On October 13, 2021 Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, reported a strategic collaboration to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases (Press release, Intellia Therapeutics, OCT 13, 2021, View Source [SID1234591177]).

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As part of this collaboration, Intellia will grant SparingVision exclusive rights to Intellia’s proprietary in vivo CRISPR/Cas9-based genome editing technology for up to three ocular targets addressing diseases with significant unmet medical need. SparingVision will lead and fund the preclinical and clinical development for the genome editing product candidates pursued under the collaboration. In addition, the parties will research and develop novel self-inactivating AAV vectors and LNP-based approaches to address delivery of CRISPR/Cas9 genome editing reagents to the retina.

As part of the licensing agreement, Intellia will receive a 10% equity ownership stake in SparingVision. Intellia will also be eligible to receive certain development and commercial milestone payments (around $200 million per product) as well as royalties on potential future sales of products arising from the collaboration. In addition, Intellia may exercise an option to obtain the US commercialization rights for product candidates arising from two of three collaboration targets. For product candidates Intellia chooses to option, Intellia will pay an opt-in fee, reimburse certain costs, share in 50% of development costs and pay royalties to SparingVision on US sales. Intellia will also maintain the ability to leverage technology advances established under this collaboration for any targets outside the partnership.

Stéphane Boissel, President and Chief Executive Officer of SparingVision, said: "SparingVision’s aim has always been to disrupt the ophthalmology field by using cutting-edge technologies to address areas of significant unmet need. This collaboration with Intellia marks a pivotal moment in this mission and is highly complementary to our already mature and growing pipeline of unique mutation-agnostic gene therapies. Intellia is the first company in history to present clinical data supporting precision editing of a disease-causing gene within the body following a single, systemic dose of CRISPR/Cas9 and we are honored to have been selected as a strategic partner. We look forward to working together with the shared goal of radically changing the treatment of blinding ocular diseases."

"Intellia has been a pioneer in utilizing CRISPR/Cas9 technology to develop potentially curative treatments for genetic diseases. Today’s announcement is another step forward in more fully leveraging the power of our genome editing technology to address diseases inadequately treated with existing medicines," said Intellia President and Chief Executive Officer John Leonard, M.D. "We have been thoroughly impressed with the team at SparingVision, particularly regarding their unparalleled understanding of retinal diseases and track-record for developing novel therapies for patients with ocular diseases. We believe SparingVision will be an excellent partner to expand our genome editing capabilities into the field of ophthalmology and we look forward to our new partnership."

The transaction is expected to close in the fourth quarter and is subject to certain closing conditions.

SparingVision’s management team will be holding a webcast to discuss the collaboration today (13th October) at 16:00 CEST / 15:00 BST / 10:00 ET, which will include a live Q&A session. Please find a link to join this webcast here: View Source

On October 13, 2021 Geneseeq Technology Inc. ("Geneseeq") and Zai Lab, Ltd. ("Zai Lab") (Nasdaq: ZLAB)(HKEX: 9688) reported that new strategic collaboration in Shanghai, China (Press release, Zai Laboratory, OCT 13, 2021, View Source [SID1234591198]). The companies will collaborate to facilitate the R&D and commercialization of Zai Lab’s cancer drugs through business partnerships in clinical trial patient recruitment, central laboratory testing, companion diagnostics development and marketing activities . The announcement was made at the Zai Lab Innovation Summit on October 12th in Shanghai.

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Marc Zhao, the co-founder and Chief Operating Officer of Geneseeq delivered a keynote speech on the landscape of gastrointestinal tumor detection and diagnosis during the conference. To conclude, Zhao said that Geneseeq and Zai Lab hope to use this strategic collaboration as the cornerstone to combine resources of both companies to provide high-quality cancer diagnostics and treatment solutions. I look forward to further joint actions between the two parties to improve cancer patient outcomes and generate positive societal impact.

William Liang , Chief Commercial Officer of Zai Lab and President of Greater China said: "In recent years, advances in genetic testing technology have boosted the continuous improvement of precision medicine and treatment programs. Zai Lab has three products listed in China with 10-15 more products expected to be approved. Many of them rely on biomarker testing. We hope to work with genetic testing partners to help patients achieve precise and personalized treatment. At the same time, we also look forward to working with these partners to improve the efficiency of drug research and development, shorten the development cycle, and accelerate product approvals."

The companies look forward to a strong alliance in providing more accurate and efficient treatment plans for patients.

On October 13, 2021 Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, reported that it has priced an underwritten public offering of 6,172,840 shares of its common stock at a price to the public of $81.00 per share (Press release, Sarepta Therapeutics, OCT 13, 2021, View Source [SID1234591243]). In addition, Sarepta has granted the underwriters a 30-day option to purchase up to an additional 925,926 shares of its common stock on the same terms and conditions as the initial shares sold to the underwriters. Sarepta anticipates the gross proceeds from the offering, before deducting underwriting discounts and commissions and other offering expenses, to be approximately $500.0 million, excluding any exercise of the underwriters’ option to purchase additional shares. The offering is expected to close on or about October 18, 2021, subject to customary closing conditions.

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Goldman Sachs & Co. LLC, J.P. Morgan Securities LLC, Morgan Stanley & Co. LLC and Credit Suisse Securities (USA) LLC are acting as joint book-running managers for the offering. RBC Capital Markets, LLC, Robert W. Baird & Co. Incorporated and Cantor Fitzgerald & Co. are acting as co-managers for the offering.

Sarepta intends to use the net proceeds from the offering principally for the continuation of, and initiation of further, clinical trials, commercialization, manufacturing, business development activities, including the potential licensing or acquisition of complementary products, technologies and entities, and other general corporate purposes.

The shares are being offered by Sarepta pursuant to an effective shelf registration statement that was previously filed with the Securities and Exchange Commission (SEC). The offering is being made only by means of a written prospectus and prospectus supplement. The final prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement relating to these securities may also be obtained from the offices of Goldman Sachs & Co. LLC, at Prospectus Department, 200 West Street, New York, New York 10282, by telephone at 866-471-2526, by facsimile at 212-902-9316 or by e-mail at [email protected]; J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (866) 803-9204, or by emailing [email protected]; Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014, or by email at [email protected]; or Credit Suisse Securities (USA) LLC, Attn: Prospectus Department, 6933 Louis Stephens Drive, Raleigh, North Carolina 27560, Telephone: 1-800-221-1037, or by email at [email protected].

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

On October 13, 2021 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported it has fully accrued the initial planned number of participants in Cohort C (third-line or greater) of its Phase 2 trial studying a combination immunotherapy (Nant Cancer Vaccine) in advanced metastatic pancreatic cancer (Press release, ImmunityBio, OCT 13, 2021, View Source [SID1234591162]). The majority of participants in the study to date remain on therapy and 90% (43/48) of the evaluable patients have exceeded the approximately two-month historical survival rate. Of the 48 evaluable patients, 23 (48%) had extremely advanced disease upon enrollment (i.e. had progressed after three to six prior lines of therapy) and, of these patients, 20 out of 23 (87%) have exceeded historical survival rates. On the strength of this early data and significant unmet medical need, the company has submitted an amendment to increase enrollment in Cohort C.

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"Patients with advanced metastatic pancreatic cancer who have failed all standards of care have very grave prognoses with few treatment options. This study was to explore if the Nant Cancer Vaccine could address this unmet need," said Patrick Soon-Shiong, M.D., Founder and Global Chief Scientific and Medical Officer of ImmunityBio, Inc. "It is gratifying to note that patients in this study, who had progressed after up to six lines of prior therapy, have exceeded historical survival rates despite having very advanced pancreatic cancer upon enrollment."

QUILT 88 is an open-label study to evaluate the safety and efficacy of the Nant Cancer Vaccine, comprising ImmunityBio’s IL-15 receptor agonist Anktiva (N-803), its off-the-shelf targeted natural killer cells (PD-L1 t-haNK), and aldoxorubicin, an albumin-modulated agent, plus low-dose chemotherapy. This combination therapeutic vaccine will be randomized against standard-of-care high-dose chemotherapy for first- and second-line treatment; the third-line cohort, with a target of 50 patients, is a single arm with the primary endpoint of overall survival.

Pancreatic cancer is the fourth leading cause of cancer-related death in the United States and has one of the highest mortality rates of all major cancers, taking nearly 50,000 lives in the U.S. every year. Surgery and subsequent adjuvant chemotherapy are the preferred treatment options for pancreatic cancer today and the five-year survival rate for late-stage cases is just 3%. For the majority of patients who present with more advanced disease, treatment typically consists of chemotherapy alone or supportive care for metastatic patients, and chemotherapy with or without radiation for those with locally advanced disease.

"Achieving robust enrollment in this patient group and early promising efficacy evidence are important milestones in ImmunityBio’s effort to develop this therapeutic with the potential to improve survival rates and provide a replacement for toxic chemotherapy," continued Soon-Shiong. "As the historical survival rate for third- to sixth-line pancreatic cancer patients is approximately two months, we are encouraged by this early data and have decided to open this cohort to more patients with advanced metastatic disease who have no further treatment options."

QUILT-88 Study Details

This Phase 2, randomized, three-cohort, open-label study will evaluate the comparative efficacy and overall safety of standard-of-care chemotherapy versus standard-of-care chemotherapy, in combination with PD-L1 t-haNK, Anktiva (N-803), and aldoxorubicin in subjects with locally advanced or metastatic pancreatic cancer (NCT04390399). Each treatment setting, as well as each first- and second-line or later maintenance treatment, will be evaluated independently as Cohorts A, B, and C, respectively, with Cohorts A and B having independent experimental and control arms. The study will initially enroll 298 subjects across all three cohorts. The primary objective of Cohorts A and B is progression-free survival (PFS) per RECIST V1.1, and the objective of Cohort C is overall survival (OS). Secondary objectives include initial safety and additional efficacy measures, including overall response rate (ORR), complete response (CR) rate, durability of response (DoR), disease control rate (DCR), and overall survival (OS).

Currently, three trial sites have been activated: Hoag Memorial Hospital Presbyterian in Orange County, Calif., The Chan Soon-Shiong Institute for Medicine in Los Angeles County, Calif., and Avera McKennan Hospital and University Health Center in Sioux Falls, South Dakota, which serves patients in the tri-state area (Iowa, Nebraska and South Dakota).

On October 13, 2021 Incyte (Nasdaq:INCY) reported that it has scheduled its third quarter financial results conference call and webcast for 8:00 a.m. ET on Tuesday, November 2, 2021 (Press release, Incyte, OCT 13, 2021, View Source [SID1234591178]).

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The schedule for the press release and conference call/webcast is as follows:

• Q3 2021 Press Release:

November 2, 2021 at 7:00 a.m. ET
• Q3 2021 Conference Call: November 2, 2021 at 8:00 a.m. ET
• Domestic Dial-In Number: 877-407-3042
• International Dial-In Number: 201-389-0864
• Conference ID Number:
13724199

If you are unable to participate, a replay of the conference call will be available for thirty days. The replay dial-in number for the U.S. is 877-660-6853 and the dial-in number for international callers is 201-612-7415. To access the replay you will need the conference ID number 13724199.

The live webcast with slides can be accessed at Investor.Incyte.com and will be available for replay for 90 days.