BioSpace News Archive

ADC Therapeutics to Participate in Cowen 41 st Annual Health Care Conference

Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the appointment of Cynthia Pussinen as chief technical officer (CTO).

– B-SIMPLE4 pivotal Phase 3 trial evaluating SB206 as a treatment for molluscum contagiosum progressing with top-line efficacy results targeted before the end of Q2 2021 – – Company targeting initiating preclinical studies for intranasal formulation of berdazimer sodium for COVID-19 program, SB019, in Q1 2021 – – Ongoing evaluations for strategic partnering opportunities to leverage broader pipeline to secure next phase of growth –

- Outlined strategy to build integrated cell therapy company and the Company’s “2-2-5-2” five-year core value drivers - - Reported compelling durable responses in patients with synovial sarcoma, and initial responses in lung, head and neck, liver, esophagogastric junction, and melanoma cancers confirming potential of SPEAR T-cells for solid tumors - -

Expects to report initial data from Phase 1 study of NTLA-2001, a potentially curative single-course therapy for transthyretin amyloidosis (ATTR), in 2021 On track to submit an IND or IND-equivalent in mid-2021 for NTLA-5001 for the treatment of acute myeloid leukemia (AML) Intends to submit an IND or IND-equivalent in 2H 2021 for NTLA-2002 for the treatment of hereditary angioedema (HAE)

– Completed acquisition of Eidos Therapeutics, allowing BridgeBio to deploy its full clinical and commercial infrastructure to develop and plan for potential global commercialization upon regulatory approval of Eidos’ product candidate, acoramidis, a potential best-in-class therapy for patients with amyloidosis cardiomyopathy (ATTR-CM)

Successfully launched CAPLYTA ® (lumateperone) in late March 2020 and demonstrated strong commercial execution in the midst of COVID-19. CAPLYTA achieved net product revenues of $12.4 million and $22.5 million for fourth quarter and full year 2020, respectively. Fourth quarter CAPLYTA total prescriptions (TRx) increased 77% versus the previous quarter. Pursuing CAPLYTA label expansion including bipolar depression and major depressive disorder:

--ARIKAYCE EU Launch Progresses, with Product Now Available and Reimbursed in Germany and the Netherlands-- --Frontline Clinical Trial Program of ARIKAYCE® (amikacin liposome inhalation suspension) in Nontuberculous Mycobacterial (NTM) Lung Disease Caused by Mycobacterium avium complex (MAC) Initiated in Q4 2020--

Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, today announced its financial results for the fourth quarter and year ended December 31, 2020 and provided a business update.

Axcella (Nasdaq: AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced three upcoming presentations at the NASH-TAG 2021 Conference, which is taking place March 11-13