On May 13, 2021 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported financial and operating results for the first quarter 2021 (Press release, Lineage Cell Therapeutics, MAY 13, 2021, View Source [SID1234579917]). Lineage will host a conference call today at 4:30 p.m. Eastern Time to discuss its first quarter 2021 financial results and to provide a business update.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Lineage reported significant operational progress with each of its three clinical programs during the first quarter and beyond, delivering not only continued positive clinical results with OpRegen for the treatment of dry-AMD with GA, but also validating partnerships to support our OPC1 and VAC programs," stated Brian M. Culley, Lineage CEO. "We remain encouraged by the totality of the OpRegen clinical data presented to date, which is suggestive of clinically meaningful benefits, especially in earlier stage disease dry-AMD patients. Moreover, the strategic collaborations we announced for OPC1 and VAC reflect our commitment to a comprehensive asset management approach and add external validation to the potential of our platform to create positive outcomes for patients. Additionally, the capital we brought in during the first quarter ensures that we not only are well funded to reach additional milestones, but also provide us with optionality with respect to partnership discussions."

Some of the significant events and milestones achieved to date this year include:

– Presented a positive interim clinical update from the ongoing Phase 1/2a study of OpRegen for the treatment of dry-AMD with GA at the 2021 Association for Research in Vision and Ophthalmology Meeting: 83% of all Cohort 4 patients exhibited stable or improved Best Corrected Visual Acuity (BCVA) while visual acuity declined in the majority of untreated eyes;

– Reported that the first known finding of retinal tissue restoration in a patient who received a retinal pigment epithelium (RPE) cell transplant continues to demonstrate areas of retinal restoration as of their last assessment, approximately 3 years after treatment;

– Treated a vitelliform maculopathy patient with OpRegen under named patient compassionate use: the delivery of OpRegen RPE cells via pars plana vitrectomy (PPV) was successful, with no complications arising during the procedure and the patient remains in follow-up;

– Entered into a worldwide license agreement with Immunomic Therapeutics for an allogeneic cell-based cancer immunotherapy based on Lineage’s VAC platform with a total of $2 million in upfront payments anticipated in the first year and the potential for $67 million in development and commercial milestones;

– Entered into an exclusive agreement with Neurgain Technologies to evaluate a novel delivery system for OPC1 for treatment of spinal cord injury;

– Announced the appointment of Anula Jayasuriya, M.D., Ph.D., M.B.A., a successful healthcare private equity executive and venture capitalist with extensive clinical, industry, entrepreneurial, and investment experience, to the Company’s Board of Directors; and

– Announced the appointment of Dr. Dipti Amin, MBBS, a medically trained senior executive with broad expertise in medicine, pharmacology, healthcare, research, and product development, to the Company’s Board of Directors.

Some of the events and milestones to look forward to during the remainder of 2021 include:

– OpRegen Program

Presentation of additional interim data from the Phase 1/2a study, anticipated during the second quarter of 2021;
Meeting with the U.S. Food and Drug Administration (FDA) to discuss further clinical development, anticipated in the third quarter of 2021.
– OPC1 Program

FDA Regenerative Medicine Advanced Therapy interaction to assess plans to evaluate the Neurgain Parenchymal Spinal Delivery (PSD) system, scheduled in June 2021;
Evaluation of the Neurgain PSD system;
Completion of improved manufacturing process, GMP production, and comparability testing to support a late-stage clinical trial;
FDA interaction to discuss manufacturing improvements, anticipated around the end of 2021.
– VAC Program

Completion of enrollment in the ongoing VAC2 Phase 1 non-small cell lung cancer study, anticipated in mid 2021;
Introduction of manufacturing enhancements to the VAC platform;
Reporting of results from the ongoing VAC2 Phase 1 study, anticipated in the fourth quarter of 2021;
Evaluation of opportunities for new VAC product candidates based on internally-identified or partnered tumor antigens.
– Continued evaluation of partnership opportunities and expansion of existing external collaborations and identification of new collaborations.

