On August 6, 2019 Genprex, Inc. (NASDAQ: GNPX), a clinical-stage gene therapy company, reported that its manufacturing partner, Aldevron, has successfully completed the manufacturing of the TUSC2 (Tumor Suppressor Candidate 2) plasmid DNA pursuant to the manufacturing agreement between Genprex and Aldevron that was announced in September 2018 (Press release, Genprex, AUG 6, 2019, View Source [SID1234538218]).

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Genprex’s initial product candidate, Oncoprex consists of a TUSC2 plasmid DNA manufactured by Aldevron encapsulated in a nanovesicle made from lipid molecules with a positive electrical charge. Historically, the manufacturing steps of combining the plasmid DNA with the lipid nanovesicles of Oncoprex, has been done for Genprex at MD Anderson Cancer Center. Since Genprex’s IPO, the company has been working to transfer and scale up these processes through other contract manufacturers.

"Each successful release of plasmid DNA batches is an important milestone for Genprex as it allows us to not only demonstrate continued progress in coordinating scale-up manufacturing of Oncoprex, but it takes us one step closer to re-entering the clinic with both our EGFR program and our immunotherapy combination programs. Biologics manufacturing, especially for lipid nanoparticle (or LNP) delivered gene therapy, is a complex process, but one we tackle with pride alongside dedicated partners such as Aldevron, through a similar vision to deliver innovative therapies to patients with significant unmet medical needs," said Julien Pham, MD, Genprex’s President and Chief Operating Officer.

The U.S. Food and Drug Administration (FDA) has made manufacturing a top priority for cell and gene therapy companies. The FDA’s former Commissioner, Scott Gottlieb, noted in 2018 that the agency devotes approximately 80 percent of its focus during reviews of gene therapy candidates to manufacturing and quality concerns. FDA’s Director of the Center for Drug Evaluation and Research, Janet Woodcock, M.D., noted in July 2019 that the agency is adapting to the flood of new drug applications stemming from advances in gene therapy and is shifting its stance toward drug development more than in recent years. This shift emphasizes the need for gene therapy companies to not only demonstrate clinical efficacy but also be able to manufacture these drugs on a large scale.

Genprex has made manufacturing one of its top priorities since its IPO in 2018. During that time, Genprex has been building out its manufacturing process development to support this expansion and advance its commercial scaling capabilities.

Earlier this year, Genprex strengthened its senior team with the appointment of Eric Chapdelaine as Senior Director of Pharmaceutical Sciences and Manufacturing to support the company’s manufacturing, technical operations, and supply chain management.

On August 6, 2019 Seres Therapeutics, Inc. (Nasdaq: MCRB) reported second quarter 2019 financial results and provided business updates (Press release, Seres Therapeutics, AUG 6, 2019, https:// View Source [SID1234538217]).

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"We remain focused on the execution of our clinical programs and we look forward to a data-rich 2020 with four significant expected milestones including: SER-287 Phase 2b readout in mild-to-moderate ulcerative colitis; SER-109 Phase 3 readout in recurrent C. difficile infection; SER-401 Phase 1b readout in metastatic melanoma; and the advancement of our rationally-designed, fermented SER-301 program into clinical development for ulcerative colitis," said Eric D. Shaff, President and Chief Executive Officer at Seres. "We are also pleased to have strengthened Seres’ balance sheet through a public equity offering, giving us the resources needed to operate the Company into 2021 and to reach multiple important value inflection points," continued Mr. Shaff.

