On February 23, 2022 EpicentRx, Inc., a clinical stage drug and device company, in collaboration with the Texas Children’s Cancer Center in Houston, reported the start of a Phase 1 study to examine the safety and benefit of its lead small molecule, RRx-001, plus irinotecan and temozolomide for pediatric patients with recurrent or progressive malignant solid and central nervous system tumors (Press release, EpicentRx, FEB 23, 2022, View Source [SID1234608917]). The study, PIRATE (NCT04525014) was conceived and developed by doctors Holly Lindsay and Patricia Baxter at the Texas Children’s Cancer Center and will enroll patients 1 to 21 years of age with recurrent or progressive malignant primary brain or spinal cord tumors and solid tumors, excluding lymphomas.

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The genesis for the PIRATE trial was based on several preceding clinical trials which have established potential evidence that RRx-001 increases chemotherapy delivery and uptake in tumors. A Phase 1 clinical trial called G-FORCE (NCT02871843) and a Phase 1/2 clinical trial called BRAINSTORM (NCT02215512) demonstrated the safety of RRx-001 as well as potential evidence of clinical benefit in adult patients with glioblastoma or GBM and with brain metastases, respectively.

EpicentRx and Texas Children’s Cancer Center studying the safety and benefit of EpicentRx’s lead small molecule, RRx-001

Cancer cells are programmed to travel to different tissues in the body, where they establish a barrier to prevent drug delivery. One of the hypotheses of this trial is that RRx-001 may "normalize" the tumor microenvironment, making the tumor vasculature more efficient for the delivery of irinotecan and temozolomide so that their efficacy is increased while overstimulating immune cells like macrophages to go on the attack through its antagonism of the "do not eat me" CD47 signal. Another hypothesis is that RRx-001 will protect normal tissues, but not tumors, from the toxicities of irinotecan and temozolomide.

"We are excited to launch this study with RRx-001 and to establish its role as a potential treatment for pediatric CNS tumors," said Tony R. Reid, M.D., Ph.D., Chief Executive Officer of EpicentRx. "This trial would not have been possible without the enthusiastic support of Texas Children’s Cancer Center. As clinical trials in adult CNS tumors suggest that RRx-001 may provide benefit in a pediatric population, our collaboration with them is a testament to the commitment of this company to follow the data wherever it leads."

"There is compelling preclinical and clinical rationale for the safety and potential efficacy of RRx-001 in CNS tumors," said Dr. Holly Lindsay, M.D., Assistant Professor, Department of Pediatrics, Section of Hematology-Oncology, Baylor College of Medicine and lead Investigator. "The incidence of CNS tumors in children is particularly difficult to treat, and we expect that the PIRATE study will generate valuable data to help inform future treatment decisions."

For more information about PIRATE please visit View Source

On February 23, 2022 Theravance Biopharma, Inc. ("Theravance Biopharma" or the "Company") (NASDAQ: TBPH) reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Theravance, FEB 23, 2022, View Source [SID1234608916]).

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"While COVID continues to impact communities around the country and health systems are strained, YUPELRI saw the highest quarter of net sales since its launch," said Rick E Winningham, Chief Executive Officer. "I am proud of our team and their ability to find a way to service the COPD community during this challenging time. YUPELRI once a day dosing is proving increasingly important in helping over-burdened healthcare professionals provide relief to their COPD patients. In addition, GSK’s TRELEGY sales reached their highest levels since launch driven by growth in the asthma indication."

"Strong performance by YUPELRI in Q4 and into 2022 will enable us to invest in our inhaled JAK inhibitor respiratory pipeline," continued Winningham. "Through an active business development program, we will continue to unlock value from our non-core asset portfolio throughout 2022. We remain on target to become sustainably cash-flow positive beginning the second half of this year."

