1stOncology™: Cancer Intelligence Service – Page 8733 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Compugen Reports Second Quarter 2021 Results

On July 28, 2021 Compugen Ltd. (NASDAQ: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported financial results for the second quarter ended June 30, 2021 (Press release, Compugen, JUL 28, 2021, View Source [SID1234585276]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

"Our continued execution in the clinic, which includes the recent initiation of three clinical studies, further strengthens Compugen’s leadership position in the DNAM axis space," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "These new studies expand our comprehensive evaluation of the DNAM axis and leave us uniquely positioned as the only company, in a clinical setting, evaluating anti-PVRIG, anti-TIGIT and anti-PD-1 combinations, which is our key differentiator in the TIGIT space. We expect the remainder of the year to include data readouts from our triple combination study in collaboration with Bristol Myers Squibb and COM902 monotherapy study, which we expect to expand the foundation of our clinical data generated to date."

Dr. Cohen-Dayag continued, "The updated data from our COM701 Phase 1 combination and monotherapy studies presented at ASCO (Free ASCO Whitepaper) support our continued excitement in our science and potential benefit it may bring to patients, showing durable responses and disease control in patients not eligible for or typically not responding to checkpoint inhibitors including those with prior progression. In addition, we shared our first preliminary pharmacodynamic biomarker data indicating that treatment with COM701 leads to immune activation and has the potential to drive anti-tumor activity in non-inflamed tumors as evidenced by activity in selected PD-L1 low, PVRL2 positive patients. Our combination strategy around our wholly owned assets targeting PVRIG and TIGIT give us a strong first mover advantage, and we look forward to continued progress through 2021 as we work to elucidate the potential of the DNAM axis pathways in immunotherapy."

Recent and Second Quarter 2021 Corporate Highlights

Presented updated data from the COM701 monotherapy and combination with Opdivo (nivolumab) studies at the ASCO (Free ASCO Whitepaper) 2021 Annual Meeting including:
– Durable responses beyond one year, including one complete response, in tumor types typically unresponsive to checkpoint inhibitors
– Preliminary biomarker data reveal immune activation evidenced by a trend of increased proliferation of peripheral immune cells and IFNγ. IFNγ increased with increasing doses of COM701, suggesting the observed activity is likely derived from the combination treatment and not Opdivo alone
– Preliminary anti-tumor activity in PD-L1 low, PVRL2 positive patients, with non-inflamed tumor microenvironment/immune desert phenotype
Dosed the first patient in the Phase 1b cohort expansion study of COM701 in combination with Opdivo in patients with ovarian, breast, endometrial and microsatellite-stable colorectal cancers
Dosed the first patient in the Phase 1 dual combination study of COM902 and COM701 in patients with advanced malignancies, the first clinical study of dual blockade of TIGIT and PVRIG independent of anti-PD-1
Dosed the first patient in the Phase 1/2 triple combination cohort expansion of COM701 with Opdivo and Bristol Myers Squibb’s anti-TIGIT antibody, BMS-986207
Presented research at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Targets for Cancer Immunotherapy: A Deep Dive Seminar Series, supporting PVRIG as a novel and differentiated checkpoint in the DNAM axis
Financial Results

R&D expenses for the second quarter ended June 30, 2021, were $6.8 million compared with $4.4 million for the comparable period in 2020. The increase in R&D expenses reflects the strong execution and expansion of the Phase 1 clinical programs.

General and administrative expenses for the second quarter ended June 30, 2021, were $2.7 million compared with $2.1 million for the comparable period in 2020. The increase in expenses is attributed mainly to corporate related expenses.

Net loss for the second quarter of 2021 was $9.5 million, or $0.11 per basic and diluted share, compared with a net loss of $6.2 million, or $0.08 per basic and diluted share, in the comparable period of 2020.

As of June 30, 2021, cash, cash related accounts, short-term and long-term bank deposits totaled approximately $111 million, compared with approximately $124 million on December 31, 2020. The Company has no debt.

Conference Call and Webcast Information

The Company will hold a conference call today, July 28, 2021, at 8:30 AM ET to review its second quarter 2021 results. To access the live conference call by telephone, please dial 1-866-744-5399 from the U.S., or +972-3-918-0644 internationally. The call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.

