On May 24, 2021 AMSURG, a division of Envision Healthcare and a national leader in outpatient care, and Icahn School of Medicine at Mount Sinai, world-renowned for its groundbreaking research, reported that new findings on colorectal cancer (CRC), which support several recommendations to lower the screening age from 50 to 45 for people who are at average risk for CRC (Press release, Mount Sinai Hospital, MAY 24, 2021, View Source [SID1234580499]). The findings were presented at the Digestive Disease Week 2021 virtual scientific meeting on May 23.

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The Mount Sinai team analyzed approximately 3 million colonoscopies performed at more than 120 AMSURG ambulatory surgery centers across the country in the last six years. The study specifically looked at de-identified data from patients aged 18 to 54 who received a screening or diagnostic colonoscopy and were not undergoing a colonoscopy to monitor previously detected polyps, cancer, inflammatory bowel disease or genetic conditions predisposing to cancer. Results show:

Colorectal cancer was detected in 0.58 percent of patients aged 45 to 49 and in 0.53 percent of patients aged 40 to 44
Polyps that had the greatest possibility of becoming cancerous were found in 7.5 percent of patients aged 45 to 49 and in 5.8 percent of patients aged 40 to 44
A significant portion of patients had polyps present even if they did not have a documented family history of colorectal cancer
"These findings are significant and can make a meaningful difference in the healthcare system’s ability to save patients’ lives. With early and routine colonoscopies, we can prevent polyps from becoming cancerous and even detect and remove cancerous polyps while giving patients a better chance of recovery and survival. These data support efforts to begin screening at age 45 and communicate the importance of on-time screening by early messaging to patients and providers," said John Popp, MD, Medical Director for AMSURG.

Colorectal cancer is the second leading cause of cancer death in the U.S. and one of the most preventable. The incidence of CRC in patients under 50 has nearly doubled since the early 1990s.1 Consensus in the medical community is building that patients who are at average risk for CRC should begin screening at age 45.

"This study can inform additional research directed at CRC screening in younger populations. More importantly, it helps physicians inform patients about the importance of early screening several years before they reach screening age; we believe that this will encourage younger patients to get routine screenings on time, at the appropriate age, and with close attention to the individual’s risk factors and symptoms," said primary investigator, Steven Itzkowitz, MD, Professor of Medicine, Oncological Sciences and Medical Education, and Director of the GI Fellowship Program at Icahn School of Medicine at Mount Sinai.

Risk factors include a family history of CRC or pre-malignant polyps, inflammatory bowel disease and lifestyle habits, such as diet, smoking and obesity. CRC also disproportionately impacts the Black community. While symptoms are not always easy to recognize, they can include a change in bowel habits, such as diarrhea and constipation. Additionally, rectal bleeding, abdominal discomfort or a feeling that the bowel does not completely empty can be a cause for concern. Individuals are urged to listen to their bodies and consult their physicians if they have questions or require medical care.

As a leading provider of colonoscopies, AMSURG submits a larger colonoscopy dataset than any provider to a national endoscopic registry called the GI Quality Improvement Consortium (GIQuIC).The AMSURG data reviewed for this study was obtained from the registry.

This research on CRC trends in the United States is the first of an ongoing collaboration between AMSURG and Mount Sinai.

On May 24, 2021 Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) ("Aptorum Group" or "Aptorum"), a clinical-stage biopharmaceutical company, reported positive progress in respect of: (i) its ongoing Phase I clinical trial for ALS-4 (a first in-class anti-virulence approach based small molecule targeting Staphylococcus aureus, including, but not limited to Methicillin Resistant Staphylococcus Aureus ("MRSA")), under which two initial cohorts of the single dose ascending dose (SAD) portion of the trial in healthy male and female adult subjects have been completed with no serious adverse events observed (with in total 6 cohorts for SAD and 3 cohorts for multiple ascending dose (MAD) have been planned); and (ii) its ongoing Pre-IND preparation for SACT-1, a repurposed small molecule targeting neuroblastoma, under which the Pre-IND meeting with the US FDA has been completed (Press release, Aptorum, MAY 24, 2021, View Source [SID1234580498]).

