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European Medicines Agency Grants Accelerated Assessment of Ixazomib for Patients with Relapsed/Refractory Multiple Myeloma

On July 27, 2015 Takeda Pharmaceutical Company Limited (TSE: 4502) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted an accelerated assessment to ixazomib, an investigational oral proteasome inhibitor for the treatment of patients with relapsed and/or refractory multiple myeloma (Press release, Takeda, JUL 27, 2015, View Source [SID:1234506702]).

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The EMA awards an accelerated assessment to those medicines deemed to be of major public health interest and, in particular, therapeutic innovation.

Takeda expects to submit a marketing authorisation application for ixazomib in the European Union (EU) in the coming weeks. The submission is based on the results of the first pre-specified interim analysis of the pivotal Phase 3 trial TOURMALINE-MM1. This study is an international, randomized, double-blind, placebo controlled clinical trial of 722 patients designed to evaluate the superiority of ixazomib plus lenalidomide and dexamethasone over placebo plus lenalidomide and dexamethasone in adult patients with relapsed and/or refractory multiple myeloma. Patients continue to be treated to progression in this trial and evaluated for long-term outcomes.

"We are pleased that the CHMP has granted accelerated assessment for ixazomib, and believe that this designation underscores the urgent need for therapeutic innovation in multiple myeloma treatments," said Andrew Plump, M.D., Ph.D., Takeda’s Chief Medical and Scientific Officer. "We look forward to sharing the TOURMALINE-MM1 study data from the first pre-specified interim analysis at an upcoming medical meeting, and greatly appreciate the ongoing dedication and commitment from the patients and their families who have been participating in the ixazomib clinical development program."

"Continuous therapy is emerging as a standard of care in multiple myeloma because it has demonstrated improved long-term outcomes," said Philippe Moreau, MD, University Hospital of Nantes in France. "If ixazomib is approved, for the first time physicians will have the option of an all-oral proteasome inhibitor-based regimen for the treatment of multiple myeloma, which could be a real advantage in delivering sustained therapy."

About Multiple Myeloma
Multiple myeloma is a cancer of the plasma cells, which are found in the bone marrow. In multiple myeloma, a group of plasma cells, or myeloma cells, becomes cancerous and multiplies, increasing the number of plasma cells to a higher than normal level. Because plasma cells circulate widely in the body, they have the potential to affect many bones in the body, possibly resulting in compression fractures, lytic bone lesions and related pain. Multiple myeloma can cause a number of serious health problems affecting the bones, immune system, kidneys and red blood cell count, with some of the more common symptoms including bone pain and fatigue, a symptom of anemia. Multiple myeloma is a rare form of cancer with approximately 39,000 new cases in the EU and 114,000 new cases globally per year.

About Ixazomib
Ixazomib (MLN9708) is an investigational oral proteasome inhibitor which is being studied in multiple myeloma, systemic light-chain (AL) amyloidosis, and other malignancies. Ixazomib was granted orphan drug designation in multiple myeloma in both the United States and Europe in 2011 and for AL amyloidosis in both the U.S. and Europe in 2012. Ixazomib received Breakthrough Therapy status by the U.S. Food and Drug Administration (FDA) for relapsed or refractory AL amyloidosis in 2014. It is also the first oral proteasome inhibitor to enter Phase 3 clinical trials.

Ixazomib’s clinical development program further reinforces Takeda’s ongoing commitment to developing innovative therapies for people living with multiple myeloma worldwide and the healthcare professionals who treat them. Five global Phase 3 trials are ongoing:

TOURMALINE-MM1, investigating ixazomib vs. placebo, in combination with lenalidomide and dexamethasone in relapsed and/or refractory multiple myeloma

TOURMALINE-MM2, investigating ixazomib vs. placebo, in combination with lenalidomide and dexamethasone in patients with newly diagnosed multiple myeloma

TOURMALINE-MM3, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma following induction therapy and autologous stem cell transplant (ASCT)

TOURMALINE-MM4, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma who have not undergone ASCT

TOURMALINE-AL1, investigating ixazomib plus dexamethasone vs. physician choice of selected regimens in patients with relapsed or refractory AL amyloidosis

For additional information on the ongoing Phase 3 studies please visit www.clinicaltrials.gov.

