On May 11, 2020 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 corporate updates and financial results for the first quarter ended March 31, 2020 (Press release, CymaBay Therapeutics, MAY 11, 2020, View Source [SID1234557487]).

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Sujal Shah, President and CEO of CymaBay, stated, "Last week a panel of some of the most experienced and distinguished liver pathologists and hepatologists in the world completed an independent review analyzing findings from our Phase 2b study of seladelpar in patients with NASH. I am pleased to report that the panel unanimously concluded there was no clinical, biochemical or histological evidence of seladelpar-induced liver injury in the study, and as a result they also unanimously supported the lifting of the clinical hold and re-initiation of clinical development.

While we have not yet discussed full results from our investigation nor any of the panel’s conclusions with the FDA, we are planning to re-engage with the agency as quickly as possible. At this point we cannot guarantee what the next steps or timelines will be, but we are confident that we have conducted a truly rigorous, independent review to help us definitively support the conclusion that seladelpar did not cause drug-induced liver injury in our NASH phase 2b study."

Dr. Paul Watkins, Howard Q Ferguson Distinguished Professor, Schools of Medicine, Pharmacy, and Public Health, Director, Institute for Drug Safety Sciences at the University of North Carolina, Chapel Hill, said, "I was pleased to chair this esteemed independent panel of liver experts. The panel conducted a comprehensive, systematic review and discussion of all of the clinicopathological data from the seladelpar NASH Phase 2b study. In my experience, no other drug in development for NASH has been through such rigorous scrutiny of safety data at this stage of development. As we have stated, the features noted by study pathologists at end of treatment were confirmed on this review. However, these did not differ qualitatively between baseline and end of treatment. We suspect these histologic features are underreported; however, in the experience of the pathology review subcommittee, these features may be observed in patients with NASH. The panel unanimously concluded that the data in aggregate, including the lack of significant differences in histologic features or their changes across the placebo and treatment groups, do not support injury related to seladelpar."

Dr. Stephen Harrison, Medical Director, Pinnacle Clinical Research, Visiting Professor of Hepatology at Radcliffe Department of Medicine, University of Oxford, and principal investigator of the seladelpar Phase 2b study in NASH, added, "I believe CymaBay and the FDA did the right thing in putting patient safety first when development of seladelpar was halted at the end of last year until an in-depth investigation was conducted into the findings identified by study pathologists in the NASH study. At this point, the findings and additional data collected have been thoroughly investigated by leading experts in the areas of drug-induced liver injury and hepatopathology. Given the benefit observed on both NASH resolution and fibrosis with seladelpar in the NASH Phase 2b study as well as data presented at multiple medical meetings from studies of seladelpar in PBC, I am pleased that the independent review panel is supportive of restarting clinical development pending approval from the FDA."

Recent Corporate Highlights

At the end of last week, a panel of eight of the world’s foremost expert liver pathologists and hepatologists, whose collective experience relevant to CymaBay’s investigation includes drug-induced liver injury, NASH and cholestatic liver diseases, completed a four-day independent review analyzing findings from CymaBay’s NASH Phase 2b study. The panel unanimously supported lifting the clinical hold for seladelpar and re-initiation of clinical development. In addition to the chair, Dr. Paul Watkins, the panel included:
° Pierre Bedossa, MD, PhD, Professor of Pathology at the University Paris-Diderot, France, and Medical Director and CEO of LIVERPAT
° Michael Charlton, MD, Chief of Hepatology, Director of the Center for Liver Diseases and Medical Director of the Transplant Institute at the University of Chicago
° Zachary Goodman, MD, PhD, Director of Hepatic Pathology Consultation and Research, Center for Liver Disease, Inova Healthcare Services
° Neil Kaplowitz, MD, Professor of Medicine and Thomas H. Brem Chair in Medicine, Budnick Chair of Liver Disease, Keck School of Medicine, University of Southern California
° David Kleiner, MD, PhD, Head of Histopathology and Autopsy Pathology at the NIH and the Reference Pathologist for the Drug-Induced Liver Injury Network
° Willis Maddrey, MD, Professor Emeritus of Internal Medicine at The University of Texas Southwestern Medical Center
° John Vierling, MD, Professor of Medicine and Surgery, Baylor College of Medicine

CymaBay intends to reach out to the FDA to discuss all of the data it has collected to date and the results of the panel review meetings. Once initial feedback is gathered, CymaBay intends to submit a complete response to the seladelpar clinical hold to the FDA. The CymaBay Board of Directors has worked closely with management throughout the investigation and panel review and is in support of next steps to re-engage with the FDA.

As a reminder, during the fourth quarter of 2019, management implemented a restructuring program following the placement of the seladelpar program on clinical hold pending further investigation of the histologic observations noted by study pathologists in CymaBay’s Phase 2b NASH study and pending completion of its review of strategic options.

