On 16, 2022 PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported the appointment of Meredith Manning to President of the Americas, effective immediately (Press release, PharmaEssentia, MAY 16, 2022, View Source [SID1234614674]).

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"PharmaEssentia has continued to build momentum with our strong pipeline and partnerships around the world. Now, with a world-class foundation built in the U.S. and launch underway, we are expanding our growth ambition across the American continents to address the clear gaps today in therapeutic options for the treatment of myeloproliferative neoplasms (MPNs)," said Ko-Chung Lin, PhD, co-founder and chief executive officer. "Meredith has built an incredible team of expert leaders and has established a strong infrastructure already in Boston, so they are well poised to build our presence with communities in both North and South America."

In this new scope, Ms. Manning will be supported by her U.S. leadership team to evaluate potential commercial strategies and partnerships to reach MPN patient communities throughout the American continents, with a near-term focus on Canada and Latin America. The company’s ongoing clinical trial SURPASS-ET, evaluating a potential therapy in the treatment of essential thrombocythemia (ET) has recently expanded into sites in Canada.

"The progress we’ve made in the last two years has been transformative for our mission and has established the right foundation to enable strong and sustainable growth as we expand into new markets," said Ms. Manning. "Together with our seasoned leadership team here in the U.S., I am optimistic that we can introduce our portfolio in areas that remain underserved today to help advance care of people with MPNs around the world."

Ms. Manning has served as General Manager for the U.S. subsidiary of PharmaEssentia since February 2020, expanding the team nearly three-fold since her arrival and leading the organization through the U.S. regulatory approval and commercial launch of the Company’s first product, BESREMi. With a sharp focus on data-driven market insights and commercial strategy, Ms. Manning’s career has been focused on introducing transformative therapies into underserved communities. She was previously Chief Commercial Officer at resTORbio, and prior to that served as a vice president of marketing for a seven-brand hemophilia portfolio at Baxter BioScience (now Takeda). Earlier in her career, Meredith built and managed successful commercial teams for Vertex and Pfizer.

On May 16,2022 Allarity Therapeutics, Inc. ("Allarity" or the "Company"), a clinical-stage pharmaceutical company developing novel oncology therapeutics together with drug-specific DRP companion diagnostics for personalized cancer care, reported financial results for the fourth quarter and year ended December 31, 2021, and provided a corporate update (Press release, Allarity Therapeutics, MAY 16, 2022, View Source [SID1234614753]).

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"2021 was a momentous year for Allarity Therapeutics," said Steve Carchedi, President and Chief Executive Officer. "We advanced our clinical-stage assets, raised a record amount of new investment for the Company, and completed our listing on the U.S. Nasdaq stock exchange. We are grateful for the collective support of our internal team, shareholders, and advisors, who are enabling the further development of our prioritized programs to address significant unmet needs within difficult-to-treat cancers. As we await further feedback from the United States Food and Drug Administration (FDA) regarding our NDA application for dovitinib, we remain steadfast in our mission to pair promising cancer therapeutics with our proprietary companion diagnostic technology to help significantly improve treatment outcomes for patients suffering from cancer. We look forward to sharing further updates on dovitinib and our other two priority clinical programs throughout the year."

A delay in filing the Company’s Form 10-K triggered a notification of non-compliance by Nasdaq concerning Listing Rule 5250(c)(1), as announced on April 22, 2022. Issuing a promissory note to Novartis to replace an unenforceable note issued by one of the Company’s Danish subsidiaries in 2018 and an error in the calculation of a tax Net Operating Loss allowance led to a restatement of the Company’s Form 10-Q dated September 30, 2021, and financial statements for the twelve months ended December 31, 2020, were the primary cause of the delay. The Company has filed the restated Form 10-Q for September 30, 2021, and its Form 10-K for 2021 today with the U.S. Securities and Exchange Commission (SEC). With these filings completed, the Company expects to regain compliance with Nasdaq Listing Rule 5250(c)(1) in the near future. The restatement of our financial statements for the year ended December 31, 2020, also resulted in a "Triggering Event" under our Certificate of Designations for our Series A Preferred Stock (the "Preferred Stock") because of the requirement to file a post-effective amendment to the Company’s Form S-1 registration statement that registers shares of our common stock issuable upon conversion of the Preferred Stock. As previously disclosed, the Investor that holds all of our Preferred Stock and the Company entered into a Forbearance Agreement and Waiver, dated April 27, 2022, providing the Company a forbearance period until June 4, 2022, in order to have the post-effective amendment declared effective by the SEC.

