On August 16, 2021 HDT Bio Corp., a developer of immunotherapies for oncology and infectious diseases, reported an agreement with Korean biotech Quratis Inc. to co-develop HDT’s revolutionary COVID-19 vaccine (a next generation mRNA vaccine) for distribution in South Korea and neighboring countries (Press release, HDT Bio, AUG 16, 2021, View Source [SID1234586649]). HDT previously established a collaboration with Gennova Biopharmaceuticals in India, and it plans to announce similar deals in China and Brazil soon.

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"This agreement will enable Quratis to manufacture our next-generation COVID-19 vaccine and distribute it across much of East Asia," said HDT Bio CEO Steve Reed. "Today, we have advanced both HDT’s business prospects and its global health reach."

HDT Bio’s COVID-19 vaccine uses a proprietary Lipid InOrganic Nanoparticle (LIONTM) formulation to deliver immune-stimulating RNA fragments to targeted cells. The vaccine, currently in clinical trials to establish safety and efficacy, is significantly different from current mRNA vaccines in two ways. First, its RNA payload is designed to amplify itself inside the body. As a result, the vaccine effectively activates the immune system at a much lower dose than current vaccines, enhancing safety and reducing manufacturing costs. Second, the HDT LION formulation system simplifies manufacture and enhances stability.

"Partnering with HDT Bio gives us a unique opportunity to fight COVID-19 with the world’s most advanced mRNA vaccine technology not only in Korea, but also in other nations in our region to help bring an end to this deadly pandemic," said Kwan Goo Cho, President of Quratis.

Quratis got an IND approval from Ministry of Food and Drug Safety for Phase I clinical trial of a COVID-19 vaccine in mid-July and is expected to begin evaluating QTP104 in a Phase 1 clinical trial shortly.

On August 16, 2021 Ensysce Biosciences, Inc. ("Ensysce" or the "Company") (NASDAQ: ENSC, OTC: ENSCW), a clinical-stage biotech company with proprietary technology platforms to reduce the economic and social burden of prescription drug abuse and overdose, reported financial results for the second quarter of 2021 and recent corporate updates (Press release, Ensysce Biosciences, AUG 16, 2021, View Source [SID1234586673]).

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"In the second quarter, we successfully closed our merger with Leisure Acquisition Corp., providing us with financial resources to advance our lead clinical programs and focus on expanding our pipeline of products in the pain, opioid use disorder (OUD) and ADHD space as a publicly listed company," said Dr. Lynn Kirkpatrick, CEO of Ensysce Biosciences. "We currently have two technology platforms, TAAP and MPAR, with three clinical-stage product candidates. Our pipeline of candidates provides us with the ability to grow each of these programs. Our focus is on prioritization and resource allocation to maintain an optimal balance between aggressively pursuing our more advanced clinical-stage product candidates, such as PF614, PF614-MPAR, and nafamostat, while ensuring the continued development of additional potential product candidates. We have partnered with contract development and manufacturing organizations (CDMOs) to bolster our team and facilitate multiple parallel development programs. Our view, based on data so far and feedback from experts, is that our lead assets for chronic and severe pain have the potential to be transformational."

Dr. Kirkpatrick concluded, "Over the long-term, it is our vision to develop the next generation of innovative solutions for severe pain relief while the reducing the fear of and the potential for opioid misuse, abuse and overdose."

Program Updates

TAAP – opioid abuse deterrent program:

Ensysce’s lead TAAP candidate, PF614, entered Phase 1b/Bioequivalence clinical development.
Ensysce entered into an agreement for manufacture of PF614 clinical trial material with Recro Pharma.
MPAR – opioid overdose protection program:

PF614-MPAR, our overdose protection program lead product, received an Investigational New Drug (IND) allowance from the U.S. Food and Drug Administration.
Notice of Award for year 3 of a multi-year grant was received from the National Institute on Drug Abuse (NIDA), providing Ensysce with additional resources to continue its work to bring PF614-MPAR into clinical development.
PF614-MPAR entered Phase 1 clinical development utilizing a ‘translational pharmaceutics’ approach.
Other programs:

Ensysce entered into an agreement for manufacture of nafamostat clinical trial material with Recro Pharma for its COVID-19 oral therapy program.
Ensysce received a Notice of Allowance from the United States Patent and Trademark Office for a patent entitled Compositions Comprising Enzyme-Cleavable Amphetamine Prodrugs and Inhibitors Thereof. This issuance provides the Company with additional pipeline candidates for ADHD indications.
Second Quarter 2021 Financial Results

