On June 26, 2019 TARIS Bio, a biopharmaceutical company developing transformational therapies to treat people with debilitating urological disorders, reported positive safety, tolerability and preliminary efficacy data from the full patient cohort (n=20) of its Phase 1b study of TAR-200 for the treatment of patients with muscle invasive bladder cancer (MIBC), neoadjuvant to radical cystectomy (RC) (Press release, TARIS Biomedical, JUN 26, 2019, View Source [SID1234537288]). This complete dataset builds on the initial positive signal observed in the first part of the study, announced previously. Combined data from both TAR-200 study arms indicates a robust antitumor response on final histopathologic analysis after cystectomy, favorable tolerability profile, and no detectable systemic drug exposure or associated adverse events. These data provide early evidence of the potential benefit of TAR-200 in the treatment of MIBC for both patients who can receive RC and those who are unfit for surgical intervention.

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"The TAR-200-101 study has revealed exciting results in patients with muscle invasive bladder cancer," said Siamak Daneshmand, M.D., Director of Urologic Oncology at the USC Institute of Urology, and principal investigator of the study. "Such evidence of response, while early, may enable definitive treatment in patients currently unfit for curative intent therapy, including treatment with checkpoint inhibitors. Moreover, TAR-200 may also ultimately enable organ preservation strategies in lieu of radical cystectomy in patients who are unfit or unwilling to undergo bladder removal."

Each arm in the multi-center study enrolled 10 subjects with organ-confined MIBC (defined as clinical T2-T3 N0M0 disease), who were scheduled to undergo RC and were deemed unfit for systemic platinum-based neoadjuvant chemotherapy. During the window between diagnosis and RC, the TAR-200 system, which continuously delivers gemcitabine in the bladder, was administered twice for seven days per dose separated by a two-week rest. There was no delay to RC for any patient in either arm, with surgery proceeding as planned on Day 28 in Arm 1 and Day 42 in Arm 2. Pathological assessment of pooled data from 20 subjects across the two arms demonstrates:

A 50% objective response rate (ORR), defined as no residual muscle invasive disease (greater than or equal to pT2 disease on post-surgical pathology)
A 40% complete response rate (CR), defined as only residual pT0 or pTis disease
No detectable systemic gemcitabine exposure, nor any typical gemcitabine-associated systemic adverse events
Excellent local tolerability and no evidence of dose-limiting toxicity
"These data provide strong, histopathologically confirmed evidence of TAR-200’s anti-tumor activity and highlight our product candidate’s potential to fundamentally change the management of the full spectrum of disease for patients with MIBC," said Tony Kingsley, President and CEO of TARIS. "The study results support our next stage of development that focuses first on the large portion of patients who do not receive potentially curative therapies. These patients typically have significant comorbidities and a poor overall performance status, rendering them unfit for current standards of care. With few options, these patients face rapid mortality from this unmanaged disease. We believe that TAR-200 is uniquely positioned to address this substantial and serious unmet need."

The standard of care for treatment of MIBC includes radical cystectomy (complete removal of the bladder and surrounding pelvic organs), with or without neoadjuvant chemotherapy. Radical cystectomy is a major, life-changing surgery, and many patients are medically unfit and/or unwilling to undergo this procedure. Without treatment, patients with MIBC who are not candidates for curative intent therapy quickly succumb to their disease.

TARIS is currently conducting a Phase 1b trial of TAR-200 in patients with MIBC who are ineligible for radical cystectomy, to evaluate the safety and tolerability of the system following four consecutive 21-day dosing cycles (TAR-200-103). The multicenter study rapidly over-enrolled 35 subjects – over three times the targeted number of subjects, demonstrating very high demand for a novel therapeutic option for this patient population. TARIS expects to report topline results from this study in late 2019. More information is available at www.clinicaltrials.gov, identifier # NCT03404791.

About Bladder Cancer
Bladder cancer is the fifth most common neoplasm in industrialized countries, affecting roughly 2.7 million people worldwide. The National Cancer Institute estimates approximately 81,200 new cases and 17,240 deaths due to this disease in 2018; muscle invasive bladder cancer (MIBC) accounts for 20-25% of the newly diagnosed cases and the majority of disease-related mortality. Approximately 8,500 of these patients are unfit for radical bladder removal or chemotherapy, and the only treatment option is palliative care.