Full Year 2021 and Recent Highlights

In March 2022, entered into two agreements amending the LiPlaCis program. As a result of this amendment, Allarity will be exempt from any future financial obligation associated with the further development of LiPlaCis, including the cancellation of outstanding liability of $971million. Allarity will also maintain its ability to receive possible future milestone payments of up to $3.5 million
Scheduled a Type C meeting with the FDA, to be held in Q2 2022, to discuss potential paths to approval for dovitinib. This action followed the FDA’s issuance of Refusal to File (RTF) letters in February 2022 in response to the Company’s New Drug Application (NDA) and related Pre-Market Approval (PMA) application for dovitinib
Announced licensing agreements with Oncoheroes Biosciences, which will fund and advance the clinical development of both dovitinib and stenoparib in pediatric cancers utilizing Allarity’s DRP companion diagnostics
Completed listing on the U.S. Nasdaq Stock Market under the trading symbol "ALLR"
Received a $20 million PIPE investment from an institutional investor in conjunction with U.S. Nasdaq listing
Entered into an agreement with Lonza Group for the manufacturing of dovitinib to meet anticipated future commercial production needs
Entered, in July 2021, into an agreement for the sale of our irofulven program and assets to Lantern Pharma, Inc., under which Allarity received a $1 million upfront payment; the Company is eligible to receive an additional $1 million in payments plus up to U.S. $16 million in milestone payments from Lantern over the life of the program, in addition to royalties on future commercial net sales of the product. In Q1 2022, the Company received a fee payment from Lantern Pharma under the this agreement
Completed an oversubscribed rights issue, raising SEK 102.8 million ($12.1 million) before issue costs to further finance the development of Allarity’s three priority pipeline programs
Announced positive preclinical data in osteosarcoma in which dovitinib increased the median survival time in animals by 50% compared to the control group
Initiated a Phase 2 trial of IXEMPRA in Europe for the second-line treatment of metastatic breast cancer (mBC), with trial sites opened in a number of European countries
Expanded enrollment in an ongoing Phase 2 trial of stenoparib for the treatment of ovarian cancer (OC), with new trial sites opened or planned in the U.S. and Europe.

Anticipated Milestones in 2022

Announcing results of the Type C meeting with the FDA and outlining future development plans for dovitinib and its DRP-Dovitinib companion diagnostic
Initiation of prospective clinical study of dovitinib in metastatic Renal Cell Carcinoma together with its DRP-Dovitinib companion diagnostic
Interim data readout of Phase 2 clinical trial for IXEMPRA in mBC anticipated in Q4 2022
Interim data readout of Phase 2 clinical trial for stenoparib in OC anticipated in Q4 2022
Fourth Quarter and Full Year 2021 Financial Results

Balance Sheet: As of December 31, 2021, Allarity’s cash was $19.6 million, as compared to $298 thousand as of December 31 2020. This includes net proceeds from the Company’s December 2021 initial public offering in the U.S. The Company’s current cash, are expected to fund operations through 2022.

R&D Expenses: Research and Development (R&D) expenses were $9.5 million for the three months ended December 31, 2021, and $14.2 million for the full year 2021, compared to $1.7 million and $4.2 million for comparable periods in 2020, respectively.

G&A Expenses: General and Administrative (G&A) expenses were $6.2 million for the three months ended December 31, 2021, and $12.4 million for the full year 2021, compared to $856 thousand and $4.1 million for comparable periods in 2020, respectively.

Net Loss: Net loss was $16.8 million for the three months ended December 31, 2021, and $26.6 million for the full year 2021, compared to $1.7 million and $6.6 million for comparable periods in 2020, respectively.

On May 16, 2022 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, reported its financial results for the quarter ended March 31, 2022 and provided a clinical update of its anti-cancer drug candidates currently in development for the treatment of primary and metastatic brain and CNS cancer (Press release, CNS Pharmaceuticals, MAY 16, 2022, View Source [SID1234614643]).

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"Since the start of 2022, we have significantly expanded our international presence with clinical approvals in Spain, France and Switzerland received for our potentially pivotal study of Berubicin for the treatment of GBM. Once again our team demonstrated its operational, clinical and regulatory expertise this quarter. These efforts enable us to continue building momentum with patient enrollment – the cornerstone of any successful drug development program. The unmet need in GBM is enormous and knows no geographic borders and this critically important and state of the art trial will, most importantly, advance a much needed potential treatment option to patients. We have been and remain laser focused on executing on all of our operational efforts and look forward to an exciting year ahead," commented John Climaco, CEO of CNS Pharmaceuticals.

