On March 26, 2020 Asieris Pharma, located in Taizhou’s China Medical City, reported that it has raised $14 million in a C round to advance its genito-urinary tumor treatments (Press release, Asieris Pharmaceuticals, MAR 26, 2020, View Source [SID1234555896]). Its lead candidate, a novel treatment for non-muscular invasive bladder cancer, has completed China Phase III trials and a US Phase I trial. Unlike other treatments for the condition, usually bladder resection, APL-1202 is convenient, painless and does not cause damage to the urethra. A repurposed oral inhibitor of MetAP2, APL-1202 is a 50-year-old wide spectrum antibiotic that had been shown to have an anti-angiogenesis effect.

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On March 26, 2020 Innovent Biologics, Inc. ("Innovent" or the "Company") (HKEX: 01801), a world-class biopharmaceutical company that develops and commercializes high quality medicines for the treatment of oncology, autoimmune, metabolic and other major diseases, and Alector, Inc. ("Alector", Nasdaq ticker symbol: ALEC), a clinical stage biotechnology company and leader in the discovery and development of immune system focused therapeutics, jointly reported that they have entered into a licensing agreement to develop and commercialize an anti-SIRP-alpha antibody (Alector’s project code: AL008) for the treatment of oncology indications in China (Press release, Innovent Biologics, MAR 26, 2020, View Source [SID1234555834]).

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AL008 is Alector’s novel antibody targeting the CD47-SIRP-alpha pathway, a potent survival pathway co-opted by tumors to evade the innate immune system. AL008 is a first-in-class SIRP-alpha inhibitor with a unique dual mechanism of action that non-competitively antagonizes the CD47-SIRP-alpha pathway by inducing the internalization and degradation of the inhibitory receptor on macrophages to relieve immune suppression (a "don’t eat me signal") while also engaging Fc gamma to promote immuno-stimulatory pathways that drive anti-tumor immunity.

"Alector has been a pioneer in discovering and developing first-in-class therapeutics modulating the innate immune system since their founding and AL008 represents another successful program from their pipeline," said Dr. Michael Yu, Founder, Chairman and CEO of Innovent Biologics. "Currently, we have a successfully commercialized PD-1 inhibiting antibody, Tyvyt (sintilimab injection), targeting the adaptive immune system and we believe in the importance of modulating innate immune system in oncology as well, especially the SIRP-alpha-CD47 pathway. We believe that AL008 will complement perfectly with our current pipeline, further solidifying our position in this promising space while providing more options to patients in need."

"We are excited to enter into a collaboration with Innovent, one of the premier Chinese biologics companies, which has a fully-integrated multi-functional platform. We have been impressed with the diligence and speed that the Innovent team brings to bear to advance their development programs through clinical trials and beyond. We believe AL008 has great potential and look forward to demonstrating this in the clinic," said Dr. Arnon Rosenthal, Co-Founder and CEO of Alector. "We will be collaborating closely with Innovent to rapidly advance AL008 through clinical trials in order to get this program to benefit more patients both in China and globally."

Under the agreement, Innovent will lead the development and commercialization of the molecule in China, including the manufacturing of the product. Alector will lead development of AL008 outside of China. Financial terms are not disclosed.

About AL008

AL008 is an anti-SIRP-alpha antibody that non-competitively antagonizes the CD47-SIRP-alpha pathway by inducing the internalization and degradation of the inhibitory receptor on macrophages to relieve immune suppression (a "don’t eat me signal) while also engaging Fc gamma to promote immuno-stimulatory pathways that drive anti-tumor immunity. Tumor associated macrophages are associated with poor prognosis in many cancer types and are believed to inhibit the anti-tumor immune response. Targeting the CD47-SIRP-alpha pathway has shown activity in myeloid and lymphoid cancers, but additional agents targeting this pathway are needed to enhance activity and improve safety in solid tumors. AL008 monotherapy reduces tumor growth and enhances M1 macrophage activation in a humanized pre-clinical model. In comparison with other SIRP-alpha targeting antibodies, AL008 binds to all common alleles of SIRP-alpha and has best-in-class potency in tumor cell phagocytosis. AL008 promotes T cell function and in preclinical studies is not associated with depletion of red blood cells or platelets. These data highlight the potential potency of this differentiated mechanism of simultaneously providing immune-activating signals while removing immune-checkpoint signals and demonstrate the potential activity for AL008.

