On November 9, 2022 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported financial results for the third quarter ended September 30, 2022 and provided a corporate update (Press release, Decibel Therapeutics, NOV 9, 2022, View Source [SID1234623654]).

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"Decibel continued to maintain strong momentum throughout 2022 as we achieved the goals we set forth for the year. The FDA clearance of our IND application and the submission of our CTAs in the UK and Spain for our lead gene therapy product candidate, DB-OTO, mark significant milestones for us, and we look forward to initiating the Phase 1/2 clinical trial in the first half of 2023," said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. "Looking ahead, we plan to continue to strengthen our position as a leader in the broader hearing loss space and we look forward to the opportunity to further our goal of developing transformative gene therapies for the inner ear."

Pipeline Highlights and Upcoming Milestones:

Gene Therapies for Congenital, Monogenic Hearing Loss

Received FDA Clearance of IND Application for DB-OTO: In October 2022, Decibel announced that it had received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 1/2 clinical trial in pediatric patients of DB-OTO, its lead gene therapy product candidate. DB-OTO is being developed in collaboration with Regeneron Pharmaceuticals and is an adeno-associated virus (AAV)-based dual-vector gene therapy designed to provide durable hearing to individuals with profound congenital hearing loss caused by mutations of the otoferlin gene. DB-OTO uses a proprietary, cell-selective promoter to express the otoferlin transgene in hair cells, with the goal of enabling the ear to transmit sound to the brain and provide hearing to such individuals. DB-OTO received Orphan Drug and Rare Pediatric Disease designations from the FDA in 2021.
CTA Applications Filed for DB-OTO in Europe: In November 2022, Decibel announced submission of Clinical Trial Applications (CTAs) to the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom and the Spanish Agency of Medicines and Medical Devices (AEMPS) for a Phase 1/2 clinical trial of DB-OTO in pediatric patients.
Plan to Identify a Product Candidate for AAV.103 Program in Fourth Quarter of 2022: Decibel plans to select a product candidate for further development in its AAV.103 program to restore hearing in individuals with mutations in the gap junction beta-2 (GJB2) gene, the most common cause of autosomal recessive, non-syndromic, congenital hearing loss. Decibel is developing the AAV.103 program in collaboration with Regeneron Pharmaceuticals and retains global commercial rights to the AAV.103 program.
Otoprotection Therapeutic

Intend to Provide Update on DB-020 in First Half of 2023: Decibel plans to report additional data from the interim analysis of the Phase 1b clinical trial of DB-020 at an upcoming conference. The Company continues to evaluate strategic opportunities to continue development of DB-020, including seeking feedback from regulatory authorities.
Corporate Update:

Board of Directors Update: In October 2022, Decibel announced the appointment of Kevin F. McLaughlin to its Board of Directors. Kevin brings more than 40 years of financial and operating management experience spanning the biotech, high-tech and education industries.
Third Quarter 2022 Financial Results:

Cash Position: As of September 30, 2022, cash, cash equivalents and available-for-sale securities were $111.9 million, compared to $162.3 million as of December 31, 2021.
Research and Development Expenses: Research and development expenses were $10.0 million for the third quarter of 2022, compared to $9.0 million for the same period in 2021. The increase in research and development expenses for the third quarter of 2022 was primarily due to additional costs to advance DB-OTO in support of the Company’s IND filing, higher research costs related to preclinical gene therapy programs and higher personnel-related costs due to increased headcount, wages and stock-based compensation.
General and Administrative Expenses: General and administrative expenses were $6.3 million for the third quarter of 2022, compared to $5.7 million for the same period in 2021. The increase in general and administrative expenses for the third quarter of 2022 was primarily due to higher professional fees including external consulting, advisory, legal and audit services.
Financial Guidance:

Based on its current operating and development plans, Decibel believes that its existing cash, cash equivalents and available-for-sale securities will fund its operating expenses into 2024.