Clinical Programs Update

Berubicin – Novel anthracycline

CNS’ lead product candidate, Berubicin, is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier. Berubicin is currently being evaluated in a potentially pivotal global study evaluating its efficacy and safety in the treatment of GBM. The potentially pivotal global trial is an adaptive, multicenter, open-label, randomized and controlled study in adult patients with recurrent glioblastoma multiforme (WHO Grade IV) after failure of standard first-line therapy. Approximately 243 patients with GBM after failure of standard first line therapy will be randomized in a 2:1 ratio to receive Berubicin or lomustine for the evaluation of Overall Survival, the primary endpoint of the study. Overall Survival is a rigorous endpoint that the U.S. Food and Drug Administration (FDA) has recognized as a basis for approval of oncology drugs when a statistically significant improvement can be shown relative to a randomized control arm.

A pre-planned, non-binding futility analysis will be performed after approximately 30 to 50% of all planned patients have completed the primary endpoint at 6 months. This review will include additional evaluation of safety as well as secondary efficacy endpoints. Enrollment will not be paused during this interim analysis.

The FDA recently granted CNS Pharmaceuticals Fast Track Designation for Berubicin which enables more frequent interactions with the FDA to expedite the development and review process. As previously announced, the Company also received Orphan Drug Designation from the FDA which may provide seven years of marketing exclusivity upon approval of an NDA.

For more information about the potentially pivotal Berubicin trial, visit clinicaltrials.gov and reference identifier NCT04762069.

Upcoming Milestones

Continue to expand potentially pivotal study to evaluate efficacy of Berubicin in the treatment of adult GBM into additional countries;
Interim analysis of the trial when 30-50% of the total expected patients have been on study for 6 months (expected during first half of 2023); and
Complete enrollment in potentially pivotal clinical trial for GBM.
WP1244 Portfolio – Novel class of DNA-binding agents

The Company continues to advance the development of its WP1244 drug technology portfolio, which utilizes anthracycline and distamycin-based scaffolds to create small molecule agents and is believed to be 500x more potent than daunorubicin in inhibiting tumor cell proliferation. Preclinical studies of WP1244 demonstrated high uptake in the brain with antitumor activity. The Company’s development work has produced a new mesylate salt of WP1244, now identified as WP1874. The enhanced solubility of this salt may increase its ability to be formulated for use in an IV infusion, while maintaining similar potency and toxicity characteristics. Going forward, WP1874 will be the primary focus in our development efforts of the WP1244 portfolio. CNS Pharmaceuticals is also evaluating the use of WP1244/WP1874 in the treatment of other primary brain and central nervous system cancers, as well as cancers metastatic to the brain including pancreatic, ovarian, and lymphomas.

Upcoming Milestones

File IND in 2023.
Summary of Financial Results for the First Quarter 2022

The net loss for the three months ended March 31, 2022 was approximately $2.8 million compared to approximately $3.6 million for the comparable period in 2021. The change in net loss is primarily attributable to increased drug manufacturing activities in 2021 in preparation for the commencement of the Company’s clinical trials as well as the timing of annual employee incentive compensation, partially offset by an increase in contract research organization expenses as we are now actively conducting the trial of Berubicin.

The Company reported Research and development expenses of $1.5 million for the three months ended March 31, 2022 compared to approximately $2.2 million for the comparable period in 2021. The change in net loss is primarily attributable to increased drug manufacturing activities in 2021 in preparation for the commencement of the Company’s clinical trials, as well as by the payment of annual employee incentive compensation during the three months ended March 31, 2021 and not having been paid by March 31, 2022, offset by an increase in expenses during the three months ended March 31, 2022 related to contract research organization (CRO) activites in conducting our trial of Berubicin.

General and administrative expense was approximately $1.3 million for the three months ended March 31, 2022 compared to approximately $1.4 million for the comparable period in 2021. This change is primarily due to the payment of annual employee incentive compensation during the three months ended March 31, 2021 and not having been paid by March 31, 2022.

As of March 31, 2022, the Company had cash of approximately $12.4 million and working capital of approximately $13.7 million. In early January 2022, the Company completed an offering of common stock and warrants for gross proceeds of $11.5 million. The Company’s current expectation is that the cash on hand and the proceeds from the offering during January is sufficient to fund our operations into the first quarter of 2023. The timing and costs of clinical trials are difficult to predict and trial plans may change in response to evolving circumstances and as such the foregoing estimates may prove to be inaccurate.

On May 16, 2022 Galectin Therapeutics, Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, reported financial results and provided a business update for the three months ended March 31, 2022 (Press release, Galectin Therapeutics, MAY 16, 2022, View Source [SID1234614659]). These results are included in the Company’s Quarterly Report on Form 10-Q, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov.

