On August 11, 2021 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported financial results for the second quarter ended June 30, 2021 and provided a clinical and corporate update (Press release, Milestone Pharmaceuticals, AUG 11, 2021, View Source [SID1234586354]).

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"The second quarter of 2021 was marked by continued progress advancing our ongoing programs evaluating etripamil in patients with PSVT and AFib-RVR," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "With respect to the Phase 3 RAPID trial of etripamil in patients with PSVT, we are encouraged by continued improvements in study enrollment rates. We look forward to building on this momentum in the coming quarters and remain on track to report topline data in the second half of 2022."

Mr. Oliveto added, "Beyond PSVT, recent market research with cardiologists has strengthened our belief that etripamil has the potential to become a valuable treatment option for patients experiencing episodes of AFib-RVR."

Recent Updates

Entered Exclusive License Agreement with Ji Xing Pharmaceuticals to Develop and Commercialize Etripamil for PSVT in Greater China. In May 2021, the Company announced that it entered an exclusive license and collaboration agreement with Ji Xing Pharmaceuticals (Ji Xing), a biotechnology company headquartered in Shanghai and backed by RTW Investments, LP (RTW) focused on advancing innovative medicines in China, to develop and, if approved, commercialize the investigational drug etripamil in patients with paroxysmal supraventricular tachycardia (PSVT) and additional cardiovascular conditions in Greater China. In connection with the agreement, Milestone received an upfront cash payment consisting of $15 million and a $5 million equity investment by RTW Investments. In addition, the Company could receive up to $107.5 million in total development and sales milestone payments as well as tiered royalty payments on all products sold in the Greater China territory. Milestone will supply etripamil and delivery devices to Ji Xing. Ji Xing will be responsible for development and commercialization costs in Greater China.

Company Remains on Track to Report Topline Data from Pivotal Phase 3 RAPID Trial in the Second Half of 2022. Enrollment continues in the ongoing pivotal Phase 3 RAPID trial of etripamil nasal spray in patients with PSVT. As previously announced, the Company is working closely with study investigators to identify potential site-specific solutions to mitigate COVID-related enrollment and site initiation delays, and has also increased the number of participating centers. The Company continues to expect to report topline data in the second half of 2022.

The RAPID trial, which is targeting a total of 180 adjudicated PSVT events, is expected to randomize approximately 500 patients 1:1 to receive either etripamil or placebo. As previously announced, to maximize the potential treatment effect of etripamil, patients will be directed to administer a second dose of study drug if they do not experience symptom relief within 10 minutes of the first study drug administration. The primary efficacy analysis for both the RAPID trial and the completed NODE-301 trial will be time to conversion of SVT over the first 30 minutes following initial study drug administration, with a target p-value of less than 0.05 for each trial. The RAPID and NODE-301 trials could potentially serve to fulfill the efficacy requirement for a future New Drug Application (NDA) for etripamil in patients with PSVT.

Open-Label NODE-303 Safety Amended to Include Repeat Dosing of Etripamil. In May 2021, the Company announced that the U.S. Food and Drug Administration agreed to allow future patients enrolled in the ongoing NODE-303 study to utilize a repeat dose of etripamil if symptoms persist for 10 minutes after the first dose. NODE-303 is Milestone’s global open-label study, which primarily evaluates the safety of etripamil when self-administered without medical supervision during single or multiple SVT episodes. Important secondary measures include efficacy, patient quality of life, and pharmacoeconomic assessments. The Company is in the process of implementing the repeat dose regimen in the study.

Data from Study of Prevalence and Incidence of Patients with PSVT in the U.S. Published in the Journal of Cardiovascular Electrophysiology. In May 2021, findings from an observational retrospective longitudinal study using claims estimated the incidence and prevalence of PSVT in contemporary practice were published in the Journal of Cardiovascular Electrophysiology. The article, titled "Prevalence and incidence of patients with paroxysmal supraventricular tachycardia in the United States", estimated that 1.3 – 2.1 million people in the U.S. have PSVT, which includes patients with potentially comorbid atrial fibrillation/atrial flutter.

