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Advanced Chemotherapy Technologies, Inc. Awarded $4 Million NIH Grant to Pursue Treatment for Locally Advanced Non-resectable Pancreatic Cancer

On August 11, 2021 Advanced Chemotherapy Technologies (ACT), Inc., a clinical-stage drug delivery company, reported that it has been awarded a Phase IIb Small Business Innovation research (SBIR) grant expected to total $4 million over two years from the National Cancer Institute, part of the National Institutes of Health (Press release, Advanced Chemotherapy Technologies, AUG 11, 2021, View Source [SID1234586353]). The grant, in combination with prior financing, supports development of the company’s ACT-IOP-003 local drug delivery system for the treatment of locally advanced non-resectable and borderline resectable pancreatic cancer.

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Pancreatic cancer is a devastating disease with 5-year survival rates of only 10%. New treatment modalities are desperately needed and ACT is developing the ACT-IOP-003 system for the targeted delivery of the FDA-approved chemotherapy treatment gemcitabine. ACT-IOP-003 enables the delivery of significantly higher concentrations directly to the tumor than achievable in systemic delivery, while also minimizing the systemic exposure and toxicity. This approach offers three major advantages over traditional systemic chemotherapy: (1) superior delivery of chemotherapy to the targeted tumor cells that are often shrouded in stroma within growing tumors, greatly increasing the amount of drug to treat the growing tumor, (2) tumor shrinkage that can enable surgical resection, the only curative treatment for pancreatic cancer, and (3) greatly decreased systemic toxicity so that the patient can better tolerate conventional approaches to their treatment.

William Daunch PhD., ACT’s Chief Technology Officer will serve as the Principal Investigator on the grant. Dr. Daunch says, "We are thrilled and encouraged that NCI has chosen to award us this highly competitive Phase IIb grant. It demonstrates the NIH’s positive recognition of the work we completed in our earlier phases, as well as their continued confidence in our program to accelerate this potentially life extending treatment to patients."

NIH sponsored grant programs are an integral source of capital for early-stage U.S. small businesses that are creating innovative technologies to improve human health. These programs help small businesses break into the federal research and development arena, create life-saving technologies, and stimulate economic growth. ACT is honored to be a recipient of this competitive award from the NIH/NCI and looks forward to advancing treatment for pancreatic cancer patients and expanding its local drug delivery technology in new indications

New Paper Highlights Capability of Personal Genome Diagnostics’ elio™ plasma resolve Assay to Detect Microsatellite Instability

On August 11, 2021 Personal Genome Diagnostics Inc. (PGDx) reported the publication of a research paper in The Oncologist, titled "Validation of a ctDNA-based next-generation sequencing assay in a cohort of solid tumor patients: a proposed solution for decentralized plasma testing," which assessed the performance of the company’s elio plasma resolve assay (Press release, Personal Genome Diagnostics, AUG 11, 2021, View Source [SID1234586352]). This is the first study validating that a decentralized plasma-based next-generation sequencing (NGS) test can detect microsatellite instability (MSI) status along with the comprehensive landscape of sequence and structural alterations encountered across solid tumors.

The study, from Weill Cornell Medicine’s Englander Institute for Precision Medicine, profiled DNA from matched tissue and plasma samples from 75 cancer patients and showed that elio plasma resolve detected 77% of sequence alterations, amplifications, and fusions that were found in metastatic samples compared to 45% of those alterations found in the primary tumor samples. There was 87% agreement for MSI status between elio plasma resolve and tumor tissue results. In 3 cases, the assay’s identification of MSI-high circulating tumor DNA (ctDNA) correlated with response to immunotherapy. In addition, the PGDx kit revealed an FGFR2 amplification that was not detected in tumor tissue from a patient with metastatic gastric cancer.

"We are elated to see that in this study, elio plasma resolve demonstrated the comprehensive ability to identify MSI-high DNA samples from cancer patients," said Megan Bailey, Chief Executive Officer of PGDx. "We believe these results show tremendous potential for elio plasma resolve and the overall benefits of cell-free DNA testing, and we are hopeful that this testing can become routine practice in the treatment of cancer."

PGDx currently offers three pan-cancer NGS kitted solutions – elio tissue complete, an FDA cleared kit, elio plasma complete, a comprehensive liquid biopsy solution, and elio plasma resolve, which has received FDA breakthrough device designation – that provide researchers and clinicians with the ability to identify biomarkers and profile tumors through advanced genomic sequencing within their own hospital systems and laboratories. elio plasma resolve is designated for research use only in the U.S. and with a CE-IVD mark in Europe.