European Medicines Agency Validates and Grants Accelerated Assessment of Marketing Authorization Application for Empliciti (elotuzumab) For the Treatment of Multiple Myeloma in Patients Who Have Received One or More Prior Therapies

On July 27, 2015 Bristol-Myers Squibb Company (NYSE:BMY) and AbbVie (NYSE:ABBV) reported the European Medicines Agency (EMA) validated for review the Marketing Authorization Application (MAA) for Empliciti, an investigational Signaling Lymphocyte Activation Molecule (SLAMF7)-directed immunostimulatory antibody, for the treatment of multiple myeloma as combination therapy in adult patients who have received one or more prior therapies (Press release, Bristol-Myers Squibb, JUL 27, 2015, View Source [SID:1234506698]).

The application was granted accelerated assessment by the EMA’s Committee for Medicinal Products for Human Use (CHMP).

Bristol-Myers Squibb and AbbVie are co-developing Empliciti, with Bristol-Myers Squibb solely responsible for commercial activities. Bristol-Myers Squibb has proposed the name Empliciti which, if approved by health authorities, will serve as the trade name for elotuzumab.

"The MAA validation marks a significant milestone in Bristol-Myers Squibb’s mission to advance the science and impact the treatment of hematologic malignancies through our Immuno-Oncology leadership," said Michael Giordano, MD, senior vice president, head of Oncology Development, Bristol-Myers Squibb. "We believe the CHMP’s acceptance for an accelerated assessment reflects the need for a new treatment option for multiple myeloma, a largely incurable disease. We are proud to be one step closer to bringing Empliciti to patients with relapsed or refractory multiple myeloma in Europe."

The MAA is primarily supported by data from two randomized clinical trials, each combining Empliciti with a different standard of care regimen for multiple myeloma. ELOQUENT-2, a Phase 3, randomized, open-label study, evaluated Empliciti in combination with lenalidomide and dexamethasone versus lenalidomide and dexamethasone alone. The results of this trial were published in The New England Journal of Medicine on June 2. Additionally, a Phase 2, randomized, open-label study (Study CA004-009) evaluated Empliciti with bortezomib and dexamethasone versus bortezomib and dexamethasone alone. These Phase 2 results were presented in an oral session (Abstract #S103) at the 20th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).

Empliciti previously obtained orphan drug designation in the European Union (EU). An orphan medicinal product must be intended for the treatment, prevention or diagnosis of a disease that is life threatening and chronically debilitating; the prevalence in the EU must not be more than five in 10,000. The medicine must be of significant benefit to those affected by the condition. If maintained, orphan drug designation allows sponsors to access a number of incentives including protocol assistance and receive market exclusivity for a ten-year period following approval.

About Empliciti

Empliciti is an investigational immunostimulatory antibody targeted against SLAMF7, a cell-surface glycoprotein that is highly and uniformly expressed on myeloma cells and Natural Killer (NK) cells, but is not detected on normal solid tissues or on hematopoietic stem cells. The safety and efficacy of Empliciti have not been evaluated by the FDA or any other health authority.

About Multiple Myeloma

Multiple myeloma is a hematologic, or blood, cancer that develops in the bone marrow. It occurs when a plasma cell, a type of cell in the soft center of bone marrow, becomes cancerous and multiplies uncontrollably. Despite advances in multiple myeloma treatment over the last decade, only 45% of patients have a ten-year survival rate. A common characteristic for many patients is that they experience a cycle of remission and relapse, in which they stop treatment for a short time, but eventually return to a treatment shortly after. Following relapse, less than 20% of patients are alive after five years. It is estimated that annually more than 114,200 new cases of multiple myeloma are diagnosed globally and annually more than 79,000 people die from the disease globally.

