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Aptorum Group Announces the Development of Microbiome Drug Candidate Targeting Obesity and Repurposed Drug Candidates Targeting Neuroblastoma

On September 9, 2019 Aptorum Group Limited (Nasdaq: APM) ("Aptorum Group"), a biopharmaceutical company focused on the development of novel therapeutics to address global unmet medical needs, reported the development of two preclinical drug candidates which target obesity and neuroblastoma, respectively (Press release, Aptorum, SEP 9, 2019, View Source [SID1234539378]).

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About CLS-1: Treatment of obesity via modulation of chemical signaling relating to gut microbiota

Under the recently-announced microbiota modulation platform operated by Aptorum Group’s wholly-owned subsidiary Claves Life Sciences Limited, we have commenced the preclinical development of macromolecule candidate CLS-1 targeting the treatment of obesity. CLS-1 is undergoing lead optimization and is expected to progress into the IND enabling stage in 2020.

The prevalence of obesity continues to escalate globally; however, there is no current optimal therapy for this condition1. For the majority of obese patients, conventional medical therapies (i.e., diet, exercise, behavioral counseling) often have a high failure rate for the long term2. We believe current pharmacotherapy has limited efficacy and is associated with substantial safety issues, and this will provide immeasurable market opportunity for CLS-1.

Chemical signaling of gut microbiota is known to be one of the major causes of obesity1. CLS-1 is an orally administered non-absorbable macromolecule that modulates the metabolite excreted by gut microbiota with high affinity and specificity. In this way, we believe the absorption of this particular metabolite, which is linked to obesity, can be inhibited.

Aptorum Group is also pursuing two further indications based on the modulation of microbiota based chemical signaling involving the above large molecule technology, which we believe to be highly scalable and we hope to be making further announcements regarding our efforts in due course.

About SACT-1: Repurposed Drug Candidates for the Treatment of Neuroblastoma

Under the recently announced Smart-ACTTM computational drug discovery platform operated by our wholly-owned subsidiary Smart Pharma Group, Aptorum Group has completed computational screening of approximately 1,615 marketed drugs against 3 therapeutic target proteins to potentially tackle poor prognosis of neuroblastoma, i.e., a rare type of children’s cancer that forms in certain types of nerve tissue and most frequently in the adrenal glands as well as spine, chest, abdomen or neck, especially in children3. For the high-risk group, the 5-year survival rate of this condition is around 40-50% as observed by the American Cancer Society.4 Aptorum Group has identified an array of repurposed candidates and has proceeded to evaluate them in cell-based and animal models in order to validate the candidates’ usage for such new indication and potential efficacies.

Aptorum Group is also pursuing two further indications under the Smart-ACTTM drug discovery platform and hopes to be making further announcements regarding our research in due course.

MATEON PARTNERS WITH WIDETRIAL FOR DATA-GENERATING EXPANDED ACCESS PROGRAMS IN CANCER

On September 9, 2019 Mateon Therapeutics Inc. (OTCQB:MATN), reported that it has executed agreements with WideTrial Inc. for an Expanded Access program (EAP) in pancreatic cancer (Press release, Mateon Therapeutics, SEP 9, 2019, View Source [SID1234539377]). Using WideTrial’s novel platform, the investigational therapeutic OT-101 will be made available for elective treatment-use to patients who do not meet the inclusion criteria of OT-101 research trials. The partnership may also support Expanded Access for OT-101 in additional patients with advanced solid tumors, excluding brain cancers (AA, GBM, and other gliomas). Separately, the companies executed data access agreements to support the development of Mateon’s AI/Blockchain technologies with WideTrial’s data assets in cancer and other therapeutic areas.

OT-101 is a first-in-class RNA therapeutic targeting TGF beta and is the lead immune-oncology drug candidate of Oncotelic, a wholly owned subsidiary of Mateon Therapeutics Inc.. The investigational drug exhibited single-agent activity in relapsed/refractory cancer patients during multiple Phase 2 clinical trials.

Like research trials, cohort Expanded Access programs (EAPs) are centrally monitored trials that enroll patients at designated sites under a single protocol. They differ from research trials in that their primary objective is to provide the option of treatment-use to patients who cannot take part in the product’s research trials.

"This Expanded Access program will allow us to meet the spirit of clinical trial inclusivity, as described in the 2017 FDA Reauthorization Act. We are pleased to have found a viable way to accommodate greater numbers of patients in need and the doctors who seek to treat them," said Dr. Vuong Trieu, Chairman and CEO of Mateon Therapeutics Inc.. "Though the primary objective is treatment, we intend to utilize generated data to improve the power and targeting of future research trials."

