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Horizon Discovery Expands Cell-Based CRISPR Screening Services

On April 3, 2020 Horizon Discovery Group plc (LSE: HZD) ("Horizon", "the Company" or "the Group"), a global leader in the application of gene editing and gene modulation for cell line engineering, reported the addition of an arrayed CRISPR knockout screening service for primary human B cells to its cell-based screening services (Press release, Horizon Discovery, APR 3, 2020, View Source [SID1234556128]).

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Primary human cells, which are cells that are freshly isolated from donors, are known to be difficult to study in the lab. However, working with these cells brings scientists one step closer to healthy or diseased micro-environments, enabling them to better understand disease etiology and therapeutic mechanisms, and thereby advance drug discovery and development programs.

Horizon has already applied its gene editing and cell culture expertise to maintain the viability of primary human T cells to enable functional genomic screens and has been delivering data-rich information to customers working in drug discovery and development. The new B cell screening service, the first of its kind in the market, will enable researchers to identify genes that affect the function of B cells and examine how this impacts other immune cell types, particularly in infectious diseases, cancer, and auto-immune disorders, such as COVID-19, Burkitt’s lymphoma and multiple sclerosis respectively.

Terry Pizzie, CEO, Horizon Discovery said: "The interest in harnessing the immune system for effective therapies continues to grow, with the global cell therapy market predicted to reach $8.21bn by 2025*. Expanding our services to encompass screening of both primary T and B cells is another example of our commitment to apply decades of gene editing experience in support of drug discovery and development for the treatment of human disease."

UroGen Announces Positive Interim Data from Phase 2b Study of UGN-102 in Patients with Low-Grade Intermediate Risk Non-Muscle Invasive Bladder Cancer

On April 3, 2020 UroGen Pharma Ltd. (Nasdaq:URGN) reported that positive interim data analysis of UGN-102 (mitomycin) for intravesical solution in patients with low-grade intermediate risk non-muscle invasive bladder cancer (LG IR-NMIBC) (Press release, UroGen Pharma, APR 3, 2020, View Source [SID1234556127]). These data were featured in a late-breaking abstract published in the April Supplement to The Journal of Urology. The detailed results presentation will be available online via the American Urological Association (AUA) in mid-May 2020.

The Phase 2b OPTIMA II trial demonstrated a complete response (CR) rate at three months following onset of treatment of 65% (41/63 patients). In this subset of patients, 31/32 patients (97%) and 17/20 patients (85%) remained free of disease at six and nine months follow-up, respectively.

"The responses we have seen demonstrate that non-surgical primary chemoablation of low-grade intermediate risk non-muscle invasive bladder cancer using UGN-102 results in a considerable treatment response with encouraging durability," said Dr. Mark Schoenberg, Chief Medical Officer at UroGen. "There are approximately 80,000 treatable patients annually in the US, but this remains a very challenging disease with high rates of recurrence associated with currently available therapies and a need for lifelong active surveillance and repetitive surgical intervention."

The most commonly reported adverse events seen to date were reported as mild to moderate and include dysuria, hematuria, urinary frequency, fatigue, urgency and urinary tract infection.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an investigational drug formulation of mitomycin in Phase 2b development for the treatment of low-grade non-muscle invasive bladder cancer (LG NMIBC). Utilizing the RTGel Technology Platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using standard intravesical catheters. The Company completed enrollment in the Phase 2b OPTIMA II trial of UGN-102 for the treatment of LG NMIBC in September 2019 and intends to advance the program to a pivotal study to further investigate UGN-102 in the treatment of this condition.

IGM Biosciences to Participate in Canaccord Genuity Horizons in Oncology Virtual Event

On April 3, 2020 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported that Fred Schwarzer, Chief Executive Officer, will participate in a panel discussion on bispecific therapies at the Canaccord Genuity Horizons in Oncology Virtual Event on Wednesday, April 8 at 10:15 a.m. ET (Press release, IGM Biosciences, APR 3, 2020, View Source [SID1234556126]).

A live webcast of the event will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following the presentation.

