On November 6, 2018 CymaBay Therapeutics, Inc. (NASDAQ: CBAY) a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported financial results and a corporate update for the quarter and nine months ended September 30, 2018 (Press release, CymaBay Therapeutics, NOV 6, 2018, View Source [SID1234530759]).

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"We continue to make excellent progress advancing our lead candidate, seladelpar, in two indications — primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH)," said Sujal Shah, President and CEO of CymaBay. "Last week we announced the initiation of ENHANCE, a global Phase 3 registration study of seladelpar for patients with PBC. In addition, 52- and 26-week results from our Phase 2 PBC study will be featured in two late-breaking presentations at The Liver Meeting on November 12. We believe these data support the potential for seladelpar to offer patients with PBC improved efficacy and better tolerability than existing second line treatment while also significantly de-risking the ongoing Phase 3 study. Enrollment in our ongoing Phase 2b study of seladelpar for patients with NASH has been progressing well. We now expect to complete enrollment in the first quarter of 2019, one quarter ahead of our previous guidance. As the only highly selective and potent PPARδ agonist in development for liver disease, we think it may be particularly well suited to treat NASH because of its beneficial effects on lipid, glucose, and sterol metabolism, as well as its effects on inflammation and fibrogenesis. As we approach the end of 2018, our entire team remains committed to these core programs for which we look forward to providing further updates in 2019."

Third Quarter 2018 and Recent Business Highlights

Initiated ENHANCE, a global Phase 3 registration study evaluating 5 and 10 mg of seladelpar versus placebo for the treatment of PBC in patients that are inadequate responders to or are intolerant to ursodeoxycholic acid (UDCA)
The study is designed to support the submission of a global registration dossier with Health Authorities to obtain approval of seladelpar in PBC
Two late-breaking presentations featuring positive data from an ongoing Phase 2 study of seladelpar in PBC will be featured during The Liver Meeting hosted by the American Association for the Study of Liver Diseases (AASLD) in San Francisco, November 9-13, 2018
The 52-week composite responder rates in the ongoing Phase 2 study for the 5 mg / 5 mg to 10 mg titration and 10 mg seladelpar groups were 59% and 71%, respectively
Results suggest that seladelpar is not associated with drug-induced pruritus and may support the hypothesis that seladelpar decreases pruritus in PBC patients
This is the third consecutive year that data on seladelpar in PBC will be highlighted in a late-breaking presentation at The Liver Meeting
Patient recruitment in the placebo-controlled Phase 2b proof-of-concept study investigating seladelpar at three doses in biopsy-proven NASH is one quarter ahead of schedule and is now expected to be fully enrolled in the first quarter of 2019
The primary efficacy outcome is the change from baseline in liver fat content at 12 weeks as measured by magnetic resonance imaging using the proton density fat fraction method (MRI-PDFF)
The study also includes a second biopsy at 52 weeks to examine its activity on NASH and fibrosis
Appointed key individuals to expand and strengthen the development organization to execute the seladelpar Phase 3 program and deliver a high-quality registration package
Dr. Patricia Rohane, M.D., appointed Vice President, Clinical Development
Dr. Stephen Rossi, Pharm. D., appointed Vice President, Early Clinical Development
Kamal Sigel, M.S., appointed Vice President, Quality
Held $198.1 million in cash, cash equivalents and marketable securities at September 30, 2018. Existing cash is expected to fund the current operating plan into 2021.
Third Quarter 2018 Financial Results

Research and development expenses were $17.9 million in the third quarter of 2018 as compared to $4.2 million in the same period of 2017. The increase was primarily driven by increases in seladelpar-related clinical trial expenses from the expansion and extension of our PBC Phase 2 study, start-up activities related to our PBC Phase 3 study, the ongoing enrollment of our NASH Phase 2b study, and the execution of other NDA-enabling studies.
General and administrative expenses were $3.3 million in the third quarter of 2018 as compared to $2.2 million in the same period of 2017. The increase was driven primarily by employee compensation expense as we hired additional personnel to support our expanding operations.
Net loss was $18.6 million, or ($0.34) per diluted share in the third quarter of 2018, as compared to $8.2 million, or ($0.21) per diluted share in the same period of 2017. Net loss was higher primarily due to increased research and development expenses, partially offset by non-cash gains on the revaluation and extinguishment of our warrant liability.
Nine Months Ended September 30, 2018 Financial Results

