Thursday.

CSL had acquired exclusive global rights to Hemgenix, the world's most expensive treatment, in June 2020 from uniQure, which retains the rights to future milestones totaling up to $1.5 billion and maintain an interest in the therapy's royalties.

CSL late last year gained the U.S. health regulator's approval for Hemgenix, a one-time gene therapy used for treating a rare genetic blood clotting disorder, and set its list price at $3.5 million.

UniQure plans to focus on gene therapies targeting central nervous system and liver-related diseases, including its drug candidates in development for Huntington's disease, Fabry disease and a form of amyotrophic lateral sclerosis

https://finance.yahoo.com/news/1-gene-therapy-maker-uniqure-121540093.html

Merck reports positive results in late-stage trial of Keytruda as treatment for bladder cancer

 Merck & Co. Inc. (MRK) said Thursday a late-stage trial of its cancer treatment Keytruda met its main goal of disease-free survival for patients with advanced bladder cancer. The Rahway, N.J.-based drug giant said Keytruda "demonstrated a statistically significant and clinically meaningful improvement in DFS versus observation in these patients after surgery." The Phase 3 trial will continue to evaluate a dual primary endpoint of overall survival. "Up to half of patients with bladder cancer who undergo surgery will experience recurrence within a year, underscoring the need for new treatment options in the adjuvant setting," said Dr. Marjorie Green, senior vice president and head of late-stage oncology, global clinical development, Merck Research Laboratories. The stock was slightly higher premarket but is down 8% in the year to date, while the S&P 500 has gained 11%.

https://www.morningstar.com/news/marketwatch/20231005240/merck-reports-positive-results-in-late-stage-trial-of-keytruda-as-treatment-for-bladder-cancer