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Dr. Pol Boudes, Chief Medical Officer, stated: "As we hear more about difficulties of reading and interpreting liver biopsies in pre-cirrhotic NASH, the feedback we get from our investigators reinforces our belief that using the prevention of esophageal varices as our primary outcome of efficacy in NAVIGATE is the appropriate efficacy outcome. Patients that are enrolled in our program have advanced to the cirrhotic stage of NASH and have developed portal hypertension, a severe complication of cirrhosis that impacts their prognosis. This also means that many of our patients have low platelet counts, and because this increases the risk of bleeding, it makes a liver biopsy far too dangerous. These are some of the reasons why we do not believe that biopsies are appropriate for patient selection or endpoints in our target population and is also why we even removed the requirement for baseline biopsies in our NAVIGATE trial. Preventing the development of an esophageal varix, on the other hand, is a very relevant and pragmatic clinical outcome. Unfortunately, too many cirrhotic patients bleed from these varices, and this can be a life-threatening event. Preventing the development of varices eliminates this potentially significant adverse outcome related to cirrhosis. We believe the design of the NAVIGATE study is truly innovative and allows us to move clinical research for liver cirrhosis forward."

Joel Lewis, Chief Executive Officer and President, said: "I am proud of our team and their accomplishments this quarter. Most importantly, as a Company, we achieved an extremely significant milestone. Our previous phase 2 trial, NASH-RX, indicated a favorable safety profile for belapectin over one year of treatment. As of today, due to the length of our adaptively designed phase 2b/3 NAVIGATE trial, our safety profile has been further evaluated by an independent data safety monitoring board who recommended the trial continue as designed. Additionally, our innovative trial design allows trial participants to move directly into the phase 3 treatment course for an additional 18 months. As these and additional patients continue receiving treatment, we continue to expand our data on the safety profile of belapectin. The significance of this safety profile in a severely compromised patient population cannot be overstated.

"We continued to make progress towards our primary goal of completing enrollment in our adaptively designed Phase 2b/3 NAVIGATE trial for the prevention of esophageal varices in patients with NASH cirrhosis. Our strategy to further expand our trial sites in Mexico and Latin America is well underway. We recently conducted a very productive in-person investigator meeting in Mexico where we have added more than 10 new sites. I attended the meeting with my clinical operations staff, and we are extremely enthusiastic about the ability of these sites to quickly enroll patients. I am grateful to all of our investigators and their teams, as well as our consultants in Mexico, for their time and dedication to our trial. Enrollment in the United States continued steadily; however, enrollment in Europe still lags far behind our expectations. We currently expect enrollment to conclude for the Phase 2b portion around the end of the third quarter of this year."

Mr. Lewis continued: "Additionally, we are making progress and are working to compile an Investigational New Drug (IND) package, including the development of a phase 2 trial protocol, with the objective for the Company to file an IND with the FDA oncology division for the treatment of recurrent or metastatic head and neck cancer for belapectin in combination with Keytruda, an immune checkpoint inhibitor. The lack of current treatments for these patients, the low response rates of monotherapy with check-point inhibitors, the limited number of therapies in development, and the resulting very high medical need make this an important area for new combination therapies."

Financial Results

For the three months ended March 31, 2022, the Company reported a net loss applicable to common stockholders of $9.9 million, or ($0.17) per share, compared to a net loss applicable to common stockholders of $6.3 million, or ($0.11) per share for the three months ended March 31, 2021. The increase is largely due to an increase in 2022 research and development expenses related to the Company’s NAVIGATE trial.

Research and development expenses for the three months ended March 31, 2022, was $8.1 million compared with $4.9 million for the three months ended March 31, 2021. The increase was primarily due to costs related to our NAVIGATE clinical trial and other supportive activities. General and administrative expenses for the three months ended March 31, 2022, were $1.9 million, compared to $1.4 million for the three months ended March 31, 2021. The increase was primarily due to non-cash stock-based compensation expense.

As of March 31, 2022, the Company had $31.6 million of cash and cash equivalents. The Company believes it has sufficient cash to fund currently planned operations and research and development activities through at least May 16, 2023.

The Company expects that it will require more cash to fund operations after May 16, 2023, and believes it will be able to obtain additional financing as needed. Currently, we expect to require an additional approximately $40-$45 million to cover costs of the NAVIGATE trial to reach the planned interim analysis estimated to occur in mid-2024, along with drug manufacturing and other research and development activities and general and administrative costs. However, there can be no assurance that we will be successful in obtaining such new financing or, if available, that such financing will be on terms favorable to us.