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ALS-4’s first-in-human Phase I trial is a randomized, double-blinded, placebo-controlled, single and multiple ascending dose study designed to evaluate safety, tolerability, and pharmacokinetics of orally administered ALS-4 in healthy male and female adult volunteers. Dosing and safety reviews of Cohort A (25mg) and Cohort B (50mg) have been completed and eight subjects (6 received ALS-4 and 2 received placebo) were dosed in each cohort. We are pleased to announce that no human subjects were dropped out of the studies and there were no Serious Adverse Events (SAE) observed. In addition, no relevant clinical changes in respect of vital signs, ECG, clinical laboratory test results and physical examinations were observed compared to the relevant baseline. On this basis, the remaining ALS-4 Phase I study will continue to progress and as of this date, Cohort C (100mg) studies have been initiated.

SACT-1, a repurposed drug candidate for the treatment of neuroblastoma, has completed its Pre-IND meeting with the US FDA. With the guidance provided by the US FDA, SACT-1 is on track to open an IND to commence clinical studies in quarter 3 of 2021.

About ALS-4

As part of Aptorum Group’s Acticule infectious disease platform, ALS-4 is a novel first-in-class orally administered small molecule drug based on an anti-virulence approach targeting staphylococcus aureus including MRSA. ALS-4 targets the antimicrobial resistant properties of the bacteria and is believed to render the bacteria highly susceptible to the host’s immune clearance. ALS-4 is targeted for potential administration on a standalone or on a combination basis with other existing antibiotics such as vancomycin.

About SACT-1

As part of Aptorum Group’s SMART-ACT platform, SACT-1 was discovered from our SMART-ACT platform focused on orphan and unmet diseases. SACT-1 is a repurposed small molecule drug targeted for the treatment of neuroblastoma especially in combination with standard of care based chemotherapies. Our internal in vitro studies demonstrated that SACT-1’s mechanism has enhanced DNA damage and tumor cell death.

On May 24, 2021 Kiromic Biopharma, Inc. (Nasdaq: KRBP), an immuno-oncology target discovery and gene-editing company with a proprietary artificial intelligence neural network platform (Diamond AI) that is used to develop novel oncology therapeutics, reported the submission of a novel Investigational New Drug (IND) to the U.S. Food and Drug Administration (FDA) for a Phase 1 clinical trial that has the potential to be a universal CAR T-Cell therapy for any solid malignancy that expresses the biomarker Iso-mesothelin, with higher efficacy, higher safety, as well as lower manufacturing and distribution costs (Press release, Kiromic, MAY 24, 2021, View Source [SID1234580497]).

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The title of the clinical trial is: ALEXIS-ISO-1: A Phase 1, Open-label, Dose Escalation Study of KB-ISM, Allogeneic Gamma Delta T-cells Expressing a Chimeric Antigenic Receptor (CAR) Against the Isoform 2 of Mesothelin in Subjects with Metastatic or Progressive Locally Advanced Solid Malignancies.

The FDA feedback is expected within 30 days from IND submission.

Key features of the IND:

Product Name

KB-ISM

Product Type

CAR-T live cell therapy

Cancer Type

All solid tumors that express IsoMesothelin

Targeting

Isoform 2 of Mesothelin (AI algorithm predicted targets)

Patient Type

Patients with solid tumor positive for ISM

No. of Patients

30

Dosing

Dose escalation

Duration of Trial

18 months

First-in-human

3Q 2021 pending FDA authorization

First data from Trial

4Q 2021

How Our KB-ISM Live Cell Therapy CAR-T Improves CAR-T Market:

Marketed and traditional CAR-T

Kiromic KB-ISM

Malignancies
(Cancer Type)

Hematologic

Solid Tumors

Live Cell Origin

Autologous

Live Cells from pre-treatment patients

Allogenic

Live Cells from healthy donors

Live Cell Cloning
(Manufacturing)

Single batch
Single dose

Single batch
Multi dose

(aka. Off-The-Shelf)

Mass Manufacturing
on-demand
a single patient

— Will be manufactured like a traditional drug

— Kiromic proprietary manufacturing

— Kiromic proprietary cryopreservation processing techniques

This submission follows by one week the company’s prior submission of the IND for a phase 1 trial of the Gamma Delta chimeric PD1 Switch Receptor.