On November 9, 2022 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company developing targeted therapies for RAS-addicted cancers, reported that the company will participate in two upcoming investor conferences (Press release, Revolution Medicines, NOV 9, 2022, View Source [SID1234623652]). Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines, will be the featured speaker in fireside chats at the H.C. Wainwright 3rd Annual Precision Oncology Virtual Conference and the 5th Annual Evercore ISI HealthCONx Conference .

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Details of these events are as follows:

H.C. Wainwright 3rd Annual Precision Oncology Virtual Conference
Conference Date: November 14, 2022
Fireside Chat Time/Date: 7:00 a.m. Eastern on Monday, November 14, 2022
Format: Virtual conference; webcast available
5th Annual Evercore ISI HealthCONx Conference
Conference Date: November 29 – December 1, 2022
Fireside Chat Time/Date: 2:15 p.m. Eastern on Thursday, December 1, 2022
Format: Virtual conference; webcast available
Webcasts of the H.C. Wainwright and Evercore ISI fireside chats can be accessed at: View Source Archived replays of the webcasts will be available on the investors section of the company’s website for at least 14 days following the events.

On November 9, 2022 Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat cancer, reported financial results for the third quarter ended September 30, 2022 (Press release, Nkarta, NOV 9, 2022, View Source [SID1234623651]).

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"The third quarter marked significant progress as we continue to treat patients across our two lead clinical programs," said Paul J. Hastings, President and CEO of Nkarta. "We recently completed the first cycle of treatment for patients in the NKX019 monotherapy dose escalation cohort and look forward to announcing new clinical data by year-end, including data from responding patients reported in the last update. We’ve also opened enrollment in multiple dose expansion cohorts, where we intend to build on our prior experience with NKX019 in patients with autologous CAR T naïve, relapsed/refractory (r/r) LBCL, while simultaneously investigating activity in patients with LBCL who have previously received autologous CAR T. We will also explore NKX019 in combination with rituximab, regardless of prior CAR T treatment. NKX101 continues to enroll patients in monotherapy dose escalation, and we have updated the timing of NKX101 data in r/r AML to the first half of next year. We are continuing our mission to bring our allogeneic, off-the-shelf cell therapies to patients in outpatient and community oncology settings, enabling convenient multi-cycle treatment and broad access."

Anticipated Clinical Milestones

Nkarta plans to present additional clinical data from its ongoing Phase 1 clinical trial of NKX019 by year-end 2022 as part of a company-hosted announcement.
Nkarta plans to present additional clinical data from its ongoing Phase 1 clinical trial of NKX101 in the first half of 2023. Nkarta updated this timing from year-end 2022 in order to collect a more robust and interpretable data set from a heavily pre-treated and heterogenous patient population.
The data updates for NKX019 and NKX101 are expected to include safety and activity data from patients in the dose escalation cohorts who receive the 3-dose monotherapy treatment at the higher 1.5 billion CAR NK cell dose, durability of response in patients who were in response as of the April 2022 data cut-off, and safety and activity data from patients in the dose escalation cohorts who may have received one or more additional cycles of CAR NK cell therapy, including consolidation therapy.
NKX019 Dose Expansion Cohorts

Nkarta recently opened enrollment in the dose expansion portion of its Phase 1 clinical trial of NKX019. This dose expansion will investigate for the first time NKX019 as combination therapy with rituximab, an anti-CD20 monoclonal antibody, in patients with r/r non-Hodgkin lymphoma, as well as NKX019 as monotherapy in patients with large B-cell lymphoma (LBCL) who previously received autologous CD19 CAR T therapy. The dose expansion will also further investigate NKX019 as monotherapy in patients with LBCL who have not previously received autologous CD19 CAR T therapy.
Owing to the recent start of patient enrollment, data from the dose expansion cohorts are not expected to be announced in the NKX019 clinical update planned for 2022.
NKX101 Clinical Data – April 2022

