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Supplemental Application Filed for Somatuline® in Japan for Additional Indication of Neuroendocrine Tumors

On July 28, 2016 Ipsen (Euronext: IPN; ADR: IPSEY) and Teijin Pharma Limited, the core company of the Teijin Group’s healthcare business, reported that Teijin Pharma has filed a supplemental application with Pharmaceuticals and Medical Devices Agency to use Ipsen’s subcutaneous drug Somatuline (lanreotide) for the treatment of neuroendocrine tumors (NETs) (Press release, Ipsen, JUL 28, 2016, View Source [SID:1234514143]). The drug is currently available in Japan as a treatment for acromegaly and pituitary gigantism.

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Ipsen and Teijin Pharma are confident that Somatuline will provide a beneficial treatment option for patients in Japan with neuroendocrine tumors.

The application is supported by CLARINET, an investigational, Phase III randomized, double-blind, placebo-controlled study which assessed the antiproliferative effects of Somatuline 120mg in patients with metastatic gastroenteropancreatic neuroendocrine tumors (GEP-NETs) which was conducted across 14 countries in Europe and the USA. Treatment with Somatuline 120 mg achieved statistically significant prolongation of progression free survival over placebo in patients with GEP-NETs. The safety profile observed in the study is consistent with the known safety profile of Somatuline. CLARINET has been the basis of the approval of an indication for the treatment of gastroenteropancreatic NETs (GEP-NETs) in more than 40 countries, including the US and EU countries. The results complement the effects of Somatuline on existing indications relating to reduction of symptoms associated with NETs. The supplemental application submitted to the Pharmaceuticals and Medical Devices Agency is also based on results of the J-001 Phase II trial for NETs that Teijin Pharma has conducted in Japan.

About Neuroendocrine Tumors

Neuroendocrine cells, which release hormones, are widely distributed throughout the human body. Neuroendocrine tumors (NETs) are rare, slow growing cancers that can arise anywhere in the body, but most commonly occur in the gastrointestinal tract, lung or pancreas. NETs are often diagnosed at a late stage because the symptoms, if any, lack specificity. In some patients, hormones secreted from NETs can cause problems, such as abdominal pain, flushing, diarrhea or pulmonary symptoms, such as wheezing. NETs can spread to other organs of the body and can cause severe co-morbidities and also significantly decrease life expectancy.
The first choice of NET treatment is removal by surgery, but if this is not possible, or if a tumor returns following surgery, another option is treatment with drugs to inhibit tumor growth and/or reduce the associated symptoms. Currently there are limitations in the types of NET patients who can be treated, depending on the location and the grading of the primary tumor, so there is an unmet medical need for new medicines that can be prescribed for a wider variety of NET patients.

Jazz Pharmaceuticals and Pfenex Enter into a Worldwide License and Option Agreement Related to Product Candidates in Early Development for Hematological Malignancies

On July 28, 2016 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Pfenex Inc. (NYSE MKT: PFNX) reported an agreement under which Pfenex granted Jazz Pharmaceuticals worldwide rights to develop and commercialize multiple early stage hematology product candidates (Press release, Jazz Pharmaceuticals, JUL 28, 2016, View Source [SID:1234514100]). The agreement also includes an option for Jazz Pharmaceuticals to negotiate a license for a recombinant pegaspargase product candidate with Pfenex. This early development stage collaboration demonstrates Jazz Pharmaceuticals’ focus on identifying innovative technologies that may lead to the development of important therapeutic options for patients with hematological malignancies.

Under the agreement, Pfenex will receive upfront and option payments totaling $15 million and may be eligible to receive additional payments of up to $166 million based on the achievement of certain development-, regulatory-, and sales-related milestones, including up to $41 million for certain non-sales-related milestones. Pfenex may also be eligible to receive tiered royalties on worldwide sales of any products resulting from the collaboration. Both parties will be contributing to development efforts.

"The collaboration with Pfenex, including access to its unique protein expression technology, demonstrates our emphasis on diversifying and strengthening our portfolio to provide improved therapeutic options for patients." said Karen Smith M.D., Ph.D, global head of research and development and chief medical officer at Jazz Pharmaceuticals plc. "We look forward to working with Pfenex on the development of multiple product candidates that have the potential to broaden our hematology/oncology portfolio."

