On November 19, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that new animal data has shown highly improved activity against acute myeloid leukemia ("AML") when used in combination with the commonly used antileukemic drug Ara-C (also referred to as "cytarabine") versus single agent (Press release, Moleculin, NOV 19, 2020, View Source [SID1234571425]). The data is being presented at the 62nd Annual Meeting & Exposition of the American Society for Hematology ("ASH") under the title: "High Efficacy of Liposomal Annamycin (L-ANN) in Combination with Cytarabine in Syngeneic p53-null AML Mouse Model."

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This study was conducted in a highly aggressive AML mouse model where median survival is approximately 13 days. For animals treated with the combination of Annamycin and Ara-C, median survival ranged from 56 to 76 days, thus expanding median survival by 585%, with some animals being completely cured. The conclusion of the study is that these experiments support initiation of clinical development of the combination of Annamycin and Ara-C in AML patients.

"This is a very important discovery that will most likely change the course of development for L-Annamycin," commented Walter Klemp, Chairman and CEO of Moleculin. "While our current AML trials are encouraging and we are seeing significant activity with Annamycin as a single agent in relapsed AML patients, this data makes a compelling case that we should move as quickly as possible to begin a clinical trial in AML for the combination of Annamycin with Ara-C, something we are calling ‘AnnAraC.’ We believe the future for Annamycin just became even more promising."

Mr. Klemp concluded: "Annamycin has already shown human activity as a single agent in its two Phase 1 AML clinical trials, including one complete response, and showing no signs of cardiotoxicity, unlike other anthracyclines. And, it now appears, based on the observed synergy in vitro and confirmatory in vivo data, that the combination of Annamycin and Ara-C could be more effective in a clinical setting than Annamycin as a single agent. This would be consistent with current practice to use Ara-C in combination with an anthracycline like Annamycin. The current first-line therapy for AML patients is the combination of an anthracycline and Ara-C in a regimen referred to as "7+3" where Ara-C is administered daily for 7 days in parallel with 3 daily doses of an anthracycline. Simply substituting the currently used anthracycline in a similar 7+3 regimen with Annamycin would represent a familiar and well-practiced treatment modality. Beyond that, it would have the added advantages that Annamycin is active against tumor cells resistant to doxorubicin and, importantly, removes the concern for cardiotoxicity, a significant toxic side effect of currently used anthracyclines."

The study abstract, as accepted by ASH (Free ASH Whitepaper), can be viewed at: View Source

On November 19, 2020 Oncopeptides reported that (Press release, Oncopeptides, NOV 19, 2020, View Source [SID1234571424])

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Financial overview July 1 – September 30, 2020
Net sales amounted to SEK 0.0 M (0.0)
Loss for the period was SEK 383.4 M (loss: 189.8)
Loss per share, before and after dilution, was SEK 5.71 (loss: 3.53)
On September 30 cash and cash equivalents amounted to SEK 1,251.6 M (1,122.3)
Significant events during the period July 1 – September 30, 2020
The FDA granted priority review of melflufen for patients with triple-class refractory multiple myeloma and set the PDUFA date to February 28, 2021
Patient enrolment in the pivotal phase 3 OCEAN study was completed including 495 patients
Patient enrolment for the phase 1/2 study in AL-amyloidosis began, this is the first study with melflufen in an indication outside multiple myeloma
The phase 2 PORT study evaluating alternative administration of melflufen and dexamethasone in multiple myeloma started
Oncopeptides further coordinated the global and US organizational structure and appointed Mohamed Ladha as General Manager of the US Business Unit
Significant events after the reporting period
Oncopeptides announced that the company intends to submit a conditional marketing authorization application for melflufen in the EU
Oncopeptides entered into a €40 M loan agreement with the European Investment Bank (EIB)
An IND application was submitted to the FDA to initiate clinical studies with OPD5, Oncopeptides’ second drug candidate

This information is information that Oncopeptides is obliged to make public pursuant to the EU Market Abuse Regulation and the Securities Markets Act. The information was submitted for publication, through the agency of the contact persons set out above, at 08:00 CET on November 19, 2020.

