Week Ahead In Biotech: PDUFA Date Ahead For Astra Zeneca, Merck's Lynparza, Pending Clinical Readouts In Focus

Biotech stocks advanced in the penultimate week of the year, and the iShares NASDAQ Biotechnology Index IBB is on track to end the year with gains in excess of 25%.

The previous week saw FDA announcements on verdicts for several therapeutic options.

Notable among the therapeutic options that crossed the FDA hurdle were Allergan plc AGN's migraine drug; Flexion Therapeutics Inc FLXN's Zilretta, which was given a label expansion; Intra-Cellular Therapies Inc ITCI's schizophrenia drug; and two generics for Eliquis, a blood thinner marketed by Bristol-Myers Squibb Co BMY and Pfizer Inc. PFE.

However, Correvio Pharma Corp CORV's Brinavess, a drug to treat atrial fibrillation, stumbled at the FDA altar.

A big gene therapy collaboration agreement between Roche Holdings AG Basel ADR RHHBY and Sarepta Therapeutics Inc SRPT was announced. 

The following are key biotech catalysts for the unfolding week:

PDUFA Dates

The FDA is scheduled to rule on the pancreatic cancer drug candidate lynparza, which is being co-developed by AstraZeneca plc AZN and Merck & Co., Inc. MRK. The PDUFA date is set for the fourth quarter.

Clinical Readouts

Q4 Releases

• Sol Gel Technologies Ltd SLGL: Phase 3 data for the acne treatment candidate TWIN (tretinoin and benzoyl peroxide)
• Vertex Pharmaceuticals Incorporated VRTX: Phase 1 data for VX-147 (for treating APOL1-mediated focal segmental glomerulosclerosis) in healthy volunteers
• Molecular Templates Inc MTEM: updated Phase 1 data for MT-511 in HER2-positive solid tumors, updated Phase 2 data for MT-3724 in combination with chemotherapy for relapsed/refractory diffuse large B-cell lymphoma, updated Phase 2 data for MT-3724 in combination with Bristol-Myers Squibb's Revlimid in diffuse B-cell lymphoma and updated Phase 2 data for MT-3724 monotherapy in relapsed/refractory diffuse large B-cell lymphoma
• Xeris Pharmaceuticals Inc XERS: Phase 2b data for self-administered glucagon in exercise-induced hypoglycemia
• Gilead Sciences, Inc. GILD: Phase 2 data for GS-9688 for HBV
• Eloxx Pharmaceuticals Inc ELOX: Phase 2 data for ELX-02 in cystinosis
• Celsion Corporation CLSN: Phase 1/2 data for GEN-1 in ovarian cancer
• Novartis AG NVS: NDA: Phase 3 data for PDR001 in BRAFV600 mutant metastatic melanoma
• Incyte Corporation INCY: Phase 3 data for itacitinib in treatment-naïve acute GVHD
• Applied Therapeutics Inc APLT: Additional Phase 1/2 data for AT-007 in galactosemia
• Axsome Therapeutics Inc AXSM: Phase 3 data for AXS-07 in migraine
• Spero Therapeutics Inc SPRO: top-line data from a Phase 1 study of SPR206 in healthy volunteers
• DURECT Corporation DRRX: top-line data from a Phase 2 study of DUR-928 in psoriasis
• Ultragenyx Pharmaceutical Inc RARE: Phase 1/2 third cohort data for DTX301 in ornithine transcarbamylase deficiency
• Obseva SA OBSV: Interim analysis of data from Part B of a Phase 2a study of OBE022 in pre-term labor
• Principia Biopharma Inc PRNB: updated Phase 2 data for PRN1008 in pemphigus
• Oramed Pharmaceuticals, Inc. ORMP: data from the first cohort of a Phase 2 study of ORMD-0801 in non-alcoholic steatohepatitis
• Inovio Pharmaceuticals Inc INO: Phase 1/2a data for INO-4700 for treating MERS virus

