On May 13, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies with its ARCUS genome editing platform, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Precision Biosciences, MAY 13, 2021, View Source [SID1234579942]).

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"In the first quarter, we have worked diligently to advance our ARCUS-based in vivo gene editing and allogeneic CAR T pipelines," commented Matt Kane, CEO and co-founder of Precision BioSciences. "In April, we seized the opportunity to reacquire all global rights previously granted to Servier for our CAR T programs, including PBCAR0191 and PBCAR19B. We believe that this repurchase, alongside our maturing datasets, will allow us to take full advantage of our CAR T programs as they progress through clinical development."

"We are on track to initiate the Phase 1 study of our PBCAR19B this month, our first candidate incorporating immune evading stealth cell technology as an alternative potential path to deeper and more durable responses. This will precede multiple anticipated clinical and research milestones across our portfolio in 2021, including interim updates from all three clinical-stage CAR T programs: PBCAR0191, PBCAR20A, and PBCAR269A. Additionally, we look forward to providing an update on our in vivo gene editing pipeline mid-year," concluded Mr. Kane.

Recent Developments and Upcoming Milestones:

Allogeneic CAR T Portfolio:

Reacquired Global Development and Commercialization Rights from Servier: In April 2021, Precision announced that it had entered into a Program Purchase Agreement to reacquire all global development and commercialization rights for all CAR T partnered programs covered under its Development and Commercial License Agreement with Servier. This includes its two CD19-targeting allogeneic CAR T candidates, PBCAR0191 and PBCAR19B stealth cell, plus four additional CAR T targets identified by Servier in 2020.

Under the terms of the Program Purchase Agreement, Precision paid $1.25 million in cash to Servier and agreed to waive earned, but as-yet unpaid milestones totaling $18.75 million that would have otherwise been payable to Precision. Servier is also eligible to receive milestones and low- to mid-single-digit royalties subject to product development achievement.

PBCAR0191: Precision has continued to advance its Phase 1/2a study of PBCAR0191 in patients with relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL) or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). Since the December 2020 interim data update, additional patients have been dosed with PBCAR0191 following enhanced lymphodepletion (eLD)1. Precision will continue to monitor the results for durability from this eLD regimen and expects to report updated interim results in June at ASCO (Free ASCO Whitepaper) 2021.

PBCAR19B: Precision has begun adding clinical trial sites for its Phase 1 study of PBCAR19B, the Company’s next generation, stealth cell, allogeneic CAR T candidate for patients with CD19-positive malignancies such as those with R/R NHL. The study is expected to begin by the end of May 2021. PBCAR19B will be administered at flat dose levels, beginning at 2.7 × 108 cells, with the ability to dose up to 8.1 × 108 cells per patient, following standard lymphodepletion2. The primary objective of the study is to identify the maximum tolerated dose and any dose-limiting toxicities.

PBCAR20A: Precision continues to enroll patients in its Phase 1/2a clinical trial of PBCAR20A, its anti-CD20 CAR T therapy for patients with R/R NHL, including patients with R/R chronic lymphocytic leukemia or R/R small lymphocytic lymphoma. In February 2021, the study began enrolling patients into dose level 3, a fixed dose of 480 × 106 cells with a max dose of 6.0 × 106 cells/kg. Precision expects to provide an interim update for the PBCAR20A study in 2021.

PBCAR269A: Precision continues to enroll patients in its Phase 1/2a study of PBCAR269A, its CAR T candidate targeting B-cell maturation antigen (BCMA) for the treatment of R/R multiple myeloma, for which Precision has received Fast Track Designation and Orphan Drug Designation from the FDA. In February 2021, the study began enrolling patients into its highest dose cohort, dose level 3 at 6.0 × 106 cells/kg and Precision expects to provide an interim update in 2021. Precision also expects to initiate the combination arm of its ongoing Phase 1/2a clinical study with PBCAR269A and nirogacestat, SpringWorks Therapeutics’ investigational gamma secretase inhibitor, in the first half of 2021.

PBCAR269B: In April 2021, Precision introduced PBCAR269B, a next-generation, BCMA-targeted candidate incorporating stealth cell technology, for treatment of R/R multiple myeloma. Precision is conducting IND-enabling studies for PBCAR269B and expects to file an IND application in early 2022.

In Vivo Gene Correction Portfolio:

Genome Editing Research Collaboration with Eli Lilly: In January 2021, Precision announced the closing of its agreement with Eli Lilly. Under the agreement, Precision will develop up to six in vivo therapies for genetic disorders using ARCUS, with an initial focus on Duchenne muscular dystrophy and two other undisclosed gene targets.

PH1: Pre-clinical research continues to progress with Precision’s wholly-owned in vivo gene correction program using its ARCUS genome editing technology to knock out the HAO1 gene as a potential one-time treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disease. The Company expects to provide an update in mid-2021.

Corporate:

Executive Leadership: In April 2021, Precision announced that it has initiated a CEO transition plan for Co-Founder and Chief Executive Officer, Matt Kane. Mr. Kane is expected to continue in his current leadership capacity until his successor has been identified. He will also serve as an advisor for a period of time after the next CEO is hired ensuring a smooth transition.

Precision also announced the appointment of Alan List, M.D. as the Company’s Chief Medical Officer and a member of the senior leadership team. Dr. List is a world-renowned hematologist with extensive academic and clinical experience in the research and development of hematology and oncology products.

Board of Directors: Precision announced that it has strengthened its Board of Directors with the appointment of Stanley R. Frankel, M.D., a hematologist-oncologist with extensive academic and industry experience in the research, clinical development, and commercialization of immuno-oncology and cellular therapies. Dr. Frankel was most recently the Senior Vice President, Cellular Therapy Development at Bristol-Myers Squibb (BMS). Prior to the BMS acquisition of Celgene, he was Corporate Vice President, Head, Immuno-Oncology & Cellular Therapy, Clinical Research and Development Head, Cell Therapy Clinical Center of Excellence at Celgene.

Elo Life Systems:

Corporate Structure: In January 2021, Precision disclosed its intention to spin out its wholly-owned subsidiary, Elo Life Systems. Precision is continuing to explore its strategic options, and expects to complete any such spinout, sale or other treatment of Elo in 2021.

Quarter Ended March 31, 2021 Financial Results

Cash and Cash Equivalents: As of March 31, 2021, Precision had approximately $193.5 million in cash and cash equivalents, which includes the $100 million upfront cash payment and equity investment of $35 million received from Eli Lilly in January 2021. The Company expects that existing cash and cash equivalents, expected operational receipts, and available credit will be sufficient to fund its operating expenses and capital expenditure requirements into 2023.

Revenues: Total revenues for the first quarter ended March 31, 2021 were $16.3 million, as compared to $7.0 million for the same period in 2020.

Research and Development Expenses: Research and development expenses were $25.6 million for the quarter ended March 31, 2021, as compared to $24.9 million for the same period in 2020.

General and Administrative Expenses: General and administrative expenses were $9.5 million for the quarter ended March 31, 2021, as compared to $9.6 million for the same period in 2020.

Net Loss: Net loss was $18.7 million, or $(0.33) per share, for the quarter ended March 31, 2021, as compared to a net loss of $26.8 million, or $(0.52) per share, for the same period in 2020.

On May 13, 2021 Genetron Holdings Limited ("Genetron Health" or the "Company", NASDAQ:GTH), a leading precision oncology platform company in China that specializes in offering molecular profiling tests, early cancer screening products and companion diagnostics development, reported a strategic partnership with Siemens Healthineers at the China Medical Equipment Fair (Press release, Genetron Health Technologies, MAY 13, 2021, View Source [SID1234579960]).

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This partnership aims to promote large scale application of Genetron’s S5 platform and lung cancer 8-gene IVD assay in Chinese hospitals, in order to provide non-small cell lung cancer (NSCLC) patients with efficient and accurate personalized diagnosis and treatment guidance. The companies also plan to work together to standardize cancer molecular testing in hospitals.

Representing Genetron Health at the signing ceremony in Shanghai were Co-founder and CEO Sizhen Wang, and Vice President Zhaohui Yin. Elisabeth Staudinger, President of Siemens Healthineers Asia Pacific, and Guo Yiming, Vice President of Siemens Healthineers Laboratory Diagnostic System, Greater China Region, also attended the event.

"Genetron Health focuses on the innovation, clinical transformation and commercialization of cancer genomics technologies. Both our lab developed test (LDT) and in vitro diagnostic (IVD) products’ business models have been progressing well," said Sizhen Wang, Co-founder and CEO of Genetron Health. "We are pleased to collaborate with Siemens Healthineers for our S5 instrument and lung 8 IVD assay, which helps diagnose and treat cancer patients. We also expect this partnership to help standardize cancer molecular testing in China, and reach more patients in need," Wang said.

"As a global leader in healthcare technology, Siemens Healthineers stays committed to addressing the needs of patients. To help Chinese cancer patients access quality care, we are not only introducing cutting-edge testing analyzers and reagents to China, but are also working with local innovators to offer personalized diagnostics. We are excited to forge a strategic partnership with Genetron Health, an industry leading precision oncology platform company. Meanwhile, we expect to push forward precision care in the field of lung cancer, therefore contributing to the vision of a Healthy China," said Guo Yiming, Vice President of Laboratory Diagnostics, Siemens Healthineers Greater China.

At present, lung cancer is one of the most serious diseases in China, placing the highest amongst morbidity and mortality rankings of all cancer types. In China, 787,000 cases of lung cancer are diagnosed every year, with about 631,000 deaths [1] caused annually as well. NSCLC is the most common type of lung cancer. In recent years, with the development of more advanced diagnostics and the emergence of targeted drugs, more NSCLC patients have been able to receive quicker and more accurate treatment. As the "first step" of targeted therapy for lung cancer, cancer molecular gene detection is the key to guided clinical treatment and drug applications. The U.S. National Comprehensive Cancer Network (NCCN) Guidelines for NSCLC also recommend that molecular testing of lung cancer-related genes be used to guide targeted therapies for NSCLC patients. Accurate lung cancer gene detection for NSCLC patients can help clinicians opt for more precise treatment.

Based on Genetron Health’s One-Step Seq technology (Chinese invention patent ZL 201710218529.4), the lung cancer 8-gene IVD assay can detect many mutations and fusions across 8 different genes at once, for targeted therapy selection in NSCLC. The assay can offer patients rapid and accurate molecular typing, medication guidance and drug resistance monitoring. Combined with Genetron S5, it offers advantages in detection efficiency, ease of use, economic cost, sample size, and can achieve a two-day turnaround time. It is suitable for independent clinical molecular testing in Chinese hospitals at all levels.

[1] Zheng R., Sun K., Zhang S., et al. Report of cancer epidemiology in China, 2015, Chinese Journal of Oncology, 2019, 41(1):19-28.

On May 13, 2021 Transcenta Holding Limited (Transcenta), a clinical stage global biotherapeutics company with fully-integrated capabilities in discovery, development and manufacturing of antibody-based therapeutics, reported that the updated phase I clinical data on the safety and efficacy of MSB2311, a pH-dependent PD-L1 antibody, in Chinese patients with advanced solid tumors and hematological malignancies will be presented as an abstract online publication at the 57th American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Transcenta, MAY 13, 2021, View Source [SID1234580030]). The 2021 ASCO (Free ASCO Whitepaper) Annual Meeting will be held online from June 4th to June 8th EDT.

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Online Publication

Title: Updated safety and efficacy of MSB2311 (an Anti-Programmed Death-Ligand 1 Antibody) in Chinese patients with advanced solid tumors and hematological malignancies from a phase I study.

Abstract Number: e14547

First Author: Lin Shen, MD (Beijing Cancer Hospital)

About MSB2311

MSB2311 is an investigational humanized PD-L1 antibody with pH dependent binding property. PD-L1 is involved in inhibiting the immune system’s response to fight cancer. MSB2311 blocks the interaction between PD-L1 and PD-1, which reactivates the suppressed or exhausted anti-tumor effector T cell function in the tumor microenvironment. MSB2311 employs engineered IgG1 which lacks FcR binding. In addition, the binding of MSB2311 to PD-L1 results in internalization of MSB2311 and MSB2311 can dissociate from bound PD-L1 in endosome with pH level lower than 5.5. This allows MSB2311 to be recycled to plasma membrane and be reused to bind with PD-L1 on another tumor cell or immune cell. Results from preclinical studies demonstrate that MSB2311 can inhibit tumor growth of PD-L1 expressing tumor cells in syngeneic mouse-model. Two phase 1 studies of MSB2311 have been completed in the US and China. MSB2311 is currently to be tested in phase 2 trial in patients with solid tumors expressing select biomarker in China.

On May 13, 2021 MediciNova, Inc., (Nasdaq: MNOV, JASDAQ:4875), a biopharmaceutical company developing small-molecule therapeutics, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, MediciNova, MAY 13, 2021, View Source [SID1234580046]).

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"As a late-stage biotech company with two anti-inflammatory platform candidates in development, our main focus over 2021 is execution across our programs that will enable us to not only showcase their therapeutic potential but bring us closer to delivering much-needed treatment options to patients," commented Yuichi Iwaki, MD, PhD, President and Chief Executive Officer of MediciNova. "We know from the prior approval in Japan and subsequent treatment in post-stroke dizziness and asthma that our lead product candidate MN-166 is remarkably safe and well-tolerated. We are now working to demonstrate the potential of MN-166, along with MN-001, to drive therapeutic benefit across a range of neurodegenerative, inflammatory, and fibrotic indications, without compromising immunity. Recent among these expansions has been our work in acute respiratory distress syndrome to combat the high rates of hospitalization due to COVID-19, and we are pleased to have partnered with BARDA to further evaluate MN-166 against chlorine gas-induced lung damage. MN-166 is the first candidate to have received BARDA funding under the Division of Research, Innovation, and Ventures’ (DRIVe) Repurposing Drugs in Response to Chemical Threats (ReDIRECT) program, exemplifying its differentiated potential as a safe and well-established anti-inflammatory drug."

Recent Clinical Highlights

MN-166 (ibudilast)

Formed partnership with BARDA to develop MN-166 as treatment for ARDS and ALI: In March 2021, the Company entered into a partnership with the Biomedical Advanced Research and Development Authority (BARDA) to develop MN-166 as a potential treatment for chlorine gas-induced lung damage such as acute respiratory distress syndrome (ARDS) and acute lung injury (ALI). Under the partnership, BARDA will provide funding for proof-of-concept studies of MN-166. The Company also continues to enroll patients in its Phase 2 clinical trial evaluating MN-166 (ibudilast) in COVID-19 patients at risk of developing ARDS. The Phase 2 trial is a randomized, double-blind parallel group study to evaluate the efficacy and safety of MN-166 in hospitalized COVID-19 patients receiving standard of care who are at risk of developing ARDS.
Continued enrollment of Phase 3 trial in ALS: The Company continues to enroll patients in its Phase 3 clinical trial evaluating MN-166 for the treatment of amyotrophic lateral sclerosis (ALS). The Phase 3 trial is a multi-center randomized, double-blind study to evaluate the efficacy, safety, and tolerability of MN-166 in ALS patients for 12 months of treatment followed by a 6-month open-label extension phase, and the primary endpoint is change from baseline in ALSFRS-R score at month 12 and survival time. Progression into this study follows positive Phase 2 data that demonstrated MN-166 resulted in a higher rate of responders on the ALSFRS-R score, indicating less functional decline compared to the placebo group. The ALSFRS-R responders showed increased overall survival compared to non-responders and MN-166 also demonstrated a favorable safety and tolerability profile.
Partnering process ongoing for progressive MS program: The Company is engaged in a process with potential partners regarding MN-166 that could lead to funding for a Phase 3 trial in progressive multiple sclerosis (PMS). In the Phase 2b trial in PMS, MN-166 achieved both primary endpoints, demonstrating a significant 48% reduction in the rate of progression of whole brain atrophy (p=0.04) as well as a favorable safety and tolerability profile. MN-166 also showed a trend of reduced risk of confirmed disability progression, an approvable endpoint for PMS, especially among secondary progressive MS (SPMS) patients without relapse, a subgroup that represents the highest unmet need with no approved long-term treatment options and accounting for approximately 80% of all SPMS patients. Based on this encouraging data and discussions with FDA, the Phase 3 trial plan is to enroll SPMS patients without relapse with 3-month confirmed disability progression as the primary endpoint.
MN-001 (tipelukast)

Completed enrollment of Phase 2 trial in IPF: The Company has completed enrollment in its Phase 2 trial of MN-001 in idiopathic pulmonary fibrosis (IPF). The Phase 2 randomized, placebo-controlled, double-blind trial is evaluating the efficacy and safety of MN-001 in patients with IPF over the course of a 26-week treatment period followed by at 26-week open label extension. The co-primary endpoints of the trial are change from baseline of forced vital capacity (FVC) and semiannual rate of decline of disease activity based on FVC.
Preparing second Phase 2 trial in NASH: Following the early completion of its Phase 2 trial evaluating MN-001 in nonalcoholic steatohepatitis (NASH) and nonalcoholic fatty liver disease (NAFLD) due to positive interim data, the Company is now preparing to initiate a larger Phase 2 trial in NASH. In the first Phase 2 trial, MN-001 demonstrated a statistically significant reduction in the primary endpoint, mean serum triglycerides (p=.02). The Company will provide an update on this program as the new trial is initiated.
Recent Business Updates

Established MedACT (MediciNova Ancillary Clinical Trial Support): As part of its ongoing patient support and engagement, the Company has established MedACT, a patient support program that grants financial and other support as needed across its ongoing clinical trials.
Received $4 million in gene therapy milestone payments: In April 2021, the Company announced it received two milestone payments under its agreement with Genzyme Corporation, a subsidiary of Sanofi, as a result of the successful achievement of two clinical development milestones for a gene therapy product based on adeno-associated virus (AAV) vector technology.
Discontinued development of COVID-19 vaccine: In March 2021, the Company announced its plans to discontinue development of a SARS-CoV-2 vaccine for COVID-19 to focus its resources on its later-stage development programs with larger unmet medical needs and market opportunities.
Raised $20 million through private placement: In January 2021, the Company raised $20 million through a private placement of common stock sold to 3D Opportunity Master Fund, a fund managed by 3D Investment Partners Pte. Ltd.
First Quarter 2021 Financial Results

Cash Position: As of March 31, 2021, cash and cash equivalents were $76.3 million, as compared to cash and cash equivalents of $61.3 million as of March 31, 2020. This increase was primarily due to approximately $20 million received in a private placement transaction which closed in January 2021. The Company expects current cash and cash equivalent to fund operations at least through the end of 2022.
Revenues: Revenues were $4.0 million for the three months ended March 31, 2021, compared to $0.0 million for the three months ended March 31, 2020. The $4.0 million increase was due to the receipt of two milestone payments under MediciNova’s assignment agreement with Genzyme Corporation, a subsidiary of Sanofi.
Research, Development and Patents Expenses: R&D and patents expenses were $2.1 million for the three months ended March 31, 2021, compared to $1.3 million for the three months ended March 31, 2020. The increase of $0.8 million was primarily due to higher clinical trial expenses from the ongoing clinical trial of MN-166 (ibudilast) in ALS and higher stock compensation expense resulting from an increase in our stock price.
General and Administrative Expenses: G&A expenses were $2.1 million for the three months ended March 31, 2021, compared to $1.7 million for the three months ended March 31, 2020. The increase of $0.4 million was primarily due to higher stock compensation expense resulting from an increase in our stock price.
Net Loss: Net loss was $0.2 million for the three months ended March 31, 2021, or $(0.00) per basic and diluted share, as compared to a net loss of $2.7 million for the three months ended March 31, 2020, or $(0.06) per basic and diluted share.

On May 13, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas" ) and Kyoto University Innovation Capital Co., Ltd. (President and CEO; Ko Kusumi, "KYOTO-iCAP") reported that they have established a strategic alliance agreement to bring their strengths together and promote the social implementation of advanced research results from national universities by discovering, supporting, and fostering them (Press release, Astellas, MAY 13, 2021, View Source [SID1234579845]).

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Based on this agreement, the two companies will establish a system for regularly exchanging information in the drug discovery and healthcare fields. KYOTO-iCAP expects to promote activities supporting establishment and growth of promising venture companies by collaborating with Astellas, which has drug discovery capabilities. Astellas expects to increse opportunities for alliance to incorporate excellent capabilities outside and accelerate delivering cutting-edge science to society by collaborating with KYOTO-iCAP, which has a strong network with promising seeds and venture companies from national universities such as Kyoto University.

In recent years, with the diversification of drug discovery methods and treatment methods, technological fusion between different fields has been attempted. In the future, promoting cross-disciplinary activities beyond medicine and pharmacy will lead to the development of the drug discovery and healthcare fields. KYOTO-iCAP targets all of Kyoto University’s research fields, not limited to drug discovery and healthcare. It has a track record of making a wide range of investments, such as interdisciplinary fusion projects and company carve-out projects. Astellas is committed to open innovation through partnerships with multiple domestic and international academia and venture companies. Astellas has been involved in the ecosystem from Kyoto, such as establishing the "Alliance Station" as a base for joint research activities within Kyoto University in 2017 and investing in the KYOTO-iCAP No. 2 fund managed by KYOTO-iCAP in January 2021.

Through this strategic alliance agreement, the two companies will contribute to the further development of the ecosystem originating in Kyoto, where venture companies from various fields gather, and will strengthen the foundation for socially implementing the research results of national universities as new drugs and new businesses.