Balance Sheet Highlights

Cash, cash equivalents and marketable securities totaled $62.4 million as of March 31, 2021. Marketable securities of $6.2 million as of March 31, 2021 include our remaining ownership of 1,122,401 shares of common stock in OncoCyte and 169,167 shares of common stock in Hadasit Bio-Holdings Ltd.

We added to our cash position in the first quarter of 2021 with net proceeds of $19.3 million received from sales of our common shares under our ATM offering and net proceeds of $10.1 million received from selling a portion of our marketable securities.

No sales were conducted under our ATM offering from March 6, 2021 through May 12, 2021.

First Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from research grants, royalties, and licensing fees. Total revenues for the three months ended March 31, 2021 were approximately $0.4 million, a decrease of $0.1 million as compared to $0.5 million for the same period in 2020. The decrease was primarily related to an approximate $0.2 million decrease in grant income, which was primarily driven by the completion of SBIR grant-related activities, offset by a $0.1 million increase in royalty-related revenues.

Operating Expenses: Operating expenses are comprised of research and development (R&D) expenses and general and administrative (G&A) expenses. Total operating expenses for the three months ended March 31, 2021 were $7.3 million, a decrease of $0.6 million as compared to $7.9 million for the same period in 2020.

R&D Expenses: R&D expenses for the three months ended March 31, 2021 were $3.4 million, an increase of approximately $0.1 million as compared to $3.3 million for the same period in 2020. The overall increase was primarily related to increases of $0.5 million and $0.4 million in VAC and OPC1 program expenses, respectively, and a net decrease of $0.8 million in OpRegen and other ophthalmic application expenses, primarily driven by fluctuations in the timing of manufacturing activities.

G&A Expenses: G&A expenses for the three months ended March 31, 2021 were $3.9 million, a decrease of approximately $0.6 million as compared to $4.5 million for the same period in 2020. The decrease was primarily attributable to decreases of $0.4 million in expenses related to our merger with Asterias Biotherapeutics, Inc., $0.2 million in rent expense and utilities, $0.1 million in legal and patent expenses, and $0.1 million in compensation expense, offset by a $0.2 million increase in investor and public relations expenses.

Loss from Operations: Loss from operations for the three months ended March 31, 2021 was approximately $7.0 million, a decrease of $0.4 million as compared to $7.4 million for the same period in 2020.

Other Income/(Expenses), Net: Other income/(expenses), net for the three months ended March 31, 2021 reflected other income, net of $5.6 million, compared to other expense, net of ($1.0) million for the same period in 2020. The variance was primarily related to the gain on sale of marketable securities and changes in the value of marketable equity securities for the applicable periods, as well as exchange rate fluctuations related to Lineage’s international subsidiaries. The increase in the value of Lineage’s OncoCyte shares and subsequent sales during the first quarter 2021 contributed significantly to the overall net increase in other income.

Net loss attributable to Lineage: The net loss attributable to Lineage for the three months ended March 31, 2021 was $1.4 million, or $0.01 per share (basic and diluted), compared to a net loss attributable to Lineage of $8.4 million, or $0.06 per share (basic and diluted), for the same period in 2020.

Conference Call and Webcast

Lineage will host a conference call and webcast today, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its first quarter 2021 financial results and to provide a business update. Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through May 21, 2021, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 4996965.

On May 13, 2021 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) ("Infinity" or the "Company"), a clinical-stage biotechnology company developing eganelisib (IPI-549), a potentially first-in-class, oral, immuno-oncology macrophage reprogramming therapeutic, reported its first quarter 2021 financial results and provided a corporate update (Press release, Infinity Pharmaceuticals, MAY 13, 2021, View Source [SID1234579916]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2021 is poised to be a landmark year for Infinity with meaningful data updates, building on important data presentations over the last few months, which support the potential of eganelisib’s unique immune modulatory mechanism to improve outcomes across multiple solid tumor indications, lines of therapies and treatment combinations," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "Our encouraging results from MARIO-3 in front-line TNBC show a patient benefit from the addition of eganelisib to the approved standard of care regimen, Tecentriq and Abraxane. We are very much looking forward to sharing updated data from the study across a substantially larger number of patients, with an initial analysis of durability of response, in both mid-year and in the fourth quarter of this year. In order to provide the most meaningful update to our MARIO-3 TNBC presentation from SABCS in December 2020 for our mid-year TNBC update, our goal is to maximize the amount – both in terms of the number of patients and time on study – of data available by planning for a June data cut to be discussed on a July 27th webcast."

