On March 1, 2022 Xilio Therapeutics, Inc. (Nasdaq: XLO), a biotechnology company developing tumor-selective immuno-oncology therapies for people living with cancer, reported pipeline and business progress and reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Xilio Therapeutics, MAR 1, 2022, View Source [SID1234609247]).

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"Xilio made significant pipeline and business progress in 2021, becoming a clinical-stage organization and transitioning to a publicly traded company through the successful completion of our IPO," said René Russo, Pharm.D., president and chief executive officer of Xilio. "As we look to 2022, I am excited about the many opportunities ahead of us, including anticipated initial clinical data readouts for XTX101, our tumor-selective anti-CTLA-4 monoclonal antibody, and XTX202, our tumor-selective IL-2. In addition, we plan to submit an IND for XTX301, our tumor-selective IL-12, in the second half of the year and continue to generate new research programs by leveraging our robust platform. With our exceptional team and strong financial position, we believe we have the resources in place to advance our vision of bringing transformative immuno-oncology therapies to people living with cancer."

Pipeline and Business Progress

Cytokine Programs

●Xilio continues to advance enrollment in the Phase 1 portion of its Phase 1/2 clinical trial evaluating XTX202 for the treatment of patients with solid tumors. XTX202, a modified form of interleukin-2 (IL-2), is designed to localize activity in the tumor microenvironment, with the goal of overcoming the known tolerability challenges of existing IL-2 therapies while achieving enhanced anti-tumor activity. Xilio expects to report preliminary data from the Phase 1 portion of the trial in the second half of 2022.

●Xilio continues to progress preclinical studies for XTX301, a tumor-selective IL-12. The company plans to submit an investigational new drug application (IND) for XTX301 as a potential treatment for patients with solid tumors in the second half of 2022.

Checkpoint Inhibitor Program

●Xilio continues to advance enrollment in the Phase 1 portion of its Phase 1/2 clinical trial evaluating XTX101, a tumor-selective anti-CTLA-4 monoclonal antibody, as a monotherapy and in combination with pembrolizumab, an anti-PD-1, for the treatment of patients with advanced solid tumors. Xilio expects to report preliminary data from the monotherapy cohort of the Phase 1 portion of the trial in the middle of 2022 and preliminary data from the combination cohort of the Phase 1 portion of the trial in the second half of 2022.

Recent Business Highlights

●In January 2022, Xilio appointed Yuan Xu, Ph.D., to its board of directors.

Fourth Quarter and Full Year 2021 Financial Results

●Cash Position: Cash and cash equivalents were $198.1 million as of December 31, 2021, compared to $19.2 million as of December 31, 2020. The increase was primarily related to $144.9 million in net proceeds received from convertible preferred stock financings in the first quarter of 2021 and $116.4 million in net proceeds received from Xilio’s initial public offering in the fourth quarter of 2021. Net cash proceeds from financings were partially offset by operating uses of cash for the year ended December 31, 2021.

●Research & Development (R&D) Expenses: R&D expenses were $11.4 million for the quarter ended December 31, 2021, compared to $17.7 million for the quarter ended December 31, 2020. R&D expenses were $51.2 million for the year ended December 31, 2021, compared to $43.9 million for the year ended December 31, 2020. The year-over-year increase was primarily driven by higher personnel-related costs due to increased headcount and expenses associated with R&D programs.

●General & Administrative (G&A) Expenses: G&A expenses were $8.2 million for the quarter ended December 31, 2021, compared to $2.9 million for the quarter ended December 30, 2020. G&A expenses were $23.9 million for the year ended December 31, 2021, compared to $10.7 million for the year ended December 31, 2020. The year-over-year increase was primarily driven by higher personnel-related costs due to increased headcount and professional and consulting fees related to transitioning to and operating as a publicly traded company.

●Net Loss: Net loss was $19.7 million for the quarter ended December 31, 2021 and $75.8 million for the year ended December 31, 2021.

Financial Guidance

Xilio continues to believe that its existing cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into 2024.

On March 1, 2022 Avid Bioservices, Inc. (NASDAQ:CDMO), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality services to biotechnology and pharmaceutical companies, reported that it will report financial results for the third quarter of fiscal year 2022 on March 8, 2022 after market close and will host a conference call and webcast at 1:30 PM Pacific Time (4:30 PM Eastern Time) (Press release, Avid Bioservices, MAR 1, 2022, View Source [SID1234609273]). Members of Avid’s senior management will discuss financial results for the third quarter and review recent corporate developments.