Program Updates and Corporate Highlights

SER-287 Phase 2b ECO-RESET study in ulcerative colitis: SER-287 is an oral, donor-derived microbiome therapeutic candidate designed to normalize the gastrointestinal microbiome of individuals with ulcerative colitis. Seres continues to enroll the SER-287 Phase 2b ECO-RESET induction study in patients with active mild-to-moderate ulcerative colitis. The SER-287 Phase 2b ECO-RESET study was initiated in December 2018 and is expected to enroll approximately 201 patients with mild-to-moderate ulcerative colitis. Based on FDA feedback, Seres expects that with positive Phase 2b study results, the study could serve as one of two pivotal trials to enable a SER-287 Biologics License Application (BLA) submission. Seres expects to report Phase 2b ECO-RESET study top line results in the third quarter of 2020.
SER-109 Phase 3 ECOSPOR III study in recurrent C. difficile infection: SER-109 is an oral, donor-derived microbiome therapeutic candidate designed to restore the depleted, or dysbiotic, gastrointestinal microbiome of patients with recurrent C. difficile infection. The Company continues to enroll the ECOSPOR III trial. ECOSPOR III is designed to evaluate efficacy and safety in 188 patients with recurrent C. difficile infection. All patients enrolled in ECOSPOR III are required to test positive for C. difficile cytotoxin to ensure enrollment of only patients with an active C. difficile infection. Seres expects to report SER-109 ECOSPOR III top line study results in early 2020.
SER-401 Phase 1b in metastatic melanoma: SER-401 is an oral, donor-derived microbiome therapeutic candidate comprising a bacterial signature similar to that observed in checkpoint inhibitor immunotherapy responders. The ongoing Phase 1b study, supported by the Parker Institute for Cancer Immunotherapy and The University of Texas MD Anderson Cancer Center, will evaluate the potential for SER-401 to augment response to nivolumab, an approved anti-PD-1 checkpoint inhibitor therapy, and will assess a variety of biological measures of response. Seres expects to obtain SER-401 Phase 1b preliminary study results in the second half of 2020.
SER-301 preclinical candidate: Seres also continues to advance its rationally-designed, fermented microbiome drug discovery and development capabilities. These efforts are focused on advancing SER-301, a preclinical therapeutic candidate for ulcerative colitis, into clinical development. The Company is entitled to a $10 million milestone payment associated with the initiation of SER-301 clinical development from its ongoing collaboration with Nestlé Health Science. Seres expects to file an Investigational New Drug (IND) application and initiate clinical development for SER-301 in early 2020.
Public equity offering (June 2019): Seres completed a public equity offering of common stock, resulting in net proceeds of $60.6 million. The financing involved both new and existing investors, including from a new fund managed by Flagship Pioneering.
Appointment of Stephen Berenson to its Board of Directors (August 2019): Mr. Berenson is a managing partner at Flagship Pioneering and previously spent his career as an investment banker at J.P. Morgan.
Financial Results
Seres reported a net loss of $10.8 million for the second quarter of 2019, as compared to a net loss of $27.8 million for the same period in 2018. The second quarter net loss was driven primarily by clinical and development expenses, personnel expenses and ongoing development of the Company’s microbiome therapeutics platform. The second quarter net loss figure was inclusive of $12.5 million in recognized revenue associated primarily with the Company’s collaborations with Nestlé Health Science and AstraZeneca.

Research and development expenses for the second quarter of 2019 were $17.9 million, as compared to $24.1 million for the same period in 2018. The research and development expense was primarily related to Seres’ microbiome therapeutics platform, the clinical development of SER-109 and SER-287, as well as the Company’s immuno-oncology efforts.

General and administrative expenses for the second quarter of 2019 were $5.6 million, as compared to $8.7 million for the same period in 2018. General and administrative expenses were primarily due to headcount, professional fees and facility costs.

Seres ended the second quarter with approximately $102.2 million in cash and cash equivalents that included the first of three $6.7 million annual installment payments due under the terms of the collaboration with AstraZeneca. In June 2019 the Company completed a public equity offering of common stock, resulting in net proceeds of $60.6M.

Cash use in the second quarter declined relative to prior periods as a result of the cost cutting corporate changes implemented earlier this year. Seres expects the lower expense levels incurred this quarter to be a proxy for subsequent quarters leading up to the clinical readouts expected in 2020. Based on the Company’s current operating plan, cash resources are expected to fund operating expenses and capital expenditure requirements, excluding net cash flows from future business development activities or potential incoming milestone payments, into the first quarter of 2021.

Conference Call Information
Seres’ management will host a conference call today, Aug. 6, 2019, at 8:00 a.m. ET. To access the conference call, please dial 844-277-9450 (domestic) or 336-525-7139 (international) and reference the conference ID number 7773387. To join the live webcast, please visit the "Investors and Media" section of the Seres website at www.serestherapeutics.com.

A webcast replay will be available on the Seres website beginning approximately two hours after the event and will be archived for at least 21 days.