Ms. Perkins continued, "Our most recent data from MARIO-275 demonstrate the benefit of adding eganelisib to nivolumab in the advanced second line bladder cancer patient population irrespective of their PD-L1 status and especially for patients who are PD-L1 low who represent the majority of these patients and are least likely to respond to checkpoint inhibitors alone. Based on these results, we are in the planning stages of a potentially registration-enabling study, and we look forward to providing an update on our path forward in bladder cancer on our July 27th webcast based on our interactions with regulatory authorities and in the context of the recent FDA Oncologic Drugs Advisory Committee, or ODAC, meeting in which accelerated approvals for two checkpoint inhibitors in bladder cancer were reviewed."

Recent Updates and Program Guidance:

MARIO-3 Triple Negative Breast Cancer Cohort

Additional data from the triple negative breast cancer (TNBC) cohort of MARIO-3, the company’s ongoing Phase 2 study in collaboration with Roche/Genentech to evaluate eganelisib in a novel triple combination in the 1L setting, adding to Tecentriq and Abraxane, which has received accelerated approval in PD-L1 high 1L TNBC patients, are expected on July 27th and in 4Q 2021. Presentations will include increased patient numbers and early durability data, building upon the positive data presented at the 2020 San Antonio Breast Cancer Symposium which included 100% of patients who had some reduction and 69.2% of patients who had a response per RECIST 1.1, irrespective of PD-L1 status.
Completion of enrollment is expected in 2H’21
MARIO-275 and Advanced Urothelial Cancer

Presented positive data from the MARIO-275 randomized, placebo-controlled Phase 2 study evaluating eganelisib in combination with Opdivo in platinum-refractory, I/O naïve patients with advanced urothelial cancer (aUC), in collaboration with Bristol Myers Squibb (BMS) at the 2021 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU)
Combination of eganelisib with nivolumab demonstrated improved overall response rate (ORR), disease control rate (DCR), and progression free survival (PFS) versus 2L standard of care nivolumab monotherapy
Eganelisib increased the patient benefit over nivolumab monotherapy, regardless of PD-L1 status. The greatest benefit of eganelisib and nivolumab combination therapy over nivolumab monotherapy was observed in the PD-L1 low patient population (n=23) with improvement over nivolumab monotherapy (n=7): ORR of 26% vs. 14%; DCR 57% vs. 14%; and best responses of complete response (CR) 9% vs. 0%, and stable disease (SD) 30% vs. 0%
PD-L1 low patients demonstrated an extended progression free survival (PFS) with a hazard ratio of 0.54 representing a 46% reduction in probability of progression with the addition of eganelisib
The combination of eganelisib and nivolumab was well tolerated at the 30mg once daily dose
Translational data support eganelisib’s immune modulatory mechanism of action
Infinity is in the process of planning a potential registration enabling study leveraging findings from MARIO-275 and incorporating feedback from the U.S. Food and Drug Administration (FDA) including their ultimate decision following the recent ODAC reviews of checkpoint inhibitor accelerated approvals in PD-L1 high metastatic urothelial cancer patients and plans to provide an update on July 27th.
Corporate Update

Closed a $92 million public offering in February 2021 to support execution on the next phase of eganelisib development.
First Quarter 2021 Financial Results:

At March 31, 2021, Infinity had total cash, cash equivalents and available-for-sale securities of $106.8 million, compared to $34.1 million at December 31, 2020.
Research and development expense for the first quarter of 2021 was $8.2 million, compared to $7.3 million in the same period in 2020. The increase is primarily related to clinical, development and consulting expenses to support continued development of eganelisib.
General and administrative expense was $3.6 million for the first quarter of 2021, compared to $3.3 million for the same period in 2020. The increase in G&A expense is primarily due to an increase in stock compensation.
Net loss for the first quarter of 2021 was $11.6 million, or a basic and diluted loss per common share of $0.15, compared to a net loss of $10.9 million, or a basic and diluted loss per common share of $0.19 in the same period in 2020.
Financial Outlook: Infinity’s 2021 financial guidance, following the closing in February 2021 of a $92 million public offering of Infinity’s common stock is as follows:

Net Loss: Infinity expects net loss for 2021 to range from $40 million to $50 million.
Cash and Investments: Infinity expects to end 2021 with a year-end-cash, cash equivalents and available for sale securities balance ranging from $70 million to $80 million. Infinity’s financial guidance does not include additional funding or business development activities.
Conference Call Information

Infinity will host a conference call today, May 13th, 2021, at 4:30PM ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial (877) 316-5293 (domestic) and (631) 291-4526 (international) five minutes prior to start time. The conference ID number is 9988449. An archived version of the webcast will be available on Infinity’s website for 30 days.

On May 13, 2021 Brickell Biotech, Inc. ("Brickell" or the "Company") (Nasdaq: BBI), a clinical-stage pharmaceutical company focused on developing innovative and differentiated prescription therapeutics for the treatment of debilitating skin diseases, reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Vical, MAY 13, 2021, View Source [SID1234579915]).
"The beginning of 2021 has been a very productive period for the Brickell team, as we continue to execute against our development strategy for sofpironium bromide gel, 15% as a potential best-in-class treatment option for primary axillary hyperhidrosis," commented Robert Brown, Chief Executive Officer of Brickell. "Most importantly, we continue to see patient enrollment in our Phase 3 pivotal clinical studies meet expectations, with enrollment completed last month in the Cardigan I study, and 70% enrollment surpassed in the Cardigan II study. As a result, we are on track to announce topline data from both studies in the fourth quarter of 2021. If these studies are successful, we expect to proceed towards an NDA submission to the U.S. FDA in 2022."

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Mr. Brown continued, "In April 2021, we were pleased to highlight data from the ARGYLE study, our Phase 3 open-label, long-term safety study of sofpironium bromide gel, as part of the late-breaking research program at the American Academy of Dermatology’s Virtual Meeting Experience 2021. In this study, sofpironium bromide gel was generally well-tolerated with continued efficacy during 48 weeks in patients with primary axillary hyperhidrosis. These data further contribute to our understanding of the long-term use of sofpironium bromide gel as a potential novel treatment for the millions of patients suffering from this chronic and debilitating condition."
Business and Recent Developments
•Completed enrollment in the Phase 3 Cardigan I study and exceeded 70% enrollment in the Phase 3 Cardigan II study. Both randomized, double-blinded, placebo-controlled pivotal studies are evaluating sofpironium bromide gel, 15% vs. placebo (1:1 ratio) in approximately 350 subjects (per study) aged nine and older with primary axillary hyperhidrosis.
•Presented results from the Phase 3 open-label, long-term safety study of sofpironium bromide gel, 5% and 15% in a late-breaking oral presentation at the American Academy of Dermatology’s Virtual Meeting Experience 2021 ("AAD VMX 2021"), in which the safety, tolerability, and efficacy results for sofpironium bromide gel, 5% and 15% were consistent with prior clinical experience and no unexpected safety findings were observed.
•Published validation results from the Company’s proprietary patient reported outcome scale, the Hyperhidrosis Disease Severity Measure-Axillary© (HDSM-Ax), in the peer-reviewed Journal of Drugs in Dermatology. The published results conclude that the HDSM-AX scale is a well-defined and reliable measure of primary axillary hyperhidrosis.
•Hosted a KOL event in March with leading dermatologists to discuss hyperhidrosis through both the eyes of a patient and diagnosing clinician, as well as the unmet need that exists in hyperhidrosis, the current treatment landscape, and the negative quality of life impacts experienced by both pediatric and adult patients.
•Japanese development partner, Kaken Pharmaceutical Co., Ltd. ("Kaken"), continued to ramp up commercialization efforts for sofpironium bromide gel, 5% (ECCLOCK) in Japan.
•Strengthened the Company’s balance sheet to $34.8 million in cash and cash equivalents at the end of the first quarter of 2021, which includes aggregate net proceeds of $10.6 million from warrant exercises and the sale of shares under a previously filed At-The-Market Equity Offering Program during the first quarter of 2021.

