On March 25, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported its financial results for the year ended December 31, 2020 and provided a business update (Press release, Phio Pharmaceuticals, MAR 25, 2021, View Source [SID1234577147]).

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"Over the past year, we established steady momentum across our development programs and expanded our pipeline, including our new collaboration with AgonOx, Inc. to develop T cell-based cancer immunotherapies using PH-762. This progress has resulted in a steady stream of preclinical data, which we plan to present for several programs throughout 2021. Also, we are on track to initiate two clinical studies for our lead asset, PH-762, later this year," said Dr. Gerrit Dispersyn, President and CEO of Phio. "In support of the planned initiation of two clinical programs for different applications of PH-762, and of the advancement of our broader development pipeline, we recently completed two equity financings for additional gross proceeds of $21.7 million. Our stronger financial footing is expected to fund our current programs for the next two years, including clinical studies on the safety and efficacy of PH-762 in adoptive cell therapy and with direct drug therapy."

Quarter in Review and Recent Corporate Updates

Presented data at SITC (Free SITC Whitepaper) 2020 showing that the antitumoral efficacy of our PH-762, PH-790 and PH-804 INTASYL compounds can be further improved by combining them in a single drug treatment.
Presented data resulting from our collaborations with AgonOx, Inc. and the Helmholtz Zentrum München, including poster presentations at SITC (Free SITC Whitepaper) 2020.
Announced a collaboration for the clinical development of novel T cell-based cancer immunotherapies using PH-762 and AgonOx, Inc.’s "double positive" (DP) tumor-infiltrating lymphocyte (TIL) technology. Data developed in collaboration with AgonOx, Inc. showed PH-762 can improve the tumor cell killing activity of DP TILs two-fold.
Bolstered the balance sheet with two financings in Q1 2021 for gross proceeds of $21.7 million in additional capital.
Completed a $7.7 million registered direct offering of common stock priced at-the-market.
Completed a $14.0 million private placement of common stock and warrants priced at-the-market.
Upcoming Pipeline Milestones for 2021

Scheduled to present new study data regarding direct drug therapy with PH-762 at the AACR (Free AACR Whitepaper) Annual Meeting 2021.
Additional data publications on the Company’s pipeline programs, expected during Q2-Q3 2021.
Start of a first-in-human clinical study, on the use of PH-762 in adoptive cell therapy with TILs, namely, to enhance the therapeutic responses in cancer patients. The study, to be executed in collaboration with Agonox, Inc., is expected to be initiated in Q3 2021.
Start of a first-in-human clinical study on the use of PH-762 as directly administered drug in patients with advanced melanoma. The study is expected to be initiated in Q4 2021.
Financial Results

Cash Position

At December 31, 2020, the Company had cash of $14.2 million as compared with $6.9 million at December 31, 2019. Subsequent to the end of the fourth quarter of 2020, the Company raised an additional $21.7 million in gross proceeds through a registered direct offering and a private placement offering. The Company expects its current cash will be sufficient to fund currently planned operations to the second quarter of 2023.

Research and Development Expenses

Research and development expenses were approximately $4.4 million for the year ended December 31, 2020, compared to approximately $4.3 million for the year ended December 31, 2019. The increase is primarily due to an increase in the use of sponsored research organizations to support the development of the Company’s pipeline programs as compared to the prior year period.

General and Administrative Expenses

General and administrative expenses were approximately $4.4 million for the year ended December 31, 2020, compared to approximately $4.7 million for the year ended December 31, 2019. The decrease is primarily due to decreases in legal-related expenses and recruiting fees to support employee hiring activities as compared to the prior year period.

Net Loss

Net loss was $8.8 million, or $1.92 per share, for the year ended December 31, 2020, compared with $8.9 million, or $19.33 per share, for the year ended December 31, 2019. The decrease in net loss was primarily attributable to the decrease in general and administrative expenses, as described above. The change in net loss per share was primarily due to an increase in the number of shares outstanding as a result of our capital raise activities as compared to the prior year period.

On March 25, 2021 Neoleukin Therapeutics, Inc., "Neoleukin" (NASDAQ:NLTX), a biopharmaceutical company utilizing sophisticated computational methods to design de novo protein therapeutics, reported a corporate update and financial results for the year ended December 31, 2020 (Press release, Neoleukin Therapeutics, MAR 25, 2021, View Source [SID1234577146]).

