On December 4, 2018 AUM Biosciences (AUM), a Singapore headquartered oncology-focused biotechnology company, reported that it has licensed its first novel, highly selective anti-cancer drug, ETC-206, from A*STAR’s Experimental Therapeutics Centre (ETC) (Press release, AUM BioSciences, DEC 4, 2018, View Source [SID1234533612]). The Agency for Science, Technology and Research (A*STAR) is Singapore’s lead public sector agency that spearheads economic oriented research to advance scientific discovery and develop innovative technology.

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AUM has licensed the global rights to develop, commercialize and manufacture ETC-206 in all indications. ETC-206 was discovered and developed through a collaboration between A*STAR’s Experimental Therapeutics Centre (ETC) and Duke-NUS Medical School (a partnership between Duke University School of Medicine and the National University of Singapore). It advanced into first-in-man trials in December 2016. Initial clinical studies suggest that ETC-206 is very well tolerated and could be used as a thera∂ßpy across a range of liquid and solid tumor types.

The drug promises a more targeted approach by inhibiting the Mnk enzyme in cancer cells, which is a key player in promoting cancer growth when activated. This could lower mortality rates for cancer and improve quality of life for cancer patients compared to traditional chemotherapy, which kills cells in a less specific fashion and affects more than just the cancer cells.

"Due to low efficacy and steep costs associated with many cancer treatments, the demand for more precise anti-cancer treatments has become imperative. AUM’s small molecule and biomarker driven approach allows us to implement innovative trial designs enabling "multiple shots on goal", in cancer treatment," AUM’s Chief Executive Officer, Mr. Vishal Doshi said.

ETC-206 is the first in AUM’s pipeline of novel targeted therapies with the potential to be developed both independently and in combination with other therapies. The drug’s ability to isolate and target only cancerous cells promises a breakthrough opportunity globally.

"ETC-206 is a prime example of how A*STAR has been able to translate R&D into positive outcomes to create economic growth and enhance lives for Singaporeans. We look forward to working closely with more Singapore based enterprises such as AUM Biosciences as we continue to leverage our drug discovery and development capabilities to support the growth of the local biotech ecosystem in Singapore." Said Dr Damian O’Connell, Chief Executive Officer of A*STAR’s Experimental Therapeutics Centre.

"Duke-NUS scientists and researchers have led many discoveries with great potential for the welfare of people in Singapore and beyond," stated Dr. David Epstein, Director of Duke-NUS’ Centre for Technology and Development (CTeD), and the School’s Vice Dean for Innovation and Entrepreneurship, who is also an associate professor of the Cancer and Stem Cell Biology Programme. "ETC-206 is an important accomplishment exemplifying Duke-NUS’s mission of facilitating the translation of basic research into commercial entities and products. We are proud to partner with A*STAR in realizing this enterprising venture that was ‘Made in Singapore’ and is being taken forward by AUM, a Singapore-based startup. We look forward to more startups creating entrepreneurship opportunities in the sector."

ETC-206’s licensing by AUM not only puts Singapore on the map, but also enables the company to be at the forefront of emerging oncology trends. This is a significant step towards its vision of "Asia to global" by accelerating the development of innovative and affordable medicines.

"We are now speaking with investors to accelerate ETC-206’s development and our broader molecule acquisition roadmap. AUM will fund further clinical development and will be responsible for regulatory filings, and for clinical studies for this and future drugs," Mr. Doshi commented.

As a future roadmap, AUM has a strategic focus of acquiring and developing promising small molecules targeted oncology assets to unlock their potential, including those that are de-prioritized or not progressed by their originators due to strategic and financial limitations in the healthcare industry. Its mandate of "No biomarker, No drug" serves to increase the probability of success.

Cancer prevalence in Asia is estimated at 17.4 million in 2018, with about 8.8 million new cases every year, accounting for about half of the new global cancer cases. The number of new cases in Asia is expected to increase from 8.8 million in 2018 to 11.8 million by 2030 creating a huge unmet medical need in Asia.