No collaboration revenue was recognized in the nine months ended September 30, 2018. Collaboration revenue from Kowa Pharmaceuticals America, Inc. totaling $4.8 million was recognized in the same period of 2017.
Research and development expenses were $41.7 million in the nine months ended September 30, 2018 as compared to $12.3 million in the same period of 2017. The increase was primarily driven by increases in seladelpar-related clinical trial expenses from the expansion and extension of our PBC Phase 2 study, start-up activities related to our PBC Phase 3 study, the ongoing enrollment of our NASH Phase 2b study, and the execution of other NDA-enabling studies.
General and administrative expenses were $10.2 million in the nine months ended September 30, 2018, as compared to $9.5 million in the same period of 2017. The increase was driven primarily by higher compensation and consulting expenses, partially offset by decreases in severance and legal fees.
Net loss was $53.1 million, or ($0.93) per diluted share in the nine months ended September 30, 2018, as compared to $22.5 million, or ($0.71) per diluted share in the same period of 2017. Net loss was higher primarily due to increased research and development expenses and lower collaboration revenue.
Conference Call Details
CymaBay management will host a conference call today at 4:30 p.m. ET to discuss third quarter 2018 financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13683385. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

About Seladelpar
Seladelpar is a potent, selective, orally active PPARδ agonist that is in development for the treatment of the liver diseases PBC and NASH. For PBC, seladelpar has received an orphan designation from the US Food and Drug Administration and the European Medicine Agency. Seladelpar also received the PRIority MEdicine (PRIME) status from the European Medicine Agency.

On November 6, 2018 Compass Therapeutics, a biotechnology company committed to the ambitious goal of comprehensively drugging the human immune system, reported that preclinical data on CTX-471, a fully human monoclonal antibody that potently induces immune-mediated destruction of solid tumors, will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2018 Annual Meeting, which is being held this week in Washington, D.C (Press release, Compass Therapeutics, NOV 6, 2018, View Source [SID1234530778]). Preclinical data from the company’s novel NK cell engager platform will also be presented.

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"To our knowledge, CTX-471’s preclinical efficacy as a monotherapy in a very large tumor model is unprecedented for an immuno-oncology antibody. These data support our plans to advance our lead therapeutic candidate into the clinic in the first quarter of 2019," said Thomas Schuetz, M.D., Ph.D., the company’s co-founder and chief executive officer. "In addition, the data on our NK cell engager platform are very exciting. Our preclinical results underscore the power of our StitchMabs technology and our empirical approach to identifying novel combinations of therapeutic candidates that can engage the innate and adaptive immune system in unique and novel ways to eradicate tumors."

CTX-471, a CD137 Agonist

CTX-471 is a fully human monoclonal antibody that binds and stimulates a novel epitope on CD137, also known as 4-1BB — a member of the tumor necrosis factor receptor superfamily. Targeting of this unique epitope, combined with other characteristics of this antibody, give CTX-471 a very different profile compared to other antibodies targeting CD137.

IND-enabling studies for CTX-471 are complete and a Phase 1 clinical trial is planned for Q1 2019. Preclinical data to be presented at SITC (Free SITC Whitepaper) suggest that CTX-471 has the potential to become a best-in-class CD137 agonist with strong efficacy both as a monotherapy and in combination with tumor antigen-targeted antibodies and checkpoint inhibitors.

Compass will present data showing that CTX-471 potently induces immune-mediated tumor killing, including complete responses that are associated with the generation of long-term, functional immunological memory in multiple in vivo models and across a broad range of doses. Compass will also present data supporting the future clinical testing of CTX-471 in combination with multiple different therapeutics, including tumor-targeted antibodies and checkpoint inhibitors. These data sets will be presented in two posters:

Poster #407, titled "CTX-471, a novel agonistic antibody targeting CD137, eradicates very large tumors in vivo by selectively reprogramming the tumor microenvironment without causing hepatic toxicity," on display in Hall E. Presentation hours are Friday, Nov. 9, from 12:45 – 2:15 p.m. and 6:30 – 8 p.m.

