Abby Hardy, Author at BioTuesdays

Developments

FDA clears Orchard Therapeutics IND for OTL-200

The FDA cleared Orchard Therapeutics’ (NASDAQ:ORTX) IND for OTL-200, a stem cell-based gene therapy for the treatment of metachromatic leukodystrophy (MLD). MLD is caused by a mutation in the arylsulfatase-A gene, which...

November 23, 2020

Developments

FDA approves Eiger’s Zokinvy for progeria

The FDA approved Eiger BioPharmaceuticals’ (NASDAQ:EIGR) Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome and progeroid laminopathies. Progeria is an extremely rare and fatal disease caused by a...

November 23, 2020

Developments

Seelos Therapeutics SLS-005 gets FDA orphan drug designation for ALS

Seelos Therapeutics’ (NASDAQ:SEEL) SLS-005 (trehalose) received FDA orphan drug designation for the treatment of amyotrophic lateral sclerosis (ALS). Trehalose is a low molecular weight disaccharide that crosses the...

November 19, 2020

Developments

Kintara Therapeutics reports interim data from two Phase 2 trials of VAL-083

Kintara Therapeutics (NASDAQ:KTRA) reported interim data from two Phase 2 trials evaluating VAL-083 for the treatment of glioblastoma multiforme (GBM). In the first Phase 2 study, newly diagnosed patients receiving VAL...

November 19, 2020

Developments

Selecta gets FDA rare pediatric disease designation for methylmalonic acidemia gene therapy

Selecta Biosciences’ (NASDAQ:SELB) MMA-101 received FDA orphan drug designation for the treatment of methylmalonic acidemia (MMA). MMA is caused by a mutation in the methylmalonyl-CoA mutase gene, which results in the...

November 19, 2020

Developments

DSMB approves continuation of Hepion’s Phase 2a NASH trial

Hepion Pharmaceuticals (NASDAQ:HEPA) reported that an independent data safety monitoring board (DSMB) approved the continuation of the company’s Phase 2a ‘AMBITION’ clinical trial to the next dose level cohort. After...

November 18, 2020

Developments

Kazia presents positive interim data from Phase 2 glioblastoma trial

At the Society for Neuro-Oncology annual meeting, Kazia Therapeutics (NASDAQ:KZIA) presented positive interim data from its Phase 2 trial evaluating paxalisib for the treatment of diffuse intrinsic pontine glioma...

November 18, 2020

Developments

Milestone Pharma enrolls first patient in Phase 3 etripamil trial

Milestone Pharmaceuticals (NASDAQ:MIST) enrolled the first patient in its Phase 3 RAPID trial evaluating etripamil for the treatment of paroxysmal supraventricular tachycardia (PSVT). PSVT is characterized by...

November 18, 2020

Developments

Chiasma reports positive topline results from Phase 3 acromegaly trial

Chiasma (NASDAQ:CHMA) reported positive topline results from its Phase 3 trial evaluating MYCAPSSA for the treatment of acromegaly. Designed to support a planned marketing authorization application in the EU, the non...

November 18, 2020

Developments

Synthetic Biologics engages AGP to explore strategic alternatives

Synthetic Biologics (NYSE American:SYN) engaged Alliance Global Partners as an advisor to help the company evaluate strategic alternatives. The company is considering a variety of options, including an acquisition...

November 17, 2020

Author - Abby Hardy

FDA clears Orchard Therapeutics IND for OTL-200

FDA approves Eiger’s Zokinvy for progeria

Seelos Therapeutics SLS-005 gets FDA orphan drug designation for ALS

Kintara Therapeutics reports interim data from two Phase 2 trials of VAL-083

Selecta gets FDA rare pediatric disease designation for methylmalonic acidemia gene therapy

DSMB approves continuation of Hepion’s Phase 2a NASH trial

Kazia presents positive interim data from Phase 2 glioblastoma trial

Milestone Pharma enrolls first patient in Phase 3 etripamil trial

Chiasma reports positive topline results from Phase 3 acromegaly trial

Synthetic Biologics engages AGP to explore strategic alternatives

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