About Belapectin

Belapectin is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of NASH and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs, including fibrotic disorders of the liver, lung, kidney, heart and vascular system. Belapectin binds to galectin-3 and disrupts its function. Preclinical data in animals have shown that belapectin has robust treatment effects in reversing liver fibrosis and cirrhosis. A Phase 2 study showed belapectin may prevent the development of esophageal varices in NASH cirrhosis, and these results provide the basis for the conduct of the NAVIGATE trial. The NAVIGATE trial (www.NAVIGATEnash.com), titled "A Seamless Adaptive Phase 2b/3, Double-Blind, Randomized, Placebo-controlled Multicenter, International Study Evaluating the Efficacy and Safety of Belapectin (GR-MD-02) for the Prevention of Esophageal Varices in NASH Cirrhosis," began enrolling patients in June 2020, and is posted on www.clinicaltrials.gov (NCT04365868). Galectin-3 has a significant role in cancer, and the Company has supported a Phase 1b study in combined immunotherapy of belapectin and KEYTRUDA in advanced melanoma and in head and neck cancer. This trial provided a strong rationale for moving forward into a Company-sponsored Phase 2 development program, which the company is exploring.

About Fatty Liver Disease with Advanced Fibrosis and Cirrhosis

Non-alcoholic steatohepatitis (NASH) has become a common disease of the liver with the rise in obesity and other metabolic diseases. NASH is estimated to affect up to 28 million people in the U.S. It is characterized by the presence of excess fat in the liver along with inflammation and hepatocyte damage (ballooning) in people who consume little or no alcohol. Over time, patients with NASH can develop excessive fibrosis, or scarring of the liver, and ultimately liver cirrhosis. It is estimated that as many as 1 to 2 million individuals in the U.S. will develop cirrhosis as a result of NASH, for which liver transplantation is the only curative treatment available. Approximately 9,000 liver transplants are performed annually in the U.S. There are no drug therapies approved for the treatment of liver fibrosis or cirrhosis.

On MAY 16, 2022 CG Oncology, Inc., a clinical-stage biotechnology company focused on developing oncolytic immunotherapies for patients with advanced cancer, reported that interim results from the global Phase 2 study (CORE1) of CG0070 in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab), for the treatment of patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) unresponsive to Bacillus Calmette-Guerin (BCG) (Press release, CG Oncology, MAY 16, 2022, View Source [SID1234614675]).

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The results (Session MP54-03) were presented at the 2022 American Urological Association (AUA) Annual Meeting. The preliminary data adds to that presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) earlier this year and continues to show both promising early anti-tumor activity and tolerability of CG0070 in combination with pembrolizumab for patients with BCG unresponsive NMIBC.

Summary of Interim Clinical Results

As of the interim analysis, based on a data cutoff on April 28, 2022, 22 patients were evaluable for efficacy with a minimum of 3 months follow up.
91% of patients evaluable for efficacy (n=20/22) have achieved complete response (CR) at the initial 3-month timepoint. Of those patients evaluable for CR at additional timepoints, 87% (n=15) have also maintained a CR through 6 months, 80% (n=10) through 9 months and 75% (n=8) at the 12-month assessment.
Treatment related adverse events were generally limited to transient grade 1-2 local genitourinary symptoms including pollakiuria, bladder spasm, dysuria, fatigue, nocturia, hematuria, chills, and immune-related adverse events including hyperglycemia and hypothyroidism.
About the CORE1 Study

Under a previously announced clinical collaboration with Merck (known as MSD outside the US and Canada) relating to the investigation of CG0070 used in combination with pembrolizumab, the goal of CORE1, which will enroll up to 35 patients, is to evaluate the safety and efficacy of CG0070 plus pembrolizumab for the treatment of NMIBC unresponsive to BCG.

More information about the study can be found at www.clinicaltrials.gov (NCT04387461).

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About CG0700

CG0070, a selective oncolytic immunotherapy based on a modified adenovirus type 5 backbone that contains a cancer-selective promoter and a GM-CSF transgene, destroys bladder tumor cells through their defective retinoblastoma (Rb) pathway. CG0070 was designed to replicate inside tumor cells with dysfunctional Rb pathways, causing tumor cell lysis and immunogenic cell death. The rupture of cancer cells releases tumor-derived antigens and GM-CSF, which stimulates a systemic anti-tumor immune response. In advanced clinical trials, CG0070 is a safe and efficacious agent in NMIBC following BCG failure. CG0070 is currently in late-stage clinical trials across a variety of solid cancers, as a monotherapy or in combination with immune checkpoint inhibitors.