On December 4, 2018 Innate Pharma reported its updated results that support advancement of IPH4102 in refractory Sézary syndrome at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2018 annual meeting (Press release, Innate Pharma, DEC 4, 2018, View Source [SID1234531856]).

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Pierre Dodion, Chief Medical Officer, Innate Pharma, will be joined by Prof. Martine Bagot, Head of Dermatology Department at the Saint Louis Hospital, Paris and lead investigator of the study, for a live webcast and conference call with a Q&A session on Tuesday, December 4 at 5pm CET to discuss the announcement.

The presentation and access to the live webcast will be available at this link: View Source

Participants can also join the conference call using the following dial-in numbers:

Location

Purpose

Phone number

France

Participant

+33 (0)1 76 77 22 57

United Kingdom

Participant

+44 (0)330 336 9411

United States

Participant

+1 929-477-0324

Standard International Access

Participant

0800 279 7204

The participation code is: 4535688

On December 4, 2018 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) reported that additional data from an updated analysis of the pivotal Iomab-B Phase 3 SIERRA trial were highlighted last night in an oral presentation at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Actinium Pharmaceuticals, DEC 4, 2018, View Source [SID1234531873]). The SIERRA trial is the only ongoing Phase 3 trial offering a bone marrow transplant (BMT) to patients 55 years of age or older with active, relapsed or refractory acute myeloid leukemia (AML). Data in the table below were presented in the oral session and are updated from those at the time of abstract submission. Preliminary data strongly support the feasibility and safety of re-induction and targeted conditioning with Iomab-B prior to a BMT.

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Preliminary Feasibility and Safety Data

Randomized to
Iomab-B Study Arm
(N=19)

Randomized to
Conventional Care
(N=19)

Randomized to
Conventional Care,
No Remission, and
Crossed Over (N=10)

Number of Patients
Receiving BMT after
receiving therapy

100% (18/18)

(1 patient did not
receive therapeutic
Iomab-B dose)

21% (4/19)

100% (10/10)

Blast % at
Randomization

Median: 30%

Range: 4* -74

Median: 26%

Range: 6-97

At Randomization
24% (6-70)
At Crossover 45%
(10-70)

Days to BMT

(post-randomization)

Median: 28

Range: 23-38

Median: 67

Range: 66-86

Median: 66

Range: 57-161

Days to Absolute
Neutrophil
Engraftment

Median:13

Range: 9 – 22

Not entered

Median:13

Range: 9 – 20

Days to Platelet
Engraftment

Median: 16

Range: 13 – 26

Not entered

Median:17

Range: 10 – 20

100-Day Non-
Relapse Mortality

0% (0/18)

25% (1/4 – septic
shock)

10% (1/10 – diffuse
alveolar hemorrhage)

*1 patient with 4% blasts in the marrow had circulating AML blasts

Other Key Highlights:

15 of 19 (79%) patients in the control arm failed to achieve a complete response
67% (10/15) of patients eligible for crossover successfully transplanted with Iomab-B
All patients receiving Iomab-B engrafted despite active disease with high blast count (median 30%, or median 45% for crossover patients)
Patients receiving Iomab-B received a BMT more quickly post-randomization (28 days) than patients receiving conventional care (67 days)
In the conventional care arm, there was no difference in time to BMT for patients that crossed over to Iomab-B (66 days) compared to those achieving complete remission with conventional care (67 days)
No Grade 3 or 4 Iomab-B infusion related reactions with all Iomab-B infusions completed
No non-relapse mortality in patients randomized to Iomab-B arm
Abstract #1017: Targeted Conditioning of Iomab-B (131I-anti-CD45) Prior to Allogeneic Hematopoietic Cell Transplantation Versus Conventional Care in Relapsed or Refractory Acute Myeloid Leukemia (AML): Preliminary Feasibility and Safety Results from the Prospective, Randomized Phase 3 Sierra Trial

Dr. Edward Agura, Medical Director of Bone Marrow Transplantation at Baylor University Medical Center said, "Given that more than two thirds of patients who are diagnosed with AML are 55 years of age or older, there exists a significant unmet medical need to broaden transplant access and improve outcomes for these patients. The data from the SIERRA trial thus far are highly encouraging as they demonstrate that Iomab-B can enable a potentially curative transplant in patients with active disease, including those patients with progressing disease who did not achieve a response on conventional care. The nearly universal and rapid engraftment of patients receiving Iomab-B, together with no 100-day non-relapse mortality, is particularly compelling as these results have not been achieved with conventional care."