Year-end Highlights

YUPELRI (revefenacin) inhalation solution, the first and only once-daily, nebulized bronchodilator approved in the US for the maintenance treatment of patients with chronic obstructive pulmonary disease (COPD), continued to increase market share and achieved year-over-year net sales growth of 13%; its share of the long-acting nebulized COPD market increased to 23.2% through October 2021 (up from 18.2% in October 2020).
On January 10, 2022, the Company announced the enrollment of the first patient in a Phase 4 study of YUPELRI (read more about the announcement here).
Economic Interest

TRELEGY (first once-daily single inhaler triple therapy for COPD and asthma), in which the Company holds an economic interest, posted fourth quarter 2021 global net sales of $479 million (up from $315 million, in fourth quarter of 2020) achieving quarterly year-over-year sales growth of 52%, and full year 2021 global net sales of $1,674 million (up from $1,058 million, in 2020), achieving year-over-year sales growth of 58%; Theravance Biopharma is entitled to tiered payments equal to approximately 5.5% to 8.5% of TRELEGY global net sales.3
Fourth Quarter and Full Year Financial Results

Revenue: Total revenue for the fourth quarter of 2021 was $14.9 million, comprised of non-cash collaboration revenue of $2.8 million primarily attributed to our global collaboration with Janssen and $12.1 million in Viatris collaboration revenue. Total revenue for the fourth quarter represents a $3.8 million decrease over the same period in 2020. Full year 2021 revenue was $55.3 million, comprised of non-cash collaboration revenue of $11.5 million primarily attributed to our global collaboration with Janssen and $43.8 million in Viatris collaboration revenue.
YUPELRI: The Viatris collaboration revenue of $12.1 million for the fourth quarter represents amounts receivable from Viatris and is comprised of the Company’s 35% share of net sales of YUPELRI as well as its proportionate amount of the total shared costs incurred by the two companies. The non-shared YUPELRI costs incurred by Theravance Biopharma are recorded within operating expenses. While Viatris records the total net sales of YUPELRI within its financial statements, our implied 35% share of net sales of YUPELRI for the fourth quarter of 2021 was $15.3 million.
Research and Development (R&D) Expenses: R&D expenses for the fourth quarter of 2021 were $31.2 million, compared to $65.2 million in the same period in 2020. Fourth quarter R&D expenses included total non-cash share-based compensation of $3.4 million. In terms of Financial Guidance, full-year 2021 R&D expenses excluding non-cash share-based compensation and one-time restructuring costs were $168.0 million vs. Guidance of $170 million to $180 million.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses for the fourth quarter of 2021 were $21.5 million, compared to $30.1 million in the same period in 2020. Fourth quarter SG&A expenses included total non-cash share-based compensation of $5.1 million. In terms of Financial Guidance, full-year 2021 SG&A expenses excluding non-cash share-based compensation and one-time restructuring costs were $71.2 million vs. Guidance of $65 million to $75 million.
Restructuring and Related Expenses: Restructuring expenses for the fourth quarter of 2021 were $18.4 million and primarily comprised of severance costs, termination-related benefits, one-time retention costs, and share-based compensation expense. Full year 2021 restructuring and related expenses were $20.1 million. Cash restructuring expenses were $9.8 million for the fourth quarter of 2021 and $11.5 million for full-year 2021; non-cash restructuring expenses were $8.6 million for the fourth quarter of 2021 and for the full-year 2021.
Operating Loss: Operating loss for the fourth quarter of 2021 was $56.2 million compared to $76.5 million in the same period of 2020. Full year 2021 operating loss was $257.8 million, or $195.7 million excluding share-based compensation expense compared to $297.8 million, or $234.8 million excluding share-based compensation expense in 2020.
Cash Position: Cash, cash equivalents and marketable securities totaled $173.5 million as of December 31, 2021.
2022 Financial Guidance

Operating Expenses (excluding share-based compensation and one-time restructuring costs): The Company expects full year 2022 R&D expense of $45 million to $55 million and SG&A expense of $35 million to $45 million.
The Company expects to be sustainably cash-flow positive beginning 2H 2022.
Conference Call and Live Webcast Today at 5:00 pm ET

Theravance Biopharma will hold a conference call and live webcast accompanied by slides today at 5:00 pm ET / 2:00 pm PT / 10:00 pm GMT. To participate in the live call by telephone, please dial (855) 296-9648 from the US or (920) 663-6266 for international callers, using the confirmation code 7456139. Those interested in listening to the conference call live via the internet may do so by visiting Theravance Biopharma’s website at www.theravance.com, under the Investors section, Presentations and Events.

