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Regorafenib from Bayer Submitted to Health Authorities Seeking Approval in Second-Line Treatment of Liver Cancer (for specialized target groups only)

On November 7, 2016 Bayer reported the submission of applications to extend the marketing authorization for its oral multi-kinase inhibitor regorafenib in the U.S., Japan and Europe, for the second line treatment of patients with unresectable hepatocellular carcinoma (HCC)(Press release, Bayer, NOV 7, 2016, View Source [SID1234516327]). Regorafenib is already approved under the brand name Stivarga in many countries to treat metastatic colorectal cancer and metastatic gastrointestinal stromal tumors.

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"Almost 10 years ago, we brought Nexavar, the first approved systemic therapy in liver cancer, to patients. We have remained committed to improving the lives of liver cancer patients and are proud that our efforts have yielded a second therapy with an overall survival advantage for patients," said Dr. Joerg Moeller, member of the Executive Committee of Bayer AG’s Pharmaceutical Division and Head of Development. "These filings bring us a step closer to potentially being able to offer a much needed second-line option to liver cancer patients in the near future."

"Approximately 780,000 people are diagnosed with HCC worldwide each year, a number that continues to increase. Regorafenib is the first treatment to successfully deliver an overall survival benefit in the second-line setting for HCC and has the potential to change the treatment paradigm by becoming the new standard of care for patients who progress on sorafenib," said Dr. Jordi Bruix, BCLC Group, Liver Unit, Hospital Clinic, University of Barcelona, IDIBAPS, CIBEREHD, Spain. Dr. Bruix is the Principal Investigator of the RESORCE study as well as the Phase III study SHARP which investigated sorafenib in HCC.

The regulatory submissions for regorafenib are based on data from the international, multicenter, placebo-controlled Phase III RESORCE [REgorafenib after SORafenib in patients with hepatoCEllular carcinoma] trial. The trial investigated regorafenib in patients with unresectable hepatocellular carcinoma (HCC) whose disease had progressed during treatment with sorafenib (Nexavar) tablets. Results showed that regorafenib significantly improved overall survival (OS) compared to placebo (HR 0.63; 95% CI 0.50-0.79; p<0.001), which over the trial period represents a 37 percent reduction in the risk of death for patients who received regorafenib plus best supportive care (BSC) compared to patients treated with placebo plus BSC. The median OS was 10.6 months in patients treated with regorafenib, compared to 7.8 months in patients who received placebo plus BSC. The safety and tolerability was generally consistent with the known profile of regorafenib, with no clinically meaningful differences in health-related quality of life (HRQoL) between the regorafenib and placebo plus BSC groups.

In the U.S., regorafenib has received Fast Track designation, which is an expedited program designed to facilitate the development, and expedite the review of drugs to address an unmet medical need in the treatment of a serious or life-threatening condition.

About the RESORCE trial
The Phase III RESORCE [REgorafenib after SORafenib in patients with hepatoCEllular carcinoma] clinical trial enrolled 573 patients whose disease had progressed during treatment with sorafenib. Patients were randomized in a 2:1 ratio to receive either regorafenib or placebo plus best supportive care.

Patients received 160 mg regorafenib once daily or placebo, for 3 weeks on/1week off, with 28 days constituting one full treatment cycle. The primary endpoint of the study was overall survival, and secondary endpoints were time to progression, progression-free survival, objective tumor response rate and disease control rate. Health-related quality of life was assessed by the FACT-Hep and EQ-5D questionnaires. Safety and tolerability were also continuously monitored.

About Hepatocellular Carcinoma
Hepatocellular carcinoma (HCC) is the most common form of liver cancer and represents approximately 70-85 percent of liver cancer worldwide. Liver cancer is the sixth most common cancer in the world and the second leading cause of cancer-related deaths globally. More than 780,000 cases of liver cancer are diagnosed worldwide each year (52,000 in the European Union, 501,000 in the Western Pacific region and 30,000 in the United States) and the incidence rate is increasing. In 2012, approximately 746,000 people died of liver cancer including approximately 48,000 in the European Union, 477,000 in the Western Pacific region and 24,000 in the United States.

About Regorafenib (Stivarga)
Regorafenib is an oral multi-kinase inhibitor that potently blocks multiple protein kinases involved in tumor angiogenesis (VEGFR1, -2, -3, TIE2), oncogenesis (KIT, RET, RAF-1, BRAF), metastasis (VEGFR3, PDGFR, FGFR) and tumor immunity (CSF1R).

