On November 4, 2021 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, reported that Aviad Pato, Ph.D., Head of Immunology Research, presented data on two nicotinamide (NAM)-enabled NK cell therapies, GDA-501 and GDA-301, at the Protein & Antibody Engineering Summit (PEGS) Europe taking place in Barcelona, Spain, and virtually November 2-4, 2021 (Press release, Gamida Cell, NOV 4, 2021, View Source [SID1234594517]).

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 presentation included data from early-stage studies of GDA-501, Gamida Cell’s investigational cell therapy comprised of CAR-engineered NK cells designed to enhance homing and activation against cancers with HER2 overexpression such as breast, ovarian, lung, bladder, and gastric cancers. Data were also presented on GDA-301, which combines a CRISPR/Cas9 knockout of the CISH (cytokine inducible SH2 containing protein) gene in NK cells with a membrane-bound IL-15/IL-15Ra CAR, which is designed to improve tumor killing by promoting activation and inhibiting negative feedback signals and has potential application in a range of solid tumors and hematologic malignancies.

Data presented by Gamida Cell demonstrated that the engineered GDA-501 NK enhances potency and cytotoxicity against a HER2-expressing tumor cell line. Data also showed that GDA-301 has cytotoxic activity against a chronic myelogenous leukemia cell line (K562) and a multiple myeloma cell line (RPMI).

"The field of NK cell immunotherapy is advancing beyond what has previously been understood from T cell gene editing," said Yona Geffen, Ph.D., Vice President, Research and Development at Gamida Cell. "We are pleased to share this important update on two key potential therapies in Gamida Cell’s robust NK pipeline that show their potential as clinical immunotherapy agents."

The full presentation shared at PEGS Europe is available at www.gamida-cell.com.

On November 4, 2021 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage biotechnology company improving the lives of those affected by cancer with its next-generation, targeted antibody drug conjugates (ADCs) for patients with hematologic malignancies and solid tumors, reported abstracts for ZYNLONTA and ADCT-602 have been accepted for presentations at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held virtually and in Atlanta, Georgia from December 11-14, 2021 (Press release, ADC Therapeutics, NOV 4, 2021, View Source [SID1234594535]).

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!

"We look forward to sharing data on our targeted ADCs as single agents and in innovative combinations at the 2021 ASH (Free ASH Whitepaper) Annual Meeting," said Joseph Camardo, MD, Chief Medical Officer of ADC Therapeutics. "This will include presentations from investigators on subset data from our pivotal Phase 2 study and data on the use of ZYNLONTA post-CAR-T. We are also encouraged by the anti-tumor activity and manageable safety profile of ZYNLONTA in combination with ibrutinib. The Phase 2 protocol has recently been amended with a higher and more frequent dose of ZYNLONTA to potentially enhance the response and to investigate this combination in earlier lines of therapy."

Details of ADC Therapeutics’ oral presentation are as follows:

Planned Interim Analysis of a Phase 2 Study of Loncastuximab Tesirine Plus Ibrutinib in Patients with Advanced Diffuse Large B-Cell Lymphoma (LOTIS-3)
Abstract: 54
Date and Time: Saturday, December 11, 2021, 10:45 a.m. EST
Session: 627. Aggressive Lymphomas: Clinical and Epidemiological: Population data for Aggressive NHL Management
Presenter: Carmelo Carlo-Stella, MD, Department of Biomedical Sciences, Humanitas University, and Department of Oncology and Hematology, IRCCS Humanitas Research Hospital, Milan, Italy

Details of ADC Therapeutics’ poster presentations are as follows*:

Clinical Characteristics and Responses of Patients with Relapsed or Refractory High-Grade B-cell Lymphoma Treated with Loncastuximab Tesirine in the LOTIS-2 Clinical Trial
Abstract: 3575
Date: Monday, December 13, 2021
Session: 626. Aggressive Lymphomas: Prospective Therapeutic Trials: Poster III
Presenter: Juan Pablo Alderuccio, MD, Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL, USA

Combination of Loncastuximab Tesirine and Polatuzumab Vedotin Shows Increased Anti-Tumor Activity in Pre-Clinical Models of Non-Hodgkin Lymphoma
Abstract: 2273
Date: Sunday, December 12, 2021
Session: 605. Molecular Pharmacology and Drug Resistance: Lymphoid Neoplasms: Poster II
Presenter: Francesca Zammarchi, PhD, ADC Therapeutics