On November 19, 2020 Invitae (NYSE: NVTA), a leading medical genetics company, reported that study findings that show nine percent of patients with pancreatic cancer had genetic changes in DNA damage repair (DDR) genes that would make them eligible for PARP inhibitor therapy or clinical treatment trials (Press release, Invitae, NOV 19, 2020, View Source [SID1234571423]). Despite professional guidelines that recommend testing for all pancreatic cancer patients, it remains underutilized in routine care. The study was presented at the National Society of Genetic Counselors 39th Annual Conference.

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"New therapeutics have recently become available to treat pancreatic cancer for patients with certain changes in genes such as BRCA1 and BRCA2. Yet despite the availability of these treatments and professional guidelines recommending testing, utilization is still lagging," said Robert Nussbaum, M.D., chief medical officer of Invitae and study author. "Pathogenic variants in these genes are associated with an increased risk of other cancers as well, such as breast, ovarian and prostate cancer, which means that a failure to test patients with pancreatic cancer impacts not only their treatment, but also the health of their families."

Importantly, the study of over 2,000 patients found that 15% of patients with actionable genetic changes reported no family history of cancer, which underscores the limitations of using testing criteria based on reported family history. National Comprehensive Cancer Network (NCCN) guidelines recommend genetic counseling and germline genetic testing for everyone diagnosed with pancreatic cancer as well as their first degree relatives — approximately 3.5 million individuals in the United States.

In addition to evaluating the clinical relevance of genetic testing results, the study offered sponsored, no-charge testing to patients to evaluate the role of cost as a barrier to testing. Researchers found a small but significant increase (2%) in testing among African-American patients compared to typical rates among patients using health insurance, suggesting reducing cost may increase access to testing among this population.

The research was presented at the virtual annual meeting of the National Society of Genetic Counselors. The full research presentation from Invitae included:

Oral platform presentations:

Increasing access for patients with pancreatic cancer to germline genetic testing: Clinical impacts across disease stage and ethnicity. Presented by Ed Esplin, MD, PhD, FACMG, FACP
Evaluating variant reclassification in reproductive carrier screening. Presented by Julia Wilkinson, MS, LCGC
Poster presentations:

Utility of adding phenotypic criteria refinement to ACMG guidelines. Presented by Lauren Frank, MS, CGC
Comprehensive germline multigene panels change clinical care and inform treatment strategies for breast cancer patients. Presented by Sarah Nielsen, MS, LCGC
Carrier screening for X-linked conditions is common practice. Presented by Dana Neitzel, MS, CGC
In addition to its scientific presence, Invitae will again partner with NSGC to present the Heart of Genetic Counseling award, which honors excellence in genetic counseling and patient care as recognized by patients. Nominations include stories from patients that highlight both the clinical and personal impact a genetic counselor had on their lives and the lives of their families. This year’s award will be presented during a virtual ceremony on Thursday, November 17th. The finalists include:

Rachelle Manookian, MS, LCGC, City of Hope in Duarte, CA, was nominated by a man who had been searching for answers about the origin of his cancer for more than 10 years. He hoped that any identified mutations would lead to a newly developed drug or clinical trial that could attack his cancer with precision. Manookian left no stone unturned and remained in touch until she found a clinical trial. Thanks to Manookian’s persistence, he finally got answers and learned that his sons did not carry the same increased genetic risk of prostate cancer.
Donna McDonald-McGinn, MS, LCGC, Children’s Hospital of Philadelphia in Philadelphia, was nominated by the mother of a son with a rare genetic condition called 22q11.2 deletion syndrome. McDonald-McGinn has been his care coordinator and navigator for 21 years, explaining and managing the care that he has needed through his life. Always acting as his advocate, McDonald-McGinn has been there for each of his 21 surgeries and is a staunch advocate for those with 22q11.2 deletion syndrome.
Gretchen MacCarrick, MS, CGC, Johns Hopkins Medicine in Baltimore, was nominated by a mother whose daughter has Loeys-Dietz syndrome (LDS), which was discovered in 2005. For nearly 12 years, MacCarrick has been by their side guiding them. She explains what tests are needed, what the results mean and navigates their daughter’s care during their regular visits to Baltimore, including a major heart surgery. Importantly, MacCarrick has even developed a strong relationship directly with the patient who is now 16 and getting more engaged in her care.

