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A clinical quarter

3Q17 offers a full slate of clinical milestones and PDUFA decisions

Buysiders will find themselves digesting at least 23 Phase III data readouts in the next quarter, two of which could provide clarity on increasingly competitive markets in lung cancer and hemophilia.

The third quarter is also slated to deliver at least two dozen PDUFA and BsUFA dates, which could give at least two platform companies their first FDA approvals.

The number of disclosed PDUFA dates for products likely to be classified as NMEs and new BLAs -- seven -- is on par with that seen in the first two quarters of the year, and any more approvals will pad 2017’s lead over 2016. FDA has already approved 23 NMEs and new BLAs this year, compared with 22 in all of 2016.

The most eagerly awaited data are from AstraZeneca plc’s Phase III MYSTIC study of PD-L1 inhibitor Imfinzi durvalumab in combination with CTLA-4 inhibitor tremelimumab in first-line non-small cell lung cancer, which could determine AZ’s place in the first-line NSCLC market.

Buysiders who spoke with BioCentury are cautious at best on the data.

“It’s not a given that it comes out cleanly,” said LSP’s Joep Muijrers.

The investors are generally more optimistic about other upcoming readouts.

Three sets of hemophilia data from BioMarin Pharmaceutical Inc., Spark Therapeutics Inc. and Roche have the potential to change the treatment landscape, which is dominated by producers of factor replacement therapies, including Shire plc and Bioverativ Inc.

Other top-of-mind readouts include Phase III data from Alnylam Pharmaceuticals Inc. and Sanofi for RNAi therapy patisiran in hereditary transthyretin (TTR) amyloidosis and Phase II triple combination data from Vertex Pharmaceuticals Inc. for cystic fibrosis.

A noteworthy regulatory event will be FDA’s July 12 Oncologic Drugs Advisory Committee (ODAC) meeting on Novartis AG’s BLA for tisagenlecleucel-T (CTL019) to treat relapsed or refractory B cell acute lymphoblastic leukemia (ALL), as investors anticipate the first potential FDA approvals for CAR T therapies from Novartis and Kite Pharma Inc. in 4Q17.

MYSTIC’s mystique

AstraZeneca’s MYSTIC data are landing in an evolving landscape for first-line NSCLC.

Opdivo nivolumab from Bristol-Myers Squibb Co. missed the primary endpoint of the Phase III CheckMate -026 study in first-line NSCLC last August.

In May, FDA approved rival PD-1 mAb Keytruda pembrolizumab from Merck & Co. Inc. in combination with chemotherapy for first-line NSCLC regardless of PD-L1 expression. Keytruda monotherapy is marketed to treat first-line NSCLC in patients whose tumors express PD-L1.

Loncar Fund’s Brad Loncar is pessimistic about MYSTIC. He thinks “they shifted the focus of that trial

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