"Our collaboration with Jazz further validates Pfenex’s product development capability enabled by our protein expression platform technology. We look forward to working with Jazz on these assets in support of further advancement in clinical development," said Bertrand C. Liang, chief executive officer of Pfenex.

Pfizer and Western Oncolytics Announce Immuno-Oncology Research Collaboration to Investigate Novel Oncolytic Virus Technology

On July 28, 2016 Pfizer Inc. (NYSE:PFE) and Western Oncolytics reported that they have entered into a development collaboration, license and option agreement to advance Western Oncolytics’ novel oncolytic vaccinia virus, WO-12 (Press release, Pfizer, JUL 28, 2016, View Source [SID:1234514099]).

Oncolytic viruses are viruses engineered to kill cancer cells while sparing healthy cells, which subsequently elicits anti-cancer immune responses. This collaboration in oncolytic virus development adds another novel technology platform to Pfizer’s cancer vaccine efforts and provides an additional tool to bolster its immuno-oncology portfolio.

Under the terms of the agreement, Pfizer and Western Oncolytics will collaborate on preclinical and clinical development of WO-12 through Phase I trials. Following completion of Phase I trials, Pfizer has an exclusive option to acquire WO-12. Financial terms of the agreement were not disclosed.

"Our goal is to combine WO-12 with our portfolio of promising investigational immunotherapies to explore how these novel combinations could help further enhance the body’s immune response in fighting cancer cells," said James Merson, Ph.D., Chief Scientific Officer, Vaccine Immunotherapeutics at Pfizer. "We believe that the real advances in immuno-oncology will come from novel combinations, and cancer fighting viruses and vaccines could play a key role in helping transform cancer treatment and potentially enable us to treat more patients."

WO-12 is a preclinical investigational oncolytic virus. As an in vivo vaccine, it has the potential to be delivered directly to the tumor (intratumoral) or intravenously. More specifically, it is a virus engineered to replicate primarily in cancer cells while delivering several therapeutic genes that modulate the immune system to enhance efficacy against a range of cancers. By replicating inside cancer cells, it is designed to both kill the cancer cell and releases tumor antigens that direct the immune system to recognize the antigens and kill additional cancer cells. WO-12 has potential applications across multiple tumor types.

"We believe this collaboration will create a unique opportunity to accelerate and expand the clinical testing of WO-12 as well as to examine potential combinations with other immunotherapies in the Pfizer portfolio," said Steve Thorne, PhD, Chief Scientific Officer of Western Oncolytics and inventor of WO-12.

Kurt Rote, CEO of Western Oncolytics, added, "We have been very impressed with the expertise and commitment to success from everyone at Pfizer, and are excited to be partnering this promising therapy with a shared vision of improving the way patients are treated."

Adaptimmune Receives Access to Priority Medicines (PRIME) Regulatory Support for its SPEAR® T-cell Therapy Targeting NY-ESO for Treatment of Soft Tissue Sarcoma

On July 28, 2016 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported that the European Medicines Agency (EMA) has granted access to its newly-established Priority Medicines (PRIME) regulatory initiative for the company’s SPEAR T-cell therapy targeting NY-ESO for the treatment of HLA-A0201, HLA-A0205, or HLA-A0206 allele positive patients with inoperable or metastatic synovial sarcoma who have received prior chemotherapy and whose tumor expresses the NY-ESO-1 tumor antigen (Press release, Adaptimmune, JUL 28, 2016, View Source [SID:1234514096]).

The PRIME initiative provides support to optimize regulatory applications and accelerate the review of medicines that address a high unmet need.

"Access to the PRIME initiative represents an important regulatory opportunity for us. It can provide early engagement on the development program with potential for accelerated assessment of data to companies like Adaptimmune who are developing new treatment modalities for patients in Europe with few or no treatment options," said Rafael Amado, Adaptimmune’s Chief Medical Officer. "Our NY-ESO SPEAR T-cell therapy may help to address the significant unmet medical need of metastatic or unresectable synovial sarcoma. We look forward to working closely with the EMA throughout its clinical evaluation."

