On December 22, 2014 Pharmacyclics reported that the U.S Food and Drug Administration (FDA) has provided a Prescription Drug User Fee Act (PDUFA) target date of April 17, 2015 by which time the FDA is planning to finalize its review of a supplemental New Drug Application (sNDA) for IMBRUVICA (ibrutinib) as a treatment for patients with Waldenstrom’s macroglobulinemia (WM) (Press release Pharmacyclics, DEC 22, 2014, View Source [SID:1234501234]). IMBRUVICA is being jointly developed and commercialized by Pharmacyclics and Janssen Biotech, Inc.

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On December 22, 2014 Oncolytics Biotech reported that it has submitted applications for Orphan Designation to the European Medicines Agency ("EMA") for REOLYSIN for the treatment of pancreatic and ovarian cancers (Press release Oncolytics Biotech, DEC 22, 2014, View Source [SID:1234501238]).

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"This is the second jurisdiction in which we have elected to file for Orphan Designation," said Dr. Brad Thompson, President and CEO of Oncolytics. "The EMA serves all 28 member countries of the EU and securing Orphan Designation would help support the future development of REOLYSIN in the region."

The EMA grants Orphan Designation to medicines intended to treat, prevent or diagnose life threatening and debilitating disease, with a prevalence no greater than five in 10,000 in the EU, and where no satisfactory method of treatment, prevention or diagnosis exists, unless the proposed medicine offers a significant benefit to those with the condition. Following Orphan Designation, sponsors can access a number of incentives including protocol assistance, market exclusivity for a ten-year period following approval and potential fee reductions. The receipt of Orphan Designation does not change the regulatory requirements or process for obtaining marketing approval. For more information, please visit: View Source

REOLYSIN is Oncolytics’ proprietary isolate of the reovirus. Its primary mode of activity is to infect and selectively target tumours with activating Ras pathway mutations and/or over-expressions of Ras pathway elements including, amongst others, EGFR, BRAF, and KRAS. Up to 70% of pancreatic cancers have activating Ras pathway mutations and/or over-expressions.

On December 19, 2014 Myriad Genetics reported that it has received approval from the U.S. Food and Drug Administration (FDA) for BRACAnalysis CDx to be used as the only companion diagnostic in conjunction with AstraZeneca’s drug Lynparza (olaparib) (Press release Myriad Genetics, DEC 19, 2014, View Source [SID:1234501219]). Lynparza is the first poly ADP-ribose polymerase (PARP) inhibitor for patients with germline mutations in BRCA1/2 advanced ovarian cancer who have had three or more lines of chemotherapy. BRACAnalysis CDx is Myriad’s first FDA-approved companion diagnostic for use with a novel PARP inhibitor.

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"Myriad is excited to offer the first and only FDA-approved companion diagnostic for Lynparza, which we believe opens a new door in personalized medicine and represents a big step forward in tailoring treatment for women with ovarian cancer," said Mark Capone, president, Myriad Genetic Laboratories. "Less than 25 percent of ovarian cancer patients know their germline BRCA status, which is critical for any ovarian cancer patient who may be considered for treatment with Lynparza."

BRACAnalysis CDx is a highly accurate molecular companion diagnostic test that identifies deleterious or suspected deleterious mutations in the BRCA1 and BRCA2 genes, using DNA obtained from a blood sample. BRACAnalysis CDx was proven in clinical studies to effectively identify patients with BRCA mutations who would be candidates for Lynparza. The approval of BRACAnalysis CDx demonstrates Myriad’s commitment to developing companion diagnostics and is the culmination of an intensive, multiyear scientific collaboration with AstraZeneca to advance personalized medicine for women with ovarian cancer.

"Myriad has proven its ability to navigate a rigorous FDA regulatory approval process that included a comprehensive review of our DNA sequencing, large rearrangement detection and variant interpretation processes. Patients can be confident their BRACAnalysis CDx test results from Myriad are highly accurate," said Capone. "Our scientific excellence, reputation for high quality and regulatory experience are key reasons why Myriad is fast becoming the partner of choice for many biopharmaceutical companies seeking to co-develop companion diagnostic tests. We hope to expand our collaborations and further diversify our product portfolio."

Myriad is committed to being a leader in companion diagnostics for personalized healthcare. The Company isactively collaborating with several biopharmaceutical companies to further evaluate BRACAnalysis CDx as an investigational companion diagnostic for use with other PARP inhibitors and chemotherapeutic agents and for use in many other solid tumor types. BRACAnalysis CDx testing will be performed in Myriad’s laboratory in Salt Lake City, Utah. For more information, please visit www.myriad.com or call customer service at 1-800-469-7423.