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To listen to the live webcast, or access the archived webcast, please visit: View Source

To listen to the conference call, please dial (877) 312-5443 or (253) 237-1126 and request the Avid Bioservices call.

On March 1, 2022 NextRNA Therapeutics, a biotechnology company unlocking the potential of non-coding RNAs to develop transformative therapeutics, reported its launch with $9.3 million in seed financing and a $46.8 million Series A led by Cobro Ventures and Lightchain Capital, with additional participation from Circle Alternative Investments, Evans Capital, Jefferies, Rivas Capital, and Willett Advisors (Press release, NextRNA Therapeutics, MAR 1, 2022, View Source [SID1234609289]). Proceeds will be used by the company to augment its target and drug discovery engine, expand its pipeline, and advance lead programs.

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The vast majority of DNA is copied into RNAs that do not make proteins. These RNA molecules, called "non-coding RNAs," play essential roles in cells by interacting with and modulating the activities of proteins. These interactions can drive pathogenic processes in multiple disease areas, including oncology, immunology, and neurology. Non-coding RNAs, in particular long non-coding RNAs, and their interacting proteins therefore represent a vast and untapped class of novel therapeutic targets.

"NextRNA is uniquely positioned to be the leader in non-coding RNA-directed medicines," said Dominique Verhelle, Ph.D., MBA, co-founder, chief scientific officer, and interim chief executive officer, NextRNA. "We have established a robust target and drug discovery engine to systematically identify disease-relevant long non-coding RNAs and their interacting proteins. By developing selective small molecules to drug these interactions, we plan to translate discoveries of non-coding RNA targets into a robust pipeline of transformative therapies across multiple disease areas."

NextRNA was established based on pioneering work by Carl Novina, M.D., Ph.D., at Dana-Farber Cancer Institute. "By understanding the interactions between long non-coding RNAs and specific proteins, we can decode the function of long non-coding RNAs and apply it to create medicines," said Dr. Novina, co-founder of NextRNA.

Since its founding in January of 2021, NextRNA has established its initial team, built out the target and drug discovery engine, and advanced two small molecule programs in oncology and immunology.

"NextRNA is at the forefront of innovation in the field of non-coding RNAs," said Todd Kaloudis, Managing Director at Cobro Ventures. "There is growing excitement around the potential of non-coding RNA-directed medicines, and we are pleased to have such an experienced team focused on NextRNA’s vision of bringing transformative therapies to patients."

On March 1, 2022 – Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported financial results for the fourth quarter and full-year ended December 31, 2021 and recent business updates related to pre-commercialization activities ahead of a potential first-in-class U.S. launch for vadadustat, Akebia’s investigational oral therapeutic for the treatment of anemia due to chronic kidney disease (CKD) (Press release, Akebia, MAR 1, 2022, View Source [SID1234609307]). Vadadustat is currently under review by the U.S. Food and Drug Administration (FDA) with a scheduled Prescription Drug User Fee Act (PDUFA) date of March 29, 2022.

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"The PDUFA date for vadadustat is fast approaching. We recognize how transformational the potential approval would be for Akebia and, true to our purpose as a company, our team continues to work tirelessly to deliver a new oral therapeutic option for the patients we serve," said John P. Butler, Chief Executive Officer of Akebia. "We are prepared for what we believe will be a significant catalyst for the company marked by launching a potential first-in-class oral therapeutic for people living with anemia due to CKD, subject to regulatory approval."

Last month Akebia and Vifor Pharma Group (Vifor Pharma) amended and restated the terms of their license agreement, which provides important access to up to 60% of U.S. dialysis patients through existing Vifor Pharma relationships, supporting a successful commercial launch of vadadustat, if approved. Vifor Pharma completed a $20 million equity investment in Akebia, and will pay Akebia a $25 million upfront payment and contribute an initial $40 million in refundable working capital to partially fund launch supply. The companies also defined profit share economics of potential vadadustat revenue.