On April 25, 2022, Nkarta reported preliminary data from its Phase 1 clinical trial evaluating NKX101, an allogeneic, cryopreserved, off-the-shelf cancer immunotherapy candidate that uses NK cells engineered to target NKG2D ligands on cancer cells, as a multi-dose, multi-cycle monotherapy in patients with r/r acute myeloid leukemia (AML) and higher-risk myelodysplastic syndrome (MDS). As of data cut-off on April 21, 2022, 21 patients had been enrolled and dosed.
Three of five patients with heavily pre-treated AML treated at the higher dose levels in a three-dose regimen achieved a complete response (60% CR) with hematologic recovery, with two of the three responses MRD (measurable residual disease) negative.
NKX101 was well tolerated. No dose-limiting toxicities were observed. No cytokine release syndrome (CRS), graft-versus-host disease (GvHD), or immune effector cell-associated neurotoxicity syndrome (ICANS) was observed. The most common higher-grade adverse events were myelosuppression and infection, which are common in this patient population following lymphodepletion.
NKX019 Clinical Data – April 2022

On April 25, 2022, Nkarta reported preliminary data from its Phase 1 clinical trial evaluating NKX019, an allogeneic, cryopreserved, off-the-shelf cancer immunotherapy candidate that uses NK cells engineered to target the B-cell antigen CD19, as a multi-dose, multi-cycle monotherapy in patients with r/r B-cell malignancies. As of data cut-off on April 21, 2022, 13 patients had been enrolled and dosed.
Three of six patients treated at the higher dose level in a three-dose regimen showed a complete response (50% CR), including one patient with aggressive large B cell lymphoma (LBCL) and one patient with mantle cell lymphoma (MCL).
NKX019 was well tolerated. No dose-limiting toxicities were observed. No CRS, GvHD, or neurotoxicity (ICANS) was observed. The most common higher-grade adverse events were myelosuppression, which is common in this patient population following lymphodepletion.
SITC 2022

In November 2022, Nkarta presented preclinical data from its engineered NK cell platform in two posters at the annual meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper).
Poster #902, "NKX019, an Off-the-Shelf CD19 CAR-NK Cell, Mediates Improved Anti-Tumor Activity and Persistence in Combination with CD20-Directed Therapeutic mAbs," presents preclinical data on the improved anti-tumor activity demonstrated by the combination of NKX019 and a CD20-directed monoclonal antibody (mAb). This combination showed superior tumor cell killing compared to that of NKX019 and either mAb alone. Each agent was capable of direct killing of tumor cells, and when combined they further acted through ADCC, or antibody dependent cellular cytotoxicity. ADCC is a mechanism of immune defense whereby immune cells such as NK cells can recognize and kill tumor cells whose surface antigens are coated with antibodies. ADCC is one of several mechanisms by which NK cells are known to kill tumor cells.
Poster #381, "Large-Scale Expansion and Engineering of Human NK Cells Sourced from Peripheral Blood Versus Umbilical Cord Blood," presents data on the use of Nkarta’s proprietary NKSTIM cell line to expand NK cells in support of potential large-scale commercial production of therapeutic CAR NK cells. NK cells derived from the healthy-donor peripheral blood were expanded over 250 billion-fold while maintaining chromosomal integrity and potency against multiple tumor cell models, and compared favorably to NK cells derived from cord blood. Cell populations capable of superior expansion and native potency could be identified in adult donor-derived NK cells, allowing the potential selection of optimal donors and cell populations for enhanced CAR NK cell production.
Other Corporate Highlights

In August 2022, Nkarta signed amended lease agreements for its future cell therapy manufacturing facility and company headquarters and for its existing facilities. The amendments provide for approximately $15 million of additional tenant improvement allowances for the future facility, increase the rent for the future facility, and increase the rent and term of the lease for some of Nkarta’s existing facilities. These allowances are in addition to the tenant improvement allowances of $25.2 million included in the original lease agreement for the future facility. Nkarta’s facilities are located in South San Francisco, California.
Third Quarter 2022 and Recent Financial Highlights