Adaptimmune recently announced that the European Commission had designated its NY-ESO SPEAR T-cell therapy as an orphan medicinal product for the treatment of soft tissue sarcoma. The company has already received orphan drug designation and Breakthrough Therapy designation for its NY-ESO SPEAR T-cell therapy from the U.S. Food and Drug Administration.

The PRIME initiative focuses on medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. The criteria for the PRIME initiative require a medicine to show its potential to benefit patients with unmet medical needs based on early clinical data. The initiative offers early and proactive support to medicine developers to optimize the generation of robust data on a medicine’s benefits and risks and enable accelerated assessment of medicines applications, and provides appointment of a rapporteur, early dialogue on the overall development plan and regulatory strategy, scientific advice at key development milestones, a dedicated point of contact, and the potential to qualify products for accelerated assessment at the time of an application for marketing authorization.

Adaptimmune’s SPEAR T-cell candidates are novel cancer immunotherapies that have been engineered to target and destroy cancer cells by strengthening a patient’s natural T-cell response. T-cells are a type of white blood cell that play a central role in a person’s immune response. Adaptimmune’s goal is to harness the power of the T-cell and, through its multiple therapeutic candidate, significantly impact cancer treatment and clinical outcomes of patients with solid and hematologic cancers.

AbbVie Announces Initiation of Phase 3 Study of Venetoclax in Patients with Relapsed or Refractory Multiple Myeloma

On July 28, 2016 AbbVie (NYSE: ABBV), a global biopharmaceutical company, reported the initiation of a Phase 3 clinical trial to study the safety and efficacy of venetoclax in combination with bortezomib and dexamethasone in patients with relapsed or refractory multiple myeloma who are considered sensitive or naïve to proteasome inhibitors and have received one to three prior lines of therapy (Press release, AbbVie, JUL 28, 2016, View Source [SID:1234514095]). The combination of venetoclax, bortezomib and dexamethasone will be compared to treatment with bortezomib, dexamethasone and placebo.1 Bortezomib, a proteasome inhibitor, and dexamethasone, a corticosteroid, are both common therapies used to treat symptomatic multiple myeloma.3

Venetoclax is being developed by AbbVie and Genentech, a member of the Roche Group. It is jointly commercialized by the companies in the U.S. and by AbbVie outside of the U.S.

"We have a comprehensive development strategy for venetoclax, with several ongoing clinical trials across a range of hematologic malignancies and multiple lines of therapy as a single agent and in combination with other medicines," said Michael Severino, M.D., executive vice president, Research and Development and chief scientific officer, AbbVie. "This Phase 3 trial represents our commitment to identifying the full potential of this therapy through our clinical development program and is an important step in our goal to provide a possible treatment for multiple myeloma patients."

The randomized, double-blind, placebo-controlled, Phase 3 clinical trial aims to recruit approximately 240 patients. The primary efficacy endpoint of the trial is progression-free survival (PFS). Secondary pre-specified outcome measures include overall survival (OS), objective response rate (ORR) and duration of response (DoR), as well as other efficacy and safety outcome measures.1 More information on the Phase 3 trial is available at www.clinicaltrials.gov (NCT02755597).

Multiple myeloma is the second most common blood cancer and begins in plasma cells in the bone marrow. When plasma cells in the marrow become cancerous, they can grow uncontrollably and produce abnormal proteins (m proteins) which can cause tumors, typically developing in the bone. When a patient has multiple plasma cell tumors, they have multiple myeloma.2

About VENCLEXTA (venetoclax) in the U.S.
Venetoclax is an oral B-cell lymphoma-2 (BCL-2) inhibitor currently approved as VENCLEXTA (venetoclax) tablets in the U.S. and indicated for the treatment of patients with chronic lymphocytic leukemia (CLL) with 17p deletion, as detected by an FDA-approved test, who have received at least one prior therapy.4 The FDA approved this indication under accelerated approval based on overall response rate, and continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial.4

The BCL-2 protein prevents apoptosis (programmed cell death) of some cells, including lymphocytes, and can be overexpressed in CLL cells. VENCLEXTA was designed to selectively inhibit the BCL-2 protein.4

VENCLEXTA is being developed by AbbVie and Genentech, a member of the Roche Group. Together, the companies are committed to BCL-2 research with VENCLEXTA, which is currently being evaluated in Phase 3 clinical trials for the treatment of relapsed/refractory CLL, along with early phase studies in several cancers. VENCLEXTA is under evaluation by Health Authorities in multiple countries, and not approved for markets outside of the U.S. AbbVie is currently working with regulatory agencies around the world to bring this medicine to eligible patients in need.

The full prescribing information for VENCLEXTA can be found here.

Patient Assistance Program
For those who qualify, AbbVie and Genentech offer patient assistance programs for people taking VENCLEXTA in the U.S.

U.S. Important Safety Information
What is the most important information I should know about VENCLEXTA?

VENCLEXTA can cause serious side effects, including:
Tumor lysis syndrome (TLS). TLS is caused by the fast breakdown of cancer cells. TLS can cause kidney failure, the need for dialysis treatment, and may lead to death. Your doctor will do tests for TLS. It is important to keep your appointments for blood tests. You will receive other medicines before starting and during treatment with VENCLEXTA to help reduce your risk of TLS. You may also need to receive intravenous (IV) fluids into your vein. Tell your doctor right away if you have any symptoms of TLS during treatment with VENCLEXTA, including fever, chills, nausea, vomiting, confusion, shortness of breath, seizures, irregular heartbeat, dark or cloudy urine, unusual tiredness, or muscle or joint pain.

Drink plenty of water when taking VENCLEXTA to help reduce your risk of getting TLS. Drink 6 to 8 glasses (about 56 ounces total) of water each day, starting 2 days before your first dose, on the day of your first dose of VENCLEXTA, and each time your dose is increased.

Who should not take VENCLEXTA?
Certain medicines must not be taken when you first start taking VENCLEXTA and while your dose is being slowly increased.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. VENCLEXTA and other medicines may affect each other, causing serious side effects
Do not start new medicines during treatment with VENCLEXTA without first talking with your doctor
What should I tell my doctor before taking VENCLEXTA?
Before taking VENCLEXTA, tell your doctor about all of your medical conditions, including if you:

Have kidney or liver problems
Have problems with your body salts or electrolytes, such as potassium, phosphorus, or calcium
Have a history of high uric acid levels in your blood or gout
Are scheduled to receive a vaccine. You should not receive a "live vaccine" before, during, or after treatment with VENCLEXTA until your doctor tells you it is okay
Are pregnant or plan to become pregnant. VENCLEXTA may harm your unborn baby. If you are able to become pregnant, your doctor should do a pregnancy test before you start treatment with VENCLEXTA, and you should use effective birth control during treatment and for 30 days after the last dose of VENCLEXTA
Are breastfeeding or plan to breastfeed. It is not known if VENCLEXTA passes into your breast milk. Do not breastfeed during treatment with VENCLEXTA
What should I avoid while taking VENCLEXTA?
You should not drink grapefruit juice or eat grapefruit, Seville oranges (often used in marmalades), or starfruit while you are taking VENCLEXTA. These products may increase the amount of VENCLEXTA in your blood.

What are the possible side effects of VENCLEXTA?
VENCLEXTA can cause serious side effects, including:

Low white blood cell count (neutropenia). Low white blood cell counts are common with VENCLEXTA, but can also be severe. Your doctor will do blood tests to check your blood counts during treatment with VENCLEXTA. Tell your doctor right away if you have a fever or any signs of an infection
The most common side effects of VENCLEXTA include diarrhea, nausea, low red blood cell count, upper respiratory tract infection, low platelet count, and feeling tired. VENCLEXTA may cause fertility problems in males. This may affect your ability to father a child. Talk to your doctor if you have concerns about fertility. These are not all the possible side effects of VENCLEXTA. Tell your doctor if you have any side effect that bothers you or that does not go away.

Please see full Prescribing Information, including Medication Guide.