On December 19, 2018 Spirita Oncology, LLC, reported that it has entered into a sublicense agreement with JS Innopharm (Shanghai) Ltd. to initiate global clinical development of E6201, a potent MEK1 inhibitor with excellent brain penetration (Press release, Spirita Oncology, DEC 19, 2018, View Source [SID1234532159]).

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In August 2018, Strategia Holdings, LLC, a parent company of Spirita Oncology, LLC, and JS Innopharm initiated a Strategic Partnership Alliance to facilitate global drug development. This China sublicense of E6201 is conducted as part of these activities.

In November 2018, Spirita Oncology and The University of Arizona Cancer Center announced the Initiation of a Phase 1 clinical trial of the E6201 in patients with central nervous system metastases from BRAF+ or MEK-mutated metastatic melanoma. In a previous study (NCT00794781), E6201 demonstrated activity in metastatic melanoma, including a patient who maintains an exceptional ongoing durable response lasting for almost 9 years ("E6201, an intravenous MEK1 inhibitor, achieves an exceptional response in BRAF V600E-mutated metastatic malignant melanoma with brain metastases" Babiker, H.M., Byron, S.A., Hendricks, W.P.D. et al. Invest New Drugs (2018). View Source).

"This partnership is to effectively deliver new therapeutic approaches to patients worldwide. Spirita is expanding the E6201 global development to provide a more effective therapeutic by collaborating with JS Innopharm," said Keizo Koya, Ph.D., President & CEO of Spirita Oncology. "We are also planning to in-license promising oncology candidates to expand our global development with the power of the US and China coming together."

"We are very pleased to partner with Spirita Oncology to advance the global development of E6201, which allow us to bring this product candidate quickly to the Chinese patients," said Dr. Jintao Zhang, CEO of JS InnoPharm. "We look forward to working with Spirita Oncology on E6201 program, and will continue to work together to in-license and develop promising oncology candidates that have synergies with our in-house pipeline."

On December 19, 2018 Acorda Therapeutics, Inc. (Nasdaq:ACOR) reported that Ron Cohen, M.D., Acorda’s President and CEO, will present at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 9 at 8:00am PST / 5:00am EST (Press release, Acorda Therapeutics, DEC 19, 2018, View Source [SID1234532143]).

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A live audio webcast of the presentation can be accessed under "Investor Events" in the Investor section of the Acorda website at www.acorda.com, or you may use the link:

On December 19, 2018 Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) reported its results from the pivotal Phase III trial MEDALIST in early December 2018 (Press release, Celgene, DEC 19, 2018, View Source [SID1234532160]). The study evaluated the efficacy and safety of the investigational product luspatercept for the treatment of patients suffering from anemia due to ring dermoblast-positive (RS +) myelodysplastic syndrome (MDS), transfusions with packed red blood cells (EC), and where erythropoietin therapy was unsuccessful , there was an intolerance to it or such therapy was out of the question. The results were Alan F. List during the scientific plenary session on the 60th

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"Severe anemia leading to red blood cell transfusion dependence is a significant burden for patients at low risk MDS. For patients who become resistant or refractory to currently available therapies, there are only limited alternatives, "said Drs. List, President and CEO of the Moffitt Cancer Center. "The results from the MEDALIST study are very promising. They support the hypothesis that luspatercept promotes the maturation of erythroid progenitor cells, allowing patients to become transfusion-independent. Luspatercept could thus improve the treatment of anemia in RS + MDS patients. "

The MEDALIST trial reached its primary endpoint: A statistically significantly greater proportion of patients achieved RBC-TI independence of ≥ 8 weeks in the first 24 weeks of treatment with luspatercept compared to placebo. In addition, all important secondary endpoints of the study were achieved. Compared to placebo, a significantly greater proportion of patients in the luspatercept arm achieved RBC-TI ≥ 12 weeks in the first 24 and 48 weeks of study, respectively, and hematologic erythropoiesis (HI-E after IWG 2006) of 8 weeks or more.

endpoints Luspatercept placebo p-value
RBC-TI ≥ 8 weeks (Week 1-24) 37.9 % (58/153) 13.2 % (10/76) <0.0001
RBC-TI ≥ 12 weeks (Week 1-24) 28.1 % (43/153) 7.9 % (6/76) 0.0002
RBC-TI ≥ 12 weeks (Week 1-48) 33.3 % (51/153) 11.8 % (9/76) 0.0003
HI-E ≥ 8 weeks (IWG 2006, weeks 1-24) 52.9 % (81/153) 11.8 % (9/76) <0.0001

Summary of safety data from the MEDALIST study

Third or fourth degree treatment-associated adverse events (TEAEs) were observed in 42.5% (65/153) of patients receiving luspatercept and 44.7% (34/76) of patients receiving placebo. Disease progression to acute myelogenous leukemia (AML) occurred in a total of four patients: three patients (2.0%) treated with luspatercept and one patient (1.3%) receiving placebo. Five patients receiving luspatercept (3.3%) and four patients receiving a placebo (5.3%) experienced one or more TEAEs that were fatal.