CD19-mediated DNA Damage Boost in Lymphoma Cells Treated with Loncastuximab Tesirine in Combination with PARP inhibitors
Abstract: 1342
Date: Saturday, December 11, 2021
Session: 622. Lymphomas: Translational—Non-Genetic: Poster I
Presenter: Stefania Fusani, PhD, Oncohematology Division, IEO Istituto Europeo di Oncologia IRCCS, Milano, Italy

Details of an independently developed ZYNLONTA poster are as follows:

The Anti-CD19 Antibody-Drug Conjugate Loncastuximab Tesirine Achieved Responses in Patients with Diffuse Large B-cell Lymphoma Who Relapsed After Anti-CD19 CAR T-Cell Therapy
Abstract: 2489
Date: Sunday, December 12, 2021
Session: 626. Aggressive Lymphomas: Prospective Therapeutic Trials: Poster II
Presenter: Paolo F. Caimi, MD, Cleveland Clinic/Case Comprehensive Cancer Center, Cleveland, OH, USA

Details of an independently developed ADCT-602 poster are as follows:

A Phase 1 Trial of ADCT-602, a CD22 Targeting Antibody Drug Conjugate Bound to PBD Toxin in Adult Patients with Relapsed or Refractory CD22+ B-Cell Acute Lymphoblastic Leukemia
Abstract: 1237
Date: Saturday, December 11, 2021
Session: 614. Acute Lymphoblastic Leukemias: Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster I
Presenter: Nitin Jain, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

*Posters will be available in the poster exhibit hall in the Georgia World Congress Center on these dates: December 11: 9:00 a.m.–7:30 p.m. EST; December 12 & 13: 9:00 a.m.–8:00 p.m. EST. Presenters planning to attend in-person are expected to present during the final two hours of the noted viewing time.

The abstracts are available through the ASH (Free ASH Whitepaper) online meeting program and will be published in the November supplemental issue of Blood.

About ZYNLONTA (loncastuximab tesirine-lpyl)

ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) has approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.

On November 4, 2021 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported its financial results for the third quarter ended September 30, 2021 and provided an update on the Company’s operations (Press release, BridgeBio, NOV 4, 2021, View Source [SID1234594564]).

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!

BridgeBio has more than 30 programs in its pipeline for patients living with genetic diseases and cancers and 20 ongoing clinical trials underway across more than 450 sites around the world. Earlier this year BridgeBio received its first two U.S. Food and Drug Administration (FDA) drug approvals and has successfully filed 15 Investigational New Drug (IND) applications since the Company’s founding in 2015.

BridgeBio’s four core value drivers:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): Topline results from Part A of the Phase 3 ATTRibute-CM trial are expected in late 2021 and from Part B in 2023. The primary endpoint at Part A is the change from baseline in a 6-minute walk distance (6MWD) in trial participants receiving acoramidis or placebo after 12 months. If the change from baseline in 6MWD in Part A is highly statistically significant, BridgeBio expects to submit an application for regulatory approval of acoramidis in 2022 to the FDA. The study enrolled more than 600 subjects with either wild-type or variant TTR across more than 80 sites in 18 countries. ATTR-CM is a rare heart condition with a progressive and debilitating impact on quality of life likely affecting more than 400,000 patients worldwide.
Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1): BridgeBio presented updated Phase 2b data for encaleret in an oral presentation at the American Society of Bone and Mineral Research (ASBMR) 2021 Annual Meeting in October. Within five days of individualized dose titration in 13 participants, encaleret normalized mean blood calcium levels and 24-hour urine calcium excretion. Achieving simultaneous blood and urine calcium normalization is a challenge for patients with ADH1 due to the limitations of current standard-of-care. If approved, encaleret could be the first therapy on the market for ADH1, a condition caused by gain of function variants in the calcium-sensing receptor (CASR) gene estimated to be carried by 12,000 individuals in the United States alone. BridgeBio plans to initiate a Phase 3 registrational trial of encaleret in patients with ADH1 in 2022.
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Initial data from the ongoing Phase 2 dose ranging study are expected in the first half of 2022. Achondroplasia is the most common form of genetic short stature and one of the most common genetic diseases, with a prevalence of greater than 55,000 cases in the United States and European Union. Low-dose infigratinib is the only known product candidate in clinical development for achondroplasia that is designed to target the disease at its genetic source and the only orally administered product candidate in clinical-stage development.
BBP-631 – AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH): Received Fast Track designation from the FDA in May 2021. IND cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing, with initial data anticipated in mid-2022. CAH is one of the most prevalent genetic diseases potentially addressable with AAV gene therapy, with more than 75,000 cases estimated in the United States and European Union. The disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to lack of endogenous cortisol production. BridgeBio’s AAV5 gene therapy candidate is designed to provide a functional copy of the 21-hydroxylase-encoding gene (CYP21A2) and potentially address many aspects of the disease course.
Recent pipeline progress and corporate updates:

Stock repurchases: BridgeBio repurchased $148.4 million in BridgeBio common stock under its 2021 Share Repurchase Program, demonstrating the Company’s confidence in the long-term prospects of its pipeline.
LianBio IPO and partnership: China-based partner LianBio raised $325 million in its initial public offering on November 1. BridgeBio is estimated to own approximately 4.7% post-IPO. BridgeBio and LianBio announced in August that the first patient was treated in a Phase 2a trial of infigratinib in patients with gastric cancer and other advanced solid tumors. LianBio in-licensed rights from BridgeBio for infigratinib for development and commercialization in Mainland China, Hong Kong and Macau.
RAS cancer portfolio: BridgeBio announced the discovery of its next-generation KRAS G12C dual inhibitors, the first-known compounds that directly bind and inhibit KRAS in both its active (GTP bound) and inactive (GDP bound) conformations, and PI3ka:RAS breakers, small molecules that block RAS driven PI3Ka activation – a novel approach with the potential to inhibit oncogenic PI3Ka signaling without adverse effects on glucose metabolism. RAS is one of the most well-known oncogenic drivers with approximately 30% of all cancers being driven by RAS mutations, including large proportions of lung, colorectal and pancreatic tumors. BridgeBio expects to select a RAS development candidate in 2022.
BBP-812 – AAV9 gene therapy candidate for Canavan disease: BridgeBio announced that the first patient was dosed in its Phase 1/2 trial of BBP-812 for Canavan disease. If successful, BridgeBio’s gene therapy could be the first approved therapeutic option for children born with Canavan disease, a devastating and life-threatening condition. An initial Phase 1/2 data readout is expected in 2022.
BBP-818 – AAV gene therapy candidate for classic galactosemia (severe GALT deficiency): BridgeBio announced a new gene therapy program for classic galactosemia, which is caused by a severe deficiency of the enzyme galactose-1-phosphate uridylyltransferase (GALT), affecting approximately 7,000 patients in the United States and the European Union. Preclinical studies in a mouse model of classic galactosemia have shown that BridgeBio’s BBP-818 therapy restored up to 72% of wild-type levels of GALT enzyme in the brain following a single dose.
BBP-398 – SHP2 inhibitor: First publication of preclinical data for BridgeBio’s potentially best-in-class SHP2 inhibitor designed for the treatment of resistant cancer. Data demonstrated activity in non-small cell lung cancer (NSCLC) driven by RAS or other MAPK-pathway activating mutations. The results were featured in a poster presentation shared at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October.
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2i (LGMD2i): Fast Track designation granted by the FDA. The Phase 2 trial was initiated in patients with LGMD2i in the first quarter of 2021. If successful, BBP-418 could be the first approved therapy for patients with LGMD2i. With approximately 7,000 patients with potentially treatable mutations, LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein. A Phase 2 data readout is expected in 2022.
BBP-711 – Glycolate oxidase (GO) inhibitor for hyperoxaluria: BridgeBio announced preliminary Phase 1 data in which BBP-711 was well-tolerated and resulted in maximal increases in plasma glycolate exceeding those achieved by any GO-targeting agents reported in healthy adult volunteers. BBP-711 is being developed for the treatment of primary hyperoxaluria type 1 (PH1) and hyperoxaluria caused by hepatic overproduction of oxalate in recurrent kidney stone formers. A full readout of Phase 1 data in healthy adult volunteers is expected in 2022, to be followed by initiation of a Phase 2/3 trial in PH1 and a Phase 2 proof-of-concept trial in recurrent kidney stone formers.
TRUSELTIQ (infigratinib): Health Canada approved TRUSELTIQ (infigratinib), a small molecule kinase inhibitor that targets fibroblast growth factor receptor (FGFR), under the Notice of Compliance with Conditions (NOC/c) policy, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) with a FGFR2 fusion or other rearrangement. BridgeBio’s partner Helsinn Group has exclusive commercial rights for TRUSELTIQ in Canada with BridgeBio eligible for tiered royalties as a percentage of net sales as part of the global collaboration and license agreement entered into between the two companies in March 2021.
BridgeBio Pharma R&D Day: Held a virtual R&D Day on October 12, 2021. Presentation replay can be found on BridgeBio’s investor website here.
Four new independent directors added to BridgeBio’s board:
Hannah Valantine, M.D., a leader in organ transplant genomics and workforce diversity who is a professor of medicine at Stanford University School of Medicine
Fred Hassan, a pharmaceutical and financial industry leader who is the former CEO of Schering-Plough and former chairman of Bausch & Lomb
Andrea Ellis, a consumer technology innovator and the chief financial officer of Lime
Douglas Dachille, an investment management veteran and the former chief investment officer of American International Group, Inc. (AIG)
Third Quarter 2021 Financial Results:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $599.6 million as of September 30, 2021, compared to $607.1 million as of December 31, 2020. Over the past three quarters, the Company repurchased $148.4 million in BridgeBio common stock under its 2021 Share Repurchase Program and $50.0 million in BridgeBio common stock in conjunction with its issuance of the 2029 convertible notes, paid $61.3 million for capped call options related to the issuance of its 2029 convertible notes and $35.0 million of regulatory-related milestone payments in connection with its approved products. Earlier during the year, BridgeBio paid $21.3 million to Eidos shareholders who elected for cash settlement in exchange for their Eidos shares and $63.8 million of direct transaction costs arising from the merger with Eidos. These were offset by cash receipts of $731.4 million in net proceeds from the issuance of BridgeBio’s 2029 convertible notes, $65.1 million from collaboration partner, Helsinn Group, and $25.0 million in net proceeds from Hercules Capital, Inc. under an amended loan agreement. The remaining change primarily related to payments of interest and operating costs and expenses.