Upcoming Milestones
•On track to complete enrollment for the Cardigan II pivotal study in the third quarter of 2021.
•Expect to report topline results from the Cardigan I and II pivotal studies in the fourth quarter of 2021.
Financial Results
First Quarter 2021 Financial Results
The Company reported cash and cash equivalents of $34.8 million as of March 31, 2021, compared to $30.1 million as of December 31, 2020.
Revenue was approximately $17 thousand for the first quarter of 2021, compared to $1.0 million for the first quarter of 2020. Revenue in 2021 consisted of royalty revenue recognized related to sales of ECCLOCK in Japan by Kaken, while revenue in 2020 was driven by collaboration revenue recognized for research and development activities related to a license agreement with Kaken pursuant to which Kaken provided research and development funding to Brickell.
Research and development expenses were $6.1 million for the first quarter of 2021, compared to $2.7 million for the first quarter of 2020. This increase was primarily due to an increase in clinical costs related to the Phase 3 Cardigan studies, which were initiated in the fourth quarter of 2020.
General and administrative expenses were $3.0 million for the first quarter of 2021, compared to $2.5 million for the first quarter of 2020. The increase was primarily due to increases in compensation-related expense and professional fees.
Brickell’s net loss was $9.0 million for the first quarter of 2021 compared to $4.1 million for the first quarter of 2020.
Conference Call and Webcast Information
Brickell’s management will host a conference call today at 4:30 p.m. ET to discuss the financial results and recent corporate developments. The dial-in number for the conference call is 1-877-705-6003 for domestic participants and 1-201-493-6725 for international participants, with Conference ID #13718897. A live webcast of the conference call can be accessed through the "Investors" tab on the Brickell Biotech website at View Source A replay will be available on this website shortly after conclusion of the event for 90 days.
About Sofpironium Bromide
Sofpironium bromide is Brickell’s lead investigational product candidate and is a new chemical entity that belongs to a class of medications called anticholinergics. Anticholinergics block the action of acetylcholine, a chemical that transmits signals within the nervous system that are responsible for a range of bodily functions, including activation of the sweat glands. Sofpironium bromide was retrometabolically designed. Retrometabolic drugs are intended to exert their action locally and are potentially rapidly metabolized into a less active metabolite once absorbed into the blood. Sofpironium bromide gel, 15% is currently being evaluated in a U.S. pivotal Phase 3 clinical program for the treatment of primary axillary hyperhidrosis, and sofpironium bromide gel, 5% is approved in Japan for the same indication under the brand name ECCLOCK. Sofpironium bromide was discovered at Bodor Laboratories, Inc. by Dr. Nicholas Bodor D.Sc., d.h.c. (multi), HoF, Graduate Research Professor Emeritus, University of Florida.
About Hyperhidrosis
Hyperhidrosis is a debilitating, life-altering medical condition where a person sweats beyond what is physiologically required for thermoregulation of the body. More than 15 million people, or 4.8% of the population of the United States, and 12.76% of the population in Japan, are believed to suffer from hyperhidrosis1,2. Primary axillary (underarm) hyperhidrosis is the targeted first indication for sofpironium bromide and is the most common site of occurrence of hyperhidrosis, affecting an estimated 65% of patients with hyperhidrosis in the United States. Additional information can be found on the International Hyperhidrosis Society website: View Source

On May 13, 2021 Kinnate Biopharma Inc. (Nasdaq: KNTE) ("Kinnate"), a biopharmaceutical company focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers, reported the closing of a $35 million Series A financing for a joint venture in China. Established with OrbiMed Asia Partners, OrbiMed Private Investments and Foresite Capital, the joint venture will be headquartered in Shanghai and enable the potential development and commercialization of certain Kinnate targeted oncology product candidates across Greater China (mainland China, Hong Kong, Taiwan, and Macau) (Press release, Kinnate Biopharma, MAY 13, 2021, View Source [SID1234579914]). Kinnate Biopharma will be the majority shareholder in the joint venture. The company has also announced that veteran biopharmaceutical industry executive Wenn Sun, Ph.D., has been appointed as the joint venture’s Executive Chair.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Establishing operations in China creates a tremendous opportunity for Kinnate to build its global footprint and further advance our mission of expanding access to innovative targeted therapies for people battling cancer," said Nima Farzan, President and CEO of Kinnate Biopharma Inc. "OrbiMed Private Investments and Foresite Capital have been important partners in the growth of Kinnate and we are pleased to now have the support of OrbiMed Asia Partners who led this financing and brings tremendous expertise and connections in China to this new joint venture. I look forward to working with this leading team of investors and Dr. Sun to make precision medicine a reality for more people in Greater China, which is one of the world’s largest healthcare markets."

