4/3/2012

Aptar Pharma’s Device Delivers INSYS Therapeutics’ Drug

Aptar Pharma recently announced INSYS Therapeutics’ SUBSYS, which uses an Aptar Pharma Sublingual Unit-Dose Spray (UDS) device, has been launched in the US for breakthrough cancer pain in patients who are 18 years of age and older and who are already tolerant to opioid therapy.

SUBSYS (fentanyl) sublingual spray is indicated for the treatment of breakthrough cancer pain (BTCP) in opioid-tolerant adult patients who are already routinely taking other opioid pain medicines around-the-clock for cancer pain. Breakthrough Cancer Pain is often defined as a temporary flare in pain that can be quite severe. These flares occur in the context of background pain that is otherwise usually well controlled with opioids.

Until now, fentanyl has been available in many dosage forms, including nasal, sublingual dissolving tablets, and oral transmucosal. SUBSYS marks the arrival of the first-ever UDS spray for fentanyl.

Estimated at over $2 billion in 2009, the world market for mucosal systemic drug delivery has experienced strong growth in the past few years. Continued success has been seen in the nasally administered treatment of migraine attacks with Triptans, Butorphanol, and Dihydroergotamine. New types of mucosal treatment have emerged with new product launches, a recent example being an orally sprayed Zolpidem for the treatment of insomnia.

Aptar Pharma’s UDS is the preferred drug delivery technology spray platform when dose accuracy and ease of administration are critical. UDS’ primeless feature offers the patient unique ready-to-use convenience that does not exist with traditional multi-dose spray pumps, which need to be primed prior to first use and sometimes re-primed when used infrequently.

“At Aptar Pharma, we are extremely pleased with the recent approval of SUBSYS, which uses a custom designed sublingual UDS device,” said Pierre Carlotti, Vice President Marketing and Communication for Aptar Pharma Prescription Division. “We look forward to the success of SUBSYS and INSYS as a serious emerging player in the treatment of breakthrough cancer pain.”

“It is amazing to think that when the SUBSYS project first started, INSYS was a small start-up with just 8 people and a dream,” added Mike Babich, President and CEO for INSYS. “Our initial trust in Aptar Pharma and their collaborative spirit as a delivery and development partner were key in helping to get SUBSYS to market.”

For more information about INSYS, visit www.insysrx.com. For more information about Aptar, visit www.aptar.com/pharma.

PharmaForm Announces Manufacturing Agreement

PharmaForm recently announced that Corcept Therapeutics Inc. has chosen them as their primary commercial manufacturer for their newly approved drug product, Korlym. The US FDA approved Corcept’s Korlym in February 2012 for patients with endogenous Cushing’s syndrome. PharmaForm has worked with Corcept for several years as a contract provider for services in the development, optimization, and validation of the manufacturing process for Corcept’s Korlym.

PharmaForm, a wholly owned subsidiary of Akela Pharma, Inc., is a leading specialty contract manufacturer for preclinical, clinical, and commercial products. PharmaForm specializes in the area of pharmaceutical dosage form development, controlled-release, and bioavailability-enhancement technologies, such as hot-melt extrusion, spray-drying, fluid bed processing, and liquid-filled capsules.

PharmaForm’s expertise along with its ability to handle certain class potent compounds places PharmaForm as a leader in the field of specialty pharmaceutical dosage formulation and GMP manufacturing. Through its diverse offerings, PharmaForm’s solutions help pharmaceutical and biotechnology clients reach their drug development targets, reduce development costs, and accelerate time-to-market for their products. For more information, visit www.pharmaform.com.

Novel Transmission-Blocking Malaria DNA Vaccine Candidate Uses Vical’s Adjuvant

Vical Incorporated recently announced researchers at Ehime University in Japan and their collaborators have developed a Vaxfectin-formulated DNA vaccine candidate with the potential to prevent transmission of malaria. Results of the initial testing, recently published in the journal Vaccine, demonstrated that the malaria parasite life cycle was interrupted in mosquitoes fed with malaria-infected human red blood cells incubated with serum from vaccinated mice. Vical provided the DNA vaccine plasmid backbone and the adjuvant used in the research.