AstraZeneca further sharpens focus through agreement with Genzyme for rare disease medicine Caprelsa

On July 27, 2015 AstraZeneca reported that it has entered into a definitive agreement with Genzyme to divest Caprelsa (vandetanib), a rare disease medicine (Press release, AstraZeneca, JUL 27, 2015, View Source;astrazeneca-agreement-with-genzyme-for-caprelsa [SID:1234506697]).

Caprelsa was granted Orphan Drug Designation by the US FDA in 2005 and is currently available in 28 countries for the treatment of aggressive and symptomatic medullary thyroid carcinoma, with global product sales of $48 million in 2014.

Under the terms of the agreement, Genzyme will pay AstraZeneca up to $300 million, including an upfront payment of $165 million to acquire the global rights to sell and develop Caprelsa, and further development and sales milestone payments of up to $135 million. The transaction does not include the transfer of any AstraZeneca employees or facilities. As an asset divestment, upfront receipt and any subsequent payments will be reported in Other Operating Income in the Company’s financial statements.

Luke Miels, Executive Vice President, Global Product & Portfolio Strategy and Corporate Affairs, AstraZeneca, said: "Caprelsa is a rare disease therapy and the divestment to Genzyme, an expert leader in endocrinology, demonstrates our commitment to ensure patients continue to have access to this medicine while we sharpen our focus on key disease areas."

Genzyme’s President and CEO, David Meeker, MD, said: "The addition of Caprelsa represents a strong strategic fit for our rare endocrinology portfolio and underscores Genzyme’s commitment to addressing unmet needs in the thyroid community. We look forward to bringing our rare disease expertise to appropriate patients with advanced stage thyroid carcinoma."

The divestment transaction is subject to closing conditions, including the receipt of antitrust clearance from the US Federal Trade Commission. The transaction is expected to complete in the second half of 2015 and does not impact AstraZeneca’s financial guidance for 2015.

MorphoSys AG Reports Results for the First Six Months of 2015

On July 27, 2015 MorphoSys AG (FSE: MOR; Prime Standard Segment; TecDAX, OTC: MPSYY) reported its financial results for the six months ending 30 June 2015. Group revenues were EUR 82.6 million (H1 2014: EUR 30.5 million) (Press release, MorphoSys, JUL 26, 2015, View Source [SID:1234506699]). The increase is attributable to revenue booked in connection with the ending of the collaboration with Celgene on MorphoSys’s proprietary drug candidate MOR202. This comprised the full realization of deferred revenues from an up-front payment received from Celgene in 2013 together with a one-time termination payment. Earnings before interest and taxes (EBIT) amounted to EUR 46.1 million (H1 2014: EUR 0.4 million). On 30 June 2015, MorphoSys held cash and cash equivalents, marketable securities, and financial assets classified as loans and receivables of EUR 324.9 million in comparison to EUR 352.8 million on 31 December 2014.

Highlights of the Second Quarter 2015

At the end of the second quarter of 2015, MorphoSys’s product pipeline comprised a total of 102 therapeutic antibodies, including 24 clinical programs. Three partnered programs are currently in phase 3 trials.

MorphoSys presented its updated phase 2 clinical results for MOR208 in non-Hodgkin’s lymphoma (NHL) at the 2015 annual conference of the American Society of Oncology (ASCO) (Free ASCO Whitepaper). The clinical data showed that MOR208 is well tolerated with a low level of infusion reactions and demonstrated encouraging single-agent activity.

MorphoSys also presented preliminary clinical data on the safety, pharmacokinetics and efficacy of MOR202 in multiple myeloma at the 2015 ASCO (Free ASCO Whitepaper) conference. MOR202 proved to be safe and well tolerated and showed early signs of clinical activity and cases of long-lasting tumor control.

In May 2015, MorphoSys acquired all outstanding shares in the Dutch biopharmaceutical company Lanthio Pharma. The acquisition added new development candidates to MorphoSys’s proprietary portfolio, including a preclinical program for fibrotic diseases (MOR107).

