On March 10, 2022 TRACON Pharmaceuticals (Nasdaq: TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the United States, reported that the first patient has been dosed following the approval of the amended ENVASARC protocol by the U.S. Food and Drug Administration ("FDA") (Press release, Tracon Pharmaceuticals, MAR 10, 2022, View Source [SID1234609854]).

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In December 2021, based on the highly tolerable safety profile and the significantly higher objective response rate (ORR) observed in lower weight patients in ENVASARC, the IDMC recommended increasing the dose of envafolimab to 600 mg every three weeks (Q3W), which is double the original envafolimab dose of 300 mg Q3W. Given the robust activity demonstrated by higher doses of envafolimab in previously completed studies, including in the pivotal trial in MSI-H/dMMR cancer that was the basis for approval of envafolimab in China, TRACON submitted the amended ENVASARC protocol in January that was cleared by the FDA in February. Patient dosing is now underway at this 600 mg Q3W dose.

"We are pleased to have initiated envafolimab dosing at 600 mg following FDA approval of the amended ENVASARC protocol," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "Given the ENVASARC data to date, particularly the highly tolerable safety profile and response rate in lower weight patients, we believe a doubling of the dose will be well tolerated and result in higher envafolimab exposures, thereby optimizing envafolimab’s efficacy for the largest number of sarcoma patients. The new envafolimab dose is higher than the dose that produced the 45% ORR in MSI-H/dMMR cancer that was the basis for approval in China. We look forward to the interim ENVASARC efficacy data review by the IDMC which we expect will occur in the second half of 2022."

About Envafolimab

Envafolimab (KN035), a single-domain antibody against PD-L1 invented by Alphamab Oncology, is the first subcutaneously injected PD-(L)1 inhibitor approved by the Chinese NMPA in November 2021 in adult patients with MSI-H/dMMR advanced solid tumors who failed systemic treatment and have no satisfactory alternative treatment options. In December 2019, Alphamab Oncology, 3D Medicines and TRACON entered into a collaboration whereby TRACON has the right to develop and commercialize envafolimab in soft tissue sarcoma in North America. Envafolimab is currently being studied in the pivotal ENVASARC Phase 2 trial in the United States sponsored by TRACON and a Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multicenter, open label, randomized, non-comparative, parallel cohort study at approximately 25 top cancer centers in the United States that began dosing in December 2020. TRACON expects the trial to enroll more than 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into a cohort of treatment with single agent envafolimab at 600 mg every three weeks and 80 patients enrolled into a cohort of treatment with envafolimab at 600 mg every three weeks with Yervoy. The primary endpoint is overall response rate by central review with duration of response a key secondary endpoint.

On March 10, 2022 Scenic Biotech BV ("Scenic"), a pioneer in the discovery of genetic modifiers to enable the development of disease modifying therapeutics for rare genetic disorders and other devastating illnesses, reported it has closed a Series A financing of ~$31 million (€28 million) (Press release, Scenic Biotech, MAR 10, 2022, View Source [SID1234609853]).

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The funds will be used to accelerate Scenic’s transition into a development stage company, advancing its in-house pipeline and supporting the creation of new programs based on its Cell-Seq genetic modifier target discovery platform including new industry collaborations.

The investment round was co-led by seasoned European investors, Eir Ventures, a Nordic Life Science venture fund, Switzerland-based BioMedPartners and Luxemburg-based Vesalius Biocapital. Existing Dutch and UK investors Inkef Capital, BioGeneration Ventures and Oxford Science Enterprises participated, together with Scenic’s founders and management.

Scenic will now advance its lead QPCTL small molecule immuno-oncology program into the clinic. In addition, by leveraging its Cel-Seq discovery platform, Scenic has generated a pipeline of disease modifying therapeutics to treat devastating inherited rare diseases. Three of these programs, which are based on druggable genetic modifiers, are being progressed towards IND enabling studies. The first program is centered on a small molecule to treat Niemann Pick Type C (NP-C), a rare lipid storage disorder that affects lipid metabolism, or the way fats, lipids, and cholesterol are transported in human cells. The second program is for Barth syndrome, an inherited mitochondrial disorder caused by mutations in the gene encoding Tafazzin. Barth syndrome is characterized by defects in cardiolipin, a critical component of the inner mitochondrial membrane. And the third program is to treat a severe heritable metabolic syndrome.

Alongside its in-house pipeline, Scenic has a multi-year, multi-indication strategic collaboration with Genentech, a member of the Roche Group, to discover, develop and commercialize novel therapeutics that target genetic modifiers. The collaboration has been expanded twice and now includes six scientific areas.

Following the Series A investment, Stephan Christgau PhD, General Partner Eir Ventures, Michael Wacker PhD, General Partner BioMedPartners and Stephane Verdood MSc, MBA, Managing Partner Vesalius Biocapital will join Scenic’s board of directors.

Commenting on behalf of the investors, Stephan Christgau said, "Scenic Biotech has the potential to become one of Europe’s most exciting biotech companies. It is a pioneer in the ground-breaking and promising new field of genetic modifiers. We are impressed by the power of its Cell-Seq platform and how it’s fueling a portfolio of in-house and partnered programs across multiple therapeutic areas. This financing will enable Scenic to continue to develop its platform and move its lead program into human clinical trials while bringing its rare disease programs to key value infection points."

Scenic Biotech’s CEO Oscar Izeboud said, "I am delighted to welcome our new investors to the company and to thank our existing investors for their continued support. We have made very significant progress since our seed investment, proving the value of our platform, extending its utility, and expanding our team. We look forward to continuing our growth and delivering on our promise to develop novel treatments to improve patients’ lives."