The most common TEAEs of any grade in more than 10% of patients in either study arm

Luspatercept
n = 153

placebo
n = 76

fatigue 26.8 % 13.2 %
diarrhea 22.2 % 9.2 %
asthenia 20.3 % 11.8 %
nausea 20.3 % 7.9 %
dizziness 19.6 % 5.3 %
back pain 19.0 % 6.6 %

"The results of the MEDALIST study illustrate the potential clinical benefits of Luspatercept to make patients with RS + low-risk MDS less dependent on red blood cell transfusion. This is an area where new therapies are needed. " med. Alise Reicin, President of Global Clinical Development at Celgene. "These findings reinforce our belief that this first-in-class erythropoietic-based drug can help these patients address the underlying cause of their disease-related chronic anemia."

"It is a great honor to present the results of the MEDALIST study as the first presentation at the ASH (Free ASH Whitepaper) plenary session," said Habib Dable, President and Chief Executive Officer of Acceleron. "The results of the MEDALIST study give us confidence that luspatercept can provide a relevant treatment option for patients with low-risk RS + MDS anywhere in the world. We look forward to continuing our clinical development program for MDS, beta-thalassemia and myelofibrosis, while investigating other applications of luspatercept in a variety of anemia-related diseases. "

Luspatercept is not approved in any region and for no indication. Acceleron and Celgene are planning to submit regulatory filings for Luspatercept in the US and Europe in the first half of 2019.

About MEDALIST

MEDALIST is a randomized, double-blind, placebo-controlled, multicentre phase III trial to evaluate the safety and efficacy of luspatercept in patients with very low, low or medium risk RS + myelodysplastic syndromes (MDS). All patients were RBC transfusion-dependent and either refractory to previous therapy with erythropoiesis-stimulating agents (ESA), did not tolerate them or were ESA naive with endogenous serum erythropoietin ≥200 U / L and had no previous treatment with disease modifying agents receive. The median age of the study participants was 71 years in the luspatercept treatment group and 72 years in the placebo group. The mean transfusion load in both study arms was 5 RBC units / 8 weeks. A total of 229 patients were randomized to receive either luspatercept 1.0 mg / kg (153 patients) or placebo (76 patients) every 21 days as a subcutaneous injection. The study was conducted at 65 sites in 11 countries.

About Luspatercept

Luspatercept, the erythrocyte-ripening substance, is the first active ingredient in a new erythroid maturation agent (EMA). It promotes erythrocyte maturation in the late stages of erythropoiesis. Acceleron and Celgene are developing Luspatercept as part of a global collaboration. Phase III clinical trials are evaluating the safety and efficacy of luspatercept in patients with MDS (MEDALIST trial) and patients with beta thalassemia (BELIEVE trial). The COMMANDS phase III study in low-risk first-line MDS patients, the BEYOND non-transfusion-dependent beta-thalassemia phase II study and a phase II study in myelofibrosis are ongoing. For more information, seewww.clinicaltrials.gov .

On December 19, 2018 Auron Therapeutics and Elucidata Corporation reported a scientific collaboration using Elucidata’s AI-based target discovery platform to identify and validate targets for differentiation9-based therapy for Acute Myeloid Leukemia (AML) and eight other oncology indications (Press release, Auron Healthcare, DEC 19, 2018, View Source [SID1234532161]). As part of this collaboration, Elucidata will use its data analytics platform PollyTM, to analyze transcriptomic, metabolomic and epigenetic data from biological samples, as well as disease and treatment response data from patients. This four-year collaboration is among the broadest efforts to date to apply the differentiation-based approach for oncology therapies and has the potential to improve outcomes for patients living with these diseases.

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Elucidata’s AI-based platform combines different forms of omics data using advanced computational analysis techniques to characterize healthy and diseased states at the molecular level. Auron will provide phenotypic data along with transcriptomic, metabolomic and epigenetic data from hundreds of patient samples which will be analyzed on PollyTM to identify and validate targets to develop new therapies with improved treatment efficacy and minimal side effects for a subgroup of patients. The insights generated from this collaboration will further enable stratification of subtypes of different cancers.

"I am extremely impressed by the PollyTM platform that Elucidata has built and the power it has to digest, integrate and analyze large data sets," said Kate Yen, Ph.D., Founder and CEO of Auron Therapeutics. "The unique partnership that we have allows us to work hand-in-hand with the software engineers, scientists, and program managers to rapidly develop novel hypotheses which we can test in the lab. In just a short period of time, we have made significant progress that has had a substantial impact on Auron’s growth."