Gamida Cell Reports Second Quarter 2021 Financial Results and Provides Company Update

On August 11, 2021 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, reported financial results for the quarter ended June 30, 2021 (Press release, Gamida Cell, AUG 11, 2021, View Source [SID1234586351]). Net loss for the second quarter of 2021 was $21.3 million, compared to a net loss of $15.1 million for the same period in 2020. As of June 30, 2021, Gamida Cell had total cash and cash equivalents of $150.2 million.

During the quarter, the company continued to execute on plans to submit a Biologic License Application (BLA) for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of stem cell transplant. This submission is expected to occur by the end of the year, subject to a pre-BLA meeting with the U.S. Food and Drug Administration (FDA) planned for the fourth quarter. In addition, Gamida prepared to begin a Phase 1/2 trial of GDA-201 in non-Hodgkin lymphoma (NHL), expected to occur by the end of 2021. Also, the company expanded its NAM-enabled natural killer (NK) cell pipeline targeting solid-tumor and hematological cancers, including genetically modified variants of proprietary NK therapies using both CRISPR/Cas9 and CAR methodologies.

"Our progress this quarter represents a major step forward for Gamida Cell and our mission to bring cancer patients potentially curative cell therapies," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We are delivering against key process development, quality and manufacturing milestones in preparation for a BLA submission for omidubicel while also advancing our go-to-market strategy for our planned commercial launch. In parallel, we bolstered our NAM-enabled NK pipeline both by readying to advance GDA-201 into the clinic based on its encouraging clinical data in patients with hematological cancers and by expanding our NK cell pipeline to address solid and liquid tumors."

Q2 and Recent Developments

Omidubicel: Advanced Cell Therapy

Continued advancement toward planned BLA submission for omidubicel to the FDA in the fourth quarter of this year. The company’s activities included CMC qualification requirements at both the Gamida–owned facility in Israel and at Lonza, a contract manufacturing organization that will be supplying commercial material upon FDA approval. Advancements were made in analytical methods validation, analytical comparability and clinical manufacturing for Expanded Access Program patients, which are also planned to be used for clinical comparability.
Advanced launch planning activities by expanding Gamida’s commercial, operational and medical affairs teams. Conducted further market research and health economic and outcomes research (HEOR) to support planned market entry and market access activities. Readied Gamida Cell Assist, supply chain and logistics programs to facilitate positive patient and transplant center experiences at time of launch.
Announced that results of the international, multi-center, randomized Phase 3 clinical study of omidubicel were published in Blood, the official journal of the American Society of Hematology (ASH) (Free ASH Whitepaper). This pivotal trial compared the safety and efficacy of omidubicel to standard umbilical cord blood transplant in patients with high-risk hematologic malignancies undergoing a bone marrow transplant. The results demonstrate that transplantation with omidubicel leads to faster neutrophil and platelet recovery, and results in fewer bacterial, viral and fungal infections and less time in the hospital, compared to a standard umbilical cord blood graft.
GDA-201: NAM-Enabled NK Immunotherapy

Prepared for filing of an Investigational New Drug (IND) application with the FDA.
Finalized clinical study protocol and statistical plan for a planned Phase 1/2 clinical trial of allogeneic, cryopreserved GDA-201 in patients with follicular and diffuse large B-cell lymphoma.
Conducted study start-up activities, including contract research organization (CRO) and clinical site selections.
NAM-Enabled NK Cell Pipeline Expansion

Advanced four new development programs that involve modifications intended to direct NK cells against specific tumor markers to improve their cancer killing capabilities against both hematological and solid tumors. Newly designated product candidates include:
GDA-301: Knockout of CISH (cytokine inducible SH2 containing protein) in NK cells using CRISPR/Cas9 in combination with a membrane-bound IL-15/IL-15Ra. Designed to improve tumor killing by promoting activation and inhibiting negative feedback signals. Potential applications exist across a range of solid tumors and lymphoma.
GDA-401: Undisclosed target genetically engineered to enhance NK cell survival in the solid tumor microenvironment for potential application across a broad range of solid tumors.
GDA-501: CAR-engineered NK cells to target HER2+ solid tumors with the potential to enhance homing and activation against cancers with HER2 overexpression, including breast, ovarian, lung, bladder, gastric and others.
GDA-601: Knockout of CD38 on NK cells to avoid fratricide by CD38 targeted antibodies in combination treatment of multiple myeloma, combined with a CD38 CAR designed to enhance killing of cancerous cells.
Advanced additional NAM-enabled research programs targeting immunosuppressive pathways using both CRISPR/Cas9 and CAR, with potential to treat solid tumor and blood cancers.
Corporate

