On May 8, 2017 /Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and SillaJen, Inc. (KOSDAQ: 215600) reported a new clinical and supply agreement for a Phase 1b dose-escalation study in renal cell carcinoma (RCC), or kidney cancer (Press release, Regeneron, MAY 8, 2017, View Source [SID1234518959]). The study will evaluate Regeneron’s PD-1 inhibitor, REGN2810, in combination with SillaJen’s oncolytic vaccinia virus, Pexa-Vec, in patients with previously treated metastatic or unresectable renal cell carcinoma.

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The open-label trial is expected to begin later this year, and is designed to evaulate the safety and efficacy of REGN2810 in combination with Pexa-Vec compared to treatment with REGN2810 as monotherapy. The study will initially open in Korea, with expansion to sites in the U.S.

Renal cell carcinoma is the most common type of kidney cancer in adults, and accounts for approximately three percent of adult malignancies and 90-95 percent of neoplasms arising from the kidney.

"We are pleased to announce this immuno-oncology agreement with Regeneron—another company doing groundbreaking work in oncology," said Eun Sang Moon, MD, DDS, Chief Executive Officer of SillaJen. "We believe that there may be synergies between our compounds and look forward to investigating this in our upcoming clinical trial in renal cell carcinoma."

Pexa-Vec has been shown to increase inflammation in the tumor micro-environment and potentially reverse the immunosuppressive microenvironment by activating T-cell mediated anti-tumor immune response. This may help "prime" the tumor by increasing sensitivity to anti-PD-1 treatment, such as REGN2810.

"Pairing our PD-1 inhibitor with other innovative approaches like Pexa-Vec allows us to evaluate multiple routes in the quest to provide important new treatments to people in need," Israel Lowy, MD, PhD, Vice President of Translation Sciences and Oncology, Regeneron. "Renal cell carcinoma is a devastating and currently under-treated disease, and combination therapies that enable the body’s immune response in novel ways may help improve these patients’ lives."

Under the terms of the agreement, the trial will be solely conducted and funded by SillaJen based upon a mutually developed study design," and Regeneron will provide REGN2810. Regeneron, in collaboration with Sanofi, is developing REGN2810 both alone and in combination with other therapies for the treatment of various cancers.

"Given the results reported thus far for monotherapy anti-PD1 immunotherapy and initial studies of Pexa-Vec for the treatment of advanced kidney cancer, we believe the combination of Pexa-Vec and REGN-2810 holds great promise," stated James Burke, MD, Chief Medical Officer of SillaJen. "This combination immunotherapy approach in RCC joins the oncolytic immunotherapy expertise of SillaJen with the well-known leadership of Regeneron in antibody targeted therapies."

About Pexa-Vec and the SOLVE Platform
Pexa-Vec is the most advanced product candidate from SillaJen’s proprietary SOLVE (Selective Oncolytic Vaccinia Engineering) platform. The vaccinia strain backbone of Pexa-Vec has been used safely in millions of people as part of a worldwide vaccination program, and over 300 cancer patients have been treated with Pexa-Vec to date. Pexa-Vec was engineered to target common genetic defects in cancer cells by deleting its thymidine kinase (TK) gene, thus making Pexa-Vec dependent on the cellular TK expressed at persistently high levels in cancer cells. Pexa-Vec is also engineered to express GM-CSF protein. GM-CSF complements the cancer cell lysis of the product candidate, leading to a cascade of events resulting in tumor necrosis, tumor vasculature shutdown and sustained anti-tumoral immune attack. Pexa-Vec has been shown to be effective when delivered both intratumorally and systemically by intravenous administration. Pexa-Vec is currently being evaluated in a worldwide Phase 3 clinical trial for advanced primary liver cancer, and more information can be found at View Source

About Regeneron Pharmaceuticals, Inc.
Regeneron (NASDAQ: REGN) is a leading science-based biopharmaceutical company that discovers, invents, develops, manufactures and commercializes medicines for the treatment of serious medical conditions. Regeneron commercializes medicines for eye diseases, high LDL-cholesterol, atopic dermatitis and a rare inflammatory condition and has product candidates in development in other areas of high unmet medical need, including rheumatoid arthritis, asthma, pain, cancer and infectious diseases. For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.

