This week PharmaShots’ news was all about the updates on clinical trials, regulatory, biotech, pharma, M&A, biosimilar, and DigiHealth. Check out our full report below:

The US FDA has approved Novo Nordisk’ Rivfloza (nedosiran) for children aged ≥9 years and adults with primary hyperoxaluria type 1, based on the P-II trial (PHYOX 2) trial & interim data from P-III extension study (PHYOX 3)  

Read more: Novo Nordisk

The US FDA has accepted the BLA and granted Priority Review for Rocket Pharmaceutical’s RP-L201 to treat Severe Leukocyte Adhesion Deficiency-I 

Read more: Rocket Pharmaceutical

Agomab’ AGMB-129 Receives the US FDA’s Fast Track Designation for Fibrostenosing Crohn’s Disease & announced the initiation of the P-IIa trial (STENOVA) evaluating AGMB-129

Read more: Agomab

The NICE has recommended Boehringer Ingelheim’s Jardiance for symptomatic chronic heart failure with preserved or mildly reduced ejection fraction, based on the P-III (EMPEROR-Preserved) trial

Read more: Boehringer Ingelheim

The US FDA has granted Orphan Drug Designation to GC Biopharma's GC1126A for Thrombotic Thrombocytopenic Purpura

Read more: GC Biopharma

Syndax highlighted protocol-defined pooled analysis results from the (AUGMENT-101) trial results of Revumenib for r/r KMT2Ar Acute Leukemia which met its 1EPs & showed CR or a CR with CRh rate of 23% in 57 efficacy evaluable patients in the pooled KMT2Ar acute leukemia cohort

Read more: Syndax

Kashiv BioSciences enrolled the first patient in the P-III clinical study of ADL018 (biosimilar, omalizumab) for Chronic Spontaneous Urticaria

Read more: Kashiv BioSciences

Novartis highlighted pre-specified interim analysis results from the P-III study (APPLAUSE-IgAN) for Iptacopan to treat IgA nephropathy demonstrated the superiority of iptacopan vs PBO in proteinuria reduction

Read more: Novartis

PDS Biotech highlighted updated interim results from the P-II Trial (VERSATILE-002) results of PDS0101 + Keytruda for Head and Neck Cancer

Read more: PDS Biotech

BiomX completed patient dosing in part 2 of the P-Ib/IIa study for BX004 to treat Chronic Pulmonary Infections in patients with Cystic Fibrosis

Read more: BiomX

AstraZeneca highlighted P-III (T2NOW) trial results of Forxiga (dapagliflozin) for Type 2 Diabetes, which showed a significant reduction in A1C

Read more: AstraZeneca

The US FDA has placed a partial clinical hold on the new patient’s enrolment in the P-II trial (TELLOMAK) for Innate Pharma’ Lacutamab due to one fatal case of hemophagocytic lymphohistiocytosis

Read more: Innate Pharma

Neurocrine Biosciences highlighted P-III (CAHtalyst) pediatric study results of Crinecerfont in children and adolescents for Congenital Adrenal Hyperplasia showed a reduction from baseline in serum androstenedione from baseline

Read more: Neurocrine Biosciences

Regeneron highlighted two-year (PULSAR) trial results of Eylea HD (aflibercept) for wet age-related macular degeneration demonstrated sustained visual and anatomic improvements through 2yrs.

Read more: Regeneron

Tenaya Therapeutics dosed the first patient of TN-201 in the P-Ib trial (MyPeak-1) for MYBPC3-associated hypertrophic cardiomyopathy

Read more: Tenaya Therapeutics

The US FDA has cleared Anumana’s ECG-AI Algorithm to detect low ejection fraction in patients at risk of heart failure

Read more: Anumana

Sanofi & Janssen collaborated to develop and commercialize a vaccine candidate for extraintestinal pathogenic E. Coli  

Read more: Sanofi & Janssen

ATUM extends its collaboration with Anagram Therapeutics to develop enzyme therapies for malabsorption and nutrient metabolism disorders

Read more: ATUM and Anagram Therapeutics

Twist Bioscience & Bayer collaborated to advance drug discovery where Bayer gets an exclusive right to license the Abs for commercialization in all global territories

Read more: Twist Bioscience & Bayer

Sanofi & Teva collaborated to co-develop and co-commercialize TEV ‘574 for inflammatory bowel disease

Read more: Sanofi & Teva

BridgeBio & Resilience collaborated to manufacture and advance BBP-812 for Canavan Disease and BP-631 for Congenital Adrenal Hyperplasia

Read more: BridgeBio & Resilience

Alvotech & Kashiv BioSciences collaborated to develop and commercialize AVT23, a proposed biosimilar to Xolair (omalizumab)

Read more: Alvotech & Kashiv BioSciences

Kyowa Kirin to acquire Orchard Therapeutics for ~$477.6M. The acquisitor enriches Kyowa Kirin’s portfolio and enables the development of numerous promising candidates with a clinically differentiated platform

Read more: Kyowa Kirin & Orchard Therapeutics

Related Post: PharmaShots Weekly Snapshots (September 25–29, 2023)