Both clinical trials support Kiromic’s clinical strategy of developing a "universal" off-the-shelf cell therapy against solid tumors.

This Submission was made 30 days ahead of the schedule previously announced on March 9, 2021.

This IND represents a Key Milestone of the program, which brings to the clinic the first target derived from the Kiromic’s AI and Bioinformatics target prediction platform.

Kiromic now expects that it will be able to deliver a first in human patient dosing by the end of the third quarter of 2021 Kiromic, an organization that is driven to achieve scientific breakthroughs, dedicated significant resources to the IND submission in an effort to achieve the optimal clinical trial design on the fastest possible timeline.

IQVIA (View Source) will be managing our clinical trial sites as the CRO (Clinical Research Organization). Leading global sites are lining up to be the first to dose our KB-ISM live cell therapies for solid tumors expressing IsoMesothelin.

IQVIA is an industry driver in data technology and advanced analytics, designed to produce and develop optimal clinical trial outcomes.

Site announcements are expected within the next few weeks.

Since Kiromic BioPharma’s Isoform 2 Mesothelin (KB-ISM) is off-the-shelf, it can be pre-manufactured like a typical drug, even though it is a living cellular therapy. This industry leading innovation is due to Kiromic’s proprietary manufacturing and cryopreservation processing techniques.

Kiromic is optimistic that any cancer cell that expresses the Isoform 2 Mesothelin marker will effectively activate and accelerate the ability of KB-ISM to traffic through the tumor microenvironment (TME), which thus far has proven to be an imposing barrier to effective T cell treatments in solid cancers.

————————————————

CEO of Kiromic, Dr. Maurizio Chiriva-Internati, DBSc, PhDs stated:

"Kiromic’s Off-The-Shelf (OTS) CAR Gamma Delta T-Cell (GDT) is a unique product, and I am very excited to present this IND to the FDA.

This technology is meant to potentially change the Cell and Gene Therapy landscape, helping to address the major issues on manufacturing, safety, cost and most importantly, time.

A Bioinformatics approach in the Cell Therapy & Gene Therapy field is disruptive and will help to screen and design specific binder (monoclonal Antibodies) with more specificity and avidity creating better and more robust therapy with less side effects.

Kiromic is in a unique position because its bioinformatic engine combines CancerDiff, a data-mining tool for the identification of tumor-preferred isoforms, with Diamond, an AI-driven immunogenic peptide identification system.

This combination allows Kiromic to identify targetable sections of a tumor isoform (peptides) that are a unique signature of cancer cells.

The advantage of Kiromic’s system is two-fold: 1) it saves time and money because it allows the prioritization of tumor-selective targets, and 2) it maximizes the likelihood of identifying targets that are safe by increasing the targeting specificity.

I am proud of our Translational and CMC teams that were capable of creating a feeder free expansion system with a purity of over 99% GDT, and it is very exciting that we can cryopreserve these cells in long term storage so that they are available on demand for shipment and ready to inject around the world.

I believe that our science will reshape and revolutionize the approach to Immune Oncology treatment in the years to come."

CFO of Kiromic, Mr. Tony Tontat stated:

"With this IsoMesothelin IND filing, Kiromic shows its commitment to invest in the latest AI technologies and then bring these AI predicted biomarkers into the clinic.

Going forth, we will look to announce our further investments in AI and how these investments in AI will keep the company in front of competitors in our space who are working without AI."

CSIO of Kiromic, Mr. Gianluca Rotino stated:

"This IND filing confirms the solidity of our IP portfolio, and highlights the results obtained by our Bioinformatics department and the application of our AI platform.

In combination with the ALEXIS-PRO-1 clinical trial, it represents the second element of the development of our clinical strategy, which will allow us at the end of the dose escalation phase of this trial, to select the best indication for the expansion phase that we project will ultimately lead to a BLA."

CMO of Kiromic, Dr. Scott Dahlbeck stated:

"I am looking forward to testing this novel AI discovered biomarker in metastatic cancer patients that desperately need safe and effective treatment options. This unique target was unearthed by our dedicated team of bioinformatic specialists, and if this clinical trial can validate the ability of Kiromic’s AI system to quickly discover dynamic targets, then it will meet one of the greatest needs in immunotherapy today, that of a lack of effective targets that can be used in immunotherapy treatments."

