On November 9, 2020 Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage biotechnology company focused on Alzheimer’s disease, reported financial results for the third quarter ended September 30, 2020 (Press release, Pain Therapeutics, NOV 9, 2020, View Source [SID1234570329]).

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For the third quarter ended September 30, 2020, net loss was $1.4 million, or $0.06 per share, compared to a net loss of $0.7 million, or $0.04 per share, for the same period in 2019. Net cash used in operations was $4.2 million during the first nine months of 2020. Net cash use in full-year 2020 is still expected to be approximately $5.0 million. The Company’s cash and cash equivalents were $24.1 million as of September 30, 2020, with no debt.

Financial Highlights for Third Quarter 2020

At September 30, 2020, cash and cash equivalents were $24.1 million, compared to $23.1 million at December 31, 2019, with no debt.

Cash balance included $4.6 million in proceeds from exercise of warrants in the first nine months of 2020. Approximately 0.8 million warrants remain outstanding, each with an exercise price of $1.25 per share. All warrants expire February 2021.

Net cash used in operations during the nine months ended September 30, 2020 was $4.2 million, net of reimbursements received from National Institutes of Health (NIH) research grant awards.

NIH research grant award reimbursements of $1.0 million were received and recorded as a reduction in research and development (R&D) expenses in the third quarter of 2020. This compared to $1.5 million of NIH research grant award reimbursements received for the same period in 2019.

R&D expenses were $0.4 million compared to a negative $0.1 million for the same period in 2019. The increase was due primarily to lower NIH research grant award reimbursements compared to the prior year.

General and administrative (G&A) expenses were $1.0 million compared to $0.8 million for the same period in 2019. The increase was due primarily to higher insurance expenses compared to the prior year.

On November 9, 2020 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company primarily focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported a series of recently issued patents for its Phase 2b/3 clinical-stage lead product candidate, Validive (clonidine HCl mucobuccal tablet) (Press release, Monopar Therapeutics, NOV 9, 2020, View Source [SID1234570328]). These patents, including U.S. Patent No. 10,675,271, provide claims covering "Clonidine and/or clonidine derivatives for use in the prevention and/or treatment of adverse side effects of chemotherapy".

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"These recently issued patents broaden the patent protection for the use of Validive in cancer patients," said Andrew Mazar, PhD, Chief Scientific Officer of Monopar. "Specifically, they provide protection into 2035 for the potential ability of Validive to prevent or treat common chemotherapy-associated side effects such as gastrointestinal disorders, respiratory disorders, fatigue and headache."

"According to the U.S. Centers for Disease Control and Prevention, about 650,000 cancer patients receive chemotherapy each year in the U.S., and most of these experience side-effects as a result of their treatment," said Chandler Robinson, MD, Chief Executive Officer of Monopar. "These patents expand the potential use of Validive beyond the earlier allowed claims for the prevention of oral mucositis in patients receiving chemoradiotherapy. This in turn serves to advance Monopar’s mission to develop treatments that improve quality of life in cancer patients undergoing treatment."

Monopar is currently developing Validive for the prevention of chemoradiation-induced severe oral mucositis in oropharyngeal cancer patients (OPC), an indication which currently has no FDA approved treatment. Monopar’s Phase 2b/3 clinical trial in OPC patients is on track to start before year-end. The recently issued patents would provide protection should Monopar determine in the future to conduct additional Validive development activities related to adverse side effects of chemotherapy beyond OPC.

On November 9, 2020 DCprime, the front-runner in the field of relapse vaccines, reported the presentation of preclinical results at the virtual 35th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), occurring November 9-14 (Press release, DCPrime, NOV 9, 2020, View Source [SID1234570327]). The in vitro and in vivo data presented at SITC (Free SITC Whitepaper) highlight the potential impact of the company’s lead vaccine candidate, DCP-001, on ovarian cancer treatment and post-remission therapy. The accepted abstract is listed below and is now available online on the SITC (Free SITC Whitepaper) website.

