5:20 PM
Jul 07, 2017
 |  BioCentury  |  Finance

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 to monotherapy from combinations,” which could indicate “the CTLA-4 half of the combination is out of favor.”

“Let’s assume that data are not as good as some people are thinking -- that’s a sentiment killer.”

Joep Muijrers, LSP

“Let’s assume that data are not as good as some people are thinking -- that’s a sentiment killer,” said Muijrers.

MYSTIC had been expected to read out on progression free survival (PFS) in 1H17. But in January, AZ updated the primary endpoints to include overall survival (OS) for durvalumab monotherapy in PD-L1-positive patients in addition to existing primary endpoints evaluating OS and PFS for the combination.

PFS data are now due in mid-2017, with OS data expected by next year.

BMS is evaluating the combination of Opdivo and Yervoy ipilimumab in the Phase III CheckMate -227 trial, which is slated to read out in 2018.

Table: 2H17 milestones

Selected products with clinical or regulatory milestones expected in 2H17. Source: BCIQ: BioCentury Online Intelligence

AB Science S.A. (Euronext:AB)MasitinibAmyotrophic lateral sclerosis (ALS)MAA approval4Q17
Ablynx N.V. (Euronext:ABLX) CaplacizumabThrombotic thrombocytopenic purpura (TTP)Ph III HERCULES data2H17
AcelRx Pharmaceuticals Inc. (NASDAQ:ACRX) / Gruenenthal GroupZalviso sufentanilModerate to severe acute pain in a hospital settingResubmit NDAYE17
Achaogen Inc. (NASDAQ:AKAO) / Ionis Pharmaceuticals Inc. (NASDAQ:IONS)PlazomicinCarbapenem-resistant Enterobacteriaceae (CRE) infection and complicated urinary tract infections (cUTIs)Submit NDA2H17
Aclaris Therapeutics Inc. (NASDAQ:ACRS) A-101Seborrheic keratosisSubmit MAA; PDUFA date2H17; 12/24/17
Acorda Therapeutics Inc. (NASDAQ:ACOR) CVT-301Parkinson’s diseaseSubmit MAA2H17
Adamas Pharmaceuticals Inc. (NASDAQ:ADMS)Amantadine extended releasePDPDUFA date8/24/17
Advanced Accelerator Applications S.A. (NASDAQ:AAAP)Lutathera (177-Lu-Dotatate)Gastroenteropancreatic neuroendocrine tumorsResubmit NDAMid-2017
Advaxis Inc. (NASDAQ:ADXS) / Biocon Ltd. (NSE:BIOCON; BSE:BIOCON)Axalimogene filolisbacMetastatic cervical cancerSubmit MAA2H17
Agios Pharmaceuticals Inc. (NASDAQ:AGIO)IvosidenibAcute myelogenous leukemia (AML)Submit NDAYE17
Agios Pharmaceuticals Inc. (NASDAQ:AGIO) / Celgene Corp....

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