Cash – Cash and cash equivalents were $8.0 million as of June 30, 2021. With the public listing of its common stock following the closing of the merger with Leisure Acquisition Corp. on June 30th, Ensysce now has access to a share subscription facility of up to $60 million which it entered into in December 2020. As such, Ensysce believes it has access to sufficient capital to fund its current planned operations for at least the next twelve months.
Federal Grants – Funding under federal grants was $0.4 million for the second quarter of 2021 compared to $1.8 million for the second quarter of 2020. The decrease is attributable to the timing of research activities eligible for funding.
R&D Expenses – Research and development expenses were $0.5 million for the second quarter of 2021 compared to $1.4 million for the same period in 2020. The decrease was primarily resulted from reduced external research and development costs related to preclinical programs for PF614-MPAR and Phase 1 clinical trial activities of nafamostat.
G&A Expenses – General and administrative expenses were $0.4 million for the second quarter of 2021 compared to $0.3 million for the second quarter of 2020. The increase was primarily a result of higher legal and other professional services expenses related to post-merger corporate matters.
Net Loss – Net loss for the second quarter of 2021 was $1.0 million compared to $0.7 million for the same period in 2020.

On August 16, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the second quarter ended June 30, 2021 (Press release, Mustang Bio, AUG 16, 2021, View Source [SID1234586634]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "In the first half of 2021, Mustang continued to progress the development of our CAR T therapies across multiple cancers, as well as our lentiviral gene therapies for the treatment of X-linked severe combined immunodeficiency ("XSCID"), also known as bubble boy disease. We are encouraged by the updated interim MB-106 CD20-targeted, autologous CAR T data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress ("EHA2021") in June. The data presented showed a favorable safety profile and compelling clinical activity, with a 93% overall response rate and 67% complete response rate in patients with high-risk B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL") who were treated with our modified cell manufacturing process. Additionally, the U.S. Food and Drug Administration ("FDA") accepted Mustang’s Investigational New Drug ("IND") application to initiate a multicenter Phase 1/2 clinical trial investigating the safety, tolerability and efficacy of MB-106 for relapsed or refractory B-NHL and CLL. We look forward to enrolling the first patient in the trial later this quarter and to further advancing MB-106 for patients with B-NHL and CLL."

Dr. Litchman continued, "Last week, we announced an exclusive license agreement for a novel in situ CAR T technology that may be able to transform the administration of CAR T therapies, facilitating how the treatments are delivered to patients, with the potential to be used broadly as an off-the-shelf therapy. With this collaboration with the Mayo Clinic, we gain access to an innovative platform technology that we hope will become the discovery engine for a whole new generation of CAR T products at Mustang. We also announced that the first patient was dosed at City of Hope in a clinical trial to establish the safety and feasibility of administering MB-101 (autologous IL13Rα2-targeted CAR T cells) to patients with leptomeningeal brain tumors. On the regulatory front, we are delighted that the European Medicines Agency ("EMA") recently granted Priority Medicines ("PRIME") designation to MB-107, our lentiviral gene therapy for the treatment of XSCID in newly diagnosed infants. We anticipate enrolling the first patient in the pivotal Mustang-IND MB-107 trial for newborns with XSCID shortly and also expect to file an IND for a pivotal MB-207 trial in previously transplanted XSCID patients later this quarter. We look forward to continuing to provide updates on our CAR T and gene therapy clinical programs in the second half of the year."

Recent Corporate Highlights:

In May 2021, Mustang announced that the FDA approved its IND application to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted CAR T for relapsed or refractory B-NHL and CLL.
Also in May 2021, Mustang announced that the first patient was dosed at City of Hope in a clinical trial to establish the safety and feasibility of administering MB-101 (autologous IL13Rα2-directed CAR T cells) to patients with leptomeningeal brain tumors (e.g., glioblastoma, ependymoma or medulloblastoma).
In June 2021, Mustang announced MB-106 CD20-targeted CAR T data were presented at EHA (Free EHA Whitepaper)2021. Dr. Mazyar Shadman of Fred Hutchinson Cancer Research Center presented updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL, which showed a favorable safety profile and compelling clinical activity with a 93% overall response rate and 67% complete response rate in patients treated with the modified cell manufacturing process.
Also in June 2021, Mustang hosted a key opinion leader webinar featuring a presentation from Dr. Shadman, who discussed interim results from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for B-NHL and CLL. A replay of the webinar can be found here.
Additionally in June 2021, Mustang announced that it has been awarded a $300,000 Massachusetts Life Sciences Center tax incentive based on a hiring commitment of 20 net new full-time equivalent employees for calendar year 2021 and retaining that headcount level through 2025.
Earlier this month, Mustang announced that the EMA granted PRIME designation to MB-107, its lentiviral gene therapy for the treatment of XSCID in newly diagnosed infants.
Last week, Mustang announced an exclusive license agreement with Mayo Clinic for a novel technology that may be able to transform the administration of CAR T therapies and has the potential to be used as an off-the-shelf therapy.
Financial Results:

As of June 30, 2021, Mustang’s cash and cash equivalents and restricted cash totaled $130.9 million, compared to $130.4 million at March 31, 2021 and $98.8 million as of December 31, 2020, an increase of $0.5 million for the quarter and an increase of $32.1 million year-to-date.
Research and development expenses including license acquisitions were $11.9 million for the second quarter of 2021, compared to $11.1 million for the second quarter of 2020. Non-cash, stock-based expenses included in research and development were $0.3 million for the second quarter of 2021, compared to $0.4 million for the second quarter of 2020.
General and administrative expenses were $2.5 million for the second quarter of 2021, compared to $3.0 million for the second quarter of 2020. Non-cash, stock-based expenses included in general and administrative expenses were $0.6 million for the second quarter of 2021, compared to $1.5 million for the second quarter of 2020.
Net loss attributable to common stockholders was $14.4 million, or $0.16 per share, for the second quarter of 2021, compared to a net loss attributable to common stockholders of $14.6 million, or $0.32 per share, for the second quarter of 2020.

On August 16, 2021 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the quarter ended June 30, 2021, as well as key clinical and corporate developments (Press release, Cel-Sci, AUG 16, 2021, View Source [SID1234586650]).

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Clinical and Corporate Developments include:

On June 28, 2021, CEL-SCI announced top line results from its 9.5 year global pivotal Phase 3 study for its immunotherapy Multikine (Leukocyte Interleukin, Injection)* in head and neck cancer. The Phase 3 results showed a long-term 5-year overall survival (OS) benefit in the treatment arm receiving Multikine treatment regimen followed by surgery and radiation. This survival benefit was statistically significant, robust and durable, with no safety issues, something not commonly seen with cancer drugs. In fact, the survival benefit increased over time and at 5-years the overall survival benefit reached an absolute 14.1% advantage for the Multikine treated arm over control (n=380, total study patients treated with surgery plus radiation): Multikine arm 62.7%, control arm 48.6% survival.
The OS benefit of 14.1% at 5 years for this treatment arm exceeded the >10% OS benefit set out for the study population as a whole in the protocol. The OS results for this treatment arm were significant (two-sided p=0.0236, HR=0.68) and the survival effect increased over time. The results from the Phase 3 cancer study proved that Multikine met all of the protocol required benefits stated in the study protocol in patients in the treatment arm receiving surgery and radiation as their standard therapies. Based on the results of this pivotal Phase 3 study, CEL-SCI intends to file a Biologic License Application with U.S. Food and Drug Administration (FDA) for approval of this indication. We are assembling the information required to request a pre-BLA (Biologics License Application) meeting with the FDA to discuss the adequacy of the study results to support a license application and receive FDA input on any other issues that would have to be addressed for an approval to be granted.
Worldwide there are approximately 890,000 newly diagnosed head and neck cancer patients of which CEL-SCI’s target population when filing for FDA approval is about 210,000 patients. That would mean that approximately 29,000 patients could be alive at 5-years if all 210,000 eligible patients received the Multikine treatment regimen followed by surgery and radiation compared to receiving surgery and radiation alone, the current treatment paradigm/SOC for these patients.
CEL-SCI is near completion of the expansion of its existing dedicated cGMP manufacturing facility for Multikine. The construction, which began in 2020, will double the current facility’s capacity to accommodate two shifts for increased production of Multikine.
CEL-SCI raised net proceeds of $53.6 million during the nine months ended June 30, 2021 through the sale of common stock and the exercise of warrants and options. As of June 30, 2021, CEL-SCI had $47.1 million in cash, cash equivalents and U.S. Treasury Bills.

"The results of this 10-year landmark study proved our novel concept of cancer treatment, 1) that the use of our investigational cancer immunotherapy Multikine before the usual first cancer treatments (as neoadjuvant treatment) should significantly increase survival and 2) that a cancer drug can have a very favorable safety profile. Our Phase 3 study results are the first sign of real progress in the treatment of advanced primary head and neck cancer in many decades. We estimate about 210,000 patients per year globally who could potentially benefit from this drug once approved. Our goal is to seek FDA approval based on the data from our recently concluded pivotal Phase 3 study," stated CEL-SCI CEO, Geert Kersten.

CEL-SCI reported an operating loss of $27.7 million for the nine months ended June 30, 2021, versus an operating loss of $20.5 million for the nine months ended June 30, 2020. CEL-SCI reported an operating loss of $10.5 million for the quarter ended June 30, 2021, versus an operating loss of $7.0 million for the quarter ended June 30, 2020.

