Sunesis Pharma gets European orphan drug status for vosaroxin to treat AML – pharmabiz.com

Sunesis Pharmaceuticals, Inc., a biopharmaceutical company, has received orphan drug designation from European Commission to its lead development candidate, vosaroxin, for the treatment of acute myeloid leukaemia (AML).

The designation provides for 10 years of marketing exclusivity in all EU member countries following product approval. Vosaroxin has previously received orphan drug and fast track designations from the US Food and Drug Administration (FDA).

“The European Commission’s decision reinforces the global potential of vosaroxin and recognizes the desperate need for new treatment options in AML, an indication for which treatment standards have not changed appreciably in the past thirty years,” said Dr Adam Craig, EVP Development & CMO of Sunesis. “European orphan drug designation is the latest in a series of regulatory milestones that have strengthened the commercial opportunity for vosaroxin on both sides of the Atlantic. These include the potential for market exclusivity to 2030 and an expedited review process in the US, as well as new patents issued in the European Union with exclusivity to 2025.”

As established by the European Medicines Agency (EMA), orphan designation is granted to product candidates intended for the treatment of a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union. It also provides for scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized marketing authorization, and certain financial incentives for companies developing orphan drug candidates. Orphan drugs are eligible for a reduction of fees associated with pre-authorization inspections, as well as marketing authorization application fees and certain other fees for qualifying companies.

Sunesis is currently enrolling patients in its VALOR trial, a phase III, multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial of vosaroxin in combination with cytarabine in first relapsed or refractory acute myeloid leukaemia.

VALOR is a phase III, randomized, double-blind, placebo-controlled, pivotal trial in patients with first relapsed or refractory AML. The trial is expected to enroll 450 evaluable patients at approximately 110 leading sites in the US, Canada, Europe, Australia and New Zealand. The VALOR trial is currently enrolling patients, who are randomized one to one to receive either vosaroxin on days one and four in combination with cytarabine daily for five days, or placebo in combination with cytarabine. Additionally, the VALOR trial employs an innovative, adaptive trial design that allows for a one-time sample size adjustment by the DSMB at the interim analysis to maintain adequate power across a broader range of survival outcomes. The trial’s primary endpoint is overall survival.

Vosaroxin is a first-in-class anti-cancer quinolone derivative, (AQD), a class of compounds that has not been used previously for the treatment of cancer. Vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis.

Sunesis is a biopharmaceutical company focused on the development and commercialization of new oncology therapeutics for the treatment of solid and hematologic cancers.

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Abbott to acquire Action Pharma's investigational compound, AP214 for … – pharmabiz.com

Abbott, a global, broad-based health care company, has entered into an agreement with a privately-owned Danish biotech company, Action Pharma A/S, to acquire all global rights to develop and commercialize investigational compound, AP214 for prevention of acute kidney injury (AKI).

Under the terms of the agreement, Abbott will provide a cash payment of $110 million to Action Pharma and will be responsible for funding all future development and commercialization activities regarding AP214. No later milestone payments or royalties will be paid to Action Pharma. Abbott expects to incur a one-time specified item in the second quarter of 2012, related to this payment.

AP214 is in development to prevent AKI associated with major cardiac surgery in patients at increased risk and has further potential in adjacent indications. AP214 is a hormone analogue that targets both systemic inflammation and cellular death (apoptosis) caused by hypoxia (lack of blood flow) that can occur during surgery. In September 2011, Action Pharma announced positive Phase 2b top-line results evaluating the efficacy, safety and tolerability of AP214. Abbott plans to conduct another Phase 2b study, which is expected to begin later this year.

This acquisition will enhance Abbott’s pipeline in renal care. Abbott has two investigational treatments in development for chronic kidney disease (CKD). Bardoxolone, a first-in-class anti-oxidant inflammation modulator that activates Nrf2, a pathway involved in the progression of CKD, is in phase III development with Reata Pharmaceuticals. Atrasentan, a compound discovered by Abbott scientists, is being evaluated in a phase II b study in patients with diabetic kidney disease.

“Clinical experience with AP214 in cardiac surgery patients suggests that it has the potential to be the first compound specifically approved to prevent acute kidney injury, a long-standing unmet need in the medical community,” said John Leonard, MD, senior vice president, pharmaceuticals, research and development, Abbott. “This acquisition complements and broadens Abbott’s late-stage renal care pipeline and builds on our existing experience in treating kidney disease.”