On November 19, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported a clinical update on omburtamab for the treatment of diffuse intrinsic pontine glioma ("DIPG") (Press release, Y-mAbs Therapeutics, NOV 19, 2020, View Source [SID1234571422]). Data was presented at the Society for Neuro-Oncology ("SNO") Virtual Annual Meeting held November 19 through November 21, 2020. The omburtamab data was presented by Dr. Evan Bander from Weill Cornell Medicine.

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In a poster presentation, Dr. Bander presented infusion related data from the ongoing Phase 1 study in DIPG. The study showed the possibility of repeated convection enhanced delivery ("CED") infusions into the pediatric brainstem. Past CED treatments did not negatively influence the procedural workflow, technical application of the targeted interface, accuracy of catheter placement or distribution capacity. In the study, seven patients underwent two or more sequential CED infusions.

"The potential to retreat DIPG patients with multiple doses of omburtamab is truly exciting. We are planning for a multicenter Phase 2 study in DIPG, and hope to utilize this new insight to optimize the outcome for DIPG patients, who represent a significant unmet medical need," said Thomas Gad, founder, Chairman and President.

Researchers at Memorial Sloan Kettering Cancer Center ("MSK") developed omburtamab, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests related to the compound and Y-mAbs.

On November 19, 2020 Teneobio Inc., a clinical stage biotech company focused on discovery and development of novel multi-specific biotherapeutic antibodies, reported the presentation of interim clinical data from its Phase 1 trial evaluating TNB-383B in relapsed/refractory multiple myeloma (R/R MM) at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, which will be held virtually Dec. 5-8, 2020 (Press release, TeneoBio, NOV 19, 2020, View Source;utm_medium=rss&utm_campaign=tnb-383b-anti-bcmaxcd3 [SID1234571421]). TNB-383B is a fully human bispecific antibody that targets BCMA on the surface of multiple myeloma (MM) cells and CD3 on the surface of T cells in order to trigger lysis of MM cells in R/R MM patients. The open-label multi-center trial is designed to assess the safety, pharmacokinetics and preliminary efficacy of TNB-383B administered intravenously once every 21 days.

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The interim results will be presented on Saturday Dec. 5 at 2:45 pm PT in an oral presentation by Cesar Rodriguez, M.D. (Wake Forest Baptist Health) titled "Interim Results of a Phase I Study of TNB-383B, a BCMAxCD3 T-cell Redirecting Antibody in Relapsed/Refractory Multiple Myeloma". The presentation will summarize interim data from 58 patients receiving escalating doses from 0.025 mg – 60 mg. Since the study is still ongoing, 81% of responders continue on treatment. Early results show an improved safety and efficacy profile.

"We are very pleased with the clinical data described in this presentation. It is encouraging to see the clinical benefit driven by T-cell mediated tumor lysis without the accompanying safety signals related to cytokine release that are typically associated with immune-activating therapies. The dosing schedule of once every three weeks allows for reduced clinic visits compared to other BCMAxCD3 bispecifics in development. Overall, we are excited to see the unique attributes of Teneobio’s CD3-engager platform validated in the clinic. We look forward to continued development of TNB-383B as well other bi-specific antibodies in our pipeline," said Ben Buelow, M.D., Ph.D., Chief Medical Officer of Teneobio.