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"We are approaching a pivotal time for DCprime in which we will be generating a growing body of preclinical data showing the potential of our vaccination approach in solid tumors in addition to first clinical data emerging from our ongoing ADVANCE II clinical study in Acute Myeloid Leukemia (AML) patients before year-end. Both developments are of equal importance for us today with the high unmet medical need in solid tumors being a significant long-term opportunity for value generation," commented Erik Manting, PhD, CEO of DCprime.

The preclinical studies presented at SITC (Free SITC Whitepaper) are based on a successful research collaboration between DCprime and the Department of Obstetrics and Gynecology of the University Medical Center of Groningen (UMCG), led by Professor Hans Nijman, MD, PhD. Animal studies were carried out in collaboration with TransCure Bioservices SAS, supported by a Eurostars funding.

In peripheral blood mononuclear cells from healthy donors and ovarian cancer patients, DCP-001 was shown to activate both T helper cells (CD4+) as well as cytotoxic T cells (CD8+) and was able to induce the formation of memory T cells. Importantly, CD8+ T cells derived from ovarian cancer patients stimulated by DCP-001 were shown to exert an elevated cytotoxic immune response to ovarian cancer cells. In a humanized mouse model for ovarian cancer, relapse vaccination with DCP-001, lead to a significant reduction of tumor growth, including partial and complete tumor regressions. Based on the promising preclinical data, DCprime is currently preparing together with the Nijman group at UMCG a Phase I clinical trial in ovarian cancer patients.

POSTER PRESENTATION DETAILS:
Presentation Title: #171: Preclinical studies support therapeutic application of the leukemic cell-based cancer relapse vaccine DCP-001 in ovarian cancer

Maho Nagasawa; Remco Bos; Haoxiao Zuo; Kiave Yune Ho Wang Yin; Marie-José van Lierop; Sebastien Tabruyn; Erik Manting; Marco de Bruyn; Hans Nijman; Satwinder Kaur SK. Singh.

The poster’s authors will be available for the Virtual Poster Viewing sessions to answer questions and interact with meeting attendees in real time on Wednesday, November 11 from 5:15–5:45 p.m. EST and Friday, November 13 from 4:40–5:10 p.m. EST

On November 9, 2020 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics, reported third quarter 2020 financial results and provided an overview of operational progress (Press release, Rubius Therapeutics, NOV 9, 2020, View Source [SID1234570326]).

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"Rubius Therapeutics continues to demonstrate strong execution across our pipeline of Red Cell Therapeutics for the treatment of cancer. Our IND filing for RTX-321 included, for the first time, frozen drug substance as part of the manufacturing process, resulting in a truly off-the-shelf cellular therapy with a potential shelf life of up to several years," said Pablo J. Cagnoni, M.D., president and chief executive officer of Rubius Therapeutics. "As we continue to escalate the dose in the RTX-240 solid tumor trial, we are assessing the safety profile and biological effects of RTX-240 on innate and adaptive immune responses. We believe that by demonstrating that RTX-240 is working as intended to induce anti-tumor innate and adaptive immunity, we can unlock the potential of the RED PLATFORM across our entire pipeline of cancer and autoimmune programs."

Third Quarter and Recent Highlights

RTX-240 Phase 1/2 Clinical Program for Advanced Solid Tumors or Relapsed/Refractory Acute Myeloid Leukemia (AML)

Completed dosing of the fourth cohort in the Phase 1/2 solid tumor clinical trial
Assessing the safety profile and biological effects of RTX-240 on innate and adaptive immune responses by measuring biomarkers associated with activation and proliferation of NK and T cells in both peripheral blood and paired tumor biopsy samples across dose levels
Dosed the first patient with relapsed or refractory AML in second Phase 1 arm of ongoing RTX-240 clinical trial
Demonstrating robust momentum in manufacturing and producing clinical supply of RTX-240 for both arms in the Phase 1/2 clinical trial of RTX-240
RTX-321 Artificial Antigen-Presenting Cell (aAPC) Development Program for Human Papillomavirus (HPV) 16-Positive Cancers

