On January 22, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, and Be The Match BioTherapies, a subsidiary of the National Marrow Donor Program/Be The Match offering integrated platforms and software to manage the collection and delivery of cellular therapies, reported a strategic collaboration to improve outcomes for patients undergoing allogeneic hematopoietic stem cell (bone marrow) transplantation (Press release, Gamida Cell, JAN 22, 2019, View Source [SID1234532823]). The collaboration supports NiCord, Gamida Cell’s investigational cell therapy, which has the potential to serve as a universal bone marrow donor source for patients with hematologic malignancies, or blood cancers, and bone marrow failure disorders requiring bone marrow transplantation.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Under the terms of the collaboration agreement, Gamida Cell and Be The Match BioTherapies will explore opportunities to work together across Gamida Cell’s ongoing clinical development program for NiCord, including the Phase 3 clinical study in patients with high-risk blood cancers. Be The Match BioTherapies has an extensive history of involvement in the delivery of cord blood units for transplant and broad access to cord blood banks globally.

The collaboration is expected to leverage a wide range of Be The Match BioTherapies’ research assets and services, including the Be The Match Registry, the largest in the world with more than 20 million potential donors, as well as personalized case management and logistics services.

"Gamida Cell’s work to bring a new therapeutic option to patients in need of a bone marrow transplant aligns with our core mission to help organizations deliver cellular therapies that save more lives and improve the quality of life for patients," said Amy Ronneberg, president of Be The Match BioTherapies. "This collaboration is designed to leverage our expertise in cellular therapy to support the advancement of Gamida Cell’s clinical development and product delivery efforts."

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including difficulty in finding a matched donor.1 NiCord is designed to potentially serve as a universal alternative to existing donor sources for bone marrow transplant.

"Be the Match BioTherapies is a respected leader in cell therapy, with especially deep roots in stem cell transplantation. Our two organizations share the belief that new cell therapies are needed not only to improve outcomes for patients undergoing stem cell transplants but also to offer patients unable to find a matched donor the chance for a successful transplant," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "This collaboration maximizes the strengths and expertise of our teams to help move us closer to our common goal of transforming this important area of medicine."

On January 22, 2019 Notable Labs, a startup that accelerates drug development by matching drugs with patients who are most likely to respond, reported that it has launched its first drug development program, ND-1000, which targets pediatric and adult blood cancers (Press release, Notable Labs, JAN 22, 2019, View Source [SID1234532886]). In an industry first, the company has opted to donate the commercial rights to price, manufacture, and distribute ND-1000 for pediatric leukemia to global repurposing non-profit Cures Within Reach in order to provide the drug to pediatric leukemia patients at a very low cost.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The partnership between Notable and Cures Within Reach is intended to further Cures Within Reach’s mission to develop and implement effective economic incentives for the repurposing of inexpensive generic drugs, devices, and other products which is especially critical in rare diseases. This collaborative venture will be supported by philanthropy from individuals, foundations and disease non-profits, as well as investment from for-profit stakeholders.

As a for-profit company, Notable Labs is able to donate ND-1000’s pediatric commercial rights to Cures Within Reach due to the drug’s eligibility for the FDA’s Pediatric Priority Review Voucher Program, which has a financial value due to an awarded voucher’s ability to be sold to another pharmaceutical company.

"We need to create and use incentives to secure approval for existing drugs that have additional therapeutic value," said Dr. Bruce Bloom, CEO, Cures Within Reach. "To our knowledge, this would be a unique therapeutic development partnership that creates both commercial value for shareholders and philanthropic impact at a significantly lower development cost. Our hope is that this type of alliance between a for-profit company and a non-profit organization to repurpose drugs and improve outcomes at lower costs to patients could become a model for others to use."