The malaria parasite advances through several life cycle stages in humans and through additional life cycle stages in mosquitoes. The transmission-blocking DNA vaccine candidate, formulated with Vical’s Vaxfectin adjuvant, expresses the Plasmodium vivax malaria parasite protein Pvs230, which is present in both human and mosquito stages of the parasite’s life cycle. Anti-Pvs230 antibodies generated by vaccinated mice recognized the Pvs230 protein and interrupted the parasite’s development in mosquitoes. The amino acid sequence of Pvs230 protein is also highly conserved among multiple field isolates of P. vivax, increasing the potential for a single vaccine to provide broad protection.

The mouse-generated Pvs230 antibodies, incubated with P. vivax-infected human red blood cells and then fed to mosquitoes, statistically reduced the number of parasites and the infection rate in mosquitoes. A Vaxfectin-formulated malaria vaccine therefore has the potential to interfere with the transmission of P. vivax to humans through mosquitoes. This novel transmission-blocking approach may thereby protect the broader population from widespread malaria outbreaks.

Vical researches and develops biopharmaceutical products based on its patented DNA delivery technologies for the prevention and treatment of serious or life-threatening diseases. Potential applications of the company’s DNA delivery technology include DNA vaccines for infectious diseases or cancer, in which the expressed protein is an immunogen; cancer immunotherapeutics, in which the expressed protein is an immune system stimulant; and cardiovascular therapies, in which the expressed protein is an angiogenic growth factor. The company is developing certain infectious disease vaccines and cancer therapeutics internally.

In addition, the company collaborates with major pharmaceutical companies and biotechnology companies that give it access to complementary technologies or greater resources. These strategic partnerships provide the company with mutually beneficial opportunities to expand its product pipeline and address significant unmet medical needs. Additional information on Vical is available at www.vical.com.

Allena Pharmaceuticals Licenses Worldwide Rights to Hyperoxaluria Portfolio

Allena Pharmaceuticals, Inc. and Althea Technologies recently announced a licensing agreement under which Allena has obtained the exclusive worldwide rights to Althea’s broad hyperoxaluria portfolio, including product patents, regulatory information, and development data related to oxalate degrading enzymes. This license agreement strengthens Allena’s ongoing research and development activities for ALLN-177, its orally delivered enzyme therapy, and helps advance the company’s clinical development program for patients with hyperoxaluria.

Hyperoxaluria is characterized by excessively high levels of oxalate in the urine, due to over-absorption of oxalate from the intestinal tract and abnormalities of oxalate production in the body. Hyperoxaluria can lead to kidney stones or chronic kidney disease and, if left untreated, end-stage renal disease and dialysis.

“Currently, there are no effective pharmacological treatments for hyperoxaluria or the nearly 2.6 million Americans who suffer from kidney stones annually,” said Alexey Margolin, PhD, Co-Founder, President, and CEO of Allena Pharmaceuticals. “Given this unmet need, there is significant demand for a novel, convenient therapy like ALLN-177, which we believe will reduce oxalate levels and prevent further progression to chronic kidney disease. The license of this portfolio enables us to accelerate our current hyperoxaluria program, including starting clinical trials in hyperoxaluria patients early next year.”

Allena’s founders and other members of the Allena team have considerable experience in the development of oral enzyme replacement and non-systemic protein therapeutics, including oxalate-degrading enzymes. The licensed hyperoxaluria portfolio from Althea also includes supportive Phase I clinical data in 58 healthy volunteers (originally generated by Altus Pharmaceuticals’ ALTU-237 program, which was acquired by Althea).