Enrollment Continues in ReVeRA Phase 2 Proof-of-Concept Trial Evaluating Etripamil in Patients Experiencing Atrial Fibrillation with Rapid Ventricular Rate (AFib-RVR). Enrollment is ongoing in ReVeRA, Milestone’s Phase 2 proof-of-concept study of etripamil nasal spray in patients experiencing AFib-RVR, in which patients will be randomized 1:1 to receive either 70 mg of etripamil or placebo. The Phase 2 double blind, placebo controlled, proof-of-concept in-patient study is designed to assess the safety and efficacy of etripamil nasal spray to reduce the ventricular rate in patients with AFib-RVR requiring treatment. The trial will be conducted in Canada in collaboration with the Montreal Heart Institute and other research centers. The primary endpoint will assess reduction in ventricular rate, with key secondary endpoints including the time to achieve the maximum reduction in rate and duration of the effect.
Second Quarter 2021 Financial Results

As of June 30, 2021, Milestone had cash, cash equivalents, and short-term investments of $135.8 million and 29.8 million common shares issued and outstanding and 12.3 million common shares issuable upon exercise of pre-funded warrants outstanding.

Revenue of $15 million was generated from the upfront payment under the License Agreement during the three months and six months ended June 30, 2021.

Research and development expense for the second quarter of 2021 was $9.4 million compared with $8.6 million for the prior year period. The difference is due to an increase in clinical expense of $0.8 million. For the six months ended June 30, 2021, research and development expense was $18.0 million compared with $20.5 million for the prior year period. The COVID-19 pandemic contributed to delays in new clinical site initiation and patient enrollment, which translated into lower than expected research and development spending in the six months ended June 30, 2021.

General and administrative expenses for the second quarter of 2021 and 2020 were $3.0 million. For both of the six month periods ended June 30, 2021 and 2020, respectively, general and administrative expense was $5.7 million.

Commercial expense for the second quarter of 2021 was $1.8 million compared with $1.5 million for the prior year period. During the second quarter of 2020, the Company reduced commercial costs in order to focus its efforts on an optimized clinical development pathway for etripamil. During the three months ended June 30, 2021, the Company increased our investment in commercialization activities resulting in higher commercial expenses. For the six months ended June 30, 2021, commercial expense was $3.2 million compared with $3.7 million for the prior year period. The decrease of commercial expense in the six months ended June 30, 2021 reflects efforts reducing operating expenses from pre-commercialization activities.

For the second quarter of 2021, operating income was $0.7 million compared to operating loss of $13.1 million in 2020. For the six months ended June 30, 2021, Milestone’s operating loss was $11.9 million compared to $29.9 million in the prior year period.
About Paroxysmal Supraventricular Tachycardia

Paroxysmal supraventricular tachycardia (PSVT) is a condition characterized by intermittent episodes of rapid heart beat (SVT) that starts and stops suddenly and without warning that affects approximately two million Americans. Episodes of SVT are often associated with symptoms including palpitations, sweating, chest pressure or pain, shortness of breath, sudden onset of fatigue, lightheadedness or dizziness, fainting, and anxiety. Certain calcium channel blockers have long been approved for the treatment of PSVT as well as other cardiac conditions. However, calcium channel blockers approved for the termination of SVT episodes must be administered intravenously under medical supervision, usually in an emergency department or other acute care setting.

About Atrial Fibrillation with Rapid Ventricular Rate

Atrial fibrillation (AFib) is a common arrhythmia marked by an irregular and often rapid heartbeat. AFib is estimated to affect five million patients in the United States, a prevalence projected by the Centers for Disease Control to increase to twelve million patients within the next 10 years. Atrial fibrillation with rapid ventricular rate (AFib-RVR) is a condition in which patients with AFib experience episodes of abnormally high heart rate, often with symptoms such as palpitations, shortness of breath, dizziness, and weakness. Oral calcium channel blockers and/or beta blockers are commonly used to manage heart rate in this condition. When episodes do occur, the corresponding symptoms often cause patients to seek care in the acute care setting such as the emergency department, where standard of care procedures include intravenous administration of calcium channel blockers or beta blockers under medical supervision. Milestone’s initial qualitative market research indicates that approximately 40% of patients with AFib experience one or more symptomatic episodes of RVR per year that require treatment, suggesting a target addressable market for etripamil in patients with AFib of approximately two million patients.

About Etripamil

Etripamil, Milestone’s lead investigational product, is a novel calcium channel blocker designed to be a rapid-response therapy for episodic cardiovascular conditions. As a nasal spray that is self-administered by the patient, etripamil has the potential to shift the current treatment experience for many patients from the emergency department to the at-home setting. Milestone is conducting a comprehensive development program for etripamil, with Phase 3 trials ongoing in paroxysmal supraventricular tachycardia (PSVT) and now a Phase 2 proof-of-concept trial underway in patients with atrial fibrillation and rapid ventricular rate (AFib-RVR).

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

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.

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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.

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.

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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.

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]).

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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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