Poster #386, titled "CTX-471, a novel agonistic antibody targeting CD137, enhances the anti-tumor activity of tumor antigen-targeted antibodies and immune checkpoint inhibitors when used in combination," on display in Hall E. Presentation hours are Saturday, Nov. 10 from 12:20 – 1:50 p.m. and 7:00 – 8:30 p.m.

NK Cell Engager Platform

Compass will present data on a novel class of antibodies targeting NK cells, which lowers the threshold for NK cell activation and induces potent tumor cell killing in vitro. As an example, CTX-4419, a first-in-class NKp30 x BCMA bispecific, induces NK cell activation and potent killing of tumor cells expressing high, medium and even low levels of antigen. The data further show that CTX-4419 activates NK cells only in the presence of tumor cells and does not require NK cell priming.

"We are excited about the potential applications of this platform. In addition to its activity across a broad range of antigen expression levels, CTX-4419 productively engages, but does not require, CD16A activation for its superior activity," said Piotr Bobrowicz, Compass’s chief scientific officer. "This unique characteristic is expected to overcome the reduction or loss of activity connected with CD16A polymorphism, receptor shedding or downregulation in the tumor microenvironment that can render CD16A-based therapeutic approaches ineffective."

The data will be presented in Poster #P530, titled "A novel class of NK cell engagers overcomes CD16A deficiency," which will be on display in Hall E on Saturday, Nov. 10 from 12:20 – 1:50 p.m. and 7:00 – 8:30 p.m

On November 6, 2018 QIMR Berghofer Medical Research Institute has reported its collaboration with US biopharmaceutical company Atara Biotherapeutics, entering into major agreements to manufacture and develop cellular immunotherapies for multiple sclerosis and some cancers (Press release, QIMR Berghofer Medical Research Institute, NOV 6, 2018, View Source [SID1234530846]).

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QIMR Berghofer secured the deal thanks to a $1.4 million funding injection from the Queensland Government to expand and upgrade the Institute’s world-class, regulatory-approved cell therapy manufacturing facility, Q-Gen Cell Therapeutics.

The deal with Atara will allow world-first clinical trials to be established locally and in the United States and Europe, giving Queenslanders access to cutting-edge medical treatments.

Under the agreement, Q-Gen will manufacture the immunotherapies for the clinical trials, including those using patients’ own immune cells and others using cells from healthy donors.

QIMR Berghofer and Atara have also extended an existing agreement to collaborate on research and development of new T cell immunotherapies for cancers and autoimmune disorders associated with a number of viruses.

Queensland’s Minister for State Development, Manufacturing, Infrastructure and Planning, Cameron Dick, said the two agreements would support approximately 40 full-time jobs at QIMR Berghofer and Q-Gen.

"Through our government’s support, QIMR Berghofer can now expand its cell therapy facility Q-Gen Cell Therapeutics, including purchasing specialised laboratory equipment," he said.

"As a result of this facility upgrade, Atara has agreed to extend its agreement with QIMR Berghofer to collaborate on research and development of new immunotherapies for cancers and autoimmune disorders associated with a number of viruses.

"The two agreements will support 40 full-time jobs at QIMR Berghofer and its Q-Gen facility at Herston as well as grow QIMR Berghofer’s reputation as a world-leader in the immunotherapy field.

"Our government’s investment will strengthen our state’s push into the sophisticated advanced manufacturing of cell therapy products by enabling QIMR Berghofer to secure agreements and attract additional cell therapy manufacturing from not only Atara, but also other biomedical and pharmaceutical companies.

"Projects like this underpin the Queensland Biomedical 10-Year Roadmap and Action Plan, which was developed in close consultation with industry to diversify Queensland’s industry base, create high-value knowledge-based jobs of the future and drive export growth for the industry by 2027."

The Minister for Health Steven Miles said immunotherapy had emerged as the "fourth pillar" of cancer treatment, along with surgery, chemotherapy and radiotherapy.

"Immunotherapy works by training the immune system to recognise and destroy cancer and other harmful cells," he said.

"These agreements put Queensland’s biomedical capabilities on the world stage."

Atara has also exercised its option under an existing licence agreement with QIMR Berghofer to an exclusive, worldwide licence to develop and commercialise a specific T cell immunotherapy that "turbo charges" a patient’s immune cells to treat autoimmune conditions associated with the Epstein-Barr virus, such as multiple sclerosis.