Hired Vladimir Melnikov as Senior Vice President, Global Operations and Manufacturing. Vladimir has over 25 years of experience in the biopharmaceutical industry. He previously served as general manager at Omrix Biopharmaceuticals and biologic technical operations lead at Ethicon Biosurgery, both part of a Johnson & Johnson Company. In those roles he supervised three Israeli biotech manufacturing sites and technology transfer to external partners. Vladimir will have responsibility for the company’s Israeli manufacturing site and manufacturing partnership with Lonza.
Hired Josh Patterson as General Counsel, effective August 30, 2021. Josh has over 20 years of experience as in-house legal counsel for biopharmaceutical companies. Josh will be joining Gamida Cell from Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, where he is currently General Counsel. Josh will be responsible for building, leading and managing the legal function for Gamida Cell.
Second Quarter 2021 Financial Results

Research and development expenses in the second quarter of 2021 were $13.5 million, compared to $9.3 million for the same period in 2020. The increase was mainly due to omidubicel commercial manufacturing readiness activities, and the advancement of the GDA-201 program, including broadening scientific capabilities and talent.
Commercial expenses in the second quarter of 2021 were $5.2 million, compared to $1.0 million for the second quarter of 2020. The increase was mainly attributed to progress with omidubicel commercial readiness activities.
General and administrative expenses were $3.8 million for the second quarter of 2021, compared to $2.5 million for the same period in 2020. The increase was mainly due to the hiring of key management positions to support business growth.
Finance income, net, was $1.2 million for the second quarter of 2021, compared to $2.2 million for the second quarter of 2020. The increase was primarily due to non-cash income, resulting from revaluation of warrants offset by interest expenses that resulted from the $75 million convertible note financing in February 2021.
Net loss for the second quarter of 2021 was $21.3 million, compared to a net loss of $15.1 million for the same period in 2020.

2021 Financial Guidance

Gamida Cell reiterates its prior financial guidance and expects cash used for ongoing operating activities in 2021 to range from $110 million to $120 million. The company believes that its current cash and cash equivalents will support the ongoing operating activities into the second half of 2022. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding and any business development activities that may be undertaken.

Expected 2021 Developments and Milestones

Gamida Cell plans to achieve the following key milestones during the second half of 2021:

Omidubicel

Pre-BLA meeting with FDA in the fourth quarter of 2021
BLA submission to the FDA in the fourth quarter of 2021
Commercial readiness activities ongoing for potential launch following approval
GDA-201

IND submission to FDA in third quarter 2021
Initiation of a company-sponsored Phase 1/2 clinical study in NHL before year-end 2021
NK cell pipeline expansion

Advance pipeline of NAM-enabled, genetically-modified NK cells in solid tumor and blood cancers
Conference Call Information

Gamida Cell will host a conference call today, August 11, 2021, at 8:00 a.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 5258448. A recording of the webcast will be available approximately two hours after the event, for approximately 30 days.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potentially life-saving1 allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.2,3 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.gamida-cell.com.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About GDA-201

Gamida Cell applied the capabilities of its nicotinamide (NAM)-based cell expansion technology to develop GDA-201, an innate NK cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results, as reported at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 has been in development through an investigator-sponsored study in patients with refractory NHL and multiple myeloma. For more information on the clinical study of GDA-201, please visit www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

Avacta Announces First Patient Dosed in AVA6000 Pro-Doxorubicin Phase I Clinical Trial

On August 11, 2021 Avacta Group plc (AIM: AVCT), a clinical stage biopharmaceutical company developing innovative cancer therapies and powerful diagnostics based on its proprietary Affimer and pre|CISION platforms, reported that the first patient has been dosed in its Phase I multicentre trial evaluating AVA6000, a novel pro-drug of Doxorubicin (Press release, Avacta, AUG 11, 2021, View Source [SID1234586350]).

AVA6000 is Avacta’s first therapeutic based on its proprietary pre|CISION technology. The platform incorporates a substrate that is specifically cleaved by the protease fibroblast activation protein alpha (FAPα), which is upregulated in most solid tumours and at low background levels in healthy tissue, providing an activation mechanism to ensure localised release of chemotherapeutic agents from their pro-drug form. By activating chemotoxins only within the tumour microenvironment, systemic exposure to healthy tissues is limited, improving the safety and therapeutic potential of these cancer therapies.

Incorporating the pre|CISION platform, AVA6000 is a tumour-activated form of Doxorubicin. Anthracyclines such as Doxorubicin, a generic chemotherapy for which the market is expected to grow to $1.38bn by 2024, are widely used as part of standard of care in several tumour types, but their use is limited by cumulative toxicity, particularly cardiotoxicity. Avacta’s pre|CISION pro-drug approach is designed to reduce the systemic exposure of healthy tissues to the active chemotherapy, leading to improved safety and therapeutic index, potentially resulting in improved dosing regimens, better efficacy and better outcomes for patients.