"The PointR platform applies cutting edge algorithms to clinical datasets and could be used to quickly and cost-effectively identify the best therapy tailored fit to a single individual. The ability to access EAP data is helpful to the development of this platform" said Saran Saund, CEO of PointR Data, which is becoming a wholly owned subsidiary of Mateon Therapeutics Inc.. "We are thrilled at the opportunity of vertically integrating artificial intelligence and drug development capabilities under one roof to quickly identify promising new therapeutic opportunities for various diseases and to deliver compelling business value."

"We built WideTrial to solve the economic and operational barriers that drug developers face with Expanded Access," said Jess Rabourn, CEO of WideTrial. "The new platform enables larger numbers of gravely ill patients to choose to try investigational medicines under a properly designed protocol. Although treatment is the primary objective, EAP sponsors can learn a lot from patients’ experiences in these programs."

PHIO PHARMACEUTICALS ANNOUNCES RESEARCH COLLABORATION WITH CARISMA THERAPEUTICS TO EVALUATE ITS SELF-DELIVERING RNAI TECHNOLOGY TO ENHANCE MACROPHAGE-BASED ADOPTIVE CELL THERAPY

On September 9, 2019 Phio Pharmaceuticals Corp. (NASDAQ: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported that it has entered into a research collaboration with Carisma Therapeutics to evaluate the potential of Phio’s self-delivering RNAi compounds to synergistically modify Carisma’s chimeric antigen receptor macrophages (CAR-M) (Press release, Phio Pharmaceuticals, SEP 9, 2019, View Source [SID1234539376]). Silencing of key genes using sd-rxRNA compounds may enhance the immune function of these cells as a novel adoptive cell therapy for use in cancer treatment.

"Overcoming local tumor immunosuppression remains a significant challenge in the treatment of cancer," said Dr. John A. Barrett, Chief Development Officer of Phio Pharmaceuticals. "We have established collaborations with several companies and academic institutions to validate our technology’s ability to improve the activity of T cells, TILS, NK cells and dendritic cells towards tumors. Macrophages are sentinel cells of the innate immune system that robustly accumulate in most solid tumors. Combining Phio’s gene silencing technology with Carisma’s CAR-M technology has the potential to enhance aspects of macrophage anti-tumor activity and may further tip the scale in favor of an immunostimulatory phenotype and enhance tumor cell killing. We believe we can leverage both technologies to turn cold tumors hot and ultimately improve outcomes for patients."

Dr. Gerrit Dispersyn, Phio Pharmaceuticals’ President and CEO stated "We are very excited to work with the Carisma Therapeutics team, which represents our first collaboration addressing macrophages utility as an immunotherapeutic. By combining Carisma’s unique approach to adoptive cell therapy using CAR macrophages with our self-delivering RNAi technology, we expect to demonstrate potential synergies between our platforms that could enable us to bring new and improved forms of adoptive cell therapy to patients in the future. Our overall strategy in this setting is to generate robust data that validate our platform for the weaponization of a broad range of immune cells against tumors in order to generate future partnerships and build shareholder value."

"Metastatic solid tumors have been an intractable challenge for engineered cell therapies. Our platform technology, based on the genetically engineered macrophage, has a unique opportunity to provide benefit in this disease state," said Dr. Michael Klichinsky, co-Founder and Vice President of Discovery at Carisma. "We are excited to work with Phio to evaluate sd-rxRNA mediated silencing of genes that have the potential to enhance macrophage immunotherapy."

Altimmune Appoints M. Scott Harris, M.D. as Chief Medical Officer

On September 9, 2019 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage biopharmaceutical company, reported it has appointed M. Scott Harris, M.D. as Chief Medical Officer (Press release, Altimmune, SEP 9, 2019, View Source [SID1234539375]). Dr. Harris is succeeding Dr. Sybil Tasker, who resigned June 30, 2019 to continue her professional focus on infectious diseases.

"Dr. Harris is a seasoned medical professional with extensive experience in hepatology and gastroenterology and broad expertise in managing clinical trials from early stage development through successful Phase 3 trials. He has led multidisciplinary forums on drug development and clinical trial design at national and international scientific meetings, and fostered collaborations between professional medical societies and the FDA. We are thrilled that a candidate of Dr. Harris’ caliber is joining the Altimmune team," said Vipin K. Garg, Ph.D., President and Chief Executive Officer. "We expect his clinical leadership to be pivotal to our success, and his appointment is timely as we are currently designing our ALT-801, HepTcell, and NasoShield clinical trials."