All of the immunotherapies were developed by the head of QIMR Berghofer’s Tumour Immunology Laboratory, Professor Rajiv Khanna AO, and his team.

Professor Khanna said the agreements with Atara were a win for Queensland patients.

"We are delighted to partner with Atara and the Queensland Government to bring much-needed new T cell immunotherapies to patients," he said.

"We are very excited by the possibility that in future, we might be able to offer new treatment options to patients with certain virus-associated cancers and autoimmune conditions like multiple sclerosis."

QIMR Berghofer’s Director and CEO, Professor Frank Gannon, said the Queensland Government’s funding was a major boost for advanced manufacturing in the state.

"Q-Gen is the one of the largest dedicated cell therapy manufacturing facilities in Australia," he said.

"It is already a world-class facility that has secured regulatory approval to manufacture immunotherapies for clinical trials in Australia and the United States. It will now be upgraded to also manufacture for clinical trials in Europe and to meet the demand created by this deal with Atara.

"The Queensland Government’s support will allow us to expand our cell therapy manufacturing program, providing a major boost for Queensland’s biotech sector."

Atara’s Global Head of Research and Development, Dr Dietmar Berger, said he was excited about the collaboration with QIMR Berghofer and increasing biotechnology development in the region.

"Atara is delighted that the Queensland Government is supporting QIMR Berghofer, as well as the growth of the biotechnology community in Queensland," he said.

On November 6, 2018 Oncolytics Biotech Inc. (Nasdaq: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported that it will host a conference call and live webcast for Analysts and Institutional Investors at 8:30 a.m. ET on Monday, November 12, 2018 following release of its third quarter 2018 financial results (Press release, Oncolytics Biotech, NOV 6, 2018, View Source [SID1234530862]).

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The live call may be accessed by dialing (888) 231-8191 for callers in North America. Overseas callers should contact investor relations for the toll-free dial information for their country. A replay of this call will be available approximately two hours after the call is ended at 855-859-2056, using the replay code 6463668 and will be available for six months.

A live webcast of the call will be accessible on the Investor Relations page of Oncolytics’ website at www.oncolyticsbiotech.com and will be archived for six months

On November 6, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the third quarter ended September 30, 2018, and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, NOV 6, 2018, View Source [SID1234530879]).

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"We made significant progress during the quarter across our programs. Rivo-cel remains on track for MAA filing in the E.U. in 2019 for pediatric patients with acute leukemias and nonmalignant blood diseases," said Bellicum’s President & CEO Rick Fair. "We also received and incorporated health authority input on our planned late-stage trial in adult AML and MDS and remain on track to initiate the trial by year-end."
Continued Mr. Fair: "In our GoCAR-T programs, we are nearing completion of the dose escalation portion of our Phase 1/2 study of BPX-601 in solid tumors, and expect to report preliminary results from the lower dose cohorts in patients with advanced pancreatic cancer in December. We also made substantial progress toward IND applications for two new dual-switch GoCAR-T candidates in 2019."
PROGRAM HIGHLIGHTS AND CURRENT UPDATES
On Track to Initiate Phase 2/3 Study of Rivo-cel in Adult AML and MDS by Year-end
Based on impressive clinical trial results to date with rivo-celTM (rivogenlecleucel, formerly called BPX-501) in pediatric leukemia patients, Bellicum is finalizing its plans to initiate a global Phase 2/3 trial in adult patients with intermediate/high-risk acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) by the end of the year. The Company completed review of the study protocol with the U.S. FDA in the third quarter and has incorporated its input into the design of the trial.

Rivo-cel Pediatric Registration Trials on Track for E.U. Filing in 2019; Significant Data Update at ASH (Free ASH Whitepaper) 2018
Prospective enrollment was recently completed in the BP-004 and C-004 E.U. registration trials of pediatric patients with leukemias, lymphomas and inherited blood disorders. These trials will serve as the basis for the Company’s planned 2019 European Marketing Authorization Application regulatory filing. In December, Bellicum will present interim data at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual meeting, with final results expected in early 2019. Among the highlights will be late interim analyses of the overall results from the BP-004 trial in children with acute leukemias and nonmalignant blood diseases, as well as the comparator C-004 trial-a multicenter, observational study of similar pediatric patients receiving a matched unrelated donor (MUD) transplant. Disease outcomes from several patient subsets, as well as the cumulative clinical experience of patients from BP-004 who received rimiducid to treat steroid refractory Graft-versus-Host-Disease will also be presented. ASH (Free ASH Whitepaper) 2018 is being held in San Diego, California on December 1-4.

