Curis: Partial Clinical Hold for TakeAim Leukemia Study of Emavusertib

 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, today announced that the U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the Company's TakeAim Leukemia Phase 1/2a study (NCT04278768). The TakeAim Leukemia study is a Phase 1/2a open-label, single arm dose escalation and expansion study of orally-administered emavusertib (CA-4948) as monotherapy and in combination with azacitidine or venetoclax in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS).

While the partial hold is in place, no new patients will be enrolled in the study, and current study participants benefitting from treatment may continue to be treated with emavusertib at doses of 300mg BID or lower.

With the partial hold, the FDA is requesting additional data from the study, including data related to the death of a R/R AML patient who experienced, among several conditions, rhabdomyolysis, which has previously been identified as a dose-limiting toxicity of emavusertib. Additionally, the FDA is requesting safety, efficacy, and other data, including data related to rhabdomyolysis and the Company's determination of the Recommended Phase 2 Dose for emavusertib in this study.

https://finance.yahoo.com/news/curis-announces-fda-partial-clinical-110000554.html

Alnylam: 3-Month Extension of Review Period for New Drug Application for Vutrisiran

 – New PDUFA goal date scheduled for July 14, 2022, due to amendment to address pending inspection classification at third-party secondary packaging and labeling facility –

– New facility has been identified and incorporated into existing regulatory submission which requires additional review time at FDA –

– No inspection observations were directly related to vutrisiran and no additional clinical data have been requested by FDA –

https://finance.yahoo.com/news/alnylam-announces-3-month-extension-113000217.html

Protalix, Chiesi : Positive Topline Results from Phase III Fabry Trial

 PRX-102 successfully met the primary endpoint on kidney function in active control, non-inferiority study vs. agalsidase beta

Topline results demonstrated a favorable tolerability and immunogenicity profile for PRX-102

BLA resubmission planned for the second half of 2022

https://finance.yahoo.com/news/protalix-biotherapeutics-chiesi-global-rare-105000805.html

OrthoPediatrics to Acquire MD Orthopaedics

 OrthoPediatrics Corp. (“OrthoPediatrics” or the “Company”) (Nasdaq: KIDS), a company focused exclusively on advancing the field of pediatric orthopedics, announced the acquisition of MD Orthopaedics, including its bracing product lines and the patented Mitchell Ponseti® Ankle-Foot Orthosis (AFO) system for the treatment of clubfoot. OrthoPediatrics will acquire MD Orthopaedics in a transaction valued up to $19.6 million. Under the terms of the agreement, OrthoPediatrics will acquire MD Orthopaedics for an upfront cash payment of $8.2 million and $8.9 million in company stock, with an additional $2.5 million in restricted stock which vest after three years.

Founded in 2004 in Wayland, Iowa, MD Orthopaedics is a profitable, privately held orthopedic device company that has developed and commercialized a custom fit bracing system that has supported the dissemination of the Ponseti Technique - the gold standard treatment for clubfoot. MD Orthopaedics will serve as a specialty bracing platform company within OrthoPediatrics Trauma & Deformity business. While this unique non-operative solution will maintain its focus on the treatment of clubfoot, OrthoPediatrics plans to leverage the MD Orthopaedics organization to develop several new innovative products that address the large unmet needs for specialty bracing within the pediatric orthopedic market. With an estimated 80% of pediatric orthopedic care involving non-surgical treatment, MD Orthopaedics expands OrthoPediatrics’ Total Addressable Market by an estimated $600 million. Since inception in 2004, MD Orthopaedics has helped an estimated 300,000 children across the globe.

https://finance.yahoo.com/news/orthopediatrics-corp-announces-acquisition-md-120000371.html

Precigen Fast Tracked for Acute Myeloid Leukemia

 – Acute myeloid leukemia (AML) is among the most common types of leukemia in adults –

– PRGN-3006 UltraCAR-T previously received orphan drug designation (ODD) in patients with AML by the US Food and Drug Administration (FDA) –

– PRGN-3006 UltraCAR-T has demonstrated a favorable safety profile to date with no dose-limiting toxicities or neurotoxicity, and has demonstrated dose-dependent in vivo expansion and durable persistence –

https://finance.yahoo.com/news/precigen-receives-fast-track-designation-120500568.html

Genentech Ocrevus Benefits in Disability Progression, Cognitive Decline in MS

 

• 75% of patients with secondary progressive multiple sclerosis (SPMS) and primary progressive MS (PPMS) achieved no evidence of progression (NEP) in a one-year interim analysis of CONSONANCE study
• 70% of patients with SPMS and PPMS demonstrated stable or improved cognition after one year of Ocrevus treatment in CONSONANCE
• Separate analysis on treatment disparities showed fewer Black and Hispanic patients with MS initiate high-efficacy treatments within two years of diagnosis
• Data at AAN support the body of evidence for Ocrevus more than 450,000 patient years and more than 225,000 patients treated globally

https://www.biospace.com/article/releases/new-data-for-genentech-s-ocrevus-ocrelizumab-show-benefit-in-disability-progression-and-cognitive-decline-in-both-secondary-progressive-and-primary-progressive-multiple-sclerosis/

FDA Grants Priority Review to Genentech Actemra for COVID-19 in Hospitalized

 

• If approved, Actemra would be the first U.S. FDA-approved immunomodulator for the treatment of COVID-19 in hospitalized patients
• Since the beginning of the pandemic, more than one million people hospitalized with COVID-19 have been treated with Actemra worldwide
• Actemra was previously granted Emergency Use Authorization for the treatment of COVID-19 in hospitalized adults and children

https://www.biospace.com/article/releases/u-s-fda-grants-priority-review-to-genentech-s-actemra-for-the-treatment-of-covid-19-in-hospitalized-adults/