In April 2015, MorphoSys announced that it had reached a clinical milestone with the initiation of a phase 2 study of the antibody guselkumab in psoriatic arthritis by its partner Janssen Biotech. The milestone payment was recognized in the first quarter of 2015.
Shortly after the end of the second quarter, MorphoSys announced that it had reached a clinical milestone associated with the IND filing of an antibody being developed to treat blood disorders by its partner Novartis, which was recognized in the second quarter of 2015.

At the Annual General Meeting on 8 May 2015, Ms. Wendy Johnson, Mr. Klaus Kühn and Dr. Frank Morich were newly elected to the Supervisory Board. Dr. Gerald Möller, Dr. Marc Cluzel and Ms. Karin Eastham were all re-elected to the Supervisory Board. Additionally, all resolutions proposed by the management were adopted.

MorphoSys repurchased 88,670 of its own shares in the second quarter of 2015. The shares will be used primarily for long-term incentive programs for the Management Board and Senior Management Group.

"The second quarter saw excellent progress with regard to our proprietary pipeline and the further expansion of our drug development capabilities in the biologics arena beyond antibodies. Clearly the highlight of the quarter from a pipeline perspective was the presentation of encouraging clinical data for our most advanced proprietary oncology programs MOR208 and MOR202," stated Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG.

"We are very pleased with the first half of 2015 and are on track to meet our goals for the full year. The addition of Lanthio’s portfolio of therapeutic peptides to our pipeline combined with new therapeutic antibody programs initiated by our partners have increased the number of therapeutic candidates in different stages of development for the first time beyond the 100 program mark," commented Jens Holstein, Chief Financial Officer of MorphoSys AG.

Financial Review for the First Half of 2015 (IFRS)

Group revenues for the first six months of 2015 amounted to EUR 82.6 million (H1 2014: EUR 30.5 million). Reasons for the increase were one-time effects in H1 2015 in connection with the full realization of deferred revenues from an up-front payment received from Celgene in 2013 together with a one-time termination fee. The Proprietary Development segment recorded revenues of EUR 59.6 million (H1 2014: EUR 7.7 million), all of which were recorded in connection with the co-development agreement with Celgene. Revenues in the Partnered Discovery segment comprised EUR 21.0 million in funded research and licensing fees (H1 2014: EUR 21.4 million) and EUR 2.0 million in success-based payments (H1 2014: EUR 1.4 million).

Total operating expenses for the first six months of 2015 amounted to EUR 40.9 million (H1 2014: EUR 30.1 million). Total research and development expenses were EUR 33.9 million (H1 2014: EUR 23.4 million). R&D expenses mainly consisted of costs for external lab services and personnel costs. Expenses for proprietary product and technology development amounted to EUR 25.3 million (H1 2014: EUR 14.9 million). General and administrative expenses increased to EUR 7.0 million (H1 2014: EUR 6.7 million) driven by higher expenses for personnel.

Earnings before interest and taxes (EBIT) amounted to EUR 46.1 million (H1 2014: EUR 0.4 million). The Proprietary Development segment reported a segment EBIT of EUR 40.2 million (H1 2014: EUR -5.9 million), while Partnered Discovery showed a segment EBIT of EUR 12.5 million (H1 2014: EUR 12.5 million).

For the first half of 2015, MorphoSys realized a net profit of EUR 36.5 million compared to EUR 0.6 million in the same period of the previous year. The resulting diluted earnings per share for the six months ending 30 June 2015 amounted to EUR 1.39 (H1 2014: EUR 0.02).

On 30 June 2015, the Company held liquid funds and marketable securities, as well as other financial assets (reported in the balance sheet under cash and cash equivalents, available for sale financial assets, bonds available for sale and financial assets classified as loans and receivables), in the amount of EUR 324.9 million, compared to EUR 352.8 million on 31 December 2014. The net cash inflow from operations in H1 2015 was EUR 1.1 million (H1 2014: net cash outflow of EUR 9.9 million). The number of shares issued at 30 June 2015 was 26,469,834, compared to 26,456,834 on 31 December 2014.