About SillaJen
SillaJen, Inc. (KOSDAQ: 215600) is a South Korean-based biotechnology company headquartered in Busan, South Korea, with satellite offices in Seoul, South Korea and San Francisco, CA. The company is focused on the development and commercialization of oncolytic immunotherapy products using the SOLVE platform, including its lead product Pexa-Vec, which is currently in Phase 3 trials for the treatment of advanced primary liver cancer. Additional information about SillaJen is available at www.sillajen.com.

On May 08, 2017 OncoMed Pharmaceuticals Inc. (NASDAQ:OMED), a clinical-stage biopharmaceutical company focused on discovering and developing novel anti-cancer therapeutics, reported first quarter financial results (Press release, OncoMed, MAY 8, 2017, View Source [SID1234518968]). As of March 31, 2017, cash, and short-term investments totaled $156.9 million.

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"We continue to drive forward our strong immuno-oncology R&D pipeline, including anti-TIGIT, wholly-owned GITRL-Fc trimer, and our novel undisclosed immuno-oncology discovery programs — as well as advancing programs in our Celgene collaboration to $98 million in potential opt-in payments within the next two years," said Paul J. Hastings, OncoMed’s Chairman and CEO. "With more than two years cash, we are dedicated to advancing our pipeline while exploring partnering opportunities to advance all of our programs."

DENALI Phase 2 Clinical Trial Update

The company is reporting top-line results from the three arm randomized Phase 2 "DENALI" clinical trial of demcizumab (anti-DLL4, OMP-21M18) in combination with carboplatin and pemetrexed in front-line non-squamous non-small cell lung cancer (NSCLC). DENALI was designed to assess the efficacy and safety of either one or two 70 day truncated courses of demcizumab plus carboplatin and pemetrexed versus carboplatin and pemetrexed plus placebo. The primary endpoint of the study was overall response rate (ORR) and secondary endpoints were clinical benefit rate (CBR; rate of complete and partial responses and stable disease), progression-free survival (PFS), overall survival (OS) and safety. The statistical plan for DENALI was based on the expectation of enrolling 200 patients, but enrollment was discontinued at 82 patients due to the evolving treatment landscape in NSCLC. Of these 82 patients, 25 patients received carboplatin and pemetrexed plus placebo, while 57 received carboplatin, pemetrexed plus either one or two 70 day courses of demcizumab. Although these data were not fully mature at the time of analysis, demcizumab treatment failed to meet its efficacy endpoints when compared to placebo, with better outcomes apparent in the placebo group. Specifically, the ORR was 28% versus 52% (p=0.04) and CBR was 79% versus 92% (p=0.17) in the pooled demcizumab arms and the placebo arm, respectively. Median PFS was 5.5 months versus 8.7 months (p=0.02) and mOS was 15.5 months versus not reached (p=.06) in the pooled demcizumab arms and the placebo arm, respectively. No statistically significant differences in efficacy were observed between patients receiving one course or two courses of demcizumab. Demcizumab was generally well tolerated in combination with chemotherapy with nausea, fatigue, constipation, anemia and hypertension being the most common toxicities. There were no cases of Grade 3 or greater heart failure or pulmonary hypertension in this study. The overall safety profile was consistent with that observed in our other studies, and no new safety signals were identified.

OncoMed is discontinuing the dosing of all patients on the demcizumab trials, including the demcizumab plus pembrolizumab Phase 1b study, and will conduct a complete program review in the near term with its partner Celgene.

Recent Developments

Presented data from multiple preclinical studies detailing the mechanism and anti-tumor activity of anti-TIGIT alone and in combination with checkpoint inhibitors at the AACR (Free AACR Whitepaper) Annual Meeting 2017. The first patient in a Phase 1a single-agent study of OncoMed’s anti-TIGIT antibody (OMP-313M32) was recently enrolled.

Began enrollment of patients in two Phase 1b clinical trials of anti-DLL4/VEGF bispecific antibody (OMP-305B83), now known as navicixizumab, plus standard-of-care chemotherapy for the treatment of second-line colorectal and platinum-resistant ovarian cancers.

Announced partner Bayer Pharma decided not to exercise its option to license the first-in-class Wnt pathway inhibitors vantictumab (anti-Fzd, OMP-18R5) and ipafricept (Fzd8-Fc, OMP-54F28) for strategic reasons. OncoMed announced it put both programs on hold, and that it would pursue potential partnering opportunities for Wnt/IO combinations, utilizing different dosing regimens than those used in the Phase 1b studies. OncoMed has subsequently decided to cease dosing patients in the current vantictumab and ipafricept Phase 1b studies following a recent bone adverse event that occurred in a patient receiving vantictumab plus paclitaxel in the Phase 1b HER2-negative breast cancer trial.