The 150 patient SIERRA study is a multi-center randomized trial that will compare outcomes of patients who receive Iomab-B and a BMT to those patients receiving physician’s choice of salvage chemotherapy, defined as conventional care, as no standard of care exists for this patient population. AML patients with active, relapsed or refractory AML have dismal prognoses and are typically not offered curative transplant as an option. This is largely because salvage treatments have a limited ability to produce a complete remission, which is necessary prior to BMT if conventional BMT is to be successful. However, with Iomab-B targeted conditioning, a complete remission prior to starting the Iomab-B conditioning is not necessary for a successful transplant.

Dr. Mark Berger, Actinium’s Chief Medical Officer said, "We believe that Iomab-B represents a potentially disruptive modality for targeted conditioning. The preliminary data from the SIERRA trial presented at ASH (Free ASH Whitepaper) exceeded our expectations regarding feasibility and safety, which adds to the already extensive body of research and data demonstrating the utility of Iomab-B for targeted conditioning in multiple hematologic indications. With this data in hand we are highly motivated to complete the SIERRA trial, which will serve as the beachhead for our multi-target, multi-disease pipeline for targeted conditioning, where we are committed to expanding our leadership position."

On December 4, 2018 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported the publication of data from the previously reported, multi-center Phase 1/2 clinical study evaluating the safety and efficacy of NiCord as a stand-alone, hematopoietic stem cell (bone marrow) transplant in the Journal of Clinical Oncology1,2 (Press release, Gamida Cell, DEC 4, 2018, View Source [SID1234531889]). NiCord is an investigational product candidate in Phase 3 development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies, or blood cancers.

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Results from the Phase 1/2 study showed that patients transplanted with NiCord had rapid and durable engraftment of neutrophils and platelets, as well as prompt immune reconstitution. The median time to neutrophil recovery was shortened by nearly 50 percent for patients who received NiCord compared to a retrospective cohort of patients who received standard umbilical cord blood. NiCord also demonstrated an acceptable safety profile for patients undergoing bone marrow transplant.

"In this study, patients who received NiCord had a clinically meaningful reduction in their time to neutrophil and platelet recovery compared to a retrospective cohort of patients who received a standard umbilical cord blood transplant. The neutrophil recovery observed with NiCord also resulted in fewer days spent in the hospital compared to the comparator cohort," said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. "These data suggest a potential step toward making stem cell transplantation safer and more accessible to patients with lethal blood cancers."

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients do not receive one for various reasons, including finding a matched donor.3 While umbilical cord blood provides a source of stem cells for patients who do not have a matched related donor, it provides a smaller number of stem cells, which can delay engraftment and put patients at a greater risk for prolonged hospitalizations and life-threatening infections. NiCord is designed to address these limitations by offering a therapeutic dose of expanded cells while preserving the functional characteristics of stem cells.

NiCord Phase 1/2 Study Design and Results
The publication, "Phase I/II study of stem cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide," described results from the completed multicenter, Phase 1/2 clinical trial of NiCord in 36 patients with high-risk hematologic malignancies and no readily available matched sibling or matched unrelated adult donor. The key primary endpoint was the cumulative incidence of neutrophil engraftment at 42 days. Additionally, the NiCord patient cohort was compared to a retrospective cohort of patients who received standard cord blood transplant using data from the Center for International Blood and Marrow Transplant Research (CIBMTR).