A replay of the conference call will be available on Theravance Biopharma’s website for 30 days through March 25, 2022. An audio replay will also be available through 8:00 pm ET on March 2, 2022 by dialing (855) 859-2056 from the US, or (404) 537-3406 for international callers, and then entering confirmation code 7456139.

On February 23, 2022 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, metabolic, autoimmune and other major diseases, reported that the first patient has been successfully dosed in the phase 1, first-in-human clinical trial of its proprietary CD73 antibody (development code: IBI325) in patients with advanced solid tumor (Press release, Innovent Biologics, FEB 23, 2022, View Source [SID1234608915]).

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The open-label, dose escalation phase 1a/1b study (NCT05119998) will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of IBI325 as a single agent and in combination with sintilimab in patients with advanced solid tumors. The recommended dose for further clinical studies will also be determined.

CD73 is an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine. Adenosine binds adenosine A2A andA2B receptors on immune cells and inhibits immune responses directed against tumors. IBI325, as a differentiated, humanized antibody against CD73, is expected to relief the immunosuppressive tumor micro-environment and to work in combination with other cancer therapies such as PD-1 or PD-L1 antibodies. A complete inhibition to CD73 both soluble and on cell surface has been well demonstrated in preclinical studies. And the anti-tumor activities of IBI325 were observed in various tumor models.

Professor Jinming Yu, member of the Chinese Academy of Engineering and president of Shandong Cancer Hospital & Institute, stated, "CD73 is one of the most important targets in the adenosine pathway. Preclinical studies and preliminary clinical data from drugs in the same class has demonstrated the modulation effect of anti-CD73 in immune microenvironment. This new clinical trial will further evaluate the efficacy and safety profile of IBI325 alone and in combination with anti-PD-1 antibody and other immune checkpoint inhibitors in advanced tumors."

"Our preclinical data suggested that IBI325 could completely inhibit the activity of CD73 molecule with no ‘hook effect’ compared with the other candidates in the same class. An outstanding synergistic effect of combination with IBI325 and sintilimab was also demonstrated," said Dr. Zhou hui, senior VP of Innovent, "IBI325 has the potential to become the best-in-class molecule. The new combination therapy of IBI325 and sintilimab would potentially address the existing unmet medical needs left by current immunotherapies. We are excited that IBI325 has come to the clinical development stage, and are committed to develop new safe and efficacious treatment option for patients with advanced tumor."

About IBI325

IBI325 is a differentiated, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine. Adenosine binds adenosine A2A andA2B receptors on immune cells and inhibits immune responses directed against tumors. IBI325 is expected to relief the immunosuppressive tumor micro-environment and to work in combination with other cancer therapies such as PD-1 or PD-L1 antibodies. A complete inhibition to CD73 both soluble and on cell surface has been well demonstrated in preclinical studies. And the anti-tumor activities of IBI325 were observed in various tumor models.

On February 23, 2022 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported that its management team will present at the Cowen 42nd Annual Health Care Conference on Monday, March 7, 2022 at 2:10 p.m. Eastern Time (Press release, Personalis, FEB 23, 2022, View Source [SID1234608914]).

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On February 23, 2022 Quanterix Corporation (NASDAQ: QTRX), a company digitizing biomarker analysis with the goal of advancing the science of precision health, reported that it will release its financial results for fourth quarter 2021 before the start of trading on Tuesday, March 1, 2022 (Press release, Quanterix, FEB 23, 2022, View Source [SID1234608913]). Company management will host a conference call at 8:30 a.m. EST to discuss Quanterix’ financial results and provide a business update. The call will be hosted by Kevin Hrusovsky, Chairman and Chief Executive Officer, Quanterix.

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Individuals interested in listening to the conference call may do so by dialing (833) 686-9351 for domestic callers, or (612) 979-9890 for international callers. Please reference the following Conference ID: 5230769. A live webcast will also be available at: View Source You may also access the live webcast by visiting the News & Events page within the Investors section of the Quanterix website at www.quanterix.com. The webcast will be available on the Company’s website for one year following completion of the call.