Cytocom Inc. Announces Completed Merger with Cleveland BioLabs

On July 28, 2021 Cytocom Inc. ("Cytocom" or "Company"), a leading biopharmaceutical company creating next-generation therapies that focus on immune homeostasis, reported the completion of its merger with Cleveland BioLabs, Inc (Press release, Cleveland BioLabs, JUL 28, 2021, View Source [SID1234585275]). The all-stock transaction was first announced on October 19, 2020.

Shares of the new Cytocom’s common stock will begin trading on Nasdaq under the ticker symbol "CBLI," at the opening bell on Wednesday, July 28, 2021.

As previously reported, the newly combined company will operate as "Cytocom, Inc." under the leadership of the existing Cytocom management team led by CEO Michael K. Handley. Mr. Handley now serves as the new company’s President and CEO.

"With the merger completed, we look forward to further advancing our late-stage clinical programs and expanding what we believe to be one of the largest toll-like receptor platforms in the industry," said Michael K. Handley. "This merger, coupled with our acquisition of ImQuest Life Sciences and the listing of the new Cytocom common shares on Nasdaq, represents a transformative growth opportunity and fits firmly with our goal of becoming a recognized leader in immune-modulating therapies targeting cancer, ARS, inflammatory and autoimmune diseases, and viruses, including COVID-19. Looking forward, we anticipate achieving multiple commercial, regulatory and clinical milestones over the next 12 to 18 months that should enable us to showcase the power of our drug development platform and further generate shareholder value."

McGuireWoods LLP represented Cleveland BioLabs and Troutman Pepper Hamilton Sanders LLP represented Cytocom in the merger.

CHIMERIC LICENSES NOVEL SOLID TUMOR CAR T THERAPY FROM WORLDRENOWNED CELL THERAPY CENTRE UNIVERSITY OF PENNSYLVANIA

On July 28, 2021 Chimeric Therapeutics (ASX:CHM, "Chimeric" or the "Company"), a clinical-stage cell therapy company committed to bringing the promise of cell therapy to life for more patients, reported that it has entered into an exclusive licensing agreement with world renowned cell therapy centre, the University of Pennsylvania (Penn), for the first CDH17 CAR (chimeric antigen receptor) T cell therapy (Press release, Chimeric Therapeutics, JUL 28, 2021, View Source [SID1234585274]).

The novel CDH17 CAR T cell therapy targets CDH17, an oncogenic factor associated with poor prognosis and metastasis in neuroendocrine tumors as well as the most common gastrointestinal tumors including colorectal cancer, pancreatic cancer and gastric cancer.

More than a decade of research and optimization has gone into development of the novel CDH17 CAR T cell therapy, developed by leading cellular immunotherapy scientist Xianxin Hua, MD, PhD, and his team. Dr Hua is a professor of Cancer Biology in Penn’s Perelman School of Medicine, an investigator at the Abramson Family Cancer Research Institute and a Harrington Scholar Innovator.

"Chimeric is incredibly excited to bring the CDH17 CAR T cell therapy into our pipeline. Our focus is on developing cell therapies that have the potential to be curative for patients and we believe that this CDH17 CAR T cell therapy has that transformative potential. We are very much looking forward to working with Dr Hua and his team to bring this CAR T to the clinic as soon as possible," said Jennifer Chow, Chimeric COO.

The CDH17 CAR T cell therapy is a next or 3rd generation CAR T cell therapy that utilizes novel technology to specifically address the challenges of solid tumors. Preclinical studies of the CHIMERIC THERAPEUTICS LIMITED | 2 LEVEL 3, 62 LYGON STREET CARLTON VIC 3053 AUSTRALIA CDH17 CAR T have demonstrated safety, with no toxicity to normal tissues, and promising efficacy with complete eradication of tumor cells and no relapse of the tumor.

"The preclinical studies indicate that our CDH17 CAR T marks the recognition of an entirely new class of solid tumor antigen targets, that are highly susceptible to CAR T attack on tumor cells yet masked from attack by the CAR T in normal tissues," said Dr Hua.

Development is currently underway for a phase 1 clinical trial that is planned to begin in 2022 at the University of Pennsylvania.

A three-year commitment for further research and development has been made to Dr Hua and Penn.