Late in the first quarter of 2020, the need for sustained cost containment was further underscored by the unexpected and rapid onset of the coronavirus pandemic and the associated travel restrictions and shelter-in-place orders issued by governmental authorities in jurisdictions where CymaBay, its partners, investigators, and vendors, conduct operations. In response to these measures, CymaBay has taken steps, such as enabling remote operations for all employees, which have allowed operating activities to continue as seamlessly as possible.

CymaBay will continue to closely monitor pandemic developments and their associated risks to the business, and will take actions available to mitigate them where possible. Further, all of CymaBay’s actions will be guided by a commitment to taking all steps possible to ensure the health and safety of its employees.

Held $176.2 million in cash, cash equivalents and short-term investments at March 31, 2020.
Mr. Shah continued, "As the next steps in our seladelpar investigation process become clear we will continue to keep our shareholders updated as appropriate, while also continuing to evaluate potential strategic alternatives. Further, we remain focused on cost containment and will look at additional steps we can take into fiscal year 2020 in order to closely control the Company’s operating expenses and associated cash burn."

First Quarter Ended March 31, 2020 Financial Results

Research and development expenses for the three months ended March 31, 2020 were $9.5 million, compared to $18.6 million for the three months ended March 31, 2019. Research and development expense in the first quarter of 2020 was significantly lower than the corresponding period in 2019 primarily due to declining clinical trial activities related to the Phase 3 PBC, Phase 2b NASH, and Phase 2 PSC clinical trials, and other studies, as efforts continue to scale back and shut down these studies as a result of the clinical hold on the seladelpar development program.

General and administrative expenses for the three months ended March 31, 2020 were $4.3 million, compared to $5.7 million for the three months ended March 31, 2019. General and administrative expenses in the first quarter of 2020 was lower than the corresponding period in 2019 due to lower continuing labor costs and other administrative expenses following restructuring efforts undertaken in the fourth quarter of 2019.

Net loss for the three months ended March 31, 2020 was $13.1 million, or ($0.19) per diluted share, compared to a net loss of $23.1 million, or ($0.37) per diluted share in the three months ended March 31, 2019. Net loss was lower in the first quarter of 2020 compared to the corresponding period in 2019 primarily due to a decrease in operating expenses, including clinical trial and labor related expenses.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss first quarter 2020 financial results and provide a corporate update. To access the live conference call, please dial 855-327-6837 from the U.S. and Canada, or 631-891-4304 internationally, Conference ID# 10009543. 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

On May 11, 2020 Unum Therapeutics Inc. (NASDAQ: UMRX), a biopharmaceutical company focused on developing curative cell therapies for solid tumors, reported financial results for the first quarter ended March 31, 2020, and provided corporate updates (Press release, Unum Therapeutics, MAY 11, 2020, View Source [SID1234557514]).

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Recent Program and Corporate Highlights

Announced plans to prioritize resources towards advancing its preclinical program, BOXR1030, for the treatment of solid tumor cancers: On March 2nd, Unum announced a corporate restructuring plan to prioritize resources towards advancing its preclinical program, BOXR1030, for the treatment of solid tumor cancers. Unum’s BOXR1030 expresses a glypican-3 (GPC3) targeted CAR and incorporates the novel transgene glutamic-oxaloacetic transaminase 2 (GOT2) to improve T cell function in the solid tumor microenvironment by enhancing T cell metabolism. Unum has initiated formal preclinical development activities, including preclinical safety testing and cGMP manufacturing readiness activities, to support filing an investigational new drug (IND) application for BOXR1030 in late 2020.

Entered into a common stock purchase agreement for up to $25 million with Lincoln Park Capital Fund, LLC ("LPC"): Under the terms of the purchase agreement announced on March 20th, Unum Therapeutics will have the sole discretion to direct LPC to purchase up to $25 million in shares of its common stock over the 36-month term of the agreement based on the market prices prevailing at the time of each sale to LPC. Unum Therapeutics controls the timing and amount of any future sales of its stock, subject to various limitations including those under the NASDAQ listing rules, and there is no upper limit as to the price per share that LPC may pay for future stock issuances under the purchase agreement. LPC has agreed not to cause or engage in any direct or indirect short selling or hedging of Unum Therapeutics’ common stock. Unum Therapeutics maintains the right to terminate the common stock purchase agreement at any time, at its discretion, without any additional cost or penalty.