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"Despite the challenges of the COVID-19 pandemic, our team has remained focused and committed to the advancement of our de novo technology platform, paving the way for future achievements this year and beyond," said Jonathan Drachman, M.D., Chief Executive Officer of Neoleukin. "In 2021, we look forward to advancing our first-in-human trials of NL-201 and expanding our de novo protein technology and pipeline."

Recent Updates

NL-201 Development

NL-201 is a computationally designed de novo protein that is a mimetic of natural cytokines IL-2 and IL-15. In the fourth quarter of 2020, Neoleukin submitted a Clinical Trial Notification ("CTN") for Phase 1 clinical testing of NL-201 in Australia, which is anticipated to be initiated at leading clinical research centers in the first half of 2021. In addition, in December 2020, Neoleukin announced submission of an Investigational New Drug ("IND") Application with the U.S. Food and Drug Administration ("FDA") to begin Phase 1 clinical testing of NL-201 in the U.S. The planned clinical trial of NL-201 in Australia and North America is expected to enroll patients with relapsed or refractory solid tumors. Patients will receive NL-201 as intravenous monotherapy to assess safety, pharmacokinetics, pharmacodynamics, and antitumor activity.

On January 7, 2021, Neoleukin received a clinical hold letter from the FDA related to its IND Application. The FDA requested that the company develop a new assay for methods-of-use testing and to demonstrate that this assay can more precisely measure the amount of NL-201 that is delivered to the patient when following the pharmacy preparation and administration instructions. The FDA also had additional requests not related to the clinical hold to be addressed by amendment of the IND. Neoleukin is working to address the FDA’s questions as quickly as possible and currently expects to resolve the clinical hold as well as to begin enrollment of patients in the first half of 2021.

In addition to the systemic trial, Neoleukin is planning a trial of NL-201 to test local administration in order to achieve higher drug concentrations in the tumor microenvironment. The company expects the local administration trial to begin by the end of 2021 and will provide further details as appropriate.

In November 2020, Neoleukin presented preclinical data on NL-201 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 35TH Anniversary Annual Meeting. These data highlighted the results of multiple experiments of NL-201 in combination with immunotherapies and demonstrate that NL-201 enhances activity of checkpoint inhibitors and tumor-targeting antibodies in preclinical models.

De Novo Protein Design for Coronavirus – NL-CVX1

In November 2020, Neoleukin announced publication in the journal Science of a manuscript describing novel molecules designed to treat or prevent infection by SARS-CoV-2, the virus that causes COVID-19. As reported, the optimized proteins act as decoys that bind to the SARS-CoV-2 spike protein with high affinity, preventing its association with the viral receptor hACE2 and blocking cellular entry. The lead molecule, NL-CVX1 (CTC-445.2d), was shown to prevent infection of multiple cell lines in vitro and, when administered intranasally, to prevent serious consequences of SARS-CoV-2 infection in hamsters. The rapid development of this targeted protein demonstrates the potential of the Neoleukin de novo protein design platform. Neoleukin is currently planning a first-in-human trial of NL-CVX1, and will continue to evaluate the program as the SARS-CoV-2 landscape evolves.

Other Research Updates

Neoleukin has multiple research projects underway evaluating the applications of de novo protein technology to develop agonists and antagonists of immune pathways. This includes development of potent and hyperstable inhibitors of inflammation that could be used to treat autoimmune conditions. Neoleukin currently plans to announce additional information about its pipeline program during the second half of 2021.

Summary of Financial Results

Cash Position: Cash and cash equivalents totaled $192.6 million as of December 31, 2020, compared to $143.1 million as of December 31, 2019. The increase was primarily driven by the completion of a public offering in July 2020 for net proceeds of $71.3 million. Based upon current internal infrastructure and pipeline initiatives, Neoleukin believes it has sufficient cash to fund operations into 2023.