On August 16, 2021 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported financial results for the second quarter ended June 30, 2021 and provided corporate updates (Press release, Cogent Biosciences, AUG 16, 2021, View Source [SID1234586605]).

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"People fighting advanced systemic mastocytosis live every day in need of additional treatment options. We initiated the Phase 2 APEX clinical study for our lead program bezuclastinib, designed to specifically target the underlying genetic cause of advanced systemic mastocytosis with the goal of providing new therapeutic options for patients," said Andrew Robbins, President and CEO of Cogent Biosciences. "In addition, we look forward to advancing bezuclastinib into two new clinical studies by the end of 2021, including the SUMMIT study for patients with non-advanced systemic mastocytosis, while actively progressing novel discovery programs with the recently formed Cogent Research Team."

Recent Program and Corporate Highlights

APEX Study in AdvSM Patients Initiated
Cogent initiated APEX, a Phase 2 clinical study of bezuclastinib in patients with AdvSM. APEX is an open-label, global, multicenter study that will evaluate the safety, efficacy, pharmacokinetic, and pharmacodynamic profiles of bezuclastinib. Learn more about the APEX study at www.cogentclinicaltrials.com
Cogent expects to report preliminary clinical data from the APEX study in the first half of 2022, including levels of serum tryptase, a validated biomarker of mast cell activity.
SUMMIT Study of bezuclastinib in NonAdvSM Patients on track to start 2H 2021
Following recent positive interactions with FDA, Cogent is on track to initiate SUMMIT, a randomized, double-blind placebo-controlled Phase 2 clinical study of bezuclastinib in patients with NonAdvSM.
FDA Granted Orphan Drug Designation for bezuclastinib in GIST (Gastrointestinal Stromal Tumors)
FDA granted orphan drug designation for bezuclastinib for the treatment of GIST with an estimated 4,000 to 6,000 GIST cases diagnosed annually in the United States.
Cogent remains on track to initiate a new study of bezuclastinib and sunitinib in GIST patients during 2H 2021.
Cogent Research Team continues to evolve efforts in pioneering best-in-class, small molecule therapeutics
Based in Boulder, Colorado, the Cogent Research Team continues to focus on developing best-in-class, small molecule therapeutics to expand Cogent’s pipeline and deliver novel precision therapies for patients living with unmet medical needs.
Announced long-term lease on ~40,000 sq. ft. facility which will serve as laboratory and office space headquarters for Cogent Research Team.
Formed the Cogent Scientific Advisory Board
Comprised of world-class experts involved in the discovery and development of novel therapeutics for patients with genetically-driven diseases, this group has been brought together to provide external perspective for the Cogent Research Team as it develops a robust portfolio of novel, small molecule discovery programs designed to address significant patient unmet needs.
Dr. Ryan Corcoran, MD, PhD – Director of the Gastrointestinal Cancer Center Program and Scientific Director, Termeer Center for Targeted Therapy at the Massachusetts General Hospital Cancer Center and Associate Professor of Medicine at Harvard Medical School
Dr. Michael Vasconcelles, MD – Chief Medical Officer of Flatiron Health, a healthcare technology and services company focused on accelerating cancer research and improving patient care
Dr. Srdan Verstovsek, MD, PhD – United Energy Resources, Inc. Professor of Medicine and a hematologist-oncologist at the MD Anderson Cancer Center, Houston, Texas, USA
Dr. Kwok-Kin Wong, MD, PhD – Director, Division of Hematology and Medical Oncology, Anne Murnick Cogan and David H. Cogan Professor of Oncology, Department of Medicine, Laura and Isaac Perlmutter Cancer Center, NYU Langone Health
Second Quarter 2021 Summarized Financial Results

R&D Expenses: Research and development expenses were $12.4 million for the second quarter of 2021 as compared to $5.1 million for the second quarter of 2020. R&D expenses include non-cash stock compensation expense of $1.0 million for the second quarter of 2021 compared to $0.5 million for the second quarter of 2020.
G&A Expenses: General and administrative expenses were $4.9 million for the second quarter of 2021 as compared to $2.8 million for the second quarter of 2020. G&A expenses include non-cash stock compensation expense of $1.6 million for the second quarter of 2021 compared to $0.4 million for the second quarter of 2020.
Net Loss: Net loss was $16.5 million for the second quarter of 2021 as compared to a net loss of $7.4 million for the second quarter of 2020. During the second quarter of 2021, the company spent $12.6 million of its cash and cash equivalents.
Cash and Cash Equivalents: As of June 30, 2021, Cogent had cash and cash equivalents of $218.1 million. The company believes that its cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into 2024.