On August 6, 2019 Ribon Therapeutics, a clinical stage biotechnology company, developing first-in-class therapeutics targeting novel enzyme families activated under cellular stress conditions, reported the appointments of Sudha Parasuraman, M.D., and Edward "Tad" Stewart to the newly created positions of Chief Medical Officer (CMO) and Chief Business Officer (CBO), respectively (Press release, Ribon Therapeutics, AUG 6, 2019, View Source [SID1234538216]). Dr. Parasuraman is a board-certified hematologist-oncologist with more than 20 years in the healthcare industry and broad experience across the spectrum of oncology drug development. Mr. Stewart brings more than 20 years of executive experience in the biotechnology area with a focus on business development, strategy and commercialization.

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"Sudha has had a distinguished career as a successful drug developer in the biopharmaceutical industry with expertise in leading clinical strategy, design and management of novel oncology programs, experience that aligns perfectly with our goal to bring first-in-class therapies to patients in need," said Victoria Richon, Ph.D., President and Chief Executive Officer, Ribon Therapeutics. "Tad brings to Ribon significant business development and strategy experience, perfectly suited to help us maximize the opportunities we see with our platform. On behalf of our entire team, I want to welcome both Sudha and Tad and we look forward to their contributions."

"Ribon has pioneered a completely novel area of human biology and rapidly used translational methods to bring those discoveries forward. Their lead program RBN-2397 is a testament to this approach and is now set to enter the clinic in the third quarter of this year," said Dr. Parasuraman. "This has established Ribon as the leader in developing potential first-in-class therapeutics that target stress response pathways. When activated, these enzymes are pivotal in disease progression and survival, making them very suitable for therapeutic intervention. I’m excited to join the Ribon team on their journey in bringing this exciting science to patients."

"Ribon’s platform enables thorough interrogation of stress response pathways, an expanding set of targets and rapid candidate generation," said Mr. Stewart. "Ribon has identified multiple monoPARP and NADase targets in oncology, neuro-degeneration and inflammatory diseases, and has tremendous capabilities to rapidly develop potent and selective inhibitors to target them. The speed with which the company has been able to execute on bringing their lead program toward the clinic is impressive and I look forward to helping expand the potential value of that platform for the company and for patients."

Dr. Parasuraman brings more than 20 years of industry and academic practice experience in hematology, oncology, and pediatrics to her work at Ribon. Most recently she served as CMO for X4 Pharmaceuticals, where she led their immuno-oncology and rare disease programs. Prior to X4, she was VP, Global Medical Affairs at uniQure Inc, where she built and led a team developing gene therapies for rare diseases. Prior to uniQure, she held senior positions at Novartis, where she led the early development program for the CDK4/6 inhibitor, ribociclib, and helped pave the way for accelerated registration studies. She was responsible for leading innovative clinical trials in the Signature Program and the commercial strategies for early-stage programs. Dr. Parasuraman also served as Medical Director at Millennium Pharmaceuticals (now Takeda Oncology) where she was the medical lead for various programs including the clinical development and lifecycle management of the first-in-class cancer therapy, VELCADE. Before joining industry, Dr. Parasuraman was a faculty member at Harvard Medical School and staff physician at Children’s Hospital of Boston, Dana Farber Cancer Institute, and Brigham and Women’s Hospital. Dr. Parasuraman completed her fellowship in pediatric hematology-oncology at St. Jude Children’s Research Hospital in Memphis, TN, and pediatric residencies at Children’s Hospital of Michigan and Los Angeles County/University of Southern California Medical Center. She received her medical degree from the University of Madras, India.

Mr. Stewart has more than 20 years of strategic biopharmaceutical business development experience, including over fifteen years at Merrimack Pharmaceuticals. During his time at Merrimack, Tad served in various roles as part of the executive leadership team, including leading the company’s business development efforts and heading the company’s Commercial Business Unit. Tad’s experience at Merrimack spanned its growth from a pre-clinical organization to a clinical development company and, ultimately, to a commercial enterprise, including the successful launch of a marketed oncology product (ONIVYDE) and the execution of several strategic transactions that supported the long-term growth of the company. Most recently he served as President and CEO of Commense, Inc., a company focused on developing pediatric immunomodulators based on manipulating the human microbiome. Prior to Commense, Tad served as CBO for Crescendo Biologics, a novel antibody platform company. Prior to Merrimack, Tad worked as a consultant with clients across the biotech, pharmaceutical and medical device industries. Tad holds an M.B.A. from the Johnson School at Cornell University and a B.S. in Biology from Bates College.