Regorafenib is approved under the brand name Stivarga in more than 90 countries worldwide, including the U.S., countries of the EU and Japan for the treatment of metastatic colorectal cancer (mCRC). The product is also approved in over 80 countries, including the U.S., countries of the EU and Japan, for the treatment of metastatic gastrointestinal stromal tumors (GIST). In the EU, Stivarga is indicated for the treatment of adult patients with mCRC who have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidine-based chemotherapy, an anti-VEGF therapy and an anti-EGFR therapy, as well as for the treatment of adult patients with unresectable or metastatic GIST who progressed on or are intolerant to prior treatment with imatinib and sunitinib.

Regorafenib is a compound developed by Bayer. In 2011, Bayer entered into an agreement with Onyx, now an Amgen subsidiary, under which Onyx receives a royalty on all global net sales of regorafenib in oncology.

About Sorafenib (Nexavar)
Sorafenib, an oral anti-cancer therapy, has been shown in preclinical studies to inhibit multiple kinases thought to be involved in both cell proliferation (growth) and angiogenesis (blood supply) – two important processes that enable cancer growth. These kinases include Raf kinase, VEGFR-1, VEGFR-2, VEGFR-3, PDGFR-B, KIT, FLT-3 and RET.

Sorafenib is marketed under the brand name Nexavar and is approved for the treatment of certain forms of hepatocellular carcinoma (HCC), renal cell carcinoma (RCC) and differentiated thyroid carcinoma (DTC). Whilst licenses may differ from country to country, across all indications Nexavar is approved in more than 100 countries worldwide. In countries of the EU, Nexavar is approved for the treatment of (HCC); for the treatment of patients with advanced RCC who have failed prior interferon-alpha or interleukin-2 based therapy or are considered unsuitable for such therapy; and for progressive, locally advanced or metastatic, differentiated (papillary/follicular/Hürthle cell) thyroid carcinoma, refractory to radioactive iodine.

Bayer has worldwide exclusive marketing rights for Nexavar, with Bayer paying a royalty on US sales to Amgen Inc. Outside the U.S., Bayer and Amgen share profits globally, excluding Japan.

MorphoSys AG Reports Results for the First Nine Months of 2016

On November 7, 2016 MorphoSys AG (FSE: MOR; Prime Standard Segment; TecDAX, OTC: MPSYY) reported its financial results for the first nine months of 2016, and outlined the key events for the third quarter ending September 30, 2016 (Press release, MorphoSys, NOV 6, 2016, View Source [SID1234516664]).

Financial results for the first nine months of 2016

For first nine months of 2016, group revenues totaled EUR 36.7 million and EBIT amounted to EUR -32.3 million. Previous year’s figures included a non-recurring effect of approximately EUR 59 million (9-months 2015 revenues: EUR 93.9 million; 9-months 2015 EBIT: EUR 34.7 million).
Adjusted for last year’s one-off effect, 9-months group revenues rose by 5% year-on-year.
The Group’s liquidity position on September 30, 2016 amounted to EUR 267.2 million (December 31, 2015: EUR 298.4 million).
The Company confirms its 2016 guidance for revenues in the range of EUR 47 million to EUR 52 million and EBIT between EUR -58 million and EUR -68 million.
Operating highlights of the third quarter of 2016

In early August, MorphoSys announced the successful completion of the safety run-in of its phase 2 clinical trial of MOR208 in combination with lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) (L-MIND trial).
At the beginning of September, MorphoSys disclosed that the first patient had been dosed in the safety run-in of a phase 2/3 combination trial of MOR208 with bendamustine. The B-MIND trial will evaluate the safety and efficacy of MOR208 combined with the chemotherapeutic agent bendamustine in comparison to rituximab plus bendamustine. The study is expected to transition into a pivotal phase 3 part in 2017.
At the end of September, MorphoSys and its Belgian development partner Galapagos NV announced that the first patient with atopic dermatitis was dosed in an ongoing phase 1 trial of MOR106 against IL-17C after the antibody showed favorable safety in healthy volunteers.
In early July, MorphoSys disclosed the receipt of a milestone payment from Novartis, which was recognized in the second quarter of 2016. This payment was triggered by the initiation of a phase 1 clinical study of a novel HuCAL antibody for the prevention of thrombosis.
In September, the Company announced the appointment of four experts to its newly formed Scientific Advisory Board. This international panel of scientific experts was established to advise the Company on the strategic options and future perspectives within its research and development activities.
In September, MorphoSys’s Dutch subsidiary Lanthio Pharma B.V., which specializes in the development of lanthipeptides, announced the appointment of Axel Mescheder, M.D. as its Chief Medical Officer.
In mid-October, the Company announced the receipt of a milestone payment from Novartis, which was booked in the third quarter of 2016. The payment was triggered by the start of a phase 1 clinical trial with a novel HuCAL antibody in the field of cancer.
At the end of the third quarter, MorphoSys’s pipeline comprised an all-time high of 110 therapeutic programs, 28 of which are in clinical development.
Key events after the end of the third quarter of 2016