Key findings included the following:

Patients transplanted with NiCord had rapid and durable engraftment of neutrophils and platelets, as well as prompt immune reconstitution. The age-adjusted cumulative incidence of neutrophil engraftment at 42 days following transplantation was 94 percent for NiCord recipients compared to 85 percent for the CIBMTR cohort.
Among patients who engrafted, the median time to neutrophil recovery was 11.5 days (95% CI: 9-14 days) for NiCord recipients compared to 21 days (95% CI: 20-23 days) for the CIBMTR cohort (p<0.001).
For patients achieving platelet recovery, the median time to platelet recovery was 34 days (95% CI:32-42 days) and 46 days (95% CI:42-50 days) for the NiCord and CIBMTR cohorts, respectively (p<0.001).
NiCord demonstrated an acceptable safety profile, with hypertension reported as the most common adverse event attributable to NiCord infusion, and moderate to severe chronic graft vs. host disease reported in 9.8 percent of patients at one year following transplantation.
Primary hospital discharge occurred at a median of 20 days following transplantation. NiCord recipients spent a median of 73 days alive and out of hospital during the first 100 days following UCB transplantation.
"These data demonstrate the potential of NiCord to give patients with high-risk blood cancers an opportunity for a cure, particularly patients who would otherwise not be able to receive a bone marrow transplant using a matched donor source," stated Ronit Simantov, M.D., chief medical officer at Gamida Cell. "We are actively enrolling patients in our Phase 3 study, which is designed to confirm the potential of NiCord to be an effective transplantation solution. We look forward to completing patient enrollment expected in the second half of 2019."

About NiCord
NiCord, the company’s lead clinical program, is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. NiCord has been granted breakthrough status by the U.S. Food and Drug Administration, making it the first bone marrow transplant alternative to receive this designation. It has also received U.S. and EU orphan drug designation. A Phase 3 study evaluating NiCord in patients with leukemia and lymphoma is ongoing in the United States, Europe and Asia.4 For more information on NiCord clinical trials, please visit www.clinicaltrials.gov.

On December 4, 2018 LineaRx, Inc., the biotherapeutics company based on the use of large-scale production of gene-sized DNA by Polymerase Chain Reaction ("PCR") and dedicated to revolutionizing biotherapeutics for the masses in affordability, availability, time-to-market, and reducing the risk associated with virally mediated therapy, and a wholly-owned subsidiary of Applied DNA Sciences, Inc. (NASDAQ: APDN), reported that it will host an Analyst Day via webinar on Thursday, December 6, 2018 at 12 p.m. to 1:30 p.m. EST (Press release, Applied DNA Sciences, DEC 4, 2018, View Source [SID1234531906]).

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During the 90-minute technical presentation Dr. James Hayward, CEO of LineaRx, Dr. Michael Hogan, Vice President of Life Sciences and Dr. Stephen Hughes, Director of DNA Programs will address:

Collaborative data on PCR-produced anti-cancer (anti-telomerase) vaccine show immunogenicity in mice,
Licensed CAR-T Therapy; partner data to be presented show efficacy in animals and humans when delivered via plasmid and virus, which bodes well for the prospect of similar efficacy of corresponding linear construct under development,
The concept for adoptive cell therapies without delay: "Networked DNA-producing PCR devices informed by Artificial Intelligence and located in hospitals operating under cGMP", and,
Proprietary IP should enable high levels of gene expression, self-transducing genes, transiently expressed genes without genomic integration; the sum of which should generate localized, in-hospital production of more effective adoptive cell therapies, faster, with a higher Therapeutic Index and a simpler mode of manufacture.
LineaRx Webinar Information

To participate in the LineaRx webinar, please follow the instructions below. Management will take questions from select invitees in the Q&A portion of the event.

To Participate:

Participant Toll Free: 1-844-887-9402
Participant Toll: 1-412-317-6798
Please ask to be joined to the LineaRx webinar
Live webcast: View Source
Replay (available 1 hour following the conclusion of the live call through December 13, 2018):

Participant Toll Free: 1-877-344-7529
Participant Toll: 1-412-317-0088
Participant Passcode: 10126911
Webcast replay: View Source
"This Analyst Day webinar serves as an excellent platform from which to build awareness for LineaRx’s competitive advantages and user benefits to the biotherapeutics industry at large. Anyone currently utilizing plasmid DNA and/or virus to transduce cells, in the DNA/RNA vaccine space, involved in CRISPR as well as, CAR-T Therapy could benefit greatly from our technology," stated Dr. Hayward.