Second Quarter of 2015 (IFRS)

In the second quarter of 2015, the Company generated revenues in the amount of EUR 12.2 million, compared to EUR 14.7 million in the same quarter of 2014. Total operating expenses amounted to EUR 23.2 million in Q2 2015, compared to EUR 15.6 million in the same quarter of 2014. EBIT amounted to EUR -6.7 million (Q2 2014: EUR -1.0 million). Net loss for the second quarter 2015 was EUR 4.3 million, compared to a net loss of EUR 0.5 million in the second quarter of 2014.

Outlook for 2015

MorphoSys re-confirmed its guidance for 2015. MorphoSys anticipates total Group revenues of EUR 101 million to EUR 106 million and anticipates a positive EBIT in the range of EUR 9 to EUR 16 million in 2015. Expenses for proprietary product and technology development are expected to amount to EUR 56 million to EUR 63 million.

FDA approves new treatment for most common form of advanced skin cancer

On July 24, 2015 The U.S. Food and Drug Administration reported it approved Odomzo (sonidegib) to treat patients with locally advanced basal cell carcinoma that has recurred following surgery or radiation therapy, or who are not candidates for surgery or radiation therapy (Press release, , JUL 24, 2015, View Source [SID:1234506626]).

Skin cancer is the most common cancer and basal cell carcinoma accounts for approximately 80 percent of non-melanoma skin cancers. Basal cell carcinoma starts in the top layer of the skin (called the epidermis) and usually develops in areas that have been regularly exposed to the sun and other forms of ultraviolet radiation. According to the National Cancer Institute, the number of new cases of non-melanoma skin cancer appears to be increasing every year. Locally advanced basal cell skin cancer refers to basal cancers that have not spread to other parts of the body, but cannot be curatively treated with local treatments, specifically surgery and radiation.

Odomzo is a pill taken once a day. It works by inhibiting a molecular pathway, called the Hedgehog pathway, which is active in basal cell cancers. By suppressing this pathway, Odomzo may stop or reduce the growth of cancerous lesions.

"Our increasing understanding of molecular pathways involved in cancer has led to approvals of many oncology drugs in difficult-to-treat diseases for which few therapeutic options previously existed," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "Thanks to a better understanding of the Hedgehog pathway, the FDA has now approved two drugs for the treatment of basal cell carcinoma just in the last three years." In 2012, Erivedge (vismodegib) was the first drug approved to treat locally advanced and metastatic basal cell carcinoma.

Odomzo carries a Boxed Warning alerting healthcare professionals that Odomzo may cause death or severe birth defects in a developing fetus when administered to a pregnant woman. Pregnancy status should be verified prior to the start of Odomzo treatment, and both male and female patients should be warned about these risks and advised to use effective contraception.

The efficacy of Odomzo was established in a multi-center, double-blind clinical trial, in which 66 patients with locally advanced basal cell carcinoma were randomly assigned to receive Odomzo 200 mg daily and 128 patients were assigned to receive Odomzo 800 mg daily. The study’s primary endpoint was objective response rate, which is the percentage of patients who experienced partial shrinkage or complete disappearance of their tumor(s). Results showed that 58 percent of patients treated with Odomzo 200 mg had their tumors shrink or disappear. This effect lasted at least 1.9 to 18.6 months, and approximately half of the responding patients’ tumor shrinkage lasted six months or longer. Response rates were similar in patients who received Odomzo 800 mg daily, however side effects were more common at this dose.

At a dose of 200 mg daily, the most common side effects of Odomzo were muscle spasms, alopecia (hair loss), dysgeusia (distortion in the sense of taste), fatigue, nausea, musculoskeletal pain, diarrhea, decreased weight, decreased appetite, myalgia (muscle pain), abdominal pain, headache, pain, vomiting and pruritus (itching). Odomzo also has the potential to cause serious musculoskeletal-related side effects, including increased serum creatine kinase levels [with rare reports of muscle tissue breakdown (rhabdomyolysis)], muscle spasms, and myalgia.

Odomzo is marketed by East Hanover, New Jersey-based Novartis Pharmaceuticals Corporation. Erivedge is marketed by Genentech in San Francisco, California.

The FDA, an agency within the U.S. Department of Health and Human Services, promotes and protects the public health by, among other things, assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.