Exploring strategic options to maximize shareholder value. Following a review of its business, the Company recently initiated and continues a process to explore strategic alternatives focused on maximizing shareholder value, with Ladenburg Thalmann & Co. Inc. acting as Unum Therapeutics’ strategic financial advisor during this process. Potential strategic alternatives that may be evaluated include, but are not limited to, an acquisition, merger, business combination, in-licensing, or other strategic transaction. There can be no assurance that this process will result in any such transaction. Unum Therapeutics has not set a timetable for completion of this review process and does not intend to

comment further unless or until the Board of Directors has approved a definitive course of action, the review process is concluded, or it is determined that other disclosure is appropriate.

First Quarter 2020 Financial Results

Collaboration Revenue: Collaboration revenue recognized during the first quarter ended March 31, 2020 of $7.0 million compared to $3.1 million in the same period of 2019. Collaboration revenue includes the recognition of a portion of the upfront payment received from Seattle Genetics, Inc. as reimbursements for research and development costs, and increased during the quarter ended March 31, 2020 as a result of the conclusion of the ATTCK-17-01 Phase 1 clinical trial and the termination of the collaboration agreement.

R&D Expenses: Research and development expenses of $9.5 million for the first quarter ended March 31, 2020 compared to $12.4 million for the same period of 2019. Research and development expenses relate to ongoing costs for Phase 1 trials and the BOXR1030 preclinical program, as well as personnel-related costs to support these programs.

G&A Expenses: General and administrative expenses for the first quarter ended March 31, 2020 were $3.7 million, compared to $2.5 million for the same period of 2019. The increase is primarily related to increased personnel-related costs, including severance costs, as well as expenses required to operate as a public company.

Net Loss: Net loss attributable to common stockholders was $6.1 million, or $0.20 per share, for the first quarter ended March 31, 2020 compared with a net loss attributable to common stockholders of $11.7 million, or $0.39 per share, for the same period of 2019.

Cash and Cash Equivalents: As of March 31, 2020, Unum had cash and cash equivalents of $29.6 million. Unum believes that its existing cash and cash equivalents will fund operating expenses and capital expenditure requirements into mid-2021.

About Unum’s BOXR1030 and BOXR Platform

Unum’s BOXR1030 was discovered from its Bolt-on Chimeric (BOXR) platform that is designed to discover novel "bolt-on" transgenes to be co-expressed with CARs, a T-cell receptor, or ACTR, to help T cells survive longer and perform better in the solid tumor microenvironment. BOXR candidates consist of two main components: 1) a targeting receptor that directs the T cell to attack tumor cells, which may be a traditional CAR receptor, a T-cell receptor, or Unum’s ACTR receptor, and 2) a novel "bolt-on" transgene that improves the intrinsic function of the T cell. Once discovered, BOXR transgenes are designed to be incorporated into

several different types of therapeutic T cells, including both ACTR T cells and CAR-T cells, to impart new functionality to T cells.

Unum’s first product candidate selected from the BOXR platform, BOXR1030, expresses GPC3+ targeted CAR and incorporates the bolt-on GOT2 transgene to improve T cell function in the solid tumor microenvironment (TME) by enhancing T cell metabolism. Preclinical data with BOXR1030 was presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November 2019. In preclinical studies, BOXR1030 T cells were resistant to suppressive TME-like conditions, showing improved T cell proliferation under both hypoxic and low glucose conditions compared with control GPC3+ CAR-T cells. In vivo, BOXR1030 demonstrated superior activity compared to the parental CAR-T with treated animals achieving complete tumor regressions. Tumor infiltrating lymphocytes isolated from the tumors of treated animals revealed that BOXR1030 cells were more resistant to dysfunction and had fewer markers of exhaustion as compared to the control CAR-T cells.

On May 11, 2020 Bionical Emas and Jazz Pharmaceuticals (NASDAQ: JAZZ) reported that they have entered into an agreement to provide appropriate patients in the United States (U.S.) who have relapsed Small Cell Lung Cancer (SCLC) lurbinectedin via an Expanded Access Program (EAP) (Press release, Bionical Emas, MAY 11, 2020, View Source [SID1234557531]). Lurbinectedin is an investigational drug under review by the U.S. Food and Drug Administration (FDA).

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The EAP is open to patients who are unable to enter clinical trials and for whom there are no appropriate alternative treatments while lurbinectedin is under regulatory review by the FDA. The EAP was originally launched in January 2020 between Bionical Emas and PharmaMar S.A. (PharmaMar), and now has successfully transitioned from PharmaMar to Jazz.

SCLC is an aggressive form of cancer that usually is diagnosed with advanced, often metastatic, disease, and often posing a worse prognosis when compared to other lung cancers.1 In the United States, approximately 10-15% of lung cancers are small cell.1 Approximately 30,000 new cases of SCLC are recorded in the U.S. every year.2

Lurbinectedin is a selective inhibitor of oncogenic transcription on which many cancers such as SCLC are particularly dependent.