“There is significant value and opportunity in this hyperoxaluria portfolio and a real patient need for an effective treatment. We know the Allena team and believe they are best equipped to translate the potential of this portfolio and the previous clinical findings into a new and novel protein therapy,” said Magda Marquet, PhD, Founder and Co-Chairman of Althea Technologies. “We are looking forward to working closely with the Allena team to support the development of ALLN-177.”

Hyperoxaluria is a condition resulting from high oxalate levels in the urine due to over-absorption of oxalate from the intestinal tract and abnormalities of oxalate production in the body. It can initially cause the development of kidney stones or can lead to chronic kidney disease, end-stage renal disease and dialysis. Hyperoxaluria can be caused by either excessive absorption of dietary oxalate (enteric hyperoxaluria) or increased endogenous production of oxalate (this is known as primary hyperoxaluria, a rare disease that affects approximately 5,000 to 7,000 patients worldwide). Enteric hyperoxaluria is a global problem, with nearly 2.6 million episodes of kidney stones annually in the US. Enteric hyperoxaluria can also occur in people who have intestinal diseases, such as Crohn’s Disease and inflammatory bowel disease, pancreatic insufficiency, or may occur in patients following gastric surgery.

Althea Technologies is a fully integrated, contract development and manufacturing organization located in San Diego, CA, providing clinical and commercial product development services. Althea offers cGMP drug product filling in both vials and syringes, and production of microbial-derived recombinant proteins and plasmid DNA. In conjunction with these manufacturing operations, Althea offers comprehensive development services, including upstream and downstream process development, analytical development, product release, and ICH-compliant stability testing. Althea’s formulation technology platform includes Crystalomics, a technology that offers a formulation solution for large molecule products that must be delivered at high concentrations or as sustained release formulations. For more information, visit www.altheatech.com.

Allena Pharmaceuticals, Inc. is developing and commercializing non-systemic protein therapeutics to treat metabolic and orphan diseases, with a particular focus on nephrologic and urologic conditions. Allena’s lead program, ALLN-177, is expected to enter clinical trials in patients with hyperoxaluria in early 2013. The company’s proven approach enables the design and development of oral protein therapies that remain in the GI tract, where the protein exerts its therapeutic effect by reducing toxic metabolites without being absorbed into the bloodstream. Based in Newton, MA, the company is backed by top-tier venture investors Bessemer Venture Partners, Frazier Healthcare and Third Rock Ventures. For more information, please visit www.allenapharma.com.

Sunesis Pharmaceuticals & Royalty Pharma Announce $25-Million Royalty Agreement

Sunesis Pharmaceuticals and Royalty Pharma recently announced that Royalty Pharma has agreed to pay Sunesis $25 million, under certain circumstances related to the successful development of Sunesis’ lead product candidate vosaroxin, to acquire a royalty on future worldwide net sales of vosaroxin.

Sunesis is evaluating vosaroxin in a pivotal Phase III, randomized, double-blind, placebo-controlled trial, the VALOR trial, in patients with first relapsed or refractory acute myeloid leukemia (AML). The VALOR trial employs an adaptive trial design that permits a one-time increase in sample size at the interim analysis by its Data and Safety Monitoring Board (DSMB). At the interim analysis, expected in the third quarter of 2012, the DSMB will examine pre-specified efficacy and safety data sets and decide whether to stop the study early for efficacy or futility, continue the study as planned, or implement a one-time sample size adjustment of 225 additional evaluable patients.

Under terms of the agreement, Royalty Pharma will invest $25 million immediately following VALOR’s interim analysis if: (a) the study stops early for efficacy, in exchange for a 3.6% participation payment on future net sales; or (b) the one-time sample size increase is being implemented, in exchange for a 6.75% participation payment on future net sales as well as two warrants. In the case when VALOR proceeds to its planned 450 patient enrollment, Royalty Pharma has the option to make a $25 million investment upon the unblinding of the study in exchange for a 3.6% participation payment on future net sales. The warrants issued to Royalty Pharma are exercisable if VALOR’s sample size is increased and are each to purchase 1,000,000 shares of Sunesis common stock at an exercise price of $3.48 and $4.64 per share, respectively. Sunesis currently holds all worldwide commercial rights to its vosaroxin product.