Under the terms of the license agreement, Chimeric Therapeutics acquires the exclusive rights to develop and commercialize the CDH17 CAR T cell therapy for which it has agreed to pay Penn license fees, development milestones and royalty payments based on commercial net sales. Upfront fees associated with the license agreement will be funded entirely from existing cash reserves.

The University of Pennsylvania is a globally recognized leader in cellular immunotherapy and widely known for being home to the 1st FDA approved CAR T therapy.

The Chimeric team will be leveraging its unique cell therapy expertise to ensure the timely development and commercialization of the novel CDH17 CAR T cell therapy, bringing the promise of cell therapy and curative potential to more patients with cancer.

Chimeric’s Executive Chairman Paul Hopper said "We are delighted to have secured the exclusive rights to this promising asset from one of the world’s pre-eminent cell therapy centres which has been a powerhouse in CAR T innovation for many years. We look forward to utilizing the deep cell therapy expertise of our team to develop and commercialize this CAR T."

Authorised on behalf of the Chimeric Therapeutics board of directors by Chairman Paul Hopper

Bridge Medicines Enters Exclusive License Agreement with Cornell University for UBR5 Inhibitor Program to Treat Resistant Cancers

On July 28, 2021 Bridge Medicines and Cornell University reported an exclusive license agreement to develop UBR5 Inhibitors for the treatment of certain difficult-to-treat cancers such as triple-negative breast cancer (TNBC) (Press release, Bridge Medicines, JUL 28, 2021, View Source [SID1234585273]).

UBR5 is a cellular enzyme (an E3 ubiquitin ligase) that is involved in the labeling of other proteins within a cell to designate them for destruction. UBR5 has recently been shown to be a key regulator in certain cancers, such as breast and ovarian cancers, enabling the cancer cells to survive and resist standard treatments.

Pioneering research by Dr. Xiaojing Ma and Dr. Gang Lin and their colleagues at Weill Cornell Medicine have identified a distinctive and profound role of UBR5 in the growth and metastasis of triple negative breast cancer (TNBC). An analysis of TNBC specimens revealed strong gene amplifications of UBR5 associated with the disease. UBR5 overexpression in TNBC tissues was confirmed at mRNA and protein levels. Dr. Ma and his colleagues discovered that tumor-derived UBR5 facilitated tumor growth in a manner dependent upon immune cells within the microenvironment, rendering them less able to kill the cancer cell. Consequently, attacking the UBR5 target may work well in combination with other immune regulators, such as those that target the PD-L1 pathway. The combined targeting of UBR5 and PD-L1 yields strong therapeutic benefit to tumor-bearing hosts in preclinical studies. Collectively, these data support targeting UBR5 as a novel immunotherapy to treat highly aggressive breast cancers that resist conventional therapies.

"Work in my laboratory has demonstrated the critical importance of the UBR5 pathway in enabling major cancers to resist chemo- and immuno-therapies," said Dr. Xiaojing Ma, professor of microbiology and immunology at Weill Cornell Medicine. "This resistance results in advancing cancers and ultimately death in many patients despite currently available treatments. This is an important unmet medical need. We believe that by developing an effective anti-UBR5 approach, we may be able to provide life-saving outcomes for patients who are out of options."

The Tri-Institutional Therapeutics Discovery Institute, Inc. (Tri-I TDI) advanced the pioneering discoveries from the Ma Laboratory into early-stage drug molecules. Tri-I TDI’s mission is to work with innovative scientists at its founding institutions, The Rockefeller University, Memorial Sloan Kettering Cancer Center, and Weill Cornell Medicine, in a close collaboration with Takeda Pharmaceutical Company Ltd, to accelerate the discovery of new drug molecules. Bridge Medicines will further develop and advance these molecules with the goal of bringing a new treatment to patients.

"We are very excited to bring the UBR5 program into Bridge Medicines," said Bill Polvino, CEO of Bridge Medicines. "The preclinical data are very compelling, and we look forward to developing a novel, first in class therapy for cancers that resist current treatments, such as triple negative breast and ovarian cancers."

"Drug discovery is essential for advancing healthcare, ensuring the translation of cutting-edge science into powerful medicines that are available to patients who need it most," said Dr. John Leonard, senior associate dean for innovation and initiatives at Weill Cornell Medicine. "The Tri-I TDI-Bridge Medicines continuum is invaluable to realizing that promise and serves as a vital component of the Weill Cornell Medicine Enterprise Innovation ecosystem. We are excited that the UBR5 program, based on Drs. Ma and Lin’s groundbreaking research, has achieved this important milestone with Bridge Medicines."