Cash, cash equivalents and marketable securities, excluding restricted cash decreased by $298.8 million when compared to balance as of June 30, 2021, which was $898.4 million. During the quarter, the Company repurchased $143.1 million of BridgeBio common stock and paid $35.0 million of regulatory-related milestone payments in connection with its approved products.

Operating Costs and Expenses

Operating costs and expenses for the quarter increased by $23.7 million to $151.8 million in the current quarter as compared to $128.1 million for the same period in the prior year. The increase in operating costs and expenses was due to an increase in personnel and external costs to support the progression in BridgeBio’s research and development programs and staged buildout of its commercial organization as part of commercial launch readiness activities. This increase in personnel and external costs was offset by $12.2 million in reimbursement of expenses from the cost sharing arrangement recognized under BridgeBio’s License and Collaboration Agreement with Helsinn Group. Stock-based compensation for the quarter was $16.1 million as compared to $17.7 million for the same period in the prior year.

The Company’s research and development expenses have not been significantly impacted by the global COVID-19 pandemic for the periods presented. While BridgeBio experienced some delays in certain of its clinical enrollment and trial commencement activities, it continues to adapt in this unprecedented time to enable alternative site, telehealth and home visits, at-home drug delivery, as well as mitigation strategies with its contract manufacturing organizations. The longer-term impact, if any, of COVID-19 on BridgeBio’s operating costs and expenses is currently unknown.

On November 4, 2021 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) ("Aeterna" or the "Company"), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products, reported its financial and operating results for the third quarter ended September 30, 2021 (Press release, AEterna Zentaris, NOV 4, 2021, View Source;id=215914&p=2209892&I=1206939-c7Z3G6f3m8 [SID1234594595]). The Company also provided an update on progress in its pre-clinical and clinical development programs.

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!

"Since January 2021, we have made a concerted effort to not only advance our clinical-stage program but to expand our development pipeline. We have delivered on that goal and are pleased with the progress made to date. The leadership and expertise we have established at Aeterna Zentaris, now coupled with a diversified portfolio of assets with the potential to address areas of significant medical need, has created an exciting time in the Company’s evolution and multiple potential avenues for value creation. We are diligently working to execute on the advancement of all our clinical and pre-clinical programs and are poised to continue building momentum and value in the near and long term," commented Dr. Klaus Paulini, Chief Executive Officer of Aeterna.

Recent Highlights:

Entered into an additional exclusive license with the Julius-Maximilians-University of Wuerzburg (the "University of Wuerzburg") for early pre-clinical development of AIM Biologics for the potential treatment of Parkinson’s disease.
Exercised its option under the agreement with the University of Wuerzburg to expand application of oral vaccine platform to second indication, chlamydia.
Pre-Clinical and Clinical Programs Update:

Diagnostics Development Pipeline

Macimorelin Diagnostic: Ghrelin agonist in development for diagnostic use in childhood-onset growth hormone deficiency ("CGHD")

Aeterna is currently conducting its pivotal Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of CGHD. Children and adolescents from two to less than 18 years of age with suspected growth hormone deficiency are to be included. The study is expected to include approximately 100 subjects worldwide, with at least 40 subjects in pre-pubertal and 40 subjects in pubertal status. Macimorelin growth hormone stimulation test ("GHST") will be performed twice for repeatability data and two standard GHSTs will be used as controls: arginine (i.v.) and clonidine (p.o.). On April 22, 2021, the U.S. FDA Investigational New Drug Application associated with this clinical trial became active. The first clinical sites in the U.S. are open for patient recruitment. In the EU and rest of world countries, clinical trial approval procedures and site initiation activities are ongoing.

Next Steps

Conduct and complete the DETECT-trial.
Therapeutics and Vaccine Development Pipeline

AIM Biologicals: Targeted, highly specific autoimmunity modifying therapeutics for the potential treatment of neuromyelitis optica spectrum disorder ("NMOSD") and Parkinson’s disease (PD)

In January 2021, Aeterna entered into an exclusive patent license and research agreement with the University of Wuerzburg, Germany, for worldwide rights to develop, manufacture and commercialize AIM Biologicals for the potential treatment of NMOSD. Additionally, the Company has engaged Prof. Dr. Joerg Wischhusen of the University of Wuerzburg as well as neuro-immunologist Dr. Michael Levy (MGH/Boston) as consultants, who will provide scientific support and advice in the field of inflammatory CNS disorders, autoimmune diseases of the nervous system, and NMOSD.

In September 2021, the Company entered into an additional exclusive license with the University of Wuerzburg for early pre-clinical development for the potential treatment of Parkinson’s disease.

Next Steps – NMOSD

Conduct in-vitro and in-vivo assessments to select an AIM Biologicals-based development candidate.
Manufacturing process development for selected candidate.
Next Steps – Parkinson’s Disease

Design and produce antigen-specific AIM Biologics molecules for the potential treatment of Parkinson’s disease.
Conduct in-vitro and in-vivo assessments in relevant Parkinson’s disease models.
Macimorelin Therapeutic: Ghrelin agonist in development for the treatment of ALS (Lou Gehrig’s disease)

In January 2021, the Company entered into a material transfer agreement with the University of Queensland, Australia, to provide macimorelin for the conduct of pre-clinical and subsequent clinical studies evaluating macimorelin as a potential therapeutic for the treatment of ALS (Lou Gehrig’s disease). The University of Queensland researchers have filed for supportive grants and aim to conduct pre-clinical studies in multiple pre-clinical models to demonstrate the therapeutic reach of macimorelin on disease progression and disease-specific pathology. They also plan to conduct a subsequent investigator initiated clinical trial given positive pre-clinical results.

Macimorelin, a ghrelin agonist, is an orally active small molecule that stimulates the secretion of growth hormone from the pituitary gland. Acting via this mechanism, it is believed that macimorelin may slow the progression of certain neurodegenerative diseases like ALS.

Next Steps

Work with the University of Queensland to conduct proof-of-concept studies with macimorelin in disease-specific animal models.
Assess alternative formulations.
Formalize pre-clinical development plan.
Delayed Clearance Parathyroid Hormone ("DC-PTH") Fusion Polypeptides: Potential treatment for primary hypoparathyroidism

In March 2021, Aeterna entered into an exclusive patent and know-how license agreement and research agreement with The University of Sheffield, United Kingdom, for the intellectual property relating to DC-PTH fusion polypeptides with delayed clearance for all human uses. In consultation with The University of Sheffield, Aeterna has selected AEZS-150 as the lead candidate in its DC-PTH program. AEZS-150 is being developed with the goal of providing a potential new treatment option of primary hypoparathyroidism in adults.

Next Steps

Work with The University of Sheffield to conduct in depth characterization of development candidate (in-vitro and in-vivo).
Develop manufacturing process.
Formalized pre-clinical development of AEZS-150 in preparation for a potential IND filing for conducting the first in-human clinical study.
Vaccine Platform: Potential orally active, live-attenuated bacterial vaccine with application across multiple coronavirus types, including COVID-19 (SARS-CoV-2) and chlamydia

In February 2021, Aeterna entered into an exclusive option agreement with the University of Wuerzburg to evaluate a pre-clinical, potential COVID-19 vaccine developed at the University of Wuerzburg. In March 2021, the Company exercised its option and entered into a license agreement where the Company was granted an exclusive, world-wide, license to certain patent applications and know-how owned by the University of Wuerzburg to research and develop, manufacture, and sell a potential COVID-19 vaccine. The Company’s vaccine platform is currently undergoing pre-clinical studies for the prevention of coronavirus diseases, including COVID-19 (SARS-CoV-2) with the planned start of clinical development targeted for H1 2023.

In September 2021, the Company exercised its option under the agreement with the University of Wuerzburg on a then undisclosed field, now known to be chlamydia. Chlamydia trachomatis is a sexually transmitted bacterium infecting over 130 million subjects annually. Asymptomatic disease can spread to the reproductive tract eventually inducing infertility, miscarriage, or ectopic pregnancy, which is a life-threatening condition. Ocular infections can lead to inclusion conjunctivitis or trachoma, which is the primary source of visual impairment or infectious blindness.

Additionally, the Company has entered into a Research Agreement under which the Company has engaged the University of Wuerzburg on a fee-for-service basis to conduct supplementary research activities and pre-clinical development studies on the potential vaccines, the results of which will be included within the scope of the license agreements. Additionally, Prof. Dr. Thomas Rudel of the University of Wuerzburg was engaged by the Company in September 2021 as a scientific consultant to support development of the salmonella-based vaccine platform for the coronavirus and chlamydia vaccines.

Next Steps – Coronavirus Vaccine

Evaluate administration route, dose and immunization scheme.
In-vivo immunology experiments with antigen variant candidates in relevant mice models.
Conduct virus challenge experiments in immunized transgenic animals.
Start manufacturing process assessment / development.
Conduct pre-clinical safety and toxicology assessment.
Next Steps – Chlamydia Vaccine

Design and prepare candidate vaccine strains.
Evaluate administration route, dose and immunization scheme.
In-vivo immunology experiments with candidate strains in relevant mouse models.
Summary of Third Quarter 2021 Financial Results

All amounts in this press release are in U.S. dollars unless otherwise noted.

Financing and Warrant Exercises

During the period between January 1, 2021, and September 30, 2021, holders have exercised certain of our outstanding warrants to purchase 35,011,187 of the Company’s common shares for gross proceeds of approximately $20.0 million. On October 1, 2021, the Company received notice of exercise of 100,000 warrants at $0.45 per common share for the issuance of 100,000 common shares of the Company; such exercise was completed on October 4, 2021.

Cash and cash equivalents

The Company had $68.0 million in cash and cash equivalents at September 30, 2021 (June 30, 2021 – $69.9 million).

Results of operations for the three-month period ended September 30, 2021

For the three-month period ended September 30, 2021, the Company reported a consolidated net loss of $1.7 million, or $0.01 loss per common share (basic), as compared with a consolidated net loss of $1.1 million, or $0.02 loss per common share (basic) for the three-month period ended September 30, 2020. The $0.6 million increase in net loss is primarily from an increase in total operating expenses of $0.6 million and a decline in net finance income of $0.5 million, partially offset by an increase of $0.5 million in total revenue.

Revenues

Total revenue for the three-month period ended September 30, 2021, was $0.6 million as compared with $0.1 million for the same period in 2020, representing an increase of $0.5 million. 2021 revenue was comprised of $0.52 million in licensing revenue (2020 – $0.02 million), $0.06 million in supply chain revenue (2020 – $0.09 million) and $0.02 million in royalty income (2020 – $0.02 million).
Operating expenses

The Company’s total operating expenses for the three-month period ended September 30, 2021, were $2.4 million as compared with $1.9 million for the same period in 2020, representing an increase of $0.5 million. This increase arose primarily from a $0.4 million increase in research and development costs and an increase of $0.1 million in selling expenses. This increase in total operating expenses was primarily due to the impact of the initiation of research and development projects as announced in the first quarter of 2021.
Net finance income

Net finance income for the three-month period ended September 30, 2021, was $0.1 million as compared with net finance income of $0.6 million for the same period in 2020, representing a decrease in net finance income of $0.5 million. This was primarily due to the $0.8 million decrease in the change in fair value of the warrant liability and a $0.1 million decline in the gain from changes in foreign currency exchange rates partially offset by a $0.4 million reduction in other finance costs.
Consolidated Financial Statements and Management’s Discussion and Analysis

For reference, the Management’s Discussion and Analysis of Financial Condition and Results of Operations for the third quarter of 2021, as well as the Company’s unaudited consolidated interim financial statements as of September 30, 2021, will be available on the Company’s website (www.zentaris.com) in the Investors section or at the Company’s profile at www.sedar.com and www.sec.gov.

On November 4, 2021 Beyond Air, Inc. (NASDAQ: XAIR), a clinical-stage medical device and biopharmaceutical company focused on developing inhaled nitric oxide (NO) for the treatment of patients with respiratory conditions, including serious lung infections and pulmonary hypertension, and ultra-high concentration nitric oxide (UNO) for the treatment of solid tumors, reported the separation of its oncology business into a new and independently managed, private company called Beyond Cancer, Ltd (Press release, Beyond Air, NOV 4, 2021, View Source [SID1234594627]). Beyond Air retains its respiratory business and will continue to focus on advancing the LungFit platform of nitric oxide generators and delivery systems to the market. Beyond Air’s preclinical oncology team and the exclusive right to the intellectual property portfolio utilizing UNO for the treatment of solid tumors now reside with Beyond Cancer.

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 decision to spin off our oncology business was made in close collaboration with the Board of Directors and Beyond Air leadership as part of our overall strategy to maximize efficiency and drive value creation to achieve our goals for both patients and shareholders. After a careful assessment of our active portfolio management strategy, it became clear that our oncology business would be more productive as a separate, independently managed entity with its own leadership team and investor base," said Steve Lisi, Chairman and Chief Executive Officer of Beyond Air. "Beyond Air was founded on the promise of NO in respiratory diseases, and this separation will enable a greater focus on the transformational LungFit platform technology. This will also allow for investment into other respiratory diseases where we can have a significant impact on our patients. I am confident that through an ongoing, collaborative partnership, the new Beyond Cancer leadership team will be able to leverage our knowledge of ultra-high concentration NO to treat solid tumors. We are excited and energized to enter this new era of transformation and innovation, as Beyond Cancer takes on its own mission of creating the next generation of immuNO-oncology treatments."

Strategic Rationale
Beyond Air believes the spin-off of the oncology business will create long-term value for shareholders through the following:

Beyond Air’s focus remains advancing the LungFit platform to treat respiratory diseases, and the oncology program does not use LungFit given the ultra-high concentrations and direct delivery to the site of the tumor
After this financing, Beyond Air will retain at least 80% equity ownership in Beyond Cancer
Beyond Cancer will pay Beyond Air a single digit royalty on all future revenues
Beyond Cancer will benefit from Beyond Air’s NO expertise, IP portfolio, preclinical oncology team, and regulatory progress
Beyond Cancer can initially utilize Beyond Air’s infrastructure while having its own investor base and dedicated leadership team with clinical oncology experience to accelerate and enhance its solid tumor pipeline
Leadership and Governance
Selena Chaisson, M.D., is joining Beyond Cancer as Chief Executive Officer. Previously, Dr. Chaisson was the Director of Healthcare Investments at Bailard, where she spent 16 years focusing on highly specialized, emerging healthcare opportunities with more than one-third of her portfolio dedicated to investments in oncology-related companies.

"I am honored to lead Beyond Cancer as we move forward to unlock the possibilities of using ultra-high concentration NO as a new treatment modality for solid tumors," said Dr. Chaisson. "Immuno-oncology has been hailed as a breakthrough therapy for many cancer patients, especially those suffering from hematological malignancies, such as leukemia and lymphoma. However, solid tumors represent approximately 90% of human cancers, with metastatic disease causing the overwhelming majority of cancer-related deaths. Beyond Cancer’s goal is to be at the forefront of a completely novel approach in preventing metastatic disease by harnessing the power of ultra-high concentration NO. In preclinical studies, intra-tumoral delivery of UNO elicited an immunogenic response with potent antitumor activity in addition to the immediate cytotoxic effect of NO-mediated tumor ablation, while limiting off-target effects. As CEO, I am excited to have the opportunity to build a biotech company around such an innovative approach and bring on a seasoned leadership team to spearhead this effort. I will continue Beyond Air’s legacy of investing in NO and look forward to maintaining a close partnership."

Prior to Bailard, Dr. Chaisson held senior executive roles at RCM Capital Management and Tiger Management. RCM Capital Management was acquired and then merged with Allianz Global Investors U.S. in 2013. Dr. Chaisson received a BS in Microbiology in 1987 from Louisiana State University in Baton Rouge, LA, where she graduated summa cum laude. She earned her M.B.A. and M.D. from Stanford University in 1992 and 1993, respectively.

Hila Confino, Ph.D., is joining Beyond Cancer as Chief Scientific Officer from Beyond Air, where she served as Head of Research in Israel. At Beyond Air, Dr. Confino led the oncology preclinical team and conducted in vitro and in vivo studies for ultra-high concentration nitric oxide in solid tumors. Dr. Confino has over 12 years of experience in cancer immunology research in both the academic and industry settings. Her work has been featured in numerous scientific conferences and published in multiple peer-reviewed journals. She holds a Ph.D. and Master of Science in Cancer Immunology from Tel Aviv University. Dr. Confino received a BS in Biotechnology from Bar—Ilan University in Israel, where she graduated cum laude.

The complete Beyond Cancer Board of Directors, Scientific Advisory Board and headquarters will be announced as they are finalized. The Board of Directors is expected to consist of six members, four of whom include:

Steve Lisi, Chairman of the Board, and CEO and Chairman of the Board of Beyond Air
Selena Chaisson, M.D., Director, and CEO of Beyond Cancer
Amir Avniel, Executive Director, and President, COO and Co-Founder of Beyond Air
Robert Carey, Director, and Board Member of Beyond Air
Transaction Details
Beyond Cancer has secured commitments of $23.9 million in a concurrent private placement of common shares, not to exceed $30 million, providing the investors with up to 20% equity ownership. The funding is expected to be used to accelerate ongoing preclinical work including the completion of IND-enabling studies, completion of a Phase 1 study, expansion of preclinical programs for combination studies, hiring of additional Beyond Cancer team members, and optimization of the delivery system, as well as for general corporate purposes. The concurrent private placement is expected to close later in the fourth fiscal quarter.

The common shares to be sold in the private placement have been and are being offered only to certain institutional and/or accredited investors in reliance upon an exemption from the registration requirements of the Securities Act of 1933, as amended (the "Securities Act"). The common shares have not been registered under the Securities Act or any state or other securities laws and may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements of the Securities Act and applicable state securities laws. The Securities and Exchange Commission has not passed upon the merits of or given its approval to the common shares, the terms of the private placement or the accuracy or completeness of any private placement materials. The common shares sold in the private placement are subject to legal and contractual restrictions on transfer.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification or otherwise under the securities laws of any such state or jurisdiction.

Hogan Lovells US LLP, Arthur Cox LLP and Conyers Dill & Pearman Limited serve as legal counsel to Beyond Air.

Upcoming Investor Events
Beyond Air management will provide more details on this news on Thursday, November 11, 2021, consistent with its scheduled time to report financial results for its second fiscal quarter ended September 30, 2021. Management will host a conference call and webcast at 4:30 pm Eastern Time the same day.

In addition, Steve Lisi, Chairman and Chief Executive Officer of Beyond Air, will participate in the Piper Sandler 33rd Annual Virtual Healthcare Conference being held from November 30 – December 2, 2021, and will be available for virtual one-on-one meetings.