3:45 PM
Apr 07, 2017
 |  BioCentury  |  Finance

To market, to market

Investors watching for new approvals and cancer, Orphan data in 2Q17

Investors are hoping that a slew of approvals and data readouts can maintain biotech’s momentum on the heels of several high-profile successes that came just before or after 1Q’s end.

Chief among those was Vertex Pharmaceuticals Inc.’s Phase III data for the combination of tezacaftor (VX-661) and Kalydeco ivacaftor in cystic fibrosis. Tezacaftor is expected to be part of a triple combination therapy that could potentially address a wider population of CF patients than those with delta F508 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR).

The doublet met the primary endpoint of an improvement in absolute percent predicted forced expiratory volume in one second (ppFEV1) in both the Phase III EVOLVE and Phase III EXPAND trials. EVOLVE enrolled patients with two copies of the delta F508 mutation, while EXPAND enrolled patients with residual CFTR function and one delta F508 mutation.

On March 29, Vertex jumped $18.34 (20%) to $108.01 after reporting the data, adding $4.6 billion in market cap.

Vertex is conducting Phase II trials of two separate triple combinations: CFTR corrector VX-440 plus tezacaftor/ivacaftor; and CFTR corrector VX-152 plus tezacaftor/ivacaftor. Data from both studies are expected next half.

OrbiMed Advisors’ Sven Borho told BioCentury, “The Vertex triple data is probably the most important of the year.”

“The Vertex triple data is probably the most important of the year.”

Sven Borho, OrbiMed

Two highly anticipated approvals also came just before the quarter’s end. Tesaro Inc.’s Zejula niraparib was approved three months ahead of its June 30 PDUFA date as maintenance treatment for recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer following platinum-based chemotherapy.

Several investors think approval further de-risks Tesaro and increases the likelihood that the cancer company will get taken out.

Dupixent dupilumab from partners Regeneron Pharmaceuticals Inc. and Sanofi also was approved in the last days of the quarter, for moderate to severe atopic dermatitis.

At least 17 PDUFA dates are expected in 2Q17, including several from mid-cap companies that investors believe could trigger more M&A activity (see “2Q Milestones”).

On the data side, one 2Q readout that was eagerly anticipated has already landed. On April 3, Paratek Pharmaceuticals Inc. announced that intravenous and oral formulations of omadacycline met the primary endpoints in the Phase III OPTIC trial to treat community-acquired bacterial pneumonia (CABP). Another Phase III trial of oral omadacycline is expected to report out in June.

The annual meeting of the American Society of Clinical Oncology (ASCO) is expected to yield greater clarity around which immuno-oncology combinations are working best in an increasingly complex and crowded field.

Investors will also be looking to data readouts and regulatory submissions in the Orphan disease space, and a handful of assorted other milestones in large and mid-cap names. Few small cap catalysts are on the radar for 2Q17.

Table: 2Q17 milestones

Selected products with late-stage clinical or regulatory milestones expected in 2Q17. (A) The MedImmune LLC unit of AstraZeneca plc (LSE:AZN; NYSE:AZN) has worldwide rights to tremelimumab from Pfizer Inc. (NYSE:PFE); (B) Uses Enhanze recombinant human hyaluronidase (rHuPH20) drug delivery technology from Halozyme Therapeutics Inc. (NASDAQ:HALO); Source: BCIQ: BioCentury Online Intelligence

AB Science S.A. (Euronext:AB)Masitinib Amyotrophic lateral sclerosis (ALS)Final Ph II/III dataMay
AcelRx Pharmaceuticals Inc. (NASDAQ:ACRX)Sufentanil sublingual tablet (ARX-04)Moderate to severe acute pain associated with trauma or injurySubmit MAA2Q17
Aclaris Therapeutics Inc. (NASDAQ:ACRS)A-101Seborrheic...

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