Major October Catalyst for Outlook? Chardan

 Chardan initiated coverage on Outlook Therapeutics Inc 

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 with a Buy rating and a price target of $10 based on the company's commercial potential with its lead ophthalmic formulation of ONS-5010 (bevacizumab, Avastin), an anti-VEGF antibody in development for wet age-related macular degeneration (wet AMD). 

The company recently re-submitted the marketing application with the FDA for ONS-5010. It expects to learn of a potential PDUFA date by the end of October, with the potential FDA approval in late August 2023.

Chardan notes that ONS-5010 is being developed as an ophthalmic formulation of Roche Holdings AG's 

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 Avastin (bevacizumab) that was initially approved in 2004 for metastatic colorectal cancer.

But it has been widely adopted for off-label use by eyecare practitioners as an effective treatment for several retinal diseases.

Despite several approved anti-VEGF treatments available for wet AMD, off-label Avastin remains the treatment of choice, accounting for ~50% share of the anti-VEGF market, primarily driven by comparable efficacy and safety at a much lower cost ($50-$100 for Avastin injection compared to ~$2k for treatments from Regeneron Pharmaceuticals Inc 

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 and Novartis AG 

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.

We believe it is unlikely that practitioners will move away from bevacizumab, so providing an option for an FDA-approved ophthalmic formulation of the drug at a reasonable price point would likely be met with enthusiasm. 

https://www.benzinga.com/general/biotech/22/09/28853135/chardan-initiates-coverage-on-this-eye-stock-with-bullish-stance-on-its-lead-candidate

GENFIT Gets FDA Orphan Drug Status For Liver Disease Candidate

 The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to GENFIT’s 

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 drug candidate GNS5611 (ezurpimtrostat) for the treatment of cholangiocarcinoma.

The pre-clinical studies evaluating GNS561 has been completed and a Phase 1b trial confirming the rationale for targeting cholangiocarcinoma.

GNS561 (ezurpimtrostat) is a Palmitoyl Protein Thioesterase-1 (PPT-1)  inhibitor that blocks autophagy. Autophagy is activated in tumor cells in response to certain conditions, due to a tumor cell growth in advanced cancers.

Dr Mark Yarchoan, Associate Professor of Oncology at John Hopkins Medicine, commented: “Cholangiocarcinoma is a rare cancer with a high mortality rate. Patients have limited treatment options, particularly following first line therapy. This is why new therapies are urgently needed and is one of the reasons that GNS561 was granted Orphan Drug Designation by the FDA. There is a real path forward for new options for second line treatment in cholangiocarcinoma, and GNS561 represents a strong second-line therapy candidate and hope to patients.”

The company is expected to commence a Phase 2 in the fourth quarter 2022, and dosing of subjects is expected in the first quarter 2023.

Cholangiocarcinoma is a rare liver malignancy with high mortality and limited treatment options. It occurs mostly in people over the age of 50.

https://www.benzinga.com/general/biotech/22/09/28857097/genfit-gets-fda-orphan-drug-status-for-liver-disease-candidate

aTyr fast-tracked for Interstitial Lung Disease candidate

 aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotherapeutics company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced that the U.S. Food and Drug Administration (FDA) has granted the company Fast Track designation for its lead therapeutic candidate, efzofitimod, for the treatment of systemic sclerosis (SSc, also known as scleroderma)-associated, interstitial lung disease (ILD).

Efzofitimod is a first-in-class immunomodulator that downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states via selective modulation of neuropilin-2 (NRP2). Clinical proof-of-concept was recently established for efzofitimod in a Phase 1b/2a study in patients with pulmonary sarcoidosis, a major form of ILD, and the company is currently investigating efzofitimod in patients with pulmonary sarcoidosis in a global Phase 3 study called EFZO-FIT™.

https://finance.yahoo.com/news/atyr-pharma-receives-fda-fast-120000608.html