Filed an IND with U.S. FDA for RTX-321 for the treatment of HPV 16-positive cancers
For the first time, the IND filing includes frozen drug substance as part of the manufacturing process, resulting in a truly off-the-shelf cellular therapy with a potential shelf life of up to several years
Conducting manufacturing in support of planned GMP clinical supply needs for the RTX-321 clinical trial
Leadership Team

Strengthened the leadership team by appointing Jose "Pepe" Carmona as chief financial officer
Pepe brings to Rubius extensive experience in global finance and operations, as well as a track record of financing clinical-stage and commercial biotech companies
Preclinical Data Presentations

This morning, Rubius presented preclinical data from its lead clinical oncology candidate, RTX-240, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s Annual Meeting, demonstrating:
RTX-240 increased CD8 T cell and NK cell expansion and activation in vitro when compared to a combination of 4-1BB agonist antibody plus recombinant IL-15, which was directly correlated with the percentage of 4-1BBL and IL-15TP expressed on the cell surface
RTX-240 expanded CD56dim NK cells, a cell population with high cytotoxicity
RTX-240 promoted NK cell-killing of a myeloid leukemia cell line, K562, which was accompanied by increased NK cell degranulation and activation
A murine surrogate for RTX-240, mRBC-240, promoted significant expansion of CD8 T cells and NK cells in vivo in a murine model of colorectal cancer (CT26)
mRBC-240 demonstrated potent antitumor activity in a B16F10 melanoma model that was directly correlated with the expansion of terminally differentiated NK cells in the tumors
Presented preclinical data at the Federation of Clinical Immunology Societies Annual Meeting and the American Association of Cancer Research Tumor Immunology and Immunotherapy Conference, from its lead aAPC program, RTX-321, for the treatment of HPV 16-positive tumors demonstrating:
RTX-321 and its mouse surrogates demonstrated a dual mechanism of action in vivo
Functions as an aAPC to boost antigen-specific CD8+ T cell responses
Promotes antigenindependent stimulation of both innate and adaptive immune responses
Mouse surrogates of RTX-321 promote tumor control, memory formation and epitope spreading in tumor models in vivo
Treatment with the RTX-321 mouse surrogate results in minimal, reversible effects in vivo, likely due to biodistribution to the vasculature and spleen
RTX-321 functions as an aAPC to boost HPV 16 antigen-specific T cells in vitro
RTX-321 promotes HPV 16-independent adaptive and innate immune responses in vitro
Third Quarter Financial Results

Net loss for the third quarter of 2020 was $40.9 million or $0.51 per common share, compared to $47.0 million or $0.59 per common share in the third quarter of 2019.

In the third quarter of 2020, Rubius invested $28.2 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $33.5 million in the third quarter of 2019. The year-over-year decrease was driven by a $7.5 million reduction in costs following the deprioritization of our rare disease pipeline in March 2020. The decrease in rare disease program expenses was offset by $9.6 million in incremental spend to advance our cancer programs, including costs associated with our Phase 1/2 clinical trial for RTX-240 for the treatment of solid tumors as well as costs associated with preclinical and IND-enabling activities for RTX-321. In addition, R&D expenses not allocated to programs decreased by $7.4 million driven primarily by reductions in non-program labor, laboratory supplies, research materials and contract research as pre-GMP manufacturing and discovery activities shifted to support the development of clinical candidates.

G&A expenses were $12.0 million during the third quarter of 2020, as compared to $15.0 million for the third quarter of 2019. The lower costs were principally driven by a reduction in stock-based compensation expense.

Nine Month Financial Results

Net loss for the first nine months of 2020 was $127.2 million or $1.58 per common share, compared to $119.0 million or $1.52 per common share in the first nine months of 2019.

