On October 15, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that the Company will host a webcast conference call to report its third quarter 2020 financial results and provide an update on the company’s business and outlook on Thursday, October 29, 2020 at 4:30 p.m. (ET) after the closing of the market (Press release, PTC Therapeutics, OCT 15, 2020, https://www.prnewswire.com/news-releases/ptc-therapeutics-to-host-conference-call-to-discuss-third-quarter-2020-financial-results-301147176.html [SID1234568540]).

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The call can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 7096445. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

On October 15, 2020 BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, reported financial results for the third quarter ended September 30, 2020, and provided a corporate update (Press release, BrainStorm Cell Therapeutics, OCT 15, 2020, View Source [SID1234568538]).

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"The most important near-term event for BrainStorm will be the upcoming top-line data readout for the NurOwn Phase 3 trial in ALS, expected by the end of November. A successful outcome will set us on the path to filing a Biologic License Application (BLA) for what we believe will be a valuable new treatment for ALS," said Chaim Lebovits, Chief Executive Officer of BrainStorm Cell Therapeutics. "In parallel to our preparations for upcoming data read out, we are very busy planning and executing on other pre-BLA activities. On the management front, we appointed William K. White and Dr. Anthony Waclawski, adding valuable commercial and regulatory expertise to our leadership team. This expertise will be crucial as we work towards obtaining regulatory approval for NurOwn and ensuring that, if approved, it will be readily accessible to ALS patients in need of new treatment options for this devastating disease."

NurOwn has an innovative mechanism of action that is broadly applicable across neurodegenerative diseases and BrainStorm continues to invest in clinical trials evaluating the product in conditions beyond ALS to maximize value creation for its various stakeholders. The company remains on track to complete dosing in its Phase 2 clinical trial in progressive multiple sclerosis (PMS) by the end of 2020. In addition, the Company recently unveiled a clinical development program in Alzheimer’s’ disease (AD) and is planning a Phase 2 proof-of-concept clinical trial at several leading AD centers in the Netherlands and France.

Third Quarter 2020 and Recent Corporate Highlights:

Completed dosing of all patients in the ongoing NurOwn Phase 3 clinical trial in ALS and plans remains on track to report top-line data by the end of November 2020.
Announced the publication of a manuscript titled "Effects of MSC-NTF cells on T and B regulatory cell function in ALS" in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. The manuscript describes NurOwn’s immunomodulatory effects on T and B regulatory cell function, suggesting that its mechanism of action is broadly applicable in ALS, PMS and Alzheimer’s disease.
Presented a scientific poster titled "Advancing NurOwn for ALS: Phase 3 Clinical Trial Design" at the Annual Northeast ALS (NEALS) Meeting.
A Phase 2 trial evaluating NurOwn as a treatment for progressive multiple sclerosis (PMS) is ongoing at 5 leading U.S. multiple sclerosis centers. The Company remains on track to complete dosing by year end 2020.
Presented progressive MS natural history data at the MSVirtual2020 meeting documenting an association between magnetic resonance imaging (MRI) measures and functional improvement in patients matched to the phase 2 NurOwn clinical trial.
Hosted a Key Opinion Leader (KOL) webinar on the Alzheimer’s disease program and the planned European Phase 2 trial. The webinar featured presentations by two lead investigators in the trial: Philip Scheltens, M.D., Ph.D., Professor of Cognitive Neurology and Director of the Alzheimer Centre at the VU University Medical Center in Amsterdam, Netherlands; and Bruno Dubois, M.D., Ph.D., Professor of Neurology at the Neurological Institute of the Salpétrière University Hospital in Paris, France.
Announced a groundbreaking pre-clinical study of a NurOwn derived exosome-based treatment for COVID-19 acute respiratory distress syndrome (ARDS).
Appointed Anthony Waclawski Ph.D. as Executive Vice President, Global Head of Regulatory Affairs.
Appointed William K. White as Senior Vice President, Head of Market Access and Pricing.
Received a non-dilutive bonus payment of $700,000 from California Institute for Regenerative Medicine (CIRM) for treating more California participants than originally proposed in the Phase 3 ALS trial.
Announced the grant of Japanese Patent No. 6,753,887, titled: "Methods of Generating Mesenchymal Stem Cells which Secrete Neurotrophic Factors".
Presented at the following Investor Conferences:

40th Annual Canaccord Genuity Growth Conference
Cash and Liquidity as of October 14, 2020

Total available funding as of October 14, 2020, which includes cash, cash equivalents and short-term bank deposits of approximately $33.1 million as well as remaining non-dilutive funding from CIRM, IIA and other grants, amounts to approximately $36 million.

Financial Results for the Three Months Ended September 30, 2020

Cash and cash equivalents, and short-term bank deposits amounted to approximately $28.8 million at September 30, 2020 compared to $16.2 million at June 30, 2020.
Research and development expenses, net for the three months ended September 30, 2020 and 2019 were $1.87 million and $4.01 million, respectively.
Excluding participation from IIA and CIRM under the grants and proceeds received under the hospital exemption regulatory pathway, research and development expenses decreased by $1.68 million from $5.66 million in the third quarter of 2019 to $3.98 million in the third quarter of 2020.
General and administrative expenses for the three months ended September 30, 2020 and 2019 were $2.62 million and $1.54 million, respectively.
Net loss for the three months ended on September 30, 2020 was $4.49 million, as compared to a net loss of $5.63 million for the three months ended September 30, 2019.
Net loss per share for the three months ended September 30, 2020 and 2019 was $0.14 and $0.25, respectively.
Conference Call & Webcast
Thursday, October 15, 2020 at 8 a.m. Eastern Time

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received acceptance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS) and completed enrollment in August 2020.

On October 15, 2020 I-Mab (the "Company") (Nasdaq: IMAB), a clinical stage biopharmaceutical company committed to the discovery, development and commercialization of novel biologics, reported that the Company’s abstract highlighting its U.S. phase 1 dose escalation trial data from its CD47 program, lemzoparlimab (also known as TJC4) in relapsed or refractory malignancy, will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 35th Anniversary Annual Meeting & Pre-Conference Programs (SITC 2020), taking place online November 9 – 14, 2020 (Press release, I-Mab Biopharma, OCT 15, 2020, View Source [SID1234568537]). The data to be presented will include clinical safety, pharmacokinetics (PK) & pharmacodynamics (PD), receptor occupancy (RO), and preliminary evidence of efficacy of lemzoparlimab as a monotherapy.

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Lemzoparlimab is a highly differentiated anti-CD47 monoclonal antibody originally discovered and developed by I-Mab that has been designed to minimize inherent binding to normal red blood cells while preserving its strong anti-tumor activity, a critical attribute in differentiating lemzoparlimab from other antibodies of the same class currently in clinical development. Initial results from I-Mab’s phase 1 study in U.S. described above have demonstrated the unique differentiation in drug safety and pharmacokinetics profile in cancer patients.

Details of the poster are as follows:

Title

A first-in-patient study of lemzoparlimab, a differentiated anti-CD47 antibody,
in subjects with relapsed/refractory malignancy: initial monotherapy results

Abstract #

385

Presenting Author

Jordan Berlin, MD, Vanderbilt University

The abstract is available at View Source

About CD47 and Lemzoparlimab

CD47 is a cell surface protein over-expressed in a wide variety of cancers and can act to protect tumors by delivering a "don’t eat me" signal to otherwise tumor-engulfing macrophages. CD47 antibody blocks this signal and enables macrophages to attack tumor cells, making it a potentially promising cancer drug. However, development of CD47 antibody as a cancer therapy is hampered by its hematologic side effects, such as severe anemia, caused by natural binding of CD47 antibody to red blood cells. In a scientific breakthrough, scientists at I-Mab have discovered a unique CD47 antibody, lemzoparlimab, that works efficiently to target tumor cells while exerting a minimal untoward effect on red blood cells to avoid severe anemia.

Lemzoparlimab’s hematologic safety advantage and superb anti-tumor activities have been demonstrated previously in a series of robust pre-clinical studies. The results of phase 1 clinical trial have provided further clinical validation of this differentiation in patients with cancer. I-Mab continues to advance a combination study of lemzoparlimab with Keytruda for solid tumor and with Rituxan for lymphoma in the U.S., in addition to an on-going clinical trial in patients with AML/MDS in China.

In September 2020, I-Mab and AbbVie entered into a global strategic partnership to develop and commercialize lemzoparlimab, including to design and conduct further clinical trials to evaluate lemzoparlimab in multiple cancers globally and in China. The collaboration is subject to certain pre-closing conditions.

On October 15, 2020 The National Comprehensive Cancer Network (NCCN) Oncology Research Program (ORP) reported plans to evaluate neratinib, a type of tyrosine kinase inhibitor (TKI) that works as a dual inhibitor of the epidermal growth factor receptor 1 (EGFR) and human epidermal growth factor receptor 2 (HER2) (Press release, NCCN, OCT 15, 2020, View Source [SID1234568536]). The research funding is supported by a $2-million grant from Puma Biotechnology, Inc.

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Projects may include pre-clinical, translational and clinical trials. They will focus on treatment of:

Pediatric tumors(including neurofibromatosis Type 2 and progressive Vestibular Schwannoma)
Early-stage and metastatic breast cancer, including those with HER2+ brain metastases
Other HER2-amplified tumors
EGFR-mutated glioblastoma multiforme.
Specific research areas will be determined by a group of cancer research experts from NCCN Member Institutions who form a Request for Proposals (RFP) Development Team.

"We look forward to advancing knowledge of the role this dual HER breast cancer drug may have in improving outcomes for various cancer presentations," said Wui-Jin Koh, MD, Chief Medical Officer, NCCN. "The projects we oversee will provide important information on this emerging mechanism for personalized cancer care."

"We thank the National Comprehensive Cancer Network for their inclusion of neratinib for further evaluation," said Alan H. Auerbach, Chief Executive Officer and President of Puma Biotechnology. "We are proud to support NCCN’s research projects to better understand the full potential of neratinib across a variety of indications. Our goal is to use these insights to expand the potential indications, as well as to identify patients who may benefit from neratinib."

The first phase of this project will involve the creation of an RFP, which will open in early 2021. The awarded projects will be announced following review of submitted proposals.

The NCCN ORP fosters innovation and knowledge discovery that improves the lives of people with cancer and supports preclinical, translational, and clinical research and quality improvement projects in oncology at NCCN Member Institutions. In an effort to improve collaboration in cancer research, the NCCN ORP also maintains a shared resources website and an informed consent database. For more information, visit NCCN.org/orp.

On October 15, 2020 OS Therapies, a clinical-stage biopharmaceutical company focused on discovering and developing innovative therapies to treat and cure Osteosarcoma (OS) and other deadly cancers in children and adults, reported that they have initiated their sponsorship of the FDA Investigational New Drug (IND) application for OST-HER2 a live, attenuated Listeria monocytogenes vector, expressing a tLLO-chimeric HER2 fusion protein (Press release, OS Therapies, OCT 15, 2020, View Source;phase-iib-trial-in-osteosarcoma-in-early-2021-301152798.html [SID1234568535]). This sets the pathway for initiation of the first clinical trial in Osteosarcoma patients in partnership with the Children’s Oncology Group (COG) – AOST-2121: An Open-Label Phase 2 Study of Maintenance Therapy with OST-HER2 (OST31-164) After Resection of Recurrent Osteosarcoma – in early 2021.

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A veterinary formulation of OST-HER2 had previously received accelerated approval from the USDA for Osteosarcoma in canines, and Phase I and III trials conducted in canines have had positive clinical benefit with manageable safety. The AOST-2121 clinical trial sponsored by OS Therapies and in coordination with COG will be the first trial in Adolescents and Young Adults (AYA) with Osteosarcoma, a deadly and debilitating bone cancer.

"OST-HER2 holds the promise of stimulating the patient’s immune system to seek out and combat hidden micro-metastases that can cause the osteosarcoma to recur," said Dr. Robert Petit, Chief Medical & Scientific Officer for OS Therapies. "In the Canine version of osteosarcoma, this form of treatment significantly delayed or prevented further osteosarcoma metastases and improved overall survival by three-fold. This clinical trial of OST-HER2 is an essential step towards bringing this potential clinical benefit to the children and young adults who are battling osteosarcoma."