On March 26, 2020 Celsion Corporation (NASDAQ: CLSN), a leading oncology drug development company, reported with Medidata, a Dassault Systèmes company, that examining matched patient data provided by Medidata in a synthetic control arm (SCA) with results from the Company’s completed Phase Ib dose-escalating OVATION I Study with GEN-1 in Stage III/IV ovarian cancer patients showed positive results in progression-free survival (PFS) (Press release, Celsion, MAR 26, 2020, View Source [SID1234555879]). The hazard ratio (HR) was 0.53 in the intent-to-treat (ITT) group, showing strong signals of efficacy. GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an interleukin-12 (IL-12) DNA plasmid vector encased in a non-viral nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein.

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Celsion believes these data may warrant consideration of strategies to accelerate the clinical development program for GEN-1 in newly diagnosed, advanced ovarian cancer patients by the U.S. Food and Drug Administration (FDA). In its March 2019 discussion with Celsion, the FDA noted that preliminary findings from the Phase Ib OVATION I Study were exciting but lacked a control group to evaluate GEN-1’s independent impact on impressive tumor response, surgical results and PFS. The Agency encouraged the Company to continue its GEN-1 development program and consult with FDA with new findings that may have a bearing on designations such as Fast Track and Breakthrough Therapy.

GEN-1’s strong and encouraging treatment effect, evidenced by the synthetic control arm, suggests a potentially remarkable improvement in PFS, an FDA recognized surrogate for Overall Survival, and appears to confirm the science behind IL-12’s ability to recruit the innate and adaptive elements of the immune system to fight malignancies. The strong PFS trend is supported with previously published translational data that clearly demonstrates the pro-immune changes in the tumor micro-environment associated with loco-regional GEN-1 therapy.

Celsion’s current randomized Phase II OVATION 2 Study in advanced ovarian cancer patients will commence in the 2nd half of 2020 and is designed to demonstrate a 33% improvement in PFS (HR=0.75) over current standard of care. PFS is the primary endpoint for this study.

Synthetic Control Arms have the potential to revolutionize clinical trials in certain oncology indications and some other diseases where a randomized control is not ethical or practical. SCAs are formed by carefully selecting control patients from historical clinical trials to match the demographic and disease characteristics of the patients treated with the new investigational product.

SCAs have been shown to mimic the results of traditional randomized controls so that the treatment effects of an investigational product can be visible by comparison to the SCA. SCAs can help advance the scientific validity of single arm trials, and in certain indications, reduce time and cost, and expose fewer patients to placebos or existing standard-of-care treatments that might not be effective for them. Medidata is in a unique position to create fit-for-purpose synthetic controls because of access to a pool of more than six million anonymized patients from nearly 20,000 previous clinical trials.

"Acorn AI, by Medidata, is proud to partner with Celsion to create a Synthetic Control Arm for this important clinical trial in advanced ovarian cancer patients with unmet medical needs," said Ruthie Davi, Ph.D., vice president, Data Science at Acorn AI, by Medidata. "This could have game-changing implications for patients, the medical community, and the industry. As demonstrated with the SCA for the OVATION I Study, we now have the opportunity to gain early scientific clarity to expedite the development of potentially life-saving treatments."

PFS data generated from this analysis comparing GEN-1 with SCA showed the following:

GEN-1 Population PFS Hazard Ratio (Confidence Interval)
Intent-to-treat, n=15 0.53 (95% CI 0.16, 1.73); log-rank p=0.29
Per-protocol, n=14 0.33 (95% CI 0.08, 1.37); log-rank p=0.11
"The patients in the GEN-1 arm of the OVATION I Study virtually demonstrated a doubling of control of their cancer than the Synthetic Control Arm. Although these findings are not statistically significant due to the small numbers, they are impressive nonetheless," said Dr. Nicolas Borys, Celsion’s chief medical officer. "This preliminary evidence of a strong treatment effect trend supports our commitment to the GEN-1 program, and we will aggressively explore means of accelerating its development with regulatory agencies and our investigators."

The Phase Ib OVATION I Study evaluated escalating doses of GEN-1 (36 mg/m2, 47 mg/m2, 61 mg/m2 and 79 mg/m2) administered intraperitoneally in combination with three cycles of neoadjuvant chemotherapy (NACT) prior to interval debulking surgery, followed by three cycles of NACT in the treatment of newly diagnosed patients with Stage III/IV ovarian cancer. Previously reported data demonstrated median PFS of 21 months in the per-protocol population and 17.1 months in the intent-to-treat population for all dose cohorts, comparing favorably to historically reported median PFS of 12 months.

In the OVATION I Study, complete tumor resections (R0s) were achieved for all patients receiving the highest (79 mg/m2) dose of GEN-1, and approximately 86% of patients in OVATION I had a complete or partial response. All patients experienced a clinically significant decrease in their CA-125 protein levels as of their latest study visit. CA-125 is used to monitor certain cancers during and after treatment. CA-125 is present in greater concentrations in ovarian cancer cells than in other cells. GEN-1 was well tolerated and no dose-limiting toxicities were detected. Intraperitoneal administration of GEN-1 was feasible with broad patient acceptance.

"We are extremely impressed with the high quality of the matched data from the Medidata SCA," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "They were able to provide near-perfect matches for patient characteristics in our Phase Ib OVATION I Study. Based on this capability and the remarkable potential demonstrated by GEN-1, we plan to move forward with a partial synthetic control arm for the Phase II portion of our Phase I/II OVATION 2 Study with GEN-1 in advanced ovarian cancer. Using a SCA for a portion of the study will reduce costs and should improve the rate of enrollment as patients will be more likely to receive GEN-1 rather than placebo."

About Medidata

Medidata is leading the digital transformation of life sciences, creating hope for millions of patients. Medidata helps generate the evidence and insights to help pharmaceutical, biotech, medical device and diagnostic companies, and academic researchers accelerate value, minimize risk, and optimize outcomes. More than one million registered users across 1,400 customers and partners access the world’s most-used platform for clinical development, commercial, and real-world data. Medidata, a Dassault Systèmes company (Euronext Paris: #13065, DSY.PA), is headquartered in New York City and has offices around the world to meet the needs of its customers. Discover more at www.medidata.com and follow us @Medidata , The Operating System for Life SciencesTM.

About Acorn AI

Acorn AI, by Medidata, a Dassault Systèmes company, combines data, technology, and deep expertise to help life sciences companies deliver actionable insights across the entire continuum of clinical development. Acorn AI’s advanced analytics answers the most important questions in R&D and commercialization including accelerating breakthrough innovation, optimizing study execution and commercial success, and demonstrating the value of therapies. Built upon the Medidata platform comprising nearly 20,000 trials and more than six million patients, Acorn AI products feature the industry’s largest structured, standardized clinical trial data repository connected with real world, translational, and other datasets. For more information, please visit www.medidata.com/acornai.

Medidata and Acorn AI are registered trademarks of Medidata Solutions, Inc., a wholly-owned subsidiary of Dassault Systèmes.

On March 26, 2020 IntegraGen (Paris:ALINT), a company specializing in the transformation of data from biological samples into genomic information and diagnostic tools for oncology, reported Dana-Farber Cancer Institute will utilize the company’s MERCURY cloud-based software as part of their analysis and reporting process for sequencing data obtained from tumors of cancer patients (Press release, Integragen, MAR 26, 2020, View Source [SID1234555864]). Dana-Farber plans to utilize MERCURY to assist in the analysis of sequencing data obtained from small and large targeted gene sequencing panels as well as data derived from whole exome and genome sequencing.

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"Genomic profiling of tumors can assist in the identification of pathogenic molecular alterations which drive a patient’s cancer and enable the implementation of precision medicine-based approaches to treatment," stated Annette S. Kim M.D., Ph.D., Co-Director of the Dana-Farber Cancer Institute’s new Interpretive Genomics Program within the Department of Oncologic Pathology. The program is Co-Directed by Keith L. Ligon, MD PhD, Director of the Dana-Farber Center for Patient Derived Models. "MERCURY provides us with a tool to rapidly interpret large scale and complex genomic sequencing data with the added ability of customization to meet our specific analysis and reporting needs to support clinical research and clinical trials."

"IntegraGen is excited about Dana-Faber’s decision to utilize MERCURY and look forward to interacting with another world leader in cancer care related to the utilization of our cloud-based bioinformatic tools," said Larry Yost, General Manager of IntegraGen, Inc. "We are convinced that the use of MERCURY will aid in the better understanding of the etiology of a patient’s cancer and assist with the realization of the benefits of precision medicine by transforming large-scale sequencing data into actionable results. We are also looking forward to continuing the development and expansion of our genomic interpretation software tools in North America."

MERCURY is a user-friendly genomic interpretation tool for oncology designed to assist pathologists and oncologists to rapidly transform raw data obtained via high-throughput sequencing into a clinical molecular report for clinical and research use. The cloud-based tool minimizes the complexity, time and cost associated with the clinical interpretation and identification of variants that may be of interest in the therapeutic management of patients. MERCURY utilizes the Google Cloud technology to ensure a secure environment for data analysis and storage which is compliant with the latest information security requirements.

On March 26, 2020 -BiomX Inc. (NYSE: PHGE), a clinical-stage biotechnology company developing both natural and engineered phage therapies that target specific pathogenic bacteria, reported financial results and provided a business update for the full year ended December 31, 2019 (Press release, BiomX, MAR 26, 2020, View Source [SID1234555880]).

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"2019 was a landmark year for BiomX as we advanced our lead program in acne-prone individuals into the clinic and, through our merger, listed publicly on the NYSE American stock exchange," said Jonathan Solomon, BiomX Chief Executive Officer. "Phage can target and kill specific bacteria. This property, combined with an excellent safety record, creates the potential for it to become a disruptive therapeutic modality for the treatment of chronic diseases in which bacteria play a causative role. With our phage cocktail customization capabilities, cutting-edge synthetic biology, computational biology tools, phage manufacturing facilities and a strong financial position, we believe we are well positioned to leverage phages’ unique potential and develop important and novel therapeutics. We continue to be on track to announce data from our Phase 1 study of our lead candidate BX001 in subjects with acne prone skin at the end of the first quarter of 2020 and Phase 1 data for our inflammatory bowel disease program in the fourth quarter of 2020."

COVID-19 Update

In light of the evolving COVID-19 pandemic, BiomX has implemented all recommended measures to safeguard the health and safety of its employees and the continuity of its business operations. The Company’s research and development programs currently remain on schedule. In addition BiomX enjoys a strong cash position with over $82 million in cash, cash equivalents and short-term deposits as of the end of 2019.

Recent Highlights

Development Programs

Completed enrollment of a Phase 1 cosmetic clinical study in subjects with acne-prone skin. BiomX completed enrollment in a first-in-human cosmetic clinical study of BX001 in 75 subjects with mild to moderate acne. BX001 is a topically administered gel comprised of a cocktail of naturally occurring phage designed to address the appearance of acne by targeting Cutibacterium acnes (or C. acnes, formerly taxonomically classified as P. acnes), a bacterium implicated in the pathophysiology of acne vulgaris.
Pre-IND meeting held with FDA and manufacturing initiated for BX002 for the treatment of Inflammatory Bowel Disease (IBD). BX002 is a phage cocktail targeting strains of Klebsiella pneumoniae that have been shown in animal models to be pro-inflammatory and pathogenic and may have a role in the onset and aggravation of the disease in humans1. BiomX has advanced towards the clinic with a pre-Investigational New Drug (IND) regulatory meeting and initiated manufacturing for clinical trials in this indication.
Presented preclinical results from program targeting Primary Sclerosing Cholangitis (PSC) at AASLD conference. BiomX presented in vivo data on a phage cocktail that substantially reduced bacterial burden of pathogenic Klebsiella pneumoniae strains which are thought to be disease modifying in PSC, a rare progressive liver disorder with no FDA-approved treatment2.
Presented preclinical results from colorectal cancer program at AACR (Free AACR Whitepaper) Microbiome, Viruses, and Cancer Conference. BiomX presented preclinical in vivo data on phage that target Fusobacterium nucleatum, a bacterium present in the microenvironment of colorectal cancer tumors and thought to protect tumors from the host’s immune system3. BiomX also successfully applied synthetic engineering to convert phage from temperate (lysogenic) to lytic, the state in which phage kill bacteria.
Completed construction of in-house phage manufacturing facility. The new facility, located at the Company’s headquarters, supports clinical manufacturing of candidate phage products and in the future could be expanded to support commercial manufacturing needs.
Corporate

Successfully completed merger resulting with a NYSE American stock exchange public listing and significant capital. In October 2019, BiomX announced closing of the merger with Chardan Healthcare Acquisition Corp. with $60.1 million raised.
Announced dual listing on the Tel-Aviv Stock Exchange (TASE). In February 2020, the Company’s common stock began public trading on the TASE.
Announced additions to Board of Directors and Senior Management. BiomX announced the addition of Russell G. Greig, Ph.D., as Chairman and Lynne Sullivan as Director to the BiomX Board and Chair of the Audit Committee. On October 2019, Merav Bassan, Ph.D., was appointed Chief Development Officer.
Key Upcoming Milestones

Data from the phase 1 cosmetic clinical study of BX001 in subjects with acne-prone skin to be announced at the end of the first quarter of 2020. The primary endpoints of the randomized, double-blind, dose-ranging, vehicle-controlled study are safety and tolerability. Exploratory endpoints include proof of principle by measurement of the effect of BX001 on C. acnes levels.
Data from the phase 1 study of BX002 in inflammatory bowel disease to be announced in the fourth quarter of 2020.
Engineering F. nucleatum phage with various payloads (such as immunostimulatory payloads) and initiating proof of concept in animal models by the fourth quarter of 2020.
Full Year 2019 Financial Results

Cash balance and short-term deposits as of December 31, 2019, were $82.3 million, compared to $40 million as of December 31, 2018. The increase was mainly due to funds raised through completion of the merger with Chardan Healthcare Acquisition Corp.
Research and development expenses of $13.5 million in 2019, compared to $9.1 million in 2018. The increase was attributed mostly to the manufacturing of BX001 and BX002, the Company’s product candidates for acne-prone skin, and IBD, respectively, and due to initiation of the BX001 clinical study.
General and administrative expenses of $8.7 million in 2019, compared to $3.4 million in 2018, mainly due to merger-related expenses and construction of the Company’s in-house manufacturing facility.
Net loss of $20.6 million in 2019, compared to $12.7 million in 2018.
Net cash used in operating activities of $17.6 million in 2019, compared to $11.3 million in 2018.
1 Atarashi et al. (2017), Science
2 Nakamoto et al. (2019), Nature Microbiology
3 Bullman et al. (2017), Science

About Phage Therapy

Bacteriophage, or phage, are viruses that target bacteria and are considered inert to mammalian cells. Phage are designed to target and kill specific bacterial species or strains without disrupting other bacteria or the healthy microbiota. All of BiomX’s phage-based product candidates derive from its proprietary platform, which is first used to discover and validate the association of specific bacterial strains with human diseases or conditions, and is then used to develop rationally-designed phage combinations ("cocktails") of naturally occurring or synthetic phage to target pathogenic bacteria. The phage cocktails contain multiple phage with complementary functions optimized through in vitro and in vivo testing.