Cash and Cash Equivalents: As of September 30, 2022, Nkarta had cash, cash equivalents, restricted cash, and short-term investments of $395.1 million.
R&D Expenses: Research and development (R&D) expenses were $23.4 million for the third quarter of 2022. Non-cash stock-based compensation expense included in R&D expense was $1.9 million for the third quarter of 2022.
G&A Expenses: General and administrative (G&A) expenses were $6.8 million for the third quarter of 2022. Non-cash stock-based compensation expense included in G&A expense was $2.4 million for the third quarter of 2022.
Net Loss: Net loss was $28.3 million, or $0.58 per basic and diluted share, for the third quarter of 2022. This net loss includes non-cash charges of $5.9 million that consisted primarily of share-based compensation of $4.4 million.
Financial Guidance

Nkarta expects its current cash and cash equivalents will be sufficient to fund its current operating plan into 2025.
About NKX101
NKX101 is an allogeneic, cryopreserved, off-the-shelf cancer immunotherapy candidate that uses natural killer (NK) cells derived from the peripheral blood of healthy donors. It is engineered with a chimeric antigen receptor (CAR) targeting NKG2D ligands on tumor cells. NKG2D, a key activating receptor found on naturally occurring NK cells, induces a cell-killing immune response through the detection of stress ligands that are widely expressed on cancer cells. NKX101 is also engineered with membrane-bound form of interleukin-15 (IL15) for greater persistence and activity without exogenous cytokine support. To learn more about the NKX101 clinical trial in adults with AML or MDS, please visit ClinicalTrials.gov.

About NKX019
NKX019 is an allogeneic, cryopreserved, off-the-shelf cancer immunotherapy candidate that uses natural killer (NK) cells derived from the peripheral blood of healthy adult donors. It is engineered with a humanized CD19-directed CAR for enhanced tumor cell targeting and a proprietary, membrane-bound form of interleukin-15 (IL-15) for greater persistence and activity without exogenous cytokine support. CD19 is a biomarker for normal and malignant B cells, and it is a validated target for B cell cancer therapies. To learn more about the NKX019 clinical trial in adults with advanced B cell malignancies, please visit ClinicalTrials.gov.

On November 9, 2022 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the following upcoming investor conferences (Press release, Ultragenyx Pharmaceutical, NOV 9, 2022, View Source [SID1234623649]):

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Stifel Healthcare Conference on Tuesday, November 15, 2022, at 1:50 PM EST in New York
Jefferies London Healthcare Conference on Thursday, November 17, 2022, at 10:25 AM GMT
The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On November 9, 2022 BioVaxys Technology Corp. (CSE: BIOV, FRA: 5LB, OTCQB: BVAXF) ("BioVaxys" or the "Company") reported that it has completed a non-brokered private placement (the "Private Placement") consisting of 4,050,000 units ("Units") at a price of $0.10 per Unit for total gross proceeds of $405,000 (Press release, BioVaxys Technology, NOV 9, 2022, View Source [SID1234623648]). Each Unit consists of one common share (a "Common Share") and one whole Common Share purchase warrant (a "Warrant"). Each Warrant is exercisable for one additional Common Share at an exercise price of $0.20 for a period of 48 months.

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All securities issued pursuant to the Private Placement are subject to a statutory hold period of four months and one day from the date of issuance. The Company intends to use the net proceeds of the Private Placement for working capital purposes. The private placement is subject to the approval of the Canadian Securities Exchange.

The Company paid a finder’s fee of $18,400 in cash related to the financing.

In addition, the Company announces that further to its news release dated October 27, 2022, it has settled an aggregate of $150,000 in debt through the issuance of common shares issued at a deemed price of $0.20 per common share.