In the nine months ended September 30, 2020, Rubius invested $90.5 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $81.9 million in the first nine months of 2019. The year-over-year increase was driven by $28.3 million of incremental costs to advance our cancer programs, including costs incurred for our Phase 1/2 clinical trial for RTX-240 for the treatment of solid tumors as well as costs of preclinical and IND-enabling activities for RTX-321. The increase in cancer program costs was offset by a $12.8 million decrease in expenses related to our rare disease pipeline following the deprioritization of these programs in March 2020. Additionally, costs not allocated to programs decreased by $6.9 million driven primarily by a shift in manufacturing activities towards the technical development and production of clinical supply for our oncology programs and the shift in discovery activities from the development of clinical candidates towards work to support IND enabling activities.

G&A expenses were $36.2 million during the first nine months of 2020, as compared to $42.3 million for the same period in 2019. The lower costs were principally driven by a reduction in stock-based compensation expense.

Cash Position

As of September 30, 2020, cash, cash equivalents and investments were $207.9 million as compared to $283.3 million as of December 31, 2019, providing Rubius with a cash runway into 2022. During 2020, the Company used $97.1 million of cash to fund operations and $4.5 million to fund capital expenditures, consisting mostly of payments for assets purchased in 2019. In addition, during 2020 the Company drew down the third and final tranche of $25.0 million pursuant to its $75.0 million loan agreement with Solar Capital.

On November 9, 2020 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported third quarter and year-to-date financial results for 2020 (Press release, Precigen, NOV 9, 2020, View Source [SID1234570325]).

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Business Highlights:

Clinical Pipeline and Data Update Conference Call: In light of the substantive progress made in advancing its clinical pipeline, as well as anticipated data disclosures for several key programs, Precigen will host a conference call in early December dedicated to reviewing these important milestones. The Company plans to announce timing and details about the call in the coming weeks;
UltraPorator: Precigen announced that the US Food and Drug Administration (FDA) cleared UltraPorator as a manufacturing device for its UltraCAR-T manufacturing process. The Company announced that it has successfully completed technology transfer of the UltraPorator system for the manufacturing of PRGN-3005 in ovarian cancer at the University of Washington/Fred Hutchinson Cancer Research Center and for PRGN-3006 in acute myeloid leukemia (AML) at the Moffitt Cancer Center. UltraPorator is a semi-closed, high-throughput system with a proprietary hardware and software solution designed to significantly reduce processing time and contamination risk, limitations inherent in existing electroporation devices that contribute to current hurdles for viable scale-up and commercialization of certain therapeutic programs;
PRGN-2009 AdenoVerse Immunotherapy: Precigen announced that the first patient has been dosed in the Phase I/II trial for PRGN-2009, a first-in-class, off-the-shelf investigational immunotherapy utilizing the AdenoVerse platform and designed to activate the immune system to recognize and target HPV-positive solid tumors;
PRGN-3005 UltraCAR-T: Precigen completed dosing of patients in dose level 3 of the intraperitoneal (IP) arm of the Phase 1 clinical trial of PRGN-3005 UltraCAR-T for the treatment of advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer (clinical trial identifier: NCT03907527); and
PRGN-3006 UltraCAR-T: Precigen completed dosing of patients in dose level 2 of the non-lymphodepletion arm and dose level 1 in the lymphodepletion arm of the Phase 1 trial of PRGN-3006 UltraCAR-T for treatment of patients with relapsed or refractory acute myeloid leukemia (AML) or higher-risk myelodysplastic syndromes (MDS) (clinical trial identifier: NCT03927261).
"The Precigen team has made impressive progress this quarter in driving value across our preclinical and clinical pipeline. In particular, we made several advances in our quest to meet unmet needs for patients, including dosing the first patient in our first-in-human study of PRGN-2009 AdenoVerse in HPV-positive solid tumors, and advancing our proprietary UltraPorator system towards clinical implementation," said Helen Sabzevari, PhD, President and CEO of Precigen. "In early December, we are excited to share a comprehensive update on our clinical pipeline progress towards meeting our 2020 goals laid out earlier this year as well as looking forward to the PRGN-3006 presentation at the 2020 meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) by the trial’s Principal Investigator, Dr. David Sallman from the Moffitt Cancer Center."