The initial focus of the joint venture is on advancing the development of KIN-2787 for the Greater China market. KIN-2787 is a Rapidly Accelerated Fibrosarcoma (RAF) inhibitor candidate being developed for the treatment of patients with lung cancer, melanoma, and other solid tumors. The joint venture will also pursue development of KIN-3248 for the Chinese market. KIN-3248 is a Fibroblast Growth Factor Receptors (FGFR) inhibitor candidate for the treatment of patients with intrahepatic cholangiocarcinoma (ICC), a cancer of the bile ducts in the liver, and urothelial carcinoma (UC), a cancer of the bladder lining. The joint venture, which will be subsequently named, has an exclusive license to one other Kinnate program and may also obtain rights to develop certain other new product candidates in Greater China from the Kinnate pipeline, as well as other third-party product candidates in China and other geographies.

"Since its founding, Kinnate has demonstrated impressive scientific discipline and exceptional execution in building a robust pipeline of targeted therapies for hard-to-treat cancers," said Steven D. Wang, Ph.D., CFA, Partner and Senior Managing Director, OrbiMed Asia Partners. "We are pleased to join them in establishing this joint venture in China and look forward to working closely with Dr. Sun and our other regional colleagues in bringing these potentially life-saving therapies to more patients."

Dr. Sun is the Founder and President of Precision Medicine Asia (PREMIA), an oncology-focused clinical genomic data company she founded in 2018. Previously, she was the Founder and Managing Partner for OxOnc Development, a venture company that, along with Pfizer Oncology, co-developed XALKORI in patients with ROS1 genetic alterations in Asia, including China. Dr. Sun also served as Head of Strategic Alliances for GSK Oncology, and helped build its alliances with various clinical research networks around the world. In 2003, in collaboration with the National Comprehensive Cancer Network (NCCN), she helped introduce the NCCN Guidelines to China. Dr. Sun was appointed Chief Business Development Officer at the Lurie Cancer Center of Northwestern University after her post-doctoral fellowship at University of Wisconsin-Madison.

"I am honored to join the Kinnate team and lead the joint venture’s efforts to help the patients in China for whom no genomically-targeted therapies exist or for which a resistance to targeted treatments has evolved," said Dr. Sun. "KIN-2787 has already demonstrated very promising results in pre-clinical studies and presents a significant opportunity to help address the tremendous demand for more effective cancer therapies across Greater China."

On May 13, 2021 Nkarta, Inc. (Nasdaq: NKTX), a biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat cancer, reported financial results for the first quarter ended March 31, 2021 (Press release, Nkarta, MAY 13, 2021, View Source [SID1234579913]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"As Nkarta prepares NKX019, our second co-lead CAR NK program, to enter clinical trials later this year, we look forward to the evolution of broad proof of concept for our healthy donor derived engineered CAR NK product candidates as mono and combination therapies across multiple targets and indications," said Paul J. Hastings, President and Chief Executive Officer of Nkarta. "We expect to report initial clinical data from NKX101, our first co-lead program, by the end of 2021, with additional data announcements from both programs in 2022."

Hastings continued, "As previously announced, we’re excited and proud to be working with CRISPR Therapeutics to enhance the potential of our NK cell therapy platform using their best in class genome engineering technology and expertise in allogeneic CAR T cell therapy. This collaboration brings together the complementary strengths of two leaders in cell therapy with the aim of accelerating our research and development efforts to advance important cell therapies that can be made broadly accessible to cancer patients."