Weill Cornell Medicine Enterprise Innovation accelerates the best of biomedical innovation to market, translating groundbreaking research into revolutionary care. Enterprise Innovation provides a bridge between academic researchers and industry stakeholders, brokering engagements and alliances, engaging with and educating faculty about biotech commercialization and business development—which includes patenting, licensing, and marketing—and developing entrepreneurial programming, education, mentoring, and other resources.

About TNBC
Triple-negative breast cancer (TNBC), as defined by the negative expression of the estrogen receptor (ER) and progesterone receptor (PR) as well as human epidermal growth factor receptor-2 (HER-2), represents approximately 15% to 20% of all invasive breast carcinomas. TNBCs are highly aggressive with large tumor mass, high nuclear grade, and increased lymph node involvement at the time of diagnosis, contributing to the highest risk of recurrence and metastasis of all breast cancer types at an early time. Particularly, because of the heterogeneity and no well-defined molecular targets, patients with metastatic TNBC have a poor 5-year survival despite standard treatment. It is urgent to discover new therapeutic targets that are involved in shaping the highly aggressive behavior of TNBC.

About Tri-I TDI
The Tri-I TDI connects researchers from Memorial Sloan Kettering Cancer Center, The Rockefeller University and Weill Cornell Medicine with collaborators from across the globe to remove barriers that impede drug discovery in academic settings. Tri-I TDI empowers the Tri-I community to advance their groundbreaking biological discoveries through preclinical studies by providing industrial-scale technical support for academic projects, making it possible to rapidly assess the utility of specific therapeutic targets in disease-relevant contexts. Working in close collaboration with Takeda Pharmaceutical Company, Ltd. and Bridge Medicines, Inc., Tri-I TDI accelerates the discovery of next generation drugs. For more information about Tri-I TDI, please visit www.tritdi.org or contact [email protected].

BioMarin Announces Second Quarter 2021 Financial Results and Corporate Updates

On July 28, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the second quarter ended June 30, 2021 (Press release, BioMarin, JUL 28, 2021, View Source [SID1234585271]).

"We enjoyed a strong first half of 2021, including financial results and the achievement of our key regulatory goals," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "With the positive CHMP opinion for VOXZOGO for children, 2 years of age and older, our commercial team is eagerly preparing for potential European approval and launch later this summer. If approved, VOXZOGO would be the first pharmacological treatment option for children with achondroplasia. Following potential approval in Europe later this summer, we hope to have a positive outcome in the United States pending our November 20 PDUFA target action date. Upon potential global approvals of VOXZOGO for achondroplasia, we have a tremendous opportunity to address the interest from families seeking treatment for their children."

Mr. Bienaimé continued, "We were pleased to receive validation of our MAA in Europe for valoctocogene roxaparvovec for the treatment of severe hemophilia A, paving the way for a CHMP opinion in the first half of 2022. Our data presentations at the International Society on Thrombosis and Haemostasis in July provided further evidence of hemostatic efficacy following treatment with valoctocogene roxaparvovec, including significant reductions in bleeding events and Factor VIII infusions. We look forward to two year data results from our complete Phase 3 study with 134 participants in early 2022, and anticipate submitting a BLA in the second quarter of 2022, should data be supportive. More than ever, we believe that valoctocogene roxaparvovec gene therapy will be an important treatment option for people with severe hemophilia A. Given the superior bleed control seen at one year with valoctocogene roxaparvovec, and the burden of frequent, regular infusions with standard of care prophylaxis, we believe one infusion of valoctocogene roxaparvovec gene therapy has the potential to address the unmet need for bleed control, supported by our clinical results to date."

