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Portola Pharmaceuticals Announces Collaboration With Ora, Inc. for Syk-selective Inhibitor (PRT2761) in Ophthalmic Diseases

SOUTH SAN FRANCISCO, Calif., May 5, 2015 (GLOBE NEWSWIRE) — Portola Pharmaceuticals (PTLA) today announced a collaboration with Ora, Inc. for the development of Portola's highly selective Syk inhibitor, PRT2761, in ophthalmic diseases. The inhibition of Syk may more completely prevent activation of mast cells in ocular allergy and, therefore, a Syk inhibitor could represent a promising therapeutic option.

"This collaboration for PRT2761 is part of a strategy that allows us to advance early-stage compounds outside of our key focus on thrombosis and hematologic cancer with innovative companies that have the expertise and resources to develop these agents to proof-of-concept. This collaboration provides us with the flexibility and optionality to build significant pipeline value," said William Lis, chief executive officer of Portola. "Once we achieve proof-of-concept with this asset, we will make a decision on the potential value of the program and our interest in further developing it."

"Portola's highly specific Syk inhibitor PRT2761 has the potential to deliver rapid onset of action and comprehensive treatment of both signs and symptoms of ocular allergy, including redness, inflammation, swelling and itching," said Stuart B. Abelson, president and chief executive officer of Ora. "We are impressed by its properties and preclinical data, which demonstrated activity in reducing conjunctival redness and swelling, a key clinical sign of allergic conjunctivitis that is not adequately treated by existing therapies."

Ora has a solid track record of successfully identifying and developing technologies to advance the standard of care of allergic eye disease. Over the past 30 years, Ora's preclinical and clinical Ora-CAC(TM) models have demonstrated their utility in translation and reproducibility leading to 20 ocular allergy drug approvals. Under the collaboration agreement, Ora will lead the clinical development of PRT2671, and the companies will share in the development expenses until proof-of-concept is established. Portola retains decision making oversight of the program.

About Portola Pharmaceuticals, Inc.

Portola Pharmaceuticals is a biopharmaceutical company developing product candidates that could significantly advance the fields of thrombosis and other hematologic diseases. The Company is advancing its three wholly-owned programs using novel biomarker and genetic approaches that may increase the likelihood of clinical, regulatory and commercial success of its potentially life-saving therapies. Portola's partnered programs are focused on developing selective Syk inhibitors for inflammatory conditions.


Portola's wholly-owned, oral, once-daily Factor Xa inhibitor betrixaban is being evaluated in the only biomarker-based Phase 3 study for hospital-to-home prophylaxis of venous thromboembolism (VTE) in acute medically ill patients. Betrixaban's distinct properties may have the potential to allow the agent to demonstrate efficacy without the significant increase in the rate of major bleeding that was seen in this patient population with other Factor Xa inhibitors. If approved, betrixaban could be the first anticoagulant for both hospital and post-discharge VTE prophylaxis and the standard of care in this large market of more than 20 million patients in the G7 countries alone.

Andexanet Alfa

Andexanet alfa, an FDA-designated breakthrough therapy, is a recombinant protein designed to reverse the anticoagulant effect in patients treated with an oral or injectable Factor Xa inhibitor. Andexanet alfa has the potential to be a first-in-class antidote for anticoagulated patients who suffer a major bleeding episode or require emergency surgery. Portola has entered into Phase 3 clinical collaboration agreements with all of the manufacturers of direct Factor Xa inhibitors while retaining all commercial rights to andexanet alfa. The Company is currently evaluating andexanet alfa in the Phase 3 and Phase 4 ANNEXA(TM) (Andexanet Alfa a Novel Antidote to the Anticoagulant Effects of FXa Inhibitors) registration studies.


Portola's product candidate in the area of hematologic cancer, cerdulatinib, is an orally available molecule that uniquely inhibits two validated tumor proliferation pathways — spleen tyrosine kinase (Syk) and janus kinase (JAK). It is currently being evaluated in a Phase 1/2a proof-of-concept study in patients with B cell leukemias or lymphomas with a focus on genetically-defined subtypes, as well as in patients who have failed therapy due to relapse or acquired mutations.

For more information, visit and follow the Company on Twitter @Portola_Pharma.

About Ora, Inc.

Ora, Inc. is a global, full-service ophthalmic clinical research and product development firm. Over the past 30 years, it has helped clients earn 39 FDA approvals. Ora supports a wide array of organizations, from start-ups to global pharmaceutical and device companies, to efficiently and successfully bring their new products from concept to market. Ora's pre-clinical and clinical models, unique methodologies and regulatory strategies have been refined and proven across thousands of projects both in the US and internationally. Ora brings together the world's most extensive and experienced network of ophthalmic experts and R&D professionals in order to maximize the value of new product initiatives. For additional information, please visit

Forward-Looking Statement

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: Portola's plans for future clinical studies for PRT2761, the potential indications, efficacy, safety and activity of PRT2761, and the potential market and indications for its other product candidates. Risks that contribute to the uncertain nature of the forward-looking statements include: the accuracy of Portola's estimates regarding its ability to initiate and/or complete its clinical trials; the success of Portola's clinical trials and the demonstrated efficacy of Portola's product candidates thereunder; the accuracy of Portola's estimates regarding its expenses and capital requirements; Portola's ability to manufacture andexanet alfa; regulatory developments in the United States and foreign countries; Portola's ability to obtain and maintain intellectual property protection for its product candidates; and the loss of key scientific or management personnel. These and other risks and uncertainties are described more fully in Portola's most recent filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K, which was filed on March 2, 2015. All forward-looking statements contained in this press release speak only as of the date on which they were made. Portola undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.


Investor Contact:
Michele Mantynen
Portola Pharmaceuticals
Media Contact:
Joey Fleury