On May 24, 2021 Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, reported that Boehringer Ingelheim has accepted a GalXC RNAi candidate for advancement under the existing agreement between the companies for the discovery and development of novel therapies for the treatment of chronic liver diseases (Press release, Boehringer Ingelheim, MAY 24, 2021, View Source [SID1234580496]). Referred to as DCR-LIV2, the compound will be investigated for the treatment of nonalcoholic steatohepatitis (NASH), a chronic liver disease for which there are no approved therapeutic interventions. Acceptance of DCR-LIV2 as a development candidate triggered a single-digit multimillion USD preclinical milestone payment to Dicerna, which the Company expects to receive in the second quarter of 2021

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Advancing this selected target to development demonstrates the commitment of our team and our collaborative partners at Boehringer Ingelheim to discovering new and innovative ways to treat NASH using RNAi," said Bob D. Brown, Ph.D., Dicerna’s Chief Scientific Officer and Executive Vice President of R&D. "Dicerna’s GalXC technology is the ideal platform for this vital work. What’s more, this announcement marks an important milestone for Dicerna and highlights the productivity of our RNAi discovery research engine, as all of Dicerna’s discovery-oriented collaborations have now produced GalXC-based development candidates that are advancing toward the clinic or have already entered clinical development."

Under the terms of the existing agreement between Dicerna and Boehringer Ingelheim, Dicerna is eligible to receive up to $170.0 million in potential additional development and commercial milestones related to DCR-LIV2. Dicerna is also eligible to receive tiered mid-single-digit royalties on potential global net sales.

About Nonalcoholic Steatohepatitis (NASH)

Nonalcoholic steatohepatitis (NASH) is a condition characterized by the buildup of fat in the liver, potentially leading to liver fibrosis, cirrhosis, liver failure or cancer, and has an especially high prevalence among people who are obese or have type 2 diabetes. NASH is estimated to affect approximately 1.5% to 6.5% of adults in the U.S.1

About RNAi and Dicerna’s GalXC RNAi Platform Technologies

Ribonucleic acid interference, or RNAi, provides a unique advantage to other disease inhibitor technologies, like small-molecule pharmaceuticals or monoclonal antibodies: instead of targeting proteins after they have been produced and released, RNAi silences the genes themselves via the targeted destruction of the messenger RNA (mRNA) made from the gene. Rather than seeking to inhibit a protein directly, the RNAi approach can prevent a disease-causing protein’s creation, directly impacting disease manifestation.

Dicerna’s proprietary GalXC RNAi platform aims to advance the development of next-generation RNAi-based therapies. Investigational therapeutics developed using our flagship GalXC technology utilize a proprietary N-acetyl-D-galactosamine (GalNAc)-mediated structure of double-stranded RNA molecules that are designed to bind specifically to receptors on liver cells, leading to selective hepatocyte internalization and access to the RNAi machinery within the cells. Dicerna is continuously innovating and exploring new applications for RNAi technology beyond GalNAc-mediated delivery to the liver, including alternative RNA structures and fully synthetic ligands that target other tissues and enable new therapeutic applications, referred to as GalXC-Plus.

On May 24, 2021 Sema4, a patient-centered health intelligence company leveraging AI and machine learning to derive data-driven insights, reported that members of the Sema4 management team will participate in fireside chats at two upcoming virtual investor conferences (Press release, Sema4, MAY 24, 2021, View Source [SID1234580495]). Eric Schadt, PhD, Founder and Chief Executive Officer, and Isaac Ro, Chief Financial Officer, will present at the Jefferies Virtual Healthcare Conference on Friday, June 4, 2021 and the Goldman Sachs Annual Global Healthcare Conference on Tuesday, June 8, 2021.

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Event: Jefferies Virtual Healthcare Conference
Date: Friday, June 4, 2021
Time: 11:00 a.m. ET / 8:00 a.m. PT

Event: Goldman Sachs Annual Global Healthcare Conference
Date: Tuesday, June 8, 2021
Time: 1:20 p.m. ET / 10:20 a.m. PT

Following each conference, a Form 8-K with a copy of the transcript will be filed with the U.S. Securities and Exchange Commission (SEC). A link to the SEC filing will be available on the Company’s website at View Source