Sunesis is a biopharmaceutical company focused on the development and commercialization of new oncology therapeutics for the treatment of solid and hematologic cancers. Sunesis has built a highly experienced cancer drug development organization committed to advancing its lead product candidate, Vosaroxin, in multiple indications to improve the lives of people with cancer. For additional information on Sunesis, please visit www.sunesis.com.

Royalty Pharma is an industry leader in acquiring royalty interests in marketed and late- stage biopharmaceutical products, with royalty interests in 30 approved products (including Abbott’s Humira, Johnson and Johnson’s Remicade, Merck’s Januvia, Gilead’s Atripla, Truvada, and Emtriva, Pfizer’s Lyrica, Amgen’s Neupogen and Neulasta, and Genentech’s Rituxan) valued at over $6 billion. Royalty Pharma has a 15-year history of providing value to holders of royalty interests.

Optimer Pharmaceuticals & Astellas Pharma Strike Deal Worth Up to $110 Million

Optimer Pharmaceuticals, Inc. and Tokyo-based Astellas Pharma Inc. recently announced the execution of an exclusive collaboration and license agreement to develop and commercialize fidaxomicin tablets in Japan for the treatment of Clostridium difficile Infection (CDI).

In return for the exclusive license to fidaxomicin in Japan, Optimer is entitled to receive a one-time, up-front cash payment of $20 million from Astellas. Optimer is also eligible to receive additional cash payments of up to $70 million upon the achievement of certain regulatory and commercial milestones. Optimer is further entitled to receive payments from Astellas that provide a return resulting in a double-digit percent of Astellas net sales in the territory. Astellas is responsible for all future costs associated with the development and commercialization of fidaxomicin in Japan.

“We are pleased to have entered a collaboration with Optimer to introduce fidaxomicin to Japan for the treatment of Clostridium difficile infections,” said Mr. Yoshihiko Hatanaka, President and CEO of Astellas Pharma Inc. “We believe that fidaxomicin’s highly differentiated clinical profile, which has been confirmed by clinical studies conducted in the US and EU, has the potential to provide a new and unique treatment option for patients in Japan. Astellas is committed to a focus on infectious diseases, and this collaboration is an important milestone for our franchise.”

“Astellas is a leader in the anti-infective market in Japan. Leveraging their expertise efficiently helps realize the potential of fidaxomicin to meet the serious unmet needs of CDI patients in Japan, helping to address the risk of recurrence inherent in the disease, while optimizing the market opportunity for our shareholders,” added Optimer’s President and CEO, Pedro Lichtinger. “Japan is a key territory we have prioritized for geographic market expansion and complements nearer term market opportunities in the US, Canada, and the European Union as we continue to pursue additional international market opportunities.”

In 2011, Optimer entered into a commercial partnership with Astellas Pharma Europe Ltd. to develop and market fidaxomicin under the trade name DIFICLIR in Europe and certain other countries in the Middle East, Africa, and the Commonwealth of Independent States. The European Medicines Agency granted marketing authorization to DIFICLIR in December 2011, and Astellas Pharma Europe Ltd. expects to begin selling DIFICLIR in the first of its territories starting in 2012. Fidaxomicin is approved by the US FDA for the treatment of Clostridium difficile-associated diarrhea (CDAD) in adults 18 years of age or older in the US under the trade name DIFICID.

Astellas Pharma Inc., located in Tokyo, Japan, is a pharmaceutical company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products. For more information on Astellas Pharma Inc., please visit www.astellas.com/en.

Optimer Pharmaceuticals, Inc. is a global biopharmaceutical company focused on discovering, developing, and commercializing innovative hospital specialty products that have a positive impact on society. Additional information can be found at www.optimerpharma.com.