About TAR-200
TARIS’ lead investigational product, TAR-200, is designed to release the chemotherapeutic agent gemcitabine continuously in the bladder for multiple weeks. TARIS believes that TAR-200 has the potential to harness gemcitabine’s both direct antitumor and immuno-oncologic activity without meaningful systemic drug exposure, yielding a powerful therapy and mitigating systemic side-effects. The TARIS System has been administered in clinical settings to over 450 individuals and over 900 doses across multiple urologic diseases. The U.S. Food and Drug Administration has granted Fast Track designation to TAR-200 in patients with organ-confined or locally advanced MIBC who are unfit for curative-intent therapy.

On June 26, 2019 Tikcro Technologies Ltd. (OTCQB: TIKRF), a pre-clinical stage developer of antibodies for cancer immune-therapy, reported its financial results for the first quarter ended March 31, 2019 (Press release, Tikcro, JUN 26, 2019, View Source [SID1234537286]).

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"We are focused on the progress of our cytotoxic T lymphocyte-associated antigen 4 (CTLA-4) antibody, which has shown strong comparative results in pre-clinical cancer treatment assays," said Aviv Boim, CEO of Tikcro. "At recent industry events a number of biotechnology companies and clinical centers have reported results of trial for the combination treatment of PD1 or PD-L1 antibodies with a CTLA-4 antibody. Several molecule structures and a number of experimental clinical indications in oncology were presented. These development efforts aim to broaden clinical indications, increase the efficacy and reduce immune-related adverse effects of a CTLA-4 antibody treatment. CTLA-4 antibody treatment gains additional attention, however, along with increased competition. Based on pre-clinical results, our novel CTLA-4 antibody has the potential to address the needs to offer higher efficacy and lower side effects."

Several established and emerging pharma companies, including Tikcro, are pursuing new CTLA-4 antibodies to further broaden its clinical scope and to reduce immune related adverse effects.

Financial Results for the First Quarter Ended March 31, 2019
Net loss for the first quarter of 2019 was $271,000, or $0.03 per diluted share, compared to a net loss of $377,000, or $0.04 per diluted share, for the same period last year.

As of March 31, 2019, the company reported $4.95 million in cash, cash equivalents and short-term bank deposits.

On June 26, 2019 Insmed Incorporated (Nasdaq: INSM) reported the closing on June 25, 2019 of the issuance of an additional 1,442,307 shares of common stock pursuant to the full exercise of the underwriter’s overallotment option related to the previously announced public offering of its common stock (Press release, Insmed, JUN 26, 2019, View Source [SID1234537285]). The underwriters were granted 30-day options to purchase up to an additional 1,042,307 shares of common stock from Insmed and up to 400,000 shares of common stock from William H. Lewis, the Company’s Chairman and Chief Executive Officer at a public offering price of $26.00 per share. The net proceeds to the Company from the sale of the additional shares, after deducting underwriting discounts and commissions but before expenses, were approximately $25.6 million. The Company did not receive any proceeds from the sale of Mr. Lewis’ shares.

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Insmed intends to use its net proceeds from this offering to continue to commercialize ARIKAYCE (amikacin liposome inhalation suspension); conduct further trials of ARIKAYCE, including Insmed’s required confirmatory trial to assess and describe the clinical benefit of ARIKAYCE in patients with Mycobacterium avium complex (MAC) lung disease; fund further clinical development of INS1007 and INS1009; invest in increased third‑party manufacturing capacity for ARIKAYCE; fund business expansion activities in Europe and Japan; fund working capital, potential debt repayment, capital expenditures, and general research and development; and for other general corporate purposes, which may include the acquisition or in‑license of additional compounds, product candidates, technology or businesses.

Morgan Stanley & Co. LLC, SVB Leerink LLC and Goldman Sachs & Co. LLC acted as joint book-running managers for the offering. Credit Suisse Securities (USA) LLC, Stifel, Nicolaus & Company, Incorporated and H.C. Wainwright & Co. acted as co-managers for the offering.

A shelf registration statement on Form S-3 relating to the public offering of the shares of common stock described above was filed with the Securities and Exchange Commission (SEC), as amended by Post-Effective Amendment No. 1 thereto, and became automatically effective upon filing. A final prospectus supplement relating to and describing the terms of the offering was filed with the SEC and is available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to this offering may be obtained from Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, Massachusetts 02110, telephone: 1-800-808-7525, ext. 6132 or email at [email protected]; and Goldman Sachs & Co. LLC, Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526, facsimile: 1-212-902-9316 or email at [email protected].

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 jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction.

On June 26, 2019 Yisheng Biopharma Co., Ltd. ("Yisheng Biopharma"), a biopharmaceutical company focusing on research, development, manufacturing, sales and marketing of immunological biologics and vaccines, reported that it has received Investigational New Drug (IND) clearance from the National Medical Products Administration (NMPA) of China to initiate a clinical trial of YS-ON-001, a first-in-class immuno-oncology product for the treatment of advanced solid tumors (Press release, Yisheng US Biopharma, JUN 26, 2019, View Source [SID1234537284]).

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"The IND clearance for YS-ON-001 by the China NMPA marks the third product entering clinical development based on our PIKA technology platform. This milestone once again demonstrates Yisheng’s capability and commitment to the advancement of immunological technology for biopharmaceutical market. PIKA immuno-modulating technology has been officially recognized three times since 2013 as a "National New Medical Innovation" by the Chinese government. We plan to move forward with the clinical development of YS-ON-001 in China to bring it to the market as efficiently as possible. Yisheng Biopharma will continue to expand our portfolio in new immunological therapeutics and vaccines to benefit patients in need of new options," commented by David Shao, Ph.D., President and Chief Executive Officer of Yisheng Biopharma.

About YS-ON-001

YS-ON-001 consists of a complex of protein and PIKA immunomodulating agent and is designed to reduce immunosuppressive effects within the tumor microenvironment and enhance antitumor immune responses. YS-ON-001 is capable of increasing CD4+ and CD8+ T-cell responses, increasing the proportion of natural killer and natural killer T cells and promoting higher expression level of PD-L1 receptors in tumor tissues. YS-ON-001 also showed the reduction in the number of T regulatory cells and myeloid-derived suppressor cells. YS-ON-001 can be delivered via intramuscular, subcutaneous or intratumoral injection and has demonstrated a highly effective tumor growth inhibition in preclinical studies with models of breast, lung, liver, colorectal, prostate and other cancers.

YS-ON-001 has received Orphan Drug Designation from the U.S FDA for development of the treatment for both hepatocellular cancer and pancreatic cancer, and is approved for use in Cambodia as YivykaTM.

On June 26, 2019 Morphic Holding, Inc. ("Morphic"), a biopharmaceutical company discovering and developing oral small-molecule integrin therapeutics, reported the pricing of its upsized initial public offering of 6,000,000 shares of its common stock at a price to the public of $15.00 per share (Press release, Morphic Therapeutic, JUN 26, 2019, View Source [SID1234537281]). The shares are expected to begin trading on The Nasdaq Global Market on June 27, 2019 under the symbol "MORF." The offering is expected to close on July 1, 2019, subject to customary closing conditions. The gross proceeds from the offering, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Morphic, are expected to be approximately $90.0 million. In addition, the underwriters have been granted a 30-day option to purchase up to an additional 900,000 shares of common stock.

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Jefferies LLC, Cowen and Company, LLC, BMO Capital Markets Corp. and Wells Fargo Securities, LLC are acting as joint book-running managers for the offering.

A registration statement relating to these securities has been filed with the Securities and Exchange Commission and became effective on June 26, 2019. The offering is being made only by means of a prospectus. A copy of the final prospectus relating to the offering, when available, may be obtained from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at 877-821-7388 or by email at [email protected], from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by telephone at (631) 592-5973 or by email at [email protected]; from BMO Capital Markets Corp. at 3 Times Square, New York, NY 10036, Attention: Equity Syndicate Department, by telephone at (800) 414-3627 or by email to [email protected]; or from Wells Fargo Securities, LLC 375 Park Avenue, New York, New York 10152, Attention: Equity Syndicate Department, or by calling (800) 326-5897, or by emailing [email protected].

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