The AVA6000 study is a dose-escalation Phase I study in patients with locally advanced or metastatic selected solid tumours, known to be FAP-positive. These cohorts will receive ascending doses of AVA6000 to determine the maximum tolerated dose and establish a recommended Phase II dose. The second part of the study is an expansion phase where patients receive AVA6000 to further evaluate the safety, tolerability and clinical activity at this recommended Phase II dose across selected tumour types. For more information visit www.clinicaltrials.com (NCT04969835)

The first patient has received their first dose of AVA6000 at The Royal Marsden NHS Foundation Trust, London. The Phase I study is being initiated across a small group of leading UK oncology centres with an established reputation for early clinical research in cancer. The dose escalation phase is anticipated to complete by Q2 2022 followed by completion of the dose expansion phase by Q2 2023.

Dr Alastair Smith, Chief Executive Officer of Avacta Group, commented:

"The initiation of the first in human Phase I clinical study for AVA6000 marks the transformation of Avacta Life Sciences into a clinical stage biopharmaceutical company. It is an outstanding achievement by the team and we are extremely proud of what has been achieved since we established the collaboration with Professor William Bachovchin at Tufts University Medical School to develop the pre|CISION technology for tumour targeting.

"We are delighted to be working on the AVA6000 study with global key opinion leaders in oncology drug development at world-class oncology clinical trial sites in UK on this important pro-drug approach to improving the safety and efficacy of chemotherapies.

"If the study shows that the pre|CISION technology is effective in reducing systemic toxicity of Doxorubicin in humans, then that will open up an extensive and proprietary pipeline for Avacta of next-generation pre|CISION pro-drug chemotherapies with significant clinical and commercial advantages in a chemotherapy market that is expected to exceed $74 billion by 2027.

I look forward to further updating the market when key clinical development milestones are achieved."

Neil Bell, Chief Development Officer, Avacta Life Sciences commented:

"The initiation of this AVA6000 Phase I trial is a significant and transformational milestone for Avacta . AVA6000 offers an opportunity to improve upon the current doxorubicin treatment paradigm for patients, either as a monotherapy or in combination. We look forward to the results from our AVA6000 first-in-human clinical trial as we strive to improve therapeutic index of doxorubicin for patients."

AVA6000 Principal Investigator Professor Udai Banerji, Deputy Director of the Drug Development Unit at The Institute of Cancer Research, London and The Royal Marsden NHS Foundation Trust commented:

"I am delighted that the first patient has now received AVA6000 in the first-in-human study. This drug harnesses our understanding of the tumour microenvironment to enhance drug delivery – targeting potent anticancer therapies to tumours and potentially sparing patients debilitating side effects. It is fantastic that efforts are being made to discover and develop smarter, kinder treatments."

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Immunetune Strengthens Team by Appointing Sijme Zeilemaker as Chief Executive Officer

On August 11, 2021 Immunetune, a preclinical-stage biotech developing next-generation DNA vaccines against cancer and infectious diseases, reported the appointment of Sijme Zeilemaker as Chief Executive Officer (Press release, ImmuneTune, AUG 11, 2021, View Source [SID1234586349]). With his experience in several oncology biotech companies ranging from preclinical to Phase II clinical development and skillset in business development and investor relations, he will be responsible for bringing Immunetune into the next phase of the company.

"It is great to be joining a biotech working on truly differentiated technologies to bring a potentially best-in-class program into the clinic. Having seen the forefront of innovation in immuno-oncology the last few years, Immunetune has the right approach and results to make a difference. Gerben and his team have done an impressive amount of work and it is such an exciting phase to be joining them," stated Sijme Zeilemaker.

"We are thrilled bringing Sijme on board to strengthen our team at this pivotal point in time. With his experience with both big pharma partners and international life science investors, he knows exactly what data and technologies they will be looking for," says Gerben Zondag, founder of Immunetune, who will take on the role of Chief Operating Officer. "With the rest of the management team in place, we are confident to move our company and programs forward into the clinic."

Sijme Zeilemaker is joining Immunetune having previously acted as Chief Operating Officer, Director Business Development and Head of Investor Relations at Immunicum AB, a clinical-stage biotech developing cell therapies against cancer, publicly listed at the Nasdaq Stockholm. He joined Immunicum in 2017 as part of a small team and supported the growth of the company through a collaboration with Pfizer and Merck KGaA, three financing rounds, and the merger with DCprime. Before Immunicum, Sijme was Director Business Development at InteRNA Technologies, and held business development roles at to-BBB technologies and 2-BBB Medicines, transitioning these companies from preclinical to clinical-stage.