Financial Highlights:

Total Revenues for the second quarter of 2021 increased to $501.7 million, compared to $429.5 million for the same period in 2020. The increase in Total Revenues was primarily attributed to the following:
Higher Vimizim and Naglazyme product revenues primarily driven by timing of orders from Europe and Middle East; and
Higher Palynziq product revenues primarily driven by a combination of revenue from more U.S. patients achieving maintenance dosing and new patients initiating therapy; partially offset by
Lower Kuvan product revenues primarily due to generic competition due to the loss of exclusivity in the U.S.
GAAP Net Income increased to $12.9 million for the second quarter of 2021 compared to a GAAP Net Loss of $29.2 million for the same period in 2020. The increase was primarily due to profits from higher revenues and lower research and development (R&D) expense driven by lower in-licensing expenses.
Non-GAAP Net Income for the second quarter of 2021 increased to $97.8 million compared to Non-GAAP Income of $57.4 million for the same period in 2020. The increase in Non-GAAP Income for the quarter, compared to the same period in 2020, was primarily attributed to higher gross profit partially offset by higher SG&A expense.
Commercial Portfolio (Naglazyme, Vimizim, Brineura, Palynziq, Kuvan and Aldurazyme)

Naglazyme, Vimizim and Brineura maintained robust patient compliance in the quarter and continue to grow based on strong underlying demand.
The number of patients on commercial Naglazyme and Vimizim therapy increased by over 10% each year-over-year.
The number of commercial patients on commercial Brineura therapy increased by over 30% year-over-year.
Order timing for both Naglazyme and Vimizim for the full year 2021 are expected to be more concentrated in the first half of the year.
Palynziq top-line results for the full year 2021 are expected to increase approximately 40% as compared to full-year 2020 results based on the mid-point of full-year 2021 guidance.
Palynziq growth in the European, Middle East and African regions (EMEA) has been impacted by ongoing challenges due to COVID-19 with Palynziq revenue from EMEA expected to increase when PKU clinics have more freedom to operate and start treating additional patients.
The number of U.S. patients on commercial Palynziq therapy increased approximately 30% year-over-year and is expected to continue to grow as U.S. PKU clinics are expected to re-open over the coming quarters.
Loss of Kuvan U.S. market share to generics was consistent with expectations.
Late-stage Regulatory Portfolio (VOXZOGO and valoctocogene roxaparvovec)

In Europe, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of VOXZOGO for the treatment of children, ages 2 years and older. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the third quarter of 2021.
The U.S. Food and Drug Administration (FDA) is reviewing the VOXZOGO New Drug Application (NDA) with the Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2021.
The European Medicines Agency (EMA) validated BioMarin’s MAA for valoctocogene roxaparvovec: CHMP opinion anticipated in the second quarter of 2022.
BioMarin is targeting a Biologics License Application (BLA) submission for valoctocogene roxaparvovec in the second quarter of 2022 assuming favorable results from the two-year follow-up safety and efficacy data from the GENEr8-1 study, followed by an expected 6-month review procedure by the FDA.
Earlier-stage Development Portfolio (BMN 307, BMN 255, BMN 331, DiNA-001, Allen Institute Collaboration)

BMN 307: Dose escalation in PHEarless, the Phase 1/2 study of BMN 307 continues based on encouraging Phe lowering and safety profile observed in study participants who were treated with the lowest dose.
BMN 255 for a subset of chronic renal disease: BMN 255 is a small molecule for the treatment of a subset of chronic renal disease. The Investigational New Drug application (IND) for BMN 255 is active and the Company is dosing subjects.
BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): BMN 331 is BioMarin’s third gene therapy candidate. BioMarin plans to leverage its broad expertise in developing gene therapies for severe hemophilia A and PKU to improve efficiencies in the development process of BMN 331. The IND for BMN 331 was recently cleared by FDA and is active.
BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies continue with BMN 351, an antisense oligonucleotide therapy that has demonstrated dystrophin expression levels of 30-50% of wild-type levels in the quadriceps in a DMD mouse model treated at 18.7 mg/kg/week for 13 weeks (measured 2 weeks following last administration). If results from the ongoing pre-clinical studies are supportive, BioMarin anticipates filing an IND for BMN 351 in the first half of 2022.
DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR received an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales.
On April 28, 2021, BioMarin and the Allen Institute announced a collaboration to create new gene therapies aimed at rare genetic diseases of the central nervous system (CNS). The goal is to combine the Allen Institute’s leadership in large-scale genomic science in the CNS therapeutic area with BioMarin’s expertise in developing transformational gene therapies.

BioMarin will host a conference call and webcast to discuss second quarter and full-year 2021 financial results today, Wednesday, July 28, 2021 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.