Gracell Announces China MNPA Acceptance of Investigational New Drug Application for GC007g Cell Therapy for CD19 Positive Relapsed or Refrctory B-cell Acute Lymphoblastic Leukemia

On April 3, 2020 Gracell Biotechnologies Co., Ltd. ("Gracell"), a clinical-stage immune cell & gene therapy company, is reported that China National Medical Products Administration (NMPA) has accepted Gracell’s Investigational New Drug (IND) application for GC007g, a donor-derived anti-CD19 chimeric antigen receptor (CAR-T) cell therapy (Press release, Gracell Biotechnologies, APR 3, 2020, View Source [SID1234556125]).

GC007g is an allogenic CAR-T therapy under development for B-ALL patients who relapsed or refractory from prior treatment. The therapy utilizes healthy T cells from human leukocyte antigens (HLA) matching donors, with potentially better T cell fitness, and possibly higher efficacy compared to the use of patient’s own T cells. The company plans to initiate a phase 1 clinical study in patients with CD19 positive relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) in Q2, 2020.

"The IND approval for GC007g marks a significant milestone for Gracell." said Dr. William CAO, founder and CEO of Gracell, "We are delighted that this first-of-its-kind CAR-T program will soon be evaluated in IND approved clinical trials. We expect donor-derived CAR-T therapy, GC007g may become a good alternative solution to the r/r B-ALL patients who may not be eligible for autologous CAR-T therapy due to infections and other conditions, to those who do not respond to autologous CAR-T therapy, or to those whose CAR-T cells fail to be manufactured successfully".

About GC007g
GC007g is an investigational CD19-targeted CAR-T cell therapy, where HLA matching donors’ T cells were employed to redirected to eradicate CD19 positive leukemia cells.

About B-ALL
B-ALL, a major form of acute lymphoblastic leukemia (ALL), is one of the most common forms of cancer in children between the ages of two and five and adults over the age of 501. In 2015, ALL affected around 837,000 people globally and resulted in 110,000 deaths worldwide2. It is also the most common cause of cancer and death from cancer among children. First onset ALL is typically treated with long term chemotherapy. In case of relapsed or refractory disease immunotherapy monoclonal antibodies may be an option in certain subtypes while CART therapy may be an option for patients 25 and younger.

Entry into a Material Definitive Agreement

On April 3, 2020, Inovio Pharmaceuticals, Inc. (the "Company") reported that it has entered into an At-The-Market Equity Offering Sales Agreement (the "Sales Agreement") with Stifel, Nicolaus & Company, Incorporated ("Stifel") under which the Company may offer and sell, from time to time at its sole discretion, shares of its common stock, par value $0.001 per share (the "Common Stock"), through Stifel as its sales agent (Filing, 8-K, Inovio, APR 3, 2020, View Source [SID1234556124]). The Company has filed a prospectus supplement pursuant to the Sales Agreement for the offer and sale of its Common Stock having an aggregate offering price of up to $150,000,000.

Pursuant to the Sales Agreement, sales of the Common Stock, if any, will be made under the Company’s previously filed and effective Registration Statement on Form S-3 (File No. 333-237172) and an applicable prospectus supplement, by any method that is deemed to be an "at the market offering" as defined in Rule 415(a)(4) under the Securities Act of 1933, as amended. Subject to the terms and conditions of the Sales Agreement, Stifel may sell the Common Stock by any method permitted by law deemed to be an "at the market offering" as defined in Rule 415(a)(4) of the Securities Act of 1933, as amended. Stifel will use commercially reasonable efforts to sell the Common Stock from time to time, based upon instructions from the Company (including any price, time or size limits or other customary parameters or conditions the Company may impose). The Company will pay Stifel a commission of up to three percent (3.0%) of the gross sales proceeds of any Common Stock sold through Stifel under the Sales Agreement, and also has provided Stifel with certain indemnification rights.

The foregoing description of the Sales Agreement is not complete and is qualified in its entirety by reference to the full text of the Sales Agreement, a copy of which is filed herewith as Exhibit 10.1 to this Current Report on Form 8-K and is incorporated herein by reference.

This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy the securities discussed herein, nor shall there be any offer, solicitation, or sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state.