On October 1, 2016, MorphoSys announced that its licensee Janssen Research & Development, LLC (Janssen) reported positive results from a phase 3 clinical study of guselkumab in 837 patients with moderate to severe plaque psoriasis ("VOYAGE 1" study). Guselkumab is a fully human antibody targeting IL-23 which was selected from MorphoSys’s HuCAL antibody library. According to Janssen, both co-primary endpoints were met. Janssen also reported that all major secondary endpoints achieved statistical significance in comparisons of guselkumab versus adalimumab (Humira). Following the positive study results, Janssen announced plans to apply for regulatory approval in 2016. Guselkumab is expected to be the first HuCAL antibody to reach the market.

In EURO million* 9-Months 2016 9-Months 2015

Group Revenues 36.7 93.9
Total Operating Expenses 69.1 63.6
Other Income/Expenses 0.1 4.5
Earnings Before Interest and Taxes – EBIT (32.3) 34.7
Consolidated Net Profit / (Loss) (31.6) 28.2
Total EPS, diluted, in EURO (1.21) 1.07

* Differences due to rounding

"We are excited about the phase 3 data in moderate-to-severe psoriasis that our partner Janssen has generated with guselkumab. This could become the first product based on our proprietary technology to reach the market," commented Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG. "Our therapeutic pipeline is progressing well, now with 110 programs in development, more than ever before, of which 28 are in clinical studies."

"With the results shown for the first nine months of 2016 we are on track to meet our targets for the full year," stated Jens Holstein, Chief Financial Officer of MorphoSys AG. "Based on our solid financial situation with liquidity of EUR 267.2 million at the end of the third quarter, MorphoSys will continue to invest in our promising development candidates from a position of strength."

Financial Review of the First Nine Months of 2016 (IFRS)

Group revenues in the first nine months of 2016 amounted to EUR 36.7 million, compared to EUR 93.9 million in the first nine months of 2015. The main reason for the decline compared to the previous year period is a non-recurring effect of about EUR 59 million in 2015 in connection with the termination of the collaboration with Celgene for MOR202. Adjusted for last year’s one-off effect, revenues for the first nine months rose by 5%.

The Proprietary Development segment recorded revenues of EUR 0.5 million (9-months 2015: EUR 59.9 million). Revenues in the Partnered Discovery segment reached EUR 36.2 million (9-months 2015: EUR 34.0 million). Success-based payments amounted to about 10% of total revenues, or EUR 3.5 million (9-months 2015: 3% or EUR 2.5 million).

Total operating expenses for the first nine months of 2016 amounted to EUR 69.1 million (9-months 2015: EUR 63.6 million). Total research and development expenses were EUR 58.8 million (9-months 2015: EUR 53.1 million). The increase is mainly due to intensified clinical development activities with MorphoSys’s proprietary antibody candidates, in particular the start of two phase 2 trials with MOR208 in 2016. R&D expenses mainly consisted of costs for external laboratory services and personnel costs. General and administrative expenses decreased slightly to EUR 10.3 million (9-months 2015: EUR 10.6 million).

Earnings before interest and taxes (EBIT) amounted to EUR -32.3 million (9-months 2015: EUR 34.7 million). Adjusted for the one-off effect in 2015 amounting to EUR 59 million, the operating loss (EBIT) for the first nine months rose by 33%, mainly due to the increase in R&D activities.

The Proprietary Development segment reported a segment EBIT of EUR -45.5 million (9-months 2015: EUR 26.5 million), while Partnered Discovery showed a nine months segment EBIT of EUR 22.8 million (9-months 2015: EUR 18.1 million). Proprietary R&D expenses including technology development amounted to EUR 46.2 million, the comparative figure for 9-months 2015 was EUR 39.9 million.

On September 30, 2016, the Group’s liquidity position amounted to EUR 267.2 million compared to EUR 298.4 million on December 31, 2015. The Company’s liquidity is reflected in the balance sheet items "cash and cash equivalents", "available-for-sale financial assets", "bonds, available-for-sale" and current and non-current "financial assets classified as loans and receivables". The decline in liquidity was mainly the result of the use of cash for operations in the first nine months of 2016 and the repurchase of shares for the Group’s long-term incentive program.