Third Quarter 2020 Financial Highlights:

Total revenues of $23.6 million in 2020 compared to $18.3 million in 2019;
Net loss from continuing operations of $29.5 million, or $(0.18) per basic share, of which $10.1 million was for non-cash charges in 2020, compared to net loss from continuing operations of $49.1 million, or $(0.32) per basic share, of which $15.7 million was for non-cash charges in 2019; and
Cash, cash equivalents, and short-term investments totaled $113.1 million as of September 30, 2020.
Year-to-Date 2020 Financial Highlights:

Total revenues of $83.8 million in 2020 compared to $73.7 million in 2019; and
Net loss from continuing operations of $102.8 million, or $(0.63) per basic share, of which $50.6 million was for non-cash charges in 2020 compared to net loss from continuing operations attributable to Precigen of $132.7 million, or $(0.86) per basic share, of which $36.2 million was for non-cash charges in 2019.
Third Quarter 2020 Financial Results Compared to Prior Year Period

Total revenues increased $5.3 million, or 29%, over the quarter ended September 30, 2019. Collaboration and licensing revenues increased $2.9 million primarily due to the accelerated recognition of previously deferred revenue upon the mutual termination of one of the Company’s collaboration agreements in July 2020. Product and service revenues generated by the Company’s Trans Ova and Exemplar subsidiaries increased $2.4 million due to an increase in services performed for new and existing customers and the expansion of Trans Ova’s commercial dairy business. Gross margin on products and services improved as a result of operational efficiencies gained through reductions in workforce and improved inventory management as well as a decrease in the cost of cows used in production.

Research and development expenses decreased $13.5 million, or 53%, from the quarter ended September 30, 2019. Salaries, benefits, and other personnel costs decreased $6.8 million and contract research organization costs and lab supplies decreased $5.1 million as Precigen suspended the operations of its MBP Titan subsidiary in the second quarter and deprioritized certain internal programs at its ActoBio subsidiary in the fourth quarter of 2019. Selling, general and administrative (SG&A) expenses were comparable period over period.

Year-to-Date 2020 Financial Results Compared to Prior Year Period

Total revenues increased $10.1 million, or 14%, over the nine months ended September 30, 2019 primarily due to an increase in Precigen’s collaboration and licensing revenues as the Company accelerated the recognition of previously deferred revenue upon the mutual termination of two of its collaboration agreements in 2020. Product and service revenues generated by Trans Ova and Exemplar increased $4.8 million due to an increase in services performed for new and existing customers and the expansion of Trans Ova’s commercial dairy business. Gross margin on products and services improved as a result of operational efficiencies gained through reductions in workforce, improved inventory management, a reduction in third-party royalty rate obligations for certain licensed technologies and a decrease in the cost of cows used in production.

Research and development expenses decreased $35.6 million, or 44%, from the nine months ended September 30, 2019. Salaries, benefits, and other personnel costs decreased $13.7 million and contract research organization costs and lab supplies decreased $17.9 million as Precigen suspended the operations of its MBP Titan subsidiary in the second quarter and deprioritized certain internal programs at its ActoBio subsidiary in the fourth quarter of 2019. SG&A expenses decreased $8.4 million and include a net decrease in fees payable to certain third-party vendors and a reduction of 30% in corporate headcount to support a more streamlined organization. Other corporate expenses decreased $1.9 million as part of the streamlined organization and the impact of the COVID-19 pandemic on travel. These decreases were partially offset by increased share-based compensation expense attributable to equity grants made in in the first quarter of 2020 and one-time severance costs for terminated employees. The Company also recorded $23.0 million of impairment charges for the nine months ended September 30, 2020 primarily due to the write down of goodwill and intangible assets related to the MBP Titan subsidiary.

Precigen: Advancing Medicine with Precision

Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit www.precigen.com or follow us on LinkedIn.

Trademarks

Precigen, UltraPorator, UltraCAR-T, AdenoVerse and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners.