Ultragenyx to Host Conference Call for First Quarter 2021 Financial Results and Corporate Update

On April 28, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel therapies for serious rare and ultra-rare genetic diseases, reported that it will host a conference call on Tuesday, May 4, 2021 at 5pm ET to discuss its financial results and corporate update for the first quarter ended March 31, 2021 (Press release, Ultragenyx Pharmaceutical, APR 28, 2021, https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-host-conference-call-first-quarter-2021-financial [SID1234578668]).

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The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 6883837. The replay of the call will be available for one year.

Nymox Announces $8,000,000 Private Placement

On April 28, 2021 Nymox Pharmaceutical Corporation (NASDAQ: NYMX) (the "Company") reported it has entered into a definitive agreement with institutional investors in a private placement of 3,669,724 shares of common stock and warrants to purchase 1,834,862 shares of common stock at a combined purchase price of $2.18 per share for gross proceeds of approximately $8,000,000 before deducting fees and other estimated offering expenses (Press release, Nymox, APR 28, 2021, View Source [SID1234578667]). The warrants will have an exercise price of $2.50 per share, will be immediately exercisable and will expire five years from the date of issuance.

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The Company expects to use the net proceeds from the private placement for working capital and other general corporate purposes. The private placement is expected to close on or about April 30, 2021, subject to the satisfaction of customary closing conditions.

A.G.P./Alliance Global Partners is acting as sole placement agent for the private placement.

The private placement is being made pursuant to the exemption from securities registration afforded by Section 4(a)(2) of the Securities Act of 1933, as amended, and Rule 506 of Regulation D as promulgated by the United States Securities and Exchange Commission (the "SEC") and the securities being sold in the private placement may not be offered or sold in the United States absent registration with the SEC or an applicable exemption from such registration requirements. The Company has agreed to file a registration statement on Form F-3 with the SEC covering the resale of the shares of common stock, as well as the shares of common stock issuable upon exercise of the warrants, issued in the private placement.

This 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 other 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 other jurisdiction.

TRACON to Report First Quarter 2021 Financial Results and Company Highlights on May 5, 2021

On April 28, 2021 RACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that it will report its first quarter 2021 financial and operating results after the close of U.S. financial markets on Wednesday, May 5, 2021 (Press release, Tracon Pharmaceuticals, APR 28, 2021, View Source [SID1234578665]). In addition, management will host a conference call to provide an update on corporate activities and discuss the financial results.

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Conference call and webcast:
Date: May 5, 2021
Time: 4:30 pm Eastern Time (1:30 pm Pacific Time)
Dial-in: (855) 779-9066 (Domestic) or (631) 485-4859 (International)
Passcode: 8852857
Via web: www.traconpharma.com; "Events and Presentations" section within the "Investors" section
A replay of the webcast will be available for 60 days on the website.

Biodesix Announces Intent to Launch Liquid Biopsy Next Generation Sequencing Test with Unprecedented Turnaround Time

On April 28, 2021 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported their plan to add a blood-based 52-gene next generation sequencing (NGS) test to their portfolio of molecular testing based on a recent publication (Press release, Biodesix, APR 28, 2021, View Source [SID1234578664]). Of critical importance, the publication, "Targeted Next-Generation Sequencing of Liquid Biopsy Samples from Patients with NSCLC," in Diagnostics showed that the rapid liquid biopsy testing was able to detect actionable genomic alterations in patients with non-small cell lung cancer (NSCLC) with an unmatched turnaround time of only 72 hours.

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The test has been in use for biopharma research testing and is performed in Biodesix’s ISO 13485-certified, New York Clinical Laboratory Evaluation Program (CLEP)-approved, College of American Pathologists (CAP)-accredited, Clinical Laboratory Improvement Amendments (CLIA)-high complexity certified clinical testing laboratory. With the turnaround time for existing, on-market liquid biopsy NGS tests ranging from 7-14 days, improving turnaround time to three days can be critical for patients with advanced NSCLC who are unable to undergo biopsy or whose biopsies have yielded insufficient test results.

"We observed significant agreement (95.7%–100%) with an orthogonal, high-sensitivity Droplet Digital Polymerase Chain Reaction (ddPCR) test," said Gary Pestano, Ph.D., Chief Development Officer at Biodesix. "This method offers a valuable supplement to assessing targeted mutations from blood while conserving specimens and maintaining sensitivity, with rapid turnaround time to actionable results."

The NGS test will complement the now 36-hour turnaround time that GeneStrat ddPCR and VeriStrat tests currently offer with the expanded coverage of 52-genes and broader molecular markers. The 52-gene NGS test will be used for advanced, late-stage, or recurrent cancer mutation detection, and the targeted 6-gene GeneStrat test can be used for identification of the select mutations for treatment guidance, recurrence monitoring, and detection of the development of resistance mutations over time. The company expects to begin offering this testing strategy in the first half of 2022.

"This test allows Biodesix to offer comprehensive molecular test results, with small and large genomic testing panels in combination with our immune profiling test for early and advanced NSCLC," said Scott Hutton, CEO of Biodesix. "We believe that by adding this NGS test to our current portfolio and multi-omics approach, we can enable physicians to make critical treatment decisions for their patients across the continuum of care in the shortest amount of time available today."

Nkarta Receives NKX019 IND Clearance from U.S. Food and Drug Administration for Treatment of Relapsed/Refractory B Cell Malignancies

On April 28, 2021 Nkarta, Inc. (Nasdaq: NKTX), a biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat cancer, reported that the U.S. Food & Drug Administration (FDA) has cleared an Investigational New Drug (IND) application to study NKX019 in patients with relapsed or refractory B cell malignancies (Press release, Nkarta, APR 28, 2021, View Source [SID1234578663]).

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NKX019 is an off-the-shelf cancer immunotherapy candidate that uses NK cells engineered with a CD19-directed chimeric antigen receptor (CAR). The CAR is designed to target cancer cells expressing CD19, a clinically validated target, and to enhance the innate anti-tumor activity of NK cells. In addition, NKX019 is engineered with a membrane-bound form of IL-15, an important cytokine for NK cell survival, which has been shown in preclinical models to enhance NK cell proliferation, persistence and activity.

Initiation of a Phase 1 clinical trial of NKX019 in patients with relapsed/refractory B cell malignancies, including non-Hodgkin lymphoma, chronic lymphocytic leukemia and acute lymphoblastic leukemia, is planned for the second half of 2021.

"Academic studies have highlighted the ability of donor-derived NK cells, when collected from healthy donors, to effectively target and kill cancer cells without some of the safety risks commonly associated with T cell therapies," noted Kanya Rajangam, MD, PhD, Chief Medical Officer of Nkarta. "We look forward to evaluating NKX019 in our trial as a planned multi-dose, multi-cycle treatment regimen to determine its potential for enhanced anti-tumor activity with a beneficial safety profile."

"This IND clearance, our second in less than 10-months time, builds on the strong track record of the Nkarta team to deliver on challenging goals," said Paul J. Hastings, President and Chief Executive Officer of Nkarta. "Thanks to their outstanding efforts, Nkarta will soon have two differentiated co-lead programs, NKX101 and NKX019, in clinical trials as we look forward to the continued progress of our best-in-class, healthy donor-derived NK cell platform and pipeline of novel cell therapy candidates for cancer patients."

About NKX019
NKX019 is an investigational, off-the-shelf cancer immunotherapy that uses natural killer (NK) cells derived from the peripheral blood of healthy donors and engineered with a CD19-directed chimeric antigen receptor (CAR) and a proprietary, membrane-bound form of interleukin 15 (IL-15). CD19 is a biomarker for normal and malignant B cells, and it is a validated target for B cell cancer therapies. Via its CAR, NKX019 targets and binds to CD19 and eliminates CD19-expressing cells via a robust immune response in preclinical studies. Preclinical models also demonstrate enhanced proliferation, persistence and activity of NK cells with the membrane-bound IL-15, an important cytokine for NK cell survival. Initiation of a Phase 1 clinical trial of NKX019 in patients with relapsed/refractory B cell malignancies in multiple centers in the United States and Australia is planned for the second half of 2021.

About Nkarta’s Platform and Natural Starting Materials
Nkarta’s engineering platform utilizes healthy adult donors as the source for NK cells. By enlisting this natural source of NK cells, Nkarta starts with bona fide NK cells endowed with inherent tumor-recognizing ability and potent cytotoxic function. Healthy donor-derived NK cells are also available in abundance, providing a large quantity of cells with which to begin the efficient two-week manufacturing process. Finally, healthy donor-derived adult cells consist of a diverse repertoire of NK cells, providing Nkarta with the potential to capitalize on the inherent diversity of the innate immune system in selecting donors or NK cell populations with optimal characteristics.

About Nkarta’s NK Cell Technologies
Nkarta has pioneered a novel discovery and development platform for the engineering and efficient production of allogeneic, off-the-shelf natural killer (NK) cell therapy candidates. The approach harnesses the innate ability of NK cells to identify and kill tumor cells. To enhance the inherent anti-tumor activity of NK cells, Nkarta genetically engineers the cells with a targeting receptor designed to recognize and bind to cancer cells through recognition of specific surface proteins. This receptor is fused to co-stimulatory and signaling domains, which amplify cell signaling and NK cell cytotoxicity. Upon target binding, NK cells activate and release cytotoxic granules for target cell killing as well as cytokines that enhance the cumulative immune response. All of Nkarta’s current NK cell therapy candidates are also engineered with a membrane-bound IL-15, a proprietary version of an activating cytokine important for NK cell growth and enhancement of NK cell persistence.

Nkarta’s manufacturing process generates an abundant supply of NK cells that, at commercial scale, is expected to be significantly lower in cost than other current cell therapies. Key to this efficiency is the rapid expansion of donor-derived NK cells using a proprietary NKSTIM cell line, leading to the production of hundreds of individual doses from a single manufacturing run. The platform also features the ability to freeze and store CAR NK cells for extended periods and is designed to enable immediate, off-the-shelf administration to patients at the point of care.