Notable Labs will open the first clinical trial for ND-1000 at The University of Kansas Cancer Center later this year through a partnership with the University’s Institute for Advancing Medical Innovation (IAMI). "We are thrilled to bring our drug repurposing and blood cancer clinical trial expertise to the unique partnership established by Notable Labs and Cures Within Reach," said IAMI Director Scott Weir, Pharm.D., Ph.D. "We are focused on bringing life-saving cancer treatments to patients and we are eager to enroll trial participants into the first clinical trial for ND-1000, joining this groundbreaking effort."

How Notable Labs works

Notable Labs has built an automated laboratory that rapidly evaluates thousands of drug combinations on cancer cells relative to healthy cells. A report is generated which prioritizes the drugs that are most specifically active against cancer cells. The first validation trials have been in blood cancers due to the accessibility and large number of cancer cells available.

The company was founded by Matt De Silva, whose father was diagnosed with Glioblastoma Multiforme, a deadly stage 4 brain cancer that claimed the lives of Beau Biden, John McCain, and Ted Kennedy. A hedge fund manager by background, Matt spent more than a year researching potential treatments and clinical trials, and realized that the physicians running the trials didn’t have the ability to accurately predict which medications would be most effective for his father. Unfortunately, cancer doesn’t wait, and Matt lost his father before he could find the right treatment. During that time Matt founded Notable Labs and built a platform that could solve the problem for other patients that he couldn’t for his own father.

"Our goal is to ensure that if a patient chooses to enroll on a clinical trial, they’ll have a strong chance of responding. If we can quickly and accurately match the right drugs with the right patients, the cost of clinical trials will drastically fall, enabling reduced drug prices that our healthcare system desperately needs," said De Silva, CEO and Founder of Notable Labs. "The work we do in our lab and this partnership with Cures Within Reach and the KU Cancer Center will ensure that more families and patients will have access to this therapy in the future."

About Cures Within Reach
Cures Within Reach (CWR) is a US-based global philanthropic leader that improves patient quality and length of life by leveraging the speed, safety and cost-effectiveness of medical repurposing research, driving more treatments to more patients more quickly. CWR catalyzes research to facilitate and validate repurposing opportunities that create clinical impact, and enables and facilitates conversation and action among stakeholders that help transform healthcare through repurposing opportunities. Through repurposing, CWR drives both market impact and health savings to patients and patient groups, from academia/researchers, with payers and the healthcare industry and with support from the government, philanthropy and others. CWR’s repurposing research projects have generated "new" treatments in over a dozen indications, making patient impact through off-label use in clinical practice or through a commercialization track.

CWR currently has a global portfolio of more than 20 repurposing research projects, as well as more than 180 repurposing research projects available for funding in a wide range of diseases on its CureAccelerator site. Visit cureswithinreach.org or follow CWR via Twitter @CuresWReach, LinkedIn (LinkedIn.com/company/cures-within-reach), YouTube (YouTube.com/cureswithinreach) or Facebook (Facebook.com/CuresWithinReach).

On January 21, 2019 BerGenBio ASA (OSE:BGBIO), reported that the first patient has been dosed in a phase I clinical trial evaluating the safety, tolerability, pharmacokinetics and anti-tumour efficacy of ADCT-601, an AXL-targeting antibody drug conjugate (ADC), in patients with advanced solid tumours (Press release, BerGenBio, JAN 21, 2019, View Source [SID1234532796]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

ADCT-601 is composed of a humanised monoclonal antibody against human AXL (BGB601) discovered by BerGenBio, conjugated to a pyrrolobenzodiazepine (PBD) dimer toxin. BGB601 was out-licensed for ADC development to ADC Therapeutics SA (ADCT). In preclinical studies, ADCT-601 has demonstrated potent and specific anti-tumour activity in multiple in vivo models and was stable and well tolerated, as reported by ADCT at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual congress in 2018.

The open-label, multi-centre, single arm phase I trial will enrol approximately 75 patients with selected tumour types and will be managed and sponsored by license partner ADC Therapeutics. For more information see View Source (accessing trial identifier NCT03700294).