"We are pleased to be working with Jazz on this important project allowing access to lurbinectedin to appropriate patients via an Expanded Access Program in the U.S.," said Tom Watson, Executive Vice President, Bionical Emas.

"Lurbinectedin provides further hope for patients suffering from relapsed SCLC who currently have limited treatment options. This EAP provides an important opportunity for those patients who are unable to enter clinical trials and for whom there are no appropriate alternative treatments, and we are pleased to support this program," said John Efthimiou, Chief Medical Officer, Bionical Emas.

A new drug application (NDA) for Lurbinectedin is under review by the FDA and has not yet been approved.

Healthcare professionals wishing to request access to lurbinectedin under the EAP or who would like to find out more should do so by emailing [email protected]. Further details concerning the EAP can be found on Clinicaltrials.gov.

On May 10, 2020 Hummingbird Bioscience, an innovative biotherapeutics company focused on the discovery and development of new breakthrough therapies, reported that it has closed an extended Series B funding round of US$25 million (Press release, Hummingbird Bioscience, MAY 10, 2020, View Source [SID1234557436]). This brings the total capital raised through financing activities and strategic partnerships to more than US$65 million to date.

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Leading the Series B extension is new investor SK Holdings, with participation from existing shareholders including Heritas Capital and SEEDS Capital, the investment arm of Enterprise Singapore.

The Series B round was extended to US$25 million due to significant over-subscription, with the addition of select quality and value-add investors. Hummingbird will use the new funds to accelerate development of new candidates into clinical trials and strengthen its scientific and research and development capabilities.

"We are delighted to have SK Holdings join our investor base. Hummingbird is building a strong portfolio of promising new therapies that we believe can deliver very meaningful benefit for patients across a broad spectrum of disease. These new funds give us further resources to develop our early stage pipeline, and support the clinical development of our lead programs," said Dr Piers Ingram, Chief Executive Officer and co-founder, Hummingbird Bioscience.

"Heritas Capital is pleased to continue our backing of the Hummingbird team since leading its Series A extended round," commented Chik Wai Chiew, Executive Director and Chief Executive Officer, Heritas Capital Management. "Even as the COVID-19 pandemic has resulted in a slow-down in investing, we are mindful that backing leading innovative biotech companies, especially players such as Hummingbird, to develop cures for addressing patients’ needs remains our priority."

Earlier this year Hummingbird announced publication of positive data on its lead candidate, HMBD-001, a first-in-class HER3 antibody; and manufacturing of HMBD-002, a first-in-class VISTA antibody. Regulatory submissions to initiate Phase I studies for these two candidates are expected in the second half of 2020.

On May 10, 2020 Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that latest clinical data of the company’s drug candidates, including the MDM2-p53 inhibitor APG-115, novel Bcl-2/Bcl-xL dual inhibitor APG-1252, and IAP inhibitor APG-1387, will be presented in three poster discussions and a poster at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Ascentage Pharma, MAY 10, 2020, View Source [SID1234557437]). Because of concerns about the current coronavirus (COVID-19) pandemic, this year’s annual meeting will be held in a virtual format from May 29 through May 31, 2020.

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The ASCO (Free ASCO Whitepaper) Annual Meeting is the most authoritative meeting in the oncology space. The purpose of this meeting is to disseminate premier scientific research and cutting-edge treatment approaches in oncology to the world.

"We are excited to present the clinical advances of three of our drug candidates," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "Through these results, Ascentage Pharma is demonstrating its capabilities of global clinical development in apoptosis. We are striving to offer more treatment options for patients with unmet medical needs."

Four posters will be presented at ASCO (Free ASCO Whitepaper)20, including:

Poster Discussion Sessions

Phase Ib study of a novel, small-molecule MDM2 inhibitor APG-115 combined with pembrolizumab in U.S. patients with metastatic solid tumors
Time: May 29th, 8:00–11:00am, EST

Abstract: #3512

Session: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

First-in-human study of palcitoclax (APG-1252), a novel dual Bcl-2/Bcl-xL inhibitor, demonstrated advantages in platelet safety while maintaining anticancer effect in U.S. patients with metastatic solid tumors
Time: May 29th, 8:00–11:00am, EST

Abstract: #3509

Session: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Phase Ib study of a novel bivalent IAP antagonist APG-1387 in combination of pembrolizumab for patients with advanced solid tumors
Time: May 29th, 8:00–11:00am, EST

Abstract: #3508

Session: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Poster Session

Phase I study results of APG-115, a MDM2-p53 antagonist, in Chinese patients with advanced liposarcoma and other solid tumors
Time: May 29th, 8:00–11:00am, EST

Abstract: #11542

Session: Sarcoma