See also: 8 Biotech Stocks Morgan Stanley Recommends For 2020

Second-Half 2019 Releases

• GlaxoSmithKline plc GSK: Pivotal Phase 1 data for TSR-042 in endometrial cancer

Year-End Releases

• argenx SE – ADR ARGX and Halozyme Therapeutics, Inc. HALO: Phase 1 data for efgartigimod using Halozyme's ENHANZE drug delivery technology
• Sorrento Therapeutics Inc SRNE: Phase 1b data for resiniferatoxin in osteoarthritis of knee
• MEI Pharma Inc MEIP: updated Phase 2 data for pracinostate in combination with Bristol-Myers Squibb's Vidaza in high-risk myelodysplastic syndrome
• Sellas Life Sciences Group Inc SLS: initial Phase 2 data for nelipepimut-S in ductal carcinoma in Situ
• Lineage Cell Therapeutics Inc LCTX: Phase 1 immunogenicity data for VAC2 in non-small cell lung cancer
• Amicus Therapeutics, Inc. FOLD: additional Phase 1/2 data for CLN6 gene therapy in Batten disease, additional Phase 1/2 data for ATB200 in Pompe disease
• Oncolytics Biotech, Inc. ONCY: interim data for pelareorep and Roche's Tecentriq in breast cancer
• Regenxbio Inc RGNX: updated Phase 1/2 data for RGX-501 in homozygous familial hypercholesterolemia
• Merus NV MRUS: Phase 1 data for MCLA-158 in colorectal cancer

2019 And Late 2019 Releases

• Alnylam Pharmaceuticals, Inc. ALNY: Phase 1/2 data for ALN-AAT02 in alpha-1 anti-trypsin deficiency-associated liver disease

Planned NDA/BLA filings

• Coherus Biosciences Inc CHRS: BLA submission for Lucentis biosimilar planned for the fourth quarter
• Eli Lilly And Co LLY: NDA filing for LOXO-292 in RET-fusion non-small cell lung cancer, medullary thyroid cancer and other tumors planned for year end 2019
• Morphosys Ag MOR: Rolling BLA submission for lenalidomide in combination with MOR208 in relapsed or refractory diffuse large B cell lymphoma expected to be completed by the end of 2019
• Incyte and Novartis: NDA submission for INC280 in non-small cell lung cancer due 2019
• Chiasma Inc CHMA: NDA filing for mycapssa in acromegaly due in the fourth quarter
• G1 Therapeutics Inc GTHX: Rolling submission for trilaciclib in second/third-line setting in small-cell lung cancer expected to begin in the fourth quarter
• Eyepoint Pharmaceuticals Inc EYPT: sNDA filing for a six-month formulation of Yutiq in non-infectious uveitis expected to be complete in 2019
• Bausch Health Companies Inc BHC and Eton Pharmaceuticals Inc ETON plan to make a NDA resubmission for EM-100 in allergic conjunctivitis
• Vanda Pharmaceuticals Inc. VNDA: sNDA filing for Hetlioz in SmIth-Magenis syndrome due in the fourth quarter
• Clovis Oncology Inc CLVS: sNDA filing for Rubraca in BRCA-mutant advanced prostate cancer planned for the fourth quarter
• PTC Therapeutics, Inc. PTCT: BLA filing for GT-AADC for aromatic L-amino acid decarboxylase deficiency planned for the fourth quarter
• Sanofi SA SNY and Regeneron Pharmaceuticals Inc REGN: sBLA filing for Dupixent in atopic dermatitis for 6-11-year-olds, planned for the fourth quarter
• Clovis Oncology Inc CLVS: sNDA filing for Rucaparib in castrate-resistant prostate cancer planned for the fourth quarter
• PLx Pharma Inc PLXP: sNDA filing for Aspertec in cardiovascular and stroke patients planned for late 2019
• Aquestive Therapeutics Inc AQST: NDA resubmission for APL-130277 in Parkinson's disease with motor fluctuations planned for the fourth quarter

Related Link: 10 Biotech M&A Targets Under The Scanner For 2020