R&D Expenses: Research and development expenses for the year ended December 31, 2020 were $24.3 million compared to $4.4 million for the year ended December 31, 2019. The increase in research and development expenses during the year ended December 31, 2020 was due to increased expenses incurred from IND-enabling activities related to our lead product candidate, NL-201, and in connection with the advancement of other Neoleukin technologies. Lower research and development costs during the year ended December 31, 2019 reflect the fact that prior to the merger between Aquinox Pharmaceuticals, Inc. ("Aquinox") and Neoleukin Therapeutics, Inc. ("Former Neoleukin") in August, 2019, all research and development activities with rosiptor had been suspended since June 2018.

G&A Expenses: General and administrative expenses for the year ended December 31, 2020 were $17.2 million compared to $18.8 million for the year ended December 31, 2019. The higher general and administrative expenses for the year ended December 31, 2019 as compared to the year ended December 31, 2020 were primarily due to severance costs and the recognition of stock-based compensation expense for certain options that vested as a result of the merger between Aquinox and Former Neoleukin in August 2019. The general and administrative expenses for the year ended December 31, 2020 reflect an increase in personnel related costs, facility-related costs, and professional service fees.

Acquired in-process R&D: The acquired in-process research and development expense of $47.7 million in 2019 arose from the merger between Aquinox and Former Neoleukin in 2019 and was expensed immediately in accordance with accounting standards.

Gain on Sale of Aquinox Canada: The gain relates to the sale of Aquinox Canada, a wholly owned subsidiary of the company, during the three months ended September 30, 2020. The gain of $7.8 million recognized is the total consideration of $8.2 million, less transaction costs of $0.4 million.

Net Loss: Net loss for the year ended December 31, 2020 was $33.3 million compared to a net loss of $69.4 million for the year ended December 31, 2019. The higher net loss in 2019 is primarily due to the acquired in-process research and development expense from the merger.

Conference Call Information

Neoleukin will host a conference call today to discuss 2020 financial results and provide a corporate update. Details as follows:

The archived audio webcast will be available on the Investor Relations section of the Neoleukin website approximately two hours after the event and will be available for replay for at least 30 days after the event.

About NL-201

NL-201 is a de novo receptor agonist of the IL-2 and IL-15 receptors, designed to expand cancer-fighting CD8 T cells and natural killer (NK) cells without any bias toward cells expressing the alpha receptor subunit (CD25). Previously presented preclinical data have demonstrated the ability of NL-201 to stimulate and expand CD8+ and NK cells at low doses with minimal impact on immunosuppressive regulatory T cells.

On March 25, 2021 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage precision medicine company transforming the drug discovery process by combining leading edge experimental and computational technologies, reported fourth quarter and full year 2020 financial results and operational highlights (Press release, Relay Therapeutics, MAR 25, 2021, View Source [SID1234577145]).

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"2020 was a transformational year for Relay Therapeutics. We successfully advanced multiple programs into the clinic, evolved our platform, expanded our strong team and completed a successful IPO," said Sanjiv Patel, M.D., president and chief executive officer. "While the last few years were spent validating our approach of combining leading edge experimental and computational techniques to create potentially life-saving therapies for patients, the next chapter will be about extending our platform leadership and delivering meaningful clinical results to support the success of our programs. 2021 will be a critical year of execution as we advance our mission of transforming drug discovery to address some of the most devastating diseases."

2020 Corporate Highlights

Initiated first-in-human clinical studies for RLY-1971 (SHP2 inhibitor) and RLY-4008 (FGFR2 inhibitor)
Progressed PI3Kα mutant selective program into late lead optimization
Continued to advance three additional precision oncology programs through preclinical development
Expanded research efforts into genetic diseases with two preclinical programs
Strengthened the balance sheet with $460 million gross proceeds raised in an initial public offering
Announced a worldwide license and collaboration agreement with Genentech, Inc., a member of the Roche Group, for the development and commercialization of RLY-1971
2021 Anticipated Milestones

Present preclinical data for RLY-4008 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021
Announce initial clinical data for RLY-4008 in the second half of 2021
Genentech to initiate RLY-1971 and GDC-6036 (KRAS G12C inhibitor) combination trial
Enter IND-enabling studies for PI3Kα mutant selective inhibitor and provide program update
Fourth Quarter and Full Year 2020 Financial Results

Cash, Cash Equivalents and Investments: As of December 31, 2020, cash, cash equivalents and investments totaled approximately $678.1 million, compared to $355.8 million as of December 31, 2019. The Company expects its current cash and cash equivalents, including the $75 million upfront payment received from Genentech in 2021, will be sufficient to fund its current operating plan into 2024.