Announced negative top-line trial results for YOSEMITE and PINNACLE Phase 2 studies and subsequent workforce reduction.

First Quarter 2017 Financial Results
Cash and short-term investments totaled $156.9 million as of March 31, 2017, compared to $184.6 million as of December 31, 2016.

Revenues for the first quarter 2017 totaled $6.2 million, as compared to $6.4 million in the first quarter of 2016. The slight decrease in revenue over the same period in 2016 was primarily due to slightly lower revenue recognized from reimbursement of research and development costs for services performed in the first quarter of 2017.

Research and development (R&D) expenses for the first quarter 2017 were $24.0 million compared with $28.4 million for the same period in 2016. The decrease was primarily due to lower external research and development costs attributable to the decrease in Phase 2 clinical trial costs of demcizumab and tarextumab programs, partially offset by an increase in internal program costs.

General and administrative (G&A) expenses for the quarter ended March 31, 2017 were $5.0 million, compared to $5.2 million for the same period in 2016. Decreased expenses during the first quarter 2017 were due to lower consulting and outside professional service costs.

Net loss for the first quarter 2017 was $22.6 million ($0.61 per share), compared to $27.2 million ($0.90 per share) for the same period of 2016. The change in net loss from the prior year quarter was due to lower R&D and G&A expenses.

2017 Financial Guidance
OncoMed anticipates 2017 full-year cash expenses will be approximately $90 million, including the impact of one-time severance-related charges in the range of approximately $2.6 million to approximately $3.1 million related to our previously announced workforce reduction. Based on the current plan, OncoMed anticipates that its current cash balance is sufficient to fund pipeline development and company operations through the third quarter of 2019, before considering potential opt-in milestones under our Celgene collaboration. These milestones include:

Anti-TIGIT: Potential $35 million following the completion of our ongoing Phase 1a clinical trial.

Rosmantuzumab (Anti-RSPO3): Potential $38 million following the completion of our ongoing Phase 1a and Phase 1b clinical trials, focused on RSPO3-high and RSPO gene fusion patients.

Navicixizumab (Anti-DLL4/VEGF): Potential $25 million following the completion of our ongoing Phase 1a and Phase 1b clinical trials.

Following potential opt-in, OncoMed would be eligible to co-develop and co-commercialize rosmantuzumab and/or navicixizumab with Celgene, while Celgene would assume all downstream costs and development activities for anti-TIGIT post option exercise. The company could be eligible to receive approximately $1.5 billion in downstream milestones related to these three programs, plus potential royalties and/or profit-sharing.

Conference Call Today
OncoMed management will host a conference call today beginning at 8:30 a.m. ET/5:30 a.m. PT to review first quarter 2017 financial results and recent progress.

Analysts and investors can participate in the conference call by dialing 1-855-420-0692 (domestic) and 1-484-756-4194 (international) using the conference ID# 18252865. The webcast of the conference call can be accessed live on the Investor Relations section of the OncoMed website, View Source An audio replay of the conference call can be accessed by dialing 1-855-859-2056 (domestic) or 1-404-537-3406 (international) utilizing the conference ID number listed above. The web broadcast of the conference call will be available for replay through July 31, 2017 via the OncoMed website.

On May 8, 2017 Cellular Biomedicine Group Inc. (NASDAQ: CBMG) ("CBMG" or the "Company"), clinical-stage biopharmaceutical firm engaged in the development of effective immunotherapies for cancer and stem cell therapies for degenerative diseases, reported financial results for the first quarter ended March 31, 2017 and provided business highlights (Press release, Cellular Biomedicine Group, MAY 8, 2017, View Source [SID1234519117]).