"Many of our on-going pre-commercialization activities, including amending the terms of our relationship with Vifor Pharma, are aimed at ensuring patient access for vadadustat," said Dell Faulkingham, Chief Commercial Officer of Akebia. "If approved, we will immediately initiate the process to secure reimbursement for vadadustat under the Transitional Drug Add-on Payment Adjustment (TDAPA) period for dialysis organizations, which we expect will take approximately six months. We believe TDAPA designation for vadadustat will be an important driver for adoption within U.S. dialysis organizations, if approved."

Akebia continues to optimize its sales, marketing, and payor strategies to support Auryxia (ferric citrate). Akebia ended 2021 with notable Auryxia net product revenue growth and the strongest quarter of net product revenue to date while the phosphate binder market declined by 8.1% year-over-year in the U.S., due in part to the impact of COVID-19 on kidney disease patients. Akebia achieved $142.2 million in net product revenue in 2021 due to improved commercial contracts and payor mix.

"Both the 2021 and our expected 2022 Auryxia revenue growth is due to our team’s commitment to patients and healthcare providers, even against the backdrop of the COVID-19 pandemic," added Dell Faulkingham. "Our renal focused field team has proven its ability to successfully engage with the kidney community and thoughtfully outline our value proposition. This expertise establishes a strong foundation from which to launch vadadustat, if approved."

Fourth Quarter and Full-Year 2021 Financial Results

Revenues: Total revenue was $59.6 million for the fourth quarter of 2021 compared to $56.7 million for the fourth quarter of 2020, and $213.6 million for the full-year 2021 compared to $295.3 million for the full-year 2020.

Collaboration revenue was $17.5 million for the fourth quarter of 2021 compared to $22.1 million for the fourth quarter of 2020, and $71.4 million for the full-year 2021 compared with $166.4 million for the full-year 2020. The decrease in both periods compared to the same periods in 2020 was primarily due to lower collaboration revenue from Otsuka Pharmaceuticals Co. Ltd (Otsuka) driven by lower development costs incurred subject to cost share provisions under both the Otsuka collaboration agreement for the U.S. and the Otsuka collaboration agreement for certain territories outside the U.S. as Akebia successfully completed the INNO2VATE and PRO2TECT global Phase 3 clinical programs in 2020 and is currently engaged in close-out activities with respect to the programs.
Net product revenue was $42.1 million for the fourth quarter of 2021 compared with $34.6 million for the fourth quarter of 2020, an increase of approximately 22 percent. Net product revenue was $142.2 million for the full-year 2021 compared to $128.9 million for the full-year 2020, an increase of approximately 10 percent.
COGS: Cost of goods sold was $50.4 million for the fourth quarter of 2021 compared to $63.2 million for the fourth quarter of 2020. Cost of goods sold was $153.4 million for the full-year 2021, compared with $295.9 million for the full-year 2020. Cost of goods sold includes a non-cash charge related to excess purchase commitments of $18.0 million and $33.4 million, for the fourth quarter and full year 2021, respectively.

R&D Expenses: Research and development expenses were $29.6 million for the fourth quarter of 2021 compared to $37.6 million for the fourth quarter of 2020, and $147.9 million for the full-year 2021 compared to $218.5 million for the full-year 2020. Fourth quarter 2021 expenses included a one-time credit of $8.6 million representing a reimbursement from Vifor Pharma following the sale of the Priority Review Voucher (PRV), which proceeds were subsequently paid to Otsuka as reimbursement for their contribution to purchase the PRV.

SG&A Expenses: Selling, general and administrative expenses were $44.8 million for the fourth quarter of 2021 compared to $40.3 million for the fourth quarter of 2020, and $174.2 million for the full-year 2021 compared to $153.9 million for the full-year 2020. The increase for the full year 2021 was primarily due to higher marketing expenses, increased headcount-related costs, and one-time legal costs.

Net Loss: Net loss was $70.7 million for the fourth quarter of 2021 compared to $87.0 million for the fourth quarter of 2020, and $282.8 million for the full-year 2021 compared to $383.5 million for the full-year 2020. The decrease in net loss for the full-year 2021 compared to the prior year was due primarily to higher product revenues, lower cost of goods sold and lower operating expenses, partially offset by lower collaboration revenue.