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "We congratulate ADC Therapeutics on reaching this important milestone. We are pleased to now see three of our AXL-targeting modalities in clinical development with the potential to address large patient populations. Our focus remains on completing our ongoing oncology phase II programme with bemcentinib, a first-in-class highly selective oral AXL inhibitor, and start randomised, potentially pivotal trials later this year. In the meantime, we look forward to providing updates on the development of BGB149, a therapeutic AXL antibody, and ADCT-601, an anti-AXL ADC, as they progress through phase I testing."

END

About ADCT-601
BerGenBio out-licensed two novel and proprietary anti-AXL monoclonal antibodies invented by the Company to ADC Therapeutics SA (ADCT) for the development of an antibody drug conjugate (ADC).

ADCT-601 is composed of BGB601, a humanised monoclonal antibody that binds to human AXL, conjugated using GlycoConnect technology to a linker with a pyrrolobenzodiazepine (PBD) dimer toxin. Once bound to an AXL-expressing cell, ADCT-601 is internalised into the cell where enzymes release the PBD-based warhead. The PBD-based warhead has the ability to form highly cytotoxic DNA interstrand cross-links, blocking cell division and ultimately killing the cancer cell. ADCT-601 is currently undergoing Phase I clinical testing (NCT03700294).

Under the license, a series of development, regulatory and sales-based milestones are due to BerGenBio from ADCT upon the achievement of certain specified events. The first milestone payment is triggered during the phase I clinical study.

About AXL
AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms underlying life-threatening diseases. In cancer, AXL suppresses the body’s immune response to tumours and drives cancer treatment failure across many indications. AXL inhibitors, therefore, have potential high value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities. Research has also shown that AXL mediates other aggressive diseases.

On January 21, 2019 ISA Pharmaceuticals B.V., a clinical-stage company dedicated to the development of rationally designed immunotherapeutics, reported randomization of the first patient in a Phase 2 combination trial of its lead compound ISA101b and Regeneron´s (NASDAQ: REGN) anti-PD-1 antibody cemiplimab (REGN2810) (Press release, ISA Pharmaceuticals, JAN 21, 2019, View Source [SID1234532797]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This double blind, placebo-controlled, randomized, phase 2 study (NCT03669718) will enroll approximately 164 patients with HPV16-positive oropharyngeal cancer (OPC), who have failed first-line chemotherapy. Patients will be randomly allocated on a 1:1 basis to the combination of cemiplimab/ISA101b or cemiplimab/placebo. In both study arms, patients will receive treatment in cycles of 3 weeks for up to 24 months. The primary efficacy objective of this trial is to assess whether the addition of ISA101b to cemiplimab will elicit a higher Overall Response Rate (ORR, as per RECIST 1.1 criteria) compared to cemiplimab plus placebo. Secondary endpoints include Progression Free Survival (PFS), Overall Survival (OS), as well as safety and tolerability of cemiplimab plus ISA101b (as compared to cemiplimab plus placebo). The study will be conducted at approximately 50 investigative sites in the USA and Europe.

ISA Pharmaceuticals´ lead compound ISA101 is a clinical-stage SLP immunotherapy targeting HPV16-induced diseases such as cervical cancer and head–and-neck cancer. It induces specific immune responses to the oncogenic E6 and E7 antigens of HPV16. Cemiplimab, also known as REGN2810, is currently under review by EMA and was approved by the U.S. Food and Drug Administration in September 2018 under the brand name Libtayo as monotherapy for patients with advanced cutaneous squamous cell carcinoma. Regeneron, in collaboration with Sanofi, is developing cemiplimab both alone and in combination with other therapies for the treatment of various cancers.

"We are excited about this new clinical trial with ISA101b and cemiplimab," said Dr. Leon Hooftman, CMO of ISA Pharmaceuticals. "ISA101b has already demonstrated very promising results when combined with nivolumab in a previous study in advanced HPV16-positive cancers. Particularly in patients with relapsed head-and-neck cancer the percentage of patients with a meaningful tumor response appeared to be twice as high as achieved with nivolumab alone. The objective of the current randomized trial is to confirm such clinical benefit of ISA101b in a controlled setting."