R&D Expenses: Research and development expenses were $32.1 million for the fourth quarter of 2020, as compared to $22.4 million for the fourth quarter of 2019. This increase was primarily due to $7.4 million of increased employee related costs, including $5.7 million of additional share-based compensation expense, primarily due to increases in our stock price and increased headcount, and $2.3 million in increased clinical trial expenses. Research and development expenses were $99.9 million for the full year 2020, as compared to $70.3 million for the full year 2019.

G&A Expenses: General and administrative expenses were $15.5 million for the fourth quarter of 2020, as compared to $3.4 million for the fourth quarter of 2019. This increase was primarily due to $9.1 million of increased employee related costs, including $7.5 million of additional share-based compensation expense, primarily due to increases in our stock price and increased headcount, as well as $3.0 million of increased general expenses, primarily driven by public company related costs. General and administrative expenses were $38.6 million for the full year 2020, as compared to $13.7 million for the full year 2019.

Net Income/Loss: Net income was $35.3 million for the fourth quarter of 2020, as compared to a net loss of $23.9 million for the fourth quarter of 2019. Net loss was $52.4 million for the full year 2020, or a net loss per share of $5.40, as compared to a net loss of $75.3 million for the full year 2019, or a net loss per share of $21.82.

On March 25, 2021 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the fourth quarter and fiscal year ended December 31, 2020 (Press release, CymaBay Therapeutics, MAR 25, 2021, View Source [SID1234577144]).

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In the fourth quarter of 2020 and through early March 2021, CymaBay made significant progress reinitiating the development program for seladelpar in primary biliary cholangitis (PBC). With multiple clinical sites activated, patient recruitment is underway in RESPONSE, a global Phase 3 registrational study evaluating seladelpar in patients with PBC. In addition, we have also initiated ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional safety data to support registration.

Sujal Shah, President and CEO of CymaBay, stated, "One year ago today, our work towards finding novel treatments for patients with chronic, inflammatory and metabolic diseases was on hold as we completed the important work of ensuring patient safety before moving forward. Today we have four active clinical studies ongoing across three programs. We continue to make great progress towards restarting our seladelpar development program with RESPONSE as we execute on a focused strategy to complete late-stage development of seladelpar for patients with PBC. Results from our previous Phase 3 study in PBC, ENHANCE, presented at The Liver Meeting 2020, highlighted the anti-cholestatic, anti-inflammatory and anti-pruritic effects of seladelpar in patients with PBC that we believe support the potential for seladelpar to address key unmet needs for patients suffering from this disease. In addition to our core focus in PBC, we continue to evaluate seladelpar for other indications and are excited about our early-stage pipeline maturing in the coming months."

Recent Corporate Highlights

Initiated RESPONSE, a 52-week, placebo-controlled, randomized, global, Phase 3 registrational study evaluating the safety and efficacy of seladelpar in patients with PBC. This study will target enrolling 180 patients, who have an inadequate response to, or intolerance to, ursodeoxycholic acid, in a 2:1 randomization to oral, once daily seladelpar 10 mg or placebo. The primary outcome measure is the responder rate at 52 weeks. A responder is defined as a patient who achieves an alkaline phosphatase level < 1.67 times the upper limit of normal with at least a 15% decrease from baseline and has a normal level of total bilirubin. Additional key outcomes of efficacy will compare the rate of normalization of alkaline phosphatase at 52 weeks and the level of pruritus at 6-months for patients with moderate to severe pruritus at baseline assessed by a numerical rating scale recorded with an electronic diary.

Initiated ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional long-term safety data to support registration. The first subjects will consist of patients who have participated in CymaBay’s prior studies of seladelpar in PBC, including the patients who completed the open-label Phase 2 study and enrolled into the previous long-term study and ENHANCE. Patients who complete RESPONSE, and potentially other future PBC studies with seladelpar, will also have the opportunity to enroll in ASSURE.