Cash Position: Cash and cash equivalents as of December 31, 2021 were $149.8 million. Akebia believes that its cash resources will be sufficient to fund its current operating plan through at least the next twelve months. Akebia’s base operating plan assumes a timely regulatory approval of vadadustat for the treatment of anemia due to CKD in dialysis dependent patients, as well as milestones and product revenues as an important source of funding of our cash runway.

On March 1, 2022 PMV Pharmaceuticals, Inc. (Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53, reported financial results for the fourth quarter and full year ended December 31, 2021 and provided corporate highlights (Press release, PMV Pharma, MAR 1, 2022, View Source [SID1234609336]).

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"We expect that 2022 will be another productive year for PMV Pharma, following accomplishments across multiple fronts in 2021," said David Mack, Ph.D., President and Chief Executive Officer. "We are encouraged by the steady progress in the development of our lead candidate PC14586, an investigational small molecule p53 Y220C reactivator, and look forward to reporting initial data from our ongoing Phase 1/2 trial in the first half of 2022. Our strong leadership and balance sheet position us to further advance our discovery pipeline of small molecule, tumor-agnostic precision medicine products that specifically target p53 mutants and targets where wild-type p53 is silenced."

Fourth Quarter 2021 and Corporate Highlights:

Patient enrollment in the Phase 1 portion of the Phase 1/2 clinical trial of PC14586 continues in line with the Company’s expectations. The Phase 1 dose escalation is assessing the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of PC14586 in patients with advanced solid tumors that have a p53 Y220C mutation (NCT04585750). The Company plans to disclose initial results from this study in the first half of 2022.

In addition to continuing work on its p53 mutant programs, PMV expands its pipeline by advancing WIP1 (Wild-Type p53-Induced Phosphatase) inhibitor program, into lead optimization. WIP1 is a phosphatase that negatively regulates wild-type p53 as well as other proteins involved in the DNA damage response pathway.

Promotions of Binh Vu Ph.D. to Senior Vice President, Discovery Research and CMC and Melissa Dumble Ph.D. to Senior Vice President, Preclinical Development and Translational Science. Dr. Vu joined PMV Pharma in 2013 as its first employee and has been an integral part of the company over the past 8 years. He will continue to lead our discovery research and CMC activities. Dr. Dumble joined PMV Pharma in 2017 and has played an important role in developing our pipeline. She will continue to lead our preclinical development and translational science activities.

Appointment of Kirsten Flowers to our Board of Directors. Ms. Flowers is the Chief Commercial Officer of Kura Oncology, a clinical-stage precision oncology company, who has extensive commercial experience leading top-performing oncology product launches at large pharmaceutical and biotechnology organizations. Prior to Kura Oncology, she served as Senior Vice President, Commercial Operations at Array Biopharma, where she built and led the commercial organization that delivered the successful launch of Braftovi + Mektovi for patients with BRAF-mutant melanoma.

Previously, she held various commercial leadership roles at Pfizer, including serving as the U.S. commercial lead for the launch of the blockbuster drug IBRANCE in breast cancer and for the launch of INLYTA in renal cell carcinoma.

Fourth Quarter 2021 Financial Results

PMV Pharma ended the fourth quarter with $314.1 million in cash, cash equivalents, and marketable securities, compared to $361.4 million as of December 31, 2020. Net cash used in operations was $46.6 million for the twelve months ended December 31, 2021 compared to $32.7 million for the twelve months ended December 31, 2020.

Net loss for the year ended December 31, 2021 was $57.8 million compared to $34.4 million for the year ended December 31, 2020.

Research and development (R&D) expenses were $36.5 million for the year ended December 31, 2021 compared to $23.9 million for the year ended December 31, 2020. The increase in R&D expenses was primarily related to increased headcount and clinical expenses related to advancing research on PC14586, the Company’s lead drug candidate.

General and administrative (G&A) expenses were $21.8 million for the year ended December 31, 2021, compared to $11.0 million for the year ended December 31, 2020. The increase in G&A expenses was primarily due to expanding the infrastructure necessary for operating as a public company.

About PC14586

PC14586 is a first-in-class, small molecule, p53 reactivator designed to selectively bind to the crevice present in the p53 Y220C mutant protein, hence, restoring the wild-type, or normal, p53 protein structure and tumor suppressing function. PC14586 is being developed for the treatment of patients with locally advanced or metastatic solid tumors that have a p53 Y220C mutation.