"With our platform, we are seeking to develop an atlas of differentiation paths of healthy and diseased cells that will help us identify and characterize disease mechanisms," said Abhishek Jha, Co-founder and CEO of Elucidata. "This collaboration with Auron is an incredible opportunity to realize the promise of big data analytics to discover new targets that will deliver more precise medicine to patients."

On December 19, 2018 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), reported that based on a subset of data from MD Anderson’s ongoing Phase 2 study, the U.S. Food and Drug Administration (FDA) did not grant Breakthrough Therapy Designation (BTD) to poziotinib for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have EGFR exon 20 mutations (Press release, Spectrum Pharmaceuticals, DEC 19, 2018, View Source;19.htm [SID1234532181]). The company’s overall development plan and timeline for a New Drug Application (NDA) filing based on the first cohort of the ZENITH20 trial remains unchanged.

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Breakthrough Therapy Designation is one of several FDA programs designed to expedite the review of drugs to treat serious or life threatening conditions. Spectrum’s BTD application included data from 30 patients from the MD Anderson Phase 2 study who had failed platinum-based chemotherapy. The data demonstrated a confirmed objective response rate of 40% and median duration of response of 6.6 months. The safety profile in this subset was consistent with historical data published on poziotinib and other tyrosine kinase inhibitors. The historical objective response rates for mutation specific NSCLC patients range between 0% and 8% with tyrosine kinase inhibitors and for non-mutation specific NSCLC patients range between 0.8% and 22.9% with other treatments.

"Our enthusiasm for poziotinib, our commitment to the program, and our overall development plan remain unchanged," said Joe Turgeon, President and Chief Executive Officer of Spectrum Pharmaceuticals. "We will continue to work with the FDA to achieve the fastest route to approval of poziotinib based on our ZENITH20 study."

ZENITH20 is a Spectrum-sponsored, open-label, single-arm, multi-center, global Phase 2 study evaluating more than 300 NSCLC patients with EGFR or HER2 exon 20 insertion mutations. The study consists of four cohorts, each of which is independently powered for a pre-specified statistical hypothesis.

"Poziotinib’s overall development program is robust, and the clinical profile remains very attractive in an area of high unmet need," said Francois Lebel, M.D., F.R.C.P.C, Chief Medical Officer of Spectrum Pharmaceuticals. "Data required for the NDA filing for previously treated NSCLC patients with EGFR exon 20 insertion mutations is expected to come from an 87-patient cohort in our ZENITH20 study. We expect to complete enrollment in this cohort in the first quarter of 2019, and announce topline data in the second half of 2019."

11500 S. Eastern Ave., Ste. 240 • Henderson, Nevada 89052 • Tel: 702-835-6300 • Fax: 702-260-7405 • www.sppirx.com • NASDAQ: SPPI

poziotinibfinal121918_image1.gif

Conference Call Details:
Wednesday, December 19, 2018 at 4:30 p.m. Eastern/1:30 p.m. Pacific
Domestic: (877) 837-3910, Conference ID# 1577888
International: (973) 796-5077, Conference ID# 1577888
The conference call will also be webcast live. To access the webcast, please visit the Investor Relations page of the Spectrum Pharmaceuticals website at View Source
For interested individuals unable to join the call, a replay will be available from December 19, 2018 @ 7:30 p.m. ET/4:30 p.m. PT through December 26, 2018, until 7:30 p.m. ET/4:30 p.m. PT.
Domestic Replay Dial-In: (855) 859-2056, Conference ID# 1577888
International Replay Dial-In: (404) 537-3406, Conference ID# 1577888

About Poziotinib
Poziotinib is a novel, oral Epidermal Growth Factor Receptor Tyrosine Kinase Inhibitor (EGFR TKI) that inhibits the tyrosine kinase activity of EGFR as well as HER2 and HER4. Importantly this leads to the inhibition of the proliferation of tumor cells that overexpress these receptors. Mutations or overexpression/amplification of EGFR family receptors have been associated with a number of different cancers, including non-small cell lung cancer (NSCLC), breast cancer, and gastric cancer. Spectrum received exclusive license from Hanmi Pharmaceuticals to develop, manufacture, and commercialize poziotinib worldwide, excluding Korea and China. Poziotinib is currently being investigated by Spectrum and Hanmi in several mid-stage trials in multiple solid tumors.

About ZENITH20

The ZENITH20 study is Spectrum-sponsored, Phase 2, open-label, single-arm, multi-center, global study for patients with EGFR or HER2 exon 20 insertion mutations in NSCLC. It consists of four cohorts, each of which is an independent study with pre-specified statistical hypotheses and statistical power. There are two cohorts in the previously-treated setting and two in the first-line setting. The full poziotinib targeted therapy clinical program is focused on four development areas for EGFR and HER2 mutations, including previously treated NSCLC, first-line NSCLC, treatment of other solid tumors and combination therapy.