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"The first quarter of 2017 was very productive, with several key achievements, including the commencement of our second Phase I CAR-T clinical trial utilizing CBMG’s proprietary and optimized CD19 construct, for the treatment of adult patients with relapsed or refractory CD19+ B-cell Acute Lymphoblastic Leukemia (ALL)," commented Tony Liu, Chief Executive Officer of CBMG. "The award of $2.29 million from the California Institute for Regenerative Medicine (CIRM) to support pre-clinical studies of AlloJoinTM in the U.S., moves forward our endeavor into the U.S. market and the development of an off-the-shelf stem cell product to treat Knee Osteoarthritis (KOA). The signing of a collaboration with GE Healthcare Life Sciences China to establish a joint laboratory within our own GMP facilities in Shanghai credits our GMP stature and capabilities. We are determined to build on our accomplishments from the first quarter to continue to strengthen our innovative pipelines and move our clinical assets into later stage development. We believe we are ahead of the competitive curve in addressing the manufacturing barriers to delivering consistent clinical grade cell therapies which have the potential to address the large cancer and knee osteoarthritis markets."

First Quarter 2017 Financial Performance

Cash Position:Cash and cash equivalents as of March 31, 2017 were $33.4 million compared to $39.3 million as of December 31, 2016.
Net Cash Used in Operating Activities:Net cash used in operating activities for the first quarter of 2017 was $4.86 million, compared to $3.58 million for the same period in 2016.
G&A Expenses:General and administrative expenses for the first quarter of 2017 were $3.2 million compared to $2.8 million for the same period in 2016. The increase is in large part attributed to the rental increase, which resulted from the new leased facilities located in the "Pharma Valley" of Shanghai from January 1, 2017.
R&D Expenses:Research and development expenses for the first quarter of 2017 were $3.0 million, compared to $2.4 million for the same period a year ago. The increase was primarily attributable to headcount increases of R&D staff and increased expenses related to advancing assets into clinical trials.
Net Loss:Net loss allocable to common stock holders was $6.2 million, compared to $4.2 million for the same period in 2016.
Business & Technology Highlights of 2017 To Date

Commenced CALL-1 ("CAR-T against Acute Lymphoblastic Leukemia") Phase I clinical trial in China utilizing its optimized proprietary C-CAR011 construct of CD19 chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of patients with relapsed or refractory (r/r) CD19+ B-cell Acute Lymphoblastic Leukemia (ALL);
Awarded $2.29 million by California Institute for Regenerative Medicine (CIRM), California’s stem cell agency, to support pre-clinical studies of AlloJoinTM, CBMG’s "Off-the-Shelf" Allogeneic Human Adipose-derived Mesenchymal Stem Cells for the treatment of Knee Osteoarthritis in the United States;
On May 4, 2017, the Company received $1.2 million from the CIRM grant, the first of four disbursements
Completed expansion of its 30,000 square foot facility in Huishan High Tech Park in Wuxi, China, with 20,000 square feet of the Wuxi GMP facility dedicated to advanced stem cell culturing, centralized plasmid and viral vector production, cell banking and development of reagents;
Began construction of a new GMP facility in "Pharma Valley" in Shanghai Zhangjiang High-Tech Park, which will consist of 40,000 square feet dedicated to advanced cell manufacturing;
Established a strategic research collaboration with GE Healthcare Life Sciences China to co-develop certain high-quality industrial control processes in Chimeric Antigen Receptor T-cell (CAR-T) and stem cell manufacturing and to form a joint laboratory within CBMG’s new Shanghai Zhangjiang GMP-facility dedicated to the joint research and development of a functionally integrated and automated immunotherapy cell preparation system.

Scynexis has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Scynexis, 2017, MAY 8, 2017, View Source [SID1234521707]).

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On May 8, 2017 Acceleron Pharma Inc. (NASDAQ:XLRN) and Celgene Corporation (NASDAQ:CELG), reported preliminary Phase 2 results from the ongoing three-month base and long-term extension studies with investigational drug luspatercept in patients with lower-risk myelodysplastic syndromes (MDS) at the 14th International Symposium on MDS in Valencia, Spain (Press release, Celgene, MAY 6, 2017, View Source [SID1234518896]). Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

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"These positive data presented in lower-risk MDS confirm our optimism in new opportunities for luspatercept beyond our ongoing Phase 3 trials," said Michael Pehl, President, Hematology and Oncology for Celgene. "We are now planning a Phase 3 clinical trial to expand the development of luspatercept into this lower-risk MDS patient population."

"There is a high unmet medical need for a drug to treat patients earlier in the MDS treatment paradigm," said Habib Dable, President and CEO of Acceleron. "We continue to be motivated to find additional opportunities for luspatercept to treat anemia due to rare blood disorders and remain on track to initiate Phase 2 trials in myelofibrosis and non-transfusion dependent beta-thalassemia by year-end."