Financial guidance for 2016

MorphoSys re-confirmed its guidance for 2016. MorphoSys anticipates total Group revenues in the range of EUR 47 million to EUR 52 million and expects EBIT to be in the range of EUR -58 million to EUR -68 million. Proprietary R&D expenses are expected to rise to EUR 76 million to EUR 83 million. This guidance does not include any potential in-licensing or co-development of additional development candidates.

10-Q – Quarterly report [Sections 13 or 15(d)]

Cerus has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Cerus, NOV 4, 2016, View Source [SID1234516265]).

Redx to present pre-clinical profile of its reversible BTK inhibitor at ASH 2016

On November 4, 2016 Redx Pharma Plc reported that it will present the pre-clinical profile of its reversible Bruton’s tyrosine kinase (BTK) inhibitor at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on 5 December 2016 in San Diego, California (Press release, Redx Pharma, NOV 4, 2016, View Source [SID1234524712]). The compound, named RXC005 (also known as REDX08608), is a novel, potent and selective, reversible BTK inhibitor that is equipotent against wild-type and mutant C481S BTK. C481 mutant BTK protein is currently estimated to be responsible for around 60% of the observed Ibrutinib resistance in patients with chronic lymphocytic leukaemia.

The Company is progressing studies to prepare the RXC005 program for first-in-human clinical trials. The aim is to commence these trials late 2017.

The Abstract for the presentation is available on the ASH (Free ASH Whitepaper) Conference website:
View Source;

Dr Neil Murray, CEO of Redx, said: We’re delighted to present the compelling pre-clinical profile of our reversible BTK inhibitor RXC005 at the prestigious American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December.

RXC005 has the potential to become a potent therapy for chronic lymphocytic leukaemia patients by tackling the growing resistance to Ibrutinib treatment. We aim to initiate first-in-human clinical studies for RXC005 late 2017.

Apogenix Reports Significant Progress in Collaboration with CANbridge

On November 4, 2016 Apogenix, a biopharmaceutical company developing next-generation immuno-oncology therapeutics, reported the achievement of additional milestones under its licensing agreement with CANbridge Life Sciences for the development and commercialization of lead candidate APG101 (INN: asunercept) in China, Macao, Hong Kong and Taiwan, triggering further paymentsto Apogenix (Press release, Apogenix, NOV 4, 2016, View Source [SID1234524576]). The milestones are related to the successful implementation of the technology transfer necessary for the production of this CD95 ligand inhibitor at the Chinese production site.

Over the course of the past few months, Apogenix and CANbridge have successfully transferred the necessary cell banks, assays, protocols, and know-how for the manufacturing of asunercept to China. The manufacturing process has proven to be very robust with product quality and process yields of the active substance comparable to the manufacturing process developed by Apogenix

CANbridge also reported that clinical development of asunercept is now underway in Taiwan. The recently initiated Phase I/II trial is evaluating asunercept plus temozolomide (TMZ) during and after radiation therapy in 55 patients with newly diagnosed glioblastoma. The study design consists of an open-label, dose-escalation Phase I trial, and a multi-center, double-blind, randomized, placebo-controlled Phase II trial. The Phase I trial will evaluate safety, tolerability, pharmacokinetics and preliminary efficacy. The Phase II part will evaluate efficacy and safety.

"We are very pleased with the progress of our collaboration with CANbridge and the achievement of additional milestones," said Thomas Hoeger, Ph.D., CEO of Apogenix. "With the successful technology transfer and the initiation of the Phase I/II trial in Taiwan,the clinical development of asunercept in Asia in now well underway. We look forward to CANbridge initiating further trials in China soon."

James Xue Ph.D., Chairman and CEO of CANbridge: "The complex transfer of asunercept production technology and know-how has been very efficient due to the excellent collaboration between the CANbridge and Apogenix teams. In particular, we were impressed by the exceptional level of enthusiasm and professional handling during the entire process."

About Asunercept (APG101)
Apogenix’s lead immuno-oncology candidate asunercept is a fully human fusion protein that consists of the extracellular domain of the CD95 receptor and the Fc domain of an IgG antibody. Asunercept is being developed for the treatment of solid tumors and malignant hematological diseases. By blocking the CD95 ligand, which negatively regulates erythrocyte production in myelodysplastic syndromes (MDS) patients, asunercept directly addresses the cause of the disorder and could thus potentially provide a cure for MDS. The World Health Organization (WHO) has recently assigned the international nonproprietary name (INN) "asunercept" for APG101.