About BRACAnalysis CDx

Intended Use: BRACAnalysis CDx is an in vitro diagnostic device intended for the qualitative detection and classification of variants in the protein coding regions and intron/exon boundaries of the BRCA1 and BRCA2 genes using genomic DNA obtained from whole blood specimens collected in EDTA. Single nucleotide variants and small insertions and deletions (indels) are identified by polymerase chain reaction (PCR) and Sanger sequencing. Large deletions and duplications in BRCA1 and BRCA2 are detected using multiplex PCR. Results of the test are used as an aid in identifying ovarian cancer patients with deleterious or suspected deleterious germline BRCA variants eligible for treatment with Lynparza (olaparib). This assay is for professional use only and is to be performed only at Myriad Genetic Laboratories, a single laboratory site located at 320 Wakara Way, Salt Lake City, UT 84108.

On December 19, 2014 Celgene International Sàrl, a wholly owned subsidiary of Celgene Corporation reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for continuous oral treatment with REVLIMID in adult patients with previously untreated multiple myeloma who are not eligible for stem cell transplantation (Press release Celgene, DEC 19, 2014, View Source [SID:1234501221]).

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The CHMP reviews applications for all 28 member states in the European Union (EU), as well as Norway, Liechtenstein and Iceland. The European Commission, which generally follows the recommendation of the CHMP, is expected to make its final decision in approximately two months. If approval is granted, detailed conditions for the use of this product will be described in the Summary of Product Characteristics (SmPC), which will be published in the revised European Public Assessment Report (EPAR).

Multiple myeloma is a persistent and life-threatening blood cancer that is characterised by tumour proliferation and suppression of the immune system. It is a rare but deadly disease: around 38,900 people were newly diagnosed with multiple myeloma in Europe in 2012, and 24,300 people died from the disease in the same year. On average, multiple myeloma is diagnosed between 65-74 years of age. The majority of newly diagnosed patients are not eligible for more aggressive treatment options such as high-dose chemotherapy with stem cell transplant, and there is currently no therapy option approved for continuous treatment to help manage the disease over the long term.

"When recommending a therapy at first diagnosis, our aim is to keep the disease under control for as long as possible," says Professor Thierry Facon, Services des Maladies du Sang, Hôpital Claude Huriez, and CHRU Lille, France. "The positive opinion for REVLIMID for the continuous treatment of adult patients with previously untreated multiple myeloma who are not eligible for transplant is a significant step towards bringing a new therapy that could extend the time patients live without their disease progressing."

The anticipated European Commission decision would be the latest milestone for Celgene’s flagship product in Europe and its continued focus on delivering innovative medicines for rare haematological diseases. REVLIMID is already indicated in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least one prior therapy. REVLIMID is also indicated for the treatment of patients with transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.

Tuomo Pätsi, President of Celgene in Europe, the Middle East and Africa (EMEA), said, "The CHMP opinion reflects the important role that therapies like REVLIMID play in treating rare haematological cancers including multiple myeloma. Innovative medicines have been critical in helping to improve patient outcomes, but despite tremendous progress over the last 10 years, myeloma remains incurable for the vast majority of patients, so new treatments are needed. At Celgene, we will continue to invest more than one-third of our revenues back into research and development to continue finding new treatment options for these patients. Our hope is that one day, deadly diseases like multiple myeloma could become a manageable, long-term chronic condition."

The CHMP recommendation in newly diagnosed multiple myeloma was based on the results of two pivotal studies: MM-015 and MM-020 (also known as FIRST). The results of these studies have been reported previously.

The FIRST study, MM-020,6 was the largest phase III, randomised study of 1,623 patients newly diagnosed with multiple myeloma and not eligible for stem cell transplantation. It compared lenalidomide-dexamethasone (Rd) administered in 28-day cycles until disease progression, with Rd for 72 weeks (18 cycles), and melphalan-prednisone-thalidomide (MPT) for 72 weeks.
MM-0157 was a phase III study of 459 patients that compared melphalan-prednisone-lenalidomide induction followed by lenalidomide maintenance (MPR-R) with melphalan-prednisone-lenalidomide (MPR) or melphalan-prednisone (MP) followed by placebo in patients ≥65 years or older with newly diagnosed multiple myeloma.