On August 6, 2019 Epizyme, Inc. (Nasdaq:EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, reported that Robert Bazemore, president and chief executive officer, will present at the Wedbush PacGrow 2019 Healthcare Conference on Tuesday, Aug. 13, 2019 at 10:55 a.m. ET in New York City (Press release, Epizyme, AUG 6, 2019, View Source [SID1234538215]).

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A live webcast will be available in the investor section of the company’s website at www.epizyme.com. The webcast will be archived for 60 days following the presentation.

On August 6, 2019 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that engineers next-generation human enzymes to provide solutions for diseases with unmet medical need, reported financial results for the second quarter ended June 30, 2019 and corporate highlights (Press release, Aeglea BioTherapeutics, AUG 6, 2019, View Source [SID1234538214]).

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"We made tremendous progress this quarter with pegzilarginase, our lead product candidate for the treatment of Arginase 1 Deficiency (ARG1-D)," said Anthony G. Quinn, M.B. Ch.B., Ph.D., president and chief executive officer of Aeglea. "We dosed our first patient in our global pivotal Phase 3 PEACE trial, which is a major milestone toward providing our therapy to patients. The U.S. Food and Drug Administration’s (FDA) breakthrough therapy designation highlights the clinical relevance of the emerging data from the Phase 1/2 and Phase 2 open-label extension clinical trials as well as the potential of pegzilarginase to provide meaningful clinical improvements over available therapy."

Recent Highlights

Aeglea dosed the first patient in the Company’s global pivotal Phase 3 PEACE trial. The pivotal trial is intended to further evaluate the efficacy and safety of pegzilarginase, the Company’s lead product candidate for the treatment of ARG1-D, a progressive disease presenting in early childhood that results in severe complications and early mortality. The Company expects to report topline data from the PEACE trial in the first quarter of 2021.

The FDA granted Breakthrough Therapy Designation (BTD) to the Company’s lead product candidate, pegzilarginase, for the treatment of ARG1-D. The FDA’s BTD is intended to expedite the development and review of new therapies that are aimed at treating a serious or life-threatening condition when preliminary clinical evidence demonstrates the therapy may have substantial improvement on at least one clinically significant endpoint over available therapy. The designation was based on data from the completed Phase 1/2 clinical trial and the ongoing Phase 2 open-label extension study. Aeglea expects to continue discussions with the FDA regarding the pegzilarginase program and the Company’s next steps in the fourth quarter of 2019.

Interim data from 35 patients in the Company’s Phase 1/2 combination trial of pegzilarginase and KEYTRUDA in extensive disease small cell lung cancer revealed to date one complete response, four partial responses and 11 patients with stable disease. The combination trial was well tolerated, and safety observations were consistent with prior studies of pegzilarginase in patients with cancer. The Company has concluded enrollment and intends on submitting the results for presentation or publication after the final dataset becomes available.
Upcoming Events

Aeglea will present at the following conferences, with details regarding the date and time of the presentations and webcasts to be announced prior to the events.

Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, September 3-6, Rotterdam, Netherlands
H.C. Wainwright & Co. 21st Annual Global Investment Conference, September 8-10, New York, NY
Second Quarter 2019 Financial Results

As of June 30, 2019, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $107.0 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the first quarter of 2021.

Research and development expenses totaled $14.8 million for the second quarter of 2019, compared with $9.1 million for the second quarter of 2018. The increase was primarily due to expanded personnel-related expenses, clinical development activity, investment in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase, and a ramp-up in manufacturing activities for the Company’s AEB4104 program for homocystinuria.

General and administrative expenses totaled $3.8 million for the second quarter of 2019, compared with $2.9 million for the second quarter of 2018. This increase was primarily due to additional employee headcount and compensation to support company growth.

Net loss totaled $18.0 million and $9.4 million for the second quarter of 2019 and 2018, respectively, with non-cash stock compensation expense of $1.2 million and $1.0 million for the second quarter of 2019 and 2018, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically depletes the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. Aeglea’s Phase 1/2 and Phase 2 open-label extension data evaluating pegzilarginase in patients with Arginase 1 Deficiency demonstrated clinical improvements and sustained lowering of plasma arginine. Aeglea is currently recruiting patients for its single, global pivotal Phase 3 PEACE trial designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.