Commercial Planning Activities for Rivo-cel Continue to Advance in Europe
Under the recently appointed General Manager of Europe, Thierry Darcis, M.D., M.B.A., Bellicum continues to build out an E.U.-based team to prepare for the commercialization of rivo-cel, if approved. Dr. Darcis and his leadership team have extensive experience launching orphan products in Europe, and Dr.

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Exhibit 99.1

Darcis has previously led successful product introductions for ViroPharma and NPS Pharmaceuticals. He also held leadership roles with Zogenix, Novartis and GlaxoSmithKline.

Initial Clinical Data on BPX-601 To Be Presented at ESMO (Free ESMO Whitepaper) Immuno-Oncology Conference
BPX-601 is Bellicum’s first GoCAR-T clinical candidate incorporating the co-activation domain, iMC. The Company expects to report preliminary safety and translational findings from the lower cell-dose cohorts of its Phase 1 dose-escalation safety study in late-stage pancreatic cancer patients at the European Society for Medical Oncology Immuno-Oncology Conference in Geneva, Switzerland in December. The Company is evaluating BPX-601 in adults with relapsed or refractory pancreatic, gastric, and prostate cancers who test positive for prostate stem cell antigen (PSCA).

Preclinical Dual-Switch Candidates On Track to Enter Clinic in 2019
Bellicum’s research team continues to advance its next-generation GoCAR-T projects, which have been designed with both activation and safety switch technologies to potentially enhance efficacy and safety. The Company expects to submit IND applications for two new dual-switch GoCAR-T product candidates in 2019.

Third Quarter 2018 Financial Results
Bellicum reported a net loss of $23.8 million for the third quarter of 2018 and $70.8 million for the nine months ended September 30, 2018, respectively, compared to a net loss of $23.4 million and $69.9 million for the comparable periods of 2017. The results included non-cash, share-based compensation charges of $3.7 million and $10.9 million for the third quarter and nine months ended September 30, 2018, and $3.7 million and $10.2 million for the comparable periods in 2017.
As of September 30, 2018, cash, restricted cash and investments totaled $118.4 million. Based on current operating plans, Bellicum continues to expect that current cash resources will be sufficient to meet operating requirements through 2019.
Research and development expenses were $16.4 million and $51.4 million, for the three and nine months ended September 30, 2018, respectively, compared to $18.1 million and $51.4 million during the comparable periods in 2017.
General and administrative expenses were $7.0 million and $18.0 million for the three and nine months ended September 30, 2018, respectively, compared to $4.6 million and $16.0 million during the comparable periods in 2017.
At September 30, 2018, Bellicum had 43,351,159 shares of common stock outstanding.

About Rivo-cel (BPX-501)
Rivo-celTM (rivogenlecleucel) is an allogeneic polyclonal T cell product designed to reduce relapse of leukemia following a stem cell transplant. The cell treatment contains a diverse repertoire of T cells which may contribute to a robust graft vs. leukemia effect. Rivo-cel’s antiviral benefits may also reduce morbidity and mortality in patients susceptible to infection following a transplant. The product’s CaspaCIDe safety switch enables this approach by allowing physicians to reduce the number of alloreactive cells in the event of uncontrolled GvHD. Rivo-cel addresses a major unmet need in adult and pediatric leukemia, lymphoma and genetic blood disease patients following a haploidentical stem cell transplant.
About BPX-601
BPX-601, the Company’s first GoCAR-T product candidate, incorporates iMC, Bellicum’s inducible co-activation domain. iMC (inducible MyD88/CD40) is designed to provide a powerful boost to T cell proliferation and persistence, and enable the CAR-T to override key immune inhibitory mechanisms,

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Exhibit 99.1

including PD-1 and TGF-beta. BPX-601 is being evaluated as a treatment for solid tumors expressing prostate stem cell antigen (PSCA), including pancreatic, prostate and gastric cancers.