REVLIMID is not currently indicated for the treatment of newly diagnosed multiple myeloma in any country.

On December 19, 2014 RXi Pharmaceuticals reported that it has entered into an assignment and exclusive global license agreement with Hapten Pharmaceuticals, LLC for the therapeutic use of Samcyprone (Press release RXi Pharmaceuticals, DEC 19, 2014, View Source;FID=26640465 [SID:1234501224]). Samcyprone is a proprietary gel formulation of diphenylcyclopropenone (DPCP), an immunomodulating agent that works by initiating a T-cell response. Although DPCP is not a registered drug, it is used successfully by dermatologists as a topical immunomodulator to treat dermatological diseases. Samcyprone is expected to demonstrate an improved safety and use profile over DPCP as currently applied. A Phase 2a trial to evaluate the efficacy and safety of Samcyprone for the treatment of viral warts has been completed and Phase 2a trials for the treatment of cutaneous metastases of various cancers including melanoma and for the treatment of alopecia areata are underway.

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"Because of its proprietary gel formulation, Samcyprone is expected to demonstrate an improved safety and usage profile over DPCP as currently used," said Dr. William Levis, Lifetime Professor of Dermatology at Rockefeller University. He further added, "Published reports on clinical results with the use of DPCP as an experimental tool for treatment of these three difficult to treat skin conditions support the efficacy of the compound, substantially reducing the clinical risk of the ongoing product development effort."

Under the terms of the Agreement, Hapten will sell and assign to RXi certain patent rights and related assets and rights to Samcyprone. The Agreement will become effective at a closing that is scheduled to occur in early 2015 and which is subject to the satisfaction of certain closing conditions. Once the Agreement is effective, Hapten will receive an upfront payment payable in cash and common stock, will be entitled to receive future milestone payments tied to the achievement of certain clinical and commercial objectives and will receive royalties based on product sales.

"Following the release reporting the Company’s good progress in our Phase 2a program with RXI-109, our sd-rxRNA compound for treatment of hypertrophic scars and keloids, I am very pleased to also announce the acquisition by RXi Pharmaceuticals of Samcyprone, a Phase 2a product. Samcyprone is a proprietary formulation of DPCP, a new chemical entity (NCE), being evaluated in clinical trials in the USA for treatment of alopecia areata, warts, and cutaneous metastases of malignant melanoma," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He added that, "Samcyprone, currently in clinical development by Hapten Pharmaceuticals, provides RXi with a second Phase 2 asset for its development pipeline. The published therapeutic benefits of DPCP in the targeted diseases dramatically increase the likelihood of a successful development pathway for Samcyprone which should result in favorable safety and efficacy as well as providing for adequate exclusivity once the product is marketed."

At closing, the assignment and exclusive license of Samcyprone to RXi will immediately add a second clinical development candidate that comes with some unique features:

Many world class academic dermatology centers use DPCP topically as an unregulated experimental tool in alopecia areata, warts and cutaneous metastases of malignant melanoma. This experience supports the efficacy of DPCP as the active ingredient and should significantly increase the likelihood of successful clinical development of Samcyprone as a drug product;
DPCP is a new chemical entity under a U.S. IND. Samcyprone, the proprietary formulation of DPCP, should provide a favorable safety profile while providing a consistent cGMP formulation. It is expected to achieve market exclusivity post approval; and
Multiple synergies exist between RXi’s sd-rxRNA platform and Samcyprone. The mechanism of action of Samcyprone is linked to DPCP’s ability to alter the expression of multiple genes and miRNAs involved in the immune response. These gene targets may be modulated by an RNAi approach, utilizing sd-rxRNAs, to further enhance Samcyprone’s efficacy and response rates. Additionally, this approach may result in the discovery and development of sd-rxRNA or other drugs that are more potent and selective for treatment of alopecia areata, warts or cutaneous metastases of malignant melanoma.

In addition, the Company announced that it has entered into a new purchase agreement with Lincoln Park Capital Fund, LLC, a Chicago-based institutional investor, whereby LPC is committed to purchase an aggregate of up to $10.8 million in shares of RXi common stock over a 28-month term. Prior to entering this new agreement, the Company and LPC mutually agreed to terminate the prior purchase agreement that was in place, with respect to the $18 million unsold balance. The Company plans to use the proceeds from this new agreement with LPC to support the development of Samcyprone, as well as the advancement of the Company’s ophthalmology and dermatology franchises and for other general corporate purposes.