Luspatercept Phase 2 Data in First-Line, Lower-Risk MDS Patients

In lower-risk, erythropoiesis-stimulating agent (ESA)-naïve MDS patients, 48% (11/23) of patients treated with luspatercept achieved red blood cell transfusion independence (RBC-TI) and 51% (20/39) of patients achieved a clinically meaningful erythroid hematological improvement (HI-E) response per the International Working Group’s (IWG) criteria. The response rates were positive in patients treated with luspatercept in both ESA-naïve and prior ESA-treated patients.

IWG HI-E, N=82
n (%)

RBC-TI, N=56
n (%)
ESA-Naïve 20/39 (51%) 11/23 (48%)
Prior ESA 22/43 (51%) 11/33 (33%)

Luspatercept Phase 2 Data in RS+ and RS- Lower-Risk MDS Patients

In patients with baseline erythropoietin (EPO) levels ≤ 500 international units per liter (IU/L), RBC-TI and IWG HI-E response rates were positive in both ring sideroblast-positive (RS+) and -negative (RS-) patients.

Baseline
EPO (IU/L)
RS Status
IWG HI-E, N=82
n (%)

RBC-TI, N=56
n (%)
≤ 500 RS+ 30/46 (65%) 16/29 (55%)
RS- 6/14 (43%) 4/7 (57%)
> 500 RS+ 5/9 (56%) 2/9 (22%)
RS- 1/11 (9%) 0/9 (0%)
Unknown 0/2 (0%) 0/2 (0%)
*Table includes both ESA refractory and ESA naïve patients. Patients treated at dose levels ≥ 0.75 mg/kg.

Luspatercept Phase 2 Safety Data

The majority of adverse events (AEs) were grade 1 or 2. AEs at least possibly related to study drug that occurred in at least 3 patients during the studies were fatigue, headache, hypertension, diarrhea, arthralgia, bone pain, injection site erythema, myalgia, and edema peripheral. Grade 3 non-serious AEs possibly or probably related to study drug were ascites, blast cell count increase, blood bilirubin increase, hypertension, platelet count increase, and pleural effusion. Grade 3 serious AEs possibly or probably related to study drug were general physical health deterioration and myalgia.

Luspatercept is an investigational product that is not approved for use in any country.

The oral presentation given at the 14th International Symposium on MDS is available on Acceleron’s website (www.acceleronpharma.com) under the Science tab.

Acceleron MDS Symposium Conference Call Information

Acceleron will host a conference call and live webcast to discuss data presented at the MDS Symposium and its first quarter operational and financial results on May 8, 2017, at 8:00 a.m. EDT. To participate by teleconference, please dial 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the Acceleron Earnings Call.

To access the live webcast, please select "Events & Presentations" in the Investors/Media section on Acceleron’s website (www.acceleronpharma.com) at least 10 minutes beforehand to ensure time for any downloads that may be required.

An archived webcast recording will be available on the Acceleron website beginning approximately two hours after the event.

About the MDS Phase 2 Studies

Data from two Phase 2 studies were presented at the conference: the base study in which patients received treatment with luspatercept for three months and the long-term extension study in which patients may receive treatment with luspatercept for up to an additional five years. In both the three-month base study and the long-term extension study, lower-risk MDS patients were enrolled and treated with open-label luspatercept, dosed subcutaneously once every three weeks.

The outcome measures for the studies included the proportion of patients who had an erythroid response (IWG HI-E) or achieved RBC transfusion independence (RBC-TI). IWG HI-E was defined as hemoglobin increase ≥ 1.5 g/dL sustained for ≥ 8 weeks in patients with < 4 units RBC / 8 weeks transfusion burden at baseline and hemoglobin levels below 10 g/dL. For patients with a ≥ 4 units RBC / 8 weeks transfusion burden at baseline, erythroid response was defined as a reduction of ≥ 4 units RBC sustained for ≥ 8 weeks. RBC-TI was defined as no RBC transfusions for ≥ 8 weeks in patients with a ≥ 2 units RBC / 8 weeks baseline transfusion burden.

About Luspatercept

Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the transforming growth factor-beta superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Acceleron and Celgene are enrolling Phase 3 clinical trials that are designed to evaluate the safety and efficacy of luspatercept in patients with myelodysplastic syndromes (the "MEDALIST" study) and in patients with beta-thalassemia (the "BELIEVE" study). For more information, please visit www.clinicaltrials.gov.