Scenic anticipates more than doubling its team in the coming 18 months including building its clinical capabilities. To accommodate the expansion Scenic will be expanding its facilities within the flourishing life sciences cluster at the Science Park in the East of Amsterdam.

On March 10, 2022 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology, reported financial results for the fourth quarter and year ended December 31, 2021 (Press release, RAPT Therapeutics, MAR 10, 2022, View Source [SID1234609852]).

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"2021 was a significant year for RAPT, with progress in both of our lead programs," said Brian Wong, M.D., Ph.D., President and Chief Executive Officer of RAPT Therapeutics. "We reported positive results for RPT193 from our Phase 1b clinical trial in atopic dermatitis and are excited about RPT193’s potential as a safe, once-daily oral drug for multiple inflammatory diseases. Based on those promising data, we plan to initiate a Phase 2b clinical trial of RPT193 in atopic dermatitis in the first half of 2022, as well as a Phase 2a trial in asthma in the second half of the year. For FLX475, we are continuing development in several indications including EBV+ lymphoma, nasopharyngeal cancer and head and neck cancer. We plan to provide an update on FLX475 in 2022 when we have data that are sufficiently mature from the ongoing cohorts."

Financial Results for the Fourth Quarter and Year Ended December 31, 2021

Fourth Quarter Ended December 31, 2021

Net loss for the fourth quarter of 2021 was $17.9 million, compared to $12.7 million for the fourth quarter of 2020.

Research and development expenses for the fourth quarter of 2021 were $14.3 million, compared to $10.9 million for the same period in 2020. The increase in research and development expenses was primarily due to higher costs for the FLX475 and RPT193 clinical trials, personnel, facilities and laboratory supplies, partially offset by a decrease in stock-based compensation expense.

General and administrative expenses for the fourth quarter of 2021 were $4.5 million, compared to $3.5 million for the same period in 2020. The increase in general and administrative expenses was primarily due to increases in professional fees, personnel costs and insurance expenses, partially offset by a decrease in stock-based compensation expense.

Year Ended December 31, 2021

Net loss for the year ended December 31, 2021 was $69.2 million, compared to $52.9 million for the same period in 2020.

Research and development expenses for the year ended December 31, 2021 were $57.0 million, compared to $45.5 million for the same period in 2020. The increase in research and development expenses was primarily due to higher costs for the FLX475 and RPT193 clinical trials, personnel, facilities, stock-based compensation expense and laboratory supplies.

General and administrative expenses for the year ended December 31, 2021 were $16.0 million, compared to $12.8 million for the same period of 2020. The increase in general and administrative expenses was primarily due to increases in professional fees, insurance expense, personnel costs and stock-based compensation expense.

As of December 31, 2021, the Company had cash, cash equivalents and marketable securities of $189.7 million.

On March 10, 2022 Achieve Life Sciences, Inc. (Nasdaq: ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine addiction, reported fourth quarter and year-end 2021 financial results and provided an update on the cytisinicline clinical development program (Press release, OncoGenex Pharmaceuticals, MAR 10, 2022, View Source [SID1234609851]).

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Recent Business Highlights

Initiated ORCA-3, the second Phase 3 trial, in 750 adult smokers at 15 clinical sites in the United States

Announced last subject and last follow-up visit in the Phase 3 ORCA-2 trial of cytisinicline for smoking cessation

Entered into a $25 million loan facility with Silicon Valley Bank

Facilitated Key Opinion Leader virtual roundtable on smoking and e-cigarette cessation

Received U.S. Food and Drug Administration (FDA) acceptance of Investigational New Drug (IND) application for investigation of cytisinicline as a treatment for nicotine e-cigarette/vaping cessation

"We finished 2021 strong achieving multiple milestones and are starting the new year off with continued momentum in the cytisinicline development program," commented John Bencich, Chief Executive Officer of Achieve. "We are eagerly anticipating topline results from the Phase 3 ORCA-2 trial in the coming months, as we enroll smokers in the recently initiated Phase 3 ORCA-3 trial at 15 clinical sites in the United States. Additionally, our cash position remains strong with funding into 2023, allowing us to execute on the final stages of clinical development needed to support an NDA for smoking cessation."

Phase 3 ORCA-3 Trial Initiated

Achieve announced it has initiated enrollment for the Phase 3 ORCA-3 trial at 15 clinical sites in the United States in January 2022. Similar to the ORCA-2 trial, ORCA-3 will evaluate the efficacy and safety of 3 mg cytisinicline dosed three times daily compared to placebo. Approximately 750 smokers will be randomized to one-of-three treatment arms to evaluate cytisinicline administered for either 6 or 12 weeks.

Phase 3 ORCA-2 Trial Completed Last Subject and Last Follow-up Visit

Achieve completed the last study follow-up visit for the last subject enrolled in the Phase 3 ORCA-2 trial in late December 2021. The ORCA-2 trial is the first Phase 3 trial in Achieve’s ORCA (Ongoing Research of Cytisinicline for Addiction) Program, designed to evaluate the smoking cessation effectiveness, safety, and tolerability of 3 mg cytisinicline taken three times daily for either 6 or 12 weeks, compared with placebo. ORCA-2 randomized 810 subjects across 17 clinical trial sites in the United States. Topline data regarding the primary results are expected in the second quarter of 2022.

$25 Million Loan Facility with Silicon Valley Bank

In December 2021, Achieve announced a $25 million debt agreement with Silicon Valley Bank and SVB Innovation Credit Fund VIII, L.P. (SVB). The proceeds and funds available under the debt agreement are expected to fund the completion of the cytisinicline smoking cessation