Presented results from two separate studies of seladelpar in patients with PBC and NASH at The Liver Meeting 2020 as follows:

Oral, late-breaking presentation by Professor Gideon Hirschfield, MD, on November 16 highlighting results from ENHANCE, a Phase 3 study of seladelpar in patients with PBC
"Poster of Distinction" presentation by Dr. Stephen Harrison, MD, featuring results from a Phase 2 paired-liver biopsy study of seladelpar in patients with NASH

In November 2020, we announced a Phase 2a proof-of-pharmacology study to evaluate the potential for MBX-2982, a GPR119 agonist, to prevent hypoglycemia in patients with type 1 diabetes (T1D). The study is being conducted by the AdventHealth Translational Research Institute (TRI) in Orlando, Florida and fully funded by The Leona M. and Harry B. Helmsley Charitable Trust with CymaBay retaining full rights to MBX-2982. TRI commenced startup activities in early 2021 and topline results are currently anticipated in late 2021.

Initiated a single and multiple ascending dose study of CB-0406 in healthy subjects to establish its pharmacokinetics, safety and maximum tolerated dose. CB-0406 is a non-agonist ligand of PPARγ that attenuates the expression of inflammatory genes.

Held $146.3 million in cash, cash equivalents and short-term investments on December 31, 2020. We believe that cash and investments are sufficient to fund CymaBay’s current operating plan into mid-2022.

Due to the ongoing effects of the global coronavirus pandemic, CymaBay continues to conduct its operations remotely for all employees, which has allowed business activities to continue as seamlessly as possible. CymaBay continues to closely monitor pandemic developments and their associated risks to the business, including the restarting of its clinical development of seladelpar, and will continue to take actions available to mitigate them where possible. Further, all CymaBay’s actions will continue to be guided by a commitment to ensuring the health and safety of its employees as well as patients enrolled in its clinical studies.
Fourth Quarter and Year Ended December 31, 2020 Financial Results

Research and development expenses for the three and twelve months ended December 31, 2020 were $10.7 million and $35.9 million, respectively. This compared to R&D expenses of $20.9 million and $83.8 million for the three and twelve months ended December 31, 2019, respectively. Research and development expenses in the three and twelve months ended December 31, 2020 were lower than the corresponding periods in 2019 primarily due to a decline in clinical trial activities related to the termination of our Phase 3 PBC, Phase 2b NASH, and Phase 2 PSC clinical trials, and other studies, after the seladelpar development program was placed on hold from November 2019 through July 2020. Clinical development of seladelpar in PBC was resumed in late 2020, and these clinical expense reductions were offset in part by increases in clinical trial costs associated with the commencement of RESPONSE and ASSURE, our two new global late-stage clinical trials in PBC.

General and administrative expenses for the three and twelve months ended December 31, 2020 were $5.2 million and $17.4 million, respectively. This compared to $4.5 million and $19.2 million for the three and twelve months ended December 31, 2019, respectively. General and administrative expenses in the year 2020 were lower than in 2019 due to lower employee compensation costs following the completion of a reduction-in-force plan in late 2019. General and administrative expenses in the three months ended December 31, 2020 were higher than the corresponding period in 2019 due to higher employee compensation associated with the hiring of additional personnel upon resumption of development of seladelpar in the second half of 2020.

Net loss for the three and twelve months ended December 31, 2020 was $15.8 million, or ($0.23) per diluted share, and $51.0 million, or ($0.74) per diluted share, respectively. This compared to net loss of $29.4 million, or ($0.43) per diluted share, and $102.8 million, or ($1.53) per diluted share, in the three and twelve months ended December 31, 2019, respectively. Net loss was lower largely due to decreases in clinical operating expenses which resulted from the temporary clinical hold placed on the seladelpar development program. With development of seladelpar restarted during the second half of 2020, we expect our operating expenses to increase in 2021 as we continue to execute on our clinical development plans.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss fourth quarter and fiscal year end 2020 financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13715944. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

On March 25, 2021 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported that Shehnaaz Suliman, M.D., MBA, M.Phil., president and chief operating officer of Alector, will participate in a fireside chat at Stifel’s 3rd Annual CNS Day on Thursday, April 1, 2021, at 9:30 a.m. ET (Press release, Alector, MAR 25, 2021, View Source [SID1234577143]).

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A live webcast of the fireside chat will be available on the "Events & Presentations" page within the Investors section of the Alector website at View Source A replay will be available on the Alector website for 30 days following the event.