RECENT ACCOMPLISHMENTS AND FUTURE MILESTONES

NKX019

In April 2021, the U.S. Food & Drug Administration cleared the Investigational New Drug (IND) application for NKX019, a chimeric antigen receptor (CAR) NK cell therapy candidate engineered to target tumors expressing CD19, for the treatment of relapsed/refractory B cell malignancies. Nkarta expects patient dosing in a Phase 1 clinical trial of NKX019 to initiate in the second half of 2021.
NKX101

In April 2021, the FDA approved a protocol amendment to the clinical trial of NKX101 for patients with relapsed/refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndromes (MDS). The amendment includes an overall shorter waiting period between enrollment of patients, an additional two-dose regimen to increase patient convenience and to deliver more CAR NK cells earlier in each treatment cycle, and the earlier introduction of non haplo-related, off-the-shelf NKX101 in the ongoing dose finding cohort.

Nkarta aims to present initial clinical data from its ongoing clinical trial of NKX101 by year end 2021. In the Phase 1 study, patients receive multiple doses of NKX101 during a 28-day treatment cycle and are eligible to receive subsequent cycles of treatment upon evidence of tolerability and disease response.
Pipeline and Platform

In May 2021, Nkarta and CRISPR Therapeutics announced a research and development collaboration to co-develop and co-commercialize two chimeric antigen receptor (CAR) NK cell product candidates, one targeting CD70, and a product candidate combining NK and T cells (NK+T), each enhanced with genome engineering. The collaboration also gives Nkarta a license to CRISPR/Cas9 gene editing technology for use in its own engineered NK cell therapy products.
Manufacturing

Nkarta expects to manufacture NKX019 clinical supply for the Phase 1 clinical trial at its in-house cGMP clinical manufacturing facility located in South San Francisco, California.

Nkarta has started early planning for a commercial-scale cell therapy manufacturing facility in the United States.
FIRST QUARTER 2021 FINANCIAL HIGHLIGHTS

Cash and Cash Equivalents: As of March 31, 2021, Nkarta had cash, cash equivalents, restricted cash and short-term investments of $299.7 million.

R&D Expenses: Research and development expenses were $13.5 million for the first quarter of 2021. Non-cash stock-based compensation expense included in R&D expense was $1.6 million for the first quarter of 2021.

G&A Expenses: General and administrative expenses were $5.9 million for the first quarter of 2021. Non-cash stock-based compensation expense included in G&A expense was $1.8 million for the first quarter of 2021.

Net Loss. Net loss was $19.4 million, or $0.59 per basic and diluted share, for the first quarter of 2021.
FINANCIAL GUIDANCE

Nkarta expects its current cash and cash equivalents will be sufficient to fund its current operating plan into at least the second half of 2023.
About NKX101
NKX101 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 membrane-bound IL15 and 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. By engineering NKX101 with the proprietary NKG2D-based CAR, the ability of NK cells to recognize and kill tumor cells in pre-clinical models is increased significantly compared to non-engineered NK cells. The addition of membrane-bound IL15, a proprietary version of a cytokine for activating NK cell growth, has been shown in pre-clinical models to enhance the proliferation, persistence and sustained activity of NK cells. A multi-center Phase 1 clinical trial of NKX101 in patients with relapsed/refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndromes (MDS) is currently enrolling. Additional information about the clinical trial is available on ClinicalTrials.gov, identifier NCT04623944.

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 recognize and kill tumor cells. To enhance the inherent biological activity of NK cells, Nkarta genetically engineers the cells with a targeting receptor designed to recognize and bind to specific proteins on the surface of cancerous cells. This receptor is fused to co-stimulatory and signaling domains to amplify cell signaling and NK cell cytotoxicity. Upon binding the target, NK cells become activated and release cytokines that enhance the immune response and cytotoxic granules that lead to killing of the target cell. All of Nkarta’s NK current cell therapy candidates are also engineered with a membrane-bound IL15, a proprietary version of a cytokine known for activating NK cell growth, to enhance the persistence and activity of the NK cells.

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 allogeneic and autologous 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 an extended period of time and is designed to enable immediate, off-the-shelf administration to patients at the point of care.