"In our collaboration with Regeneron that was announced in December 2017, this is the first of two indications in which we jointly aim to develop novel combination immunotherapies for cervical cancer and head-and-neck cancer," said Gerben Moolhuizen, CEO of ISA Pharmaceuticals. "I am very proud of the efforts of the joint team that allowed us to advance quickly to this stage. We look forward to the first read-out from the trial, which is expected in the second half of 2020."

Oropharyngeal cancer, a subtype of head-and-neck cancer, affects tissues of the throat (oropharynx), e.g. the base of the tongue, the tonsils, the soft palate, and the walls of the pharynx. Oropharyngeal cancers can be divided into two types: HPV-positive, which are related to human papillomavirus infection, and HPV-negative cancers, which are usually linked to alcohol or tobacco use.

On January 21, 2019 Achilles Therapeutics ("Achilles"), a biopharmaceutical company developing personalised cancer immunotherapies, reported that its Clinical Trial Application (CTA) to conduct a Phase I/II study with its lead product, a tumour-derived T cell therapy targeting clonal neoantigens, in development for the treatment of advanced Non-Small Cell Lung Cancer (NSCLC), has been approved by the UK regulatory authority, the Medicines and Healthcare products Regulatory Agency (MHRA) (Press release, Achilles Therapeutics, JAN 21, 2019, View Source [SID1234532798]). The study, an open-label, multi-centre Phase I/II trial evaluating the safety and clinical activity of clonal neoantigen T cells (cNeT) in patients with advanced NSCLC, is expected to enrol the first patient in 2H 2019.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Approval of our first CTA represents an important validation of our approach and a significant milestone for Achilles," said Dr Iraj Ali, CEO of Achilles Therapeutics. "Achilles was founded by world-leading experts in cancer evolution, bioinformatics and the delivery of cell-based immunotherapies and we are bringing together these disciplines to develop next-generation, patient-specific T cell therapies that harness the immune system to destroy cancer cells."

"The Achilles approach is a technological step forward in the immune-oncology space with the potential to bring the next wave of revolutionary new immunotherapies to cancer patients," said Dr Martin Forster, Chief Investigator for the study at University College London Hospitals (UCLH), the lead clinical site. "We are excited to be part of the study and look forward to enrolling patients into the clinical trial."

Achilles is developing personalised T cell therapies for solid tumours targeting clonal neoantigens: protein markers unique to each patient that are present on the surface of a cancer cell. Using its PELEUS bioinformatics platform, Achilles can identify clonal neoantigens from each patient’s unique tumour profile which are present on every cancer cell and can be recognised by the immune system. Achilles uses its proprietary process to manufacture clonal neoantigen T cells (cNeT) which exquisitely target the specific set of clonal neoantigens in each patient. The starting material for cNeT are tumour infiltrating lymphocytes (TILs) which are isolated from the patient’s own tumour sample. These T cells are already programmed to invade and attack the tumour, and previous clinical studies have shown that expanded TILs can debulk solid tumours with durable and potent responses. Targeting multiple clonal neoantigens that are present on all cancer cells, but not on healthy cells, reduces the risk that new mutations can induce immune evasion and therapeutic resistance, and allows individualised treatments to target and destroy tumours without harming healthy tissue.

A second clinical study in patients with metastatic or recurrent melanoma is anticipated to open later in 2019.

– Ends –

Further information:

Achilles Therapeutics
Dr Iraj Ali – Chief Executive Officer
+44 (0)1438 906 906
[email protected]

Julia Wilson – Head of Communications
+44 (0)7818 430877
[email protected]

Consilium Strategic Communications
Mary-Jane Elliott, Sukaina Virji, Melissa Gardiner
Tel: +44 (0) 203 709 5000
Email: [email protected]