Dr. Harris added, "I am delighted to be joining Altimmune, and was attracted to the opportunity based on the Company’s pipeline and strong balance sheet. My background in liver and GI drug development especially complements Altimmune’s liver disease portfolio and I look forward to guiding these programs through the clinic. This is an exciting time to join Altimmune."

Dr. Harris is an accomplished drug development scientist who has led numerous global clinical studies and programs in GI and liver diseases. Previously, he was co-founder and Chief Medical Officer of Lyric Pharmaceuticals, helping raise a $21 million Series A round in 2014. He has also served as Chief Medical Officer of Avaxia Biologics, interim Chief Medical Officer of Tranzyme Pharma, and Chief Medical Officer of Ocera Therapeutics. Dr. Harris was also Chief Medical Officer and Vice President of Clinical Affairs at Napo Pharmaceuticals where he authored the pivotal clinical study that led to the approval of crofelemer (Mytesi), the first Phase 2/3 adaptive trial design resulting in a drug approval. Earlier in his career he held senior roles in global clinical development and medical affairs at Otsuka Pharmaceuticals and Abbott. He sits on the faculty of Georgetown University School of Medicine as an Adjunct Professor, where he directs a course on drug development under a grant from the NIH. Dr. Harris has been a consultant on third-world drug development for the Bill and Melinda Gates Foundation and a speaker at national and international forums on drug development. Dr. Harris has an M.D. from Harvard Medical School and an MS in Administrative Medicine and Population Health from the University of Wisconsin Medical School. His post-graduate training includes residencies at John Hopkins Hospital and the University of Pennsylvania, and a Gastroenterology and Hepatology Fellowship at the Yale University School of Medicine.

PharmaMar presents new lurbinectedin data at the World Conference on Lung Cancer

On September 9, 2019 PharmaMar (MSE:PHM) reported new data on lurbinectedin during the World Conference on Lung Cancer (WCLC), which is taking place from 7th to 10th September in Barcelona (Press release, PharmaMar, SEP 9, 2019, View Source [SID1234539374]). The Conference, organized by the International Association for the Study of Lung Cancer (IASLC), brings together the world’s leading experts on this pathology and presents the latest advances in its treatment.

At this conference, PharmaMar presented two posters on lurbinectedin for the treatment of Small Cell Lung Cancer (SCLC).

The first of them evaluates new combinations with lurbinectedin for the treatment of this type of tumor. The poster entitled "Lurbinectedin (L) Combined with Paclitaxel (P) or Irinotecan (I) in Relapsed SCLC. Results from Two Phase Ib Trials (Abstract 1588)" shows the results of a Phase Ib trial in which combinations of lurbinectedin with paclitaxel or irinotecan were evaluated in patients with relapsed SCLC. Both combinations have shown similar activity and a predictable and manageable safety profile. The combination of lurbinectedin and irinotecan has shown encouraging activity in the third line treatment of patients. Also of interest, the combination of lurbinectedin and paclitaxel has shown higher activity in resistant patients (CTFI<90 days – patients for whom time from the last dose of first-line chemotherapy to the occurrence of progressive disease is less than 90 days).
These results warrant further evaluation of the combinations of lurbinectedin with paclitaxel or irinotecan. The expansion cohort of the irinotecan combination is already ongoing.

The second poster, entitled "Antitumor Activity of Single Agent Lurbinectedin in Patients with Relapsed SCLC Occurring ≥30 Days After Last Platinum Dose (Abstract 1710)" shows data from SCLC cohort of the Phase II Basket trial of lurbinectedin as a single agent, (presented in an oral session at the last ASCO (Free ASCO Whitepaper) Congress). Data are shown for the subset of 84 patients with a CTFI≥30 (time from the last dose of first-line chemotherapy to the occurrence of progressive disease is longer than or equal to 30 days). In this group of patients, the Overall Response Rate (ORR) was 40.5%. For the resistant patients with a CTFI of 30-89 days, the ORR was 29.2%, for whom no currently approved treatment exists. For the 60 patients that were sensitive (CTFI≥90 days; patients for whom time from the last dose of first-line chemotherapy to the occurrence of progressive disease is longer than or equal to 90 days), the ORR was 45%.

As for the safety of the compound in this group of patients, lurbinectedin has shown a safety profile that was acceptable and well tolerated. The most common treatment-related adverse effect has been neutropenia, and no unexpected toxicities have been observed.

During the Congress, Dr. Camilla L. Christensen, from Harvard University, will present in an oral session on transcription as a target in the treatment of SCLC, where, among others, the mechanism of action of lurbinectedin will be discussed. Lurbinectedin is a selective inhibitor of the oncogenic transcription programs on which many tumors are particularly dependent.