“We are very excited about the sale of AP214 to such a prominent global health care company as Abbott, which has key development competencies to continue the development of this drug candidate for the benefit of patients and physicians in the renal therapeutic area,” said Ingelise Saunders, CEO of Action Pharma. “We also wish to thank our shareholders for their continued support of Action Pharma.”

Acute kidney injury (AKI) is known to be associated with increased short- and long-term mortality and co-morbidity, as well as prolonged hospitalization and permanent decline in renal function. Currently there are no pharmaceuticals on the market for the treatment or prevention of AKI associated with major cardiac surgery.

Abbott is devoted to the discovery, development, manufacture and marketing of pharmaceuticals and medical products, including nutritionals, devices and diagnostics.

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Valeant Pharma to buy University Medical's OTC brand AcneFree and certain … – pharmabiz.com

Valeant Pharmaceuticals International, Inc.,a multinational specialty pharmaceutical company,  has agreed to acquire certain assets from University Medical Pharmaceuticals Corp., a specialty pharmaceutical company focused on skincare products, for approximately $64 million plus potential milestones based upon attainment of future revenue targets. University Medical’s main brand is AcneFree, a leading retail over-the-counter (OTC) acne treatment.

Total revenue in 2011 from the acquired assets was approximately $32 million. The transaction is expected to close by mid-year, subject to certain closing conditions including expiration of requisite regulatory waiting periods, and is expected to be immediately accretive.

“The addition of a leading OTC acne treatment will provide us with the ability to expand our OTC business,” stated J Michael Pearson, chairman and chief executive officer. “We believe that we can build upon our success with CeraVe in the retail channel through increased offerings to consumers. These new products will be immediately accretive to our operations and have not been factored into our 2012 financial guidance.”

Valeant Pharmaceuticals International, Inc. is a multinational specialty pharmaceutical company that develops, manufactures and markets a broad range of pharmaceutical products primarily in the areas of neurology, dermatology and branded generics.

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Quark Pharma extends pact with Pfizer to develop compounds containing … – pharmabiz.com

Silence Therapeutics Plc, a leading international RNAi therapeutics company, announced that its partner, Quark Pharmaceuticals, has extended its existing exclusive Licensing Agreement with Pfizer.

This amendment will enable Quark to perform a phase II a clinical study to assess the effect of PF-655 in a new indication, looking at visual function in patients with moderate and advanced Open-Angle Glaucoma (OAG). PF-655 incorporates Silence’s AtuRNAi technology and was sub-licensed to Pfizer by Quark in 2006, and on which Silence is entitled to receive a share of milestones and royalties that may be earned by Quark in the future on this compound.

The OAG study will be conducted in parallel with a Phase 2b study of PF-655 in diabetic macular oedema.

Silence has previously announced that it stood to receive up to $95m from Quark in relation to its licensing agreement with Pfizer. As a result of this amendment to the Quark/Pfizer license, Silence anticipates its share of these payments could now reach $120m. Silence has previously announced the receipt of $6m from Quark in relation to this licence.

Commenting on the announcement, Tony Sedgwick, Chief Executive Officer of Silence, said: “This is an exciting development for Silence. This will create a fifth external clinical programme using Silence’s IP and AtuRNAi, which is funded and managed by one of our partners. This will increase the potential share of milestones and royalties that Silence can earn under its agreement with Quark and is a further validation of the Silence technology.”

Quark and Pfizer have amended their existing exclusive Licensing Agreement on May 1 in order to enable Quark to perform a phase II a clinical study to assess the effect of PF-655 on visual function in patients with moderate and advanced Open-Angle Glaucoma (OAG). This study will be conducted in parallel with an ongoing phase II b study (QRK202) in diabetic macular edema (DME). The OAG study will evaluate the potential of PF-655 to enhance visual function in glaucoma. Under the amended agreement, should Pfizer assume development and potential commercialization of PF-655 in either or both indications following review of the Phase 2a PF-655 data, Quark will receive option exercise payments and be will eligible to receive development and regulatory milestones specific to each indication, as well as sales milestones and royalties. Quark may be eligible to receive additional total payments of up to approximately $165 million associated with development and approval of PF-655 for OAG.

Preclinical studies of PF-655 conducted by Quark suggest the potential of the compound as a neuroprotective and potentially neuroenhancing agent in diseases such as OAG, by preventing optic neural cell apoptosis and stimulating optic neural cell regeneration. In addition, in a Phase 2a study in patients with DME (Pfizer DEGAS study #B0451004), repeated injections of PF-655 showed a dose-dependent increase in visual acuity independent of changes in retinal thickness. The beneficial effects of PF-655 on visual function may potentially be due to effects on retinal cells themselves, rather than on vascular permeability.

The OAG study will be a Phase 2a, multi-center, double-masked, randomized, repeat dose, safety, tolerability and efficacy study in up to 108 patients with moderate and advanced OAG. In addition, Quark is currently conducting a Phase 2b study (QRK202) in DME patients testing higher doses of PF-655 alone and in combination with Lucentis(R) to further evaluate the safety and efficacy of PF- 655 in DME and to determine the optimal dose for pivotal Phase 3 studies.

Daniel Zurr, Ph.D. President and Chief Executive Officer of Quark stated: “We are very excited to evaluate the effect of PF-655 on visual loss in glaucoma in future clinical studies. The mechanism of action and biological activity of PF-655 are novel and its axon regenerating effects may provide a long awaited breakthrough in the treatment of glaucoma. We are pleased and grateful to our partner, Pfizer, for their support in pursuing this new indication.”

Quark Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company engaged in discovering and developing novel RNA interference (RNAi)-based therapeutics.

Silence Therapeutics plc is a leading biotechnology company dedicated to the discovery, development and delivery of targeted, systemic RNA interference (RNAi) therapeutics for the treatment of serious diseases.

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Sunesis Pharma gets European orphan drug status for vosaroxin to treat AML – pharmabiz.com

Sunesis Pharmaceuticals, Inc., a biopharmaceutical company, has received orphan drug designation from European Commission to its lead development candidate, vosaroxin, for the treatment of acute myeloid leukaemia (AML).

The designation provides for 10 years of marketing exclusivity in all EU member countries following product approval. Vosaroxin has previously received orphan drug and fast track designations from the US Food and Drug Administration (FDA).

“The European Commission’s decision reinforces the global potential of vosaroxin and recognizes the desperate need for new treatment options in AML, an indication for which treatment standards have not changed appreciably in the past thirty years,” said Dr Adam Craig, EVP Development & CMO of Sunesis. “European orphan drug designation is the latest in a series of regulatory milestones that have strengthened the commercial opportunity for vosaroxin on both sides of the Atlantic. These include the potential for market exclusivity to 2030 and an expedited review process in the US, as well as new patents issued in the European Union with exclusivity to 2025.”

As established by the European Medicines Agency (EMA), orphan designation is granted to product candidates intended for the treatment of a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union. It also provides for scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized marketing authorization, and certain financial incentives for companies developing orphan drug candidates. Orphan drugs are eligible for a reduction of fees associated with pre-authorization inspections, as well as marketing authorization application fees and certain other fees for qualifying companies.

Sunesis is currently enrolling patients in its VALOR trial, a phase III, multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial of vosaroxin in combination with cytarabine in first relapsed or refractory acute myeloid leukaemia.

VALOR is a phase III, randomized, double-blind, placebo-controlled, pivotal trial in patients with first relapsed or refractory AML. The trial is expected to enroll 450 evaluable patients at approximately 110 leading sites in the US, Canada, Europe, Australia and New Zealand. The VALOR trial is currently enrolling patients, who are randomized one to one to receive either vosaroxin on days one and four in combination with cytarabine daily for five days, or placebo in combination with cytarabine. Additionally, the VALOR trial employs an innovative, adaptive trial design that allows for a one-time sample size adjustment by the DSMB at the interim analysis to maintain adequate power across a broader range of survival outcomes. The trial’s primary endpoint is overall survival.

Vosaroxin is a first-in-class anti-cancer quinolone derivative, (AQD), a class of compounds that has not been used previously for the treatment of cancer. Vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis.

Sunesis is a biopharmaceutical company focused on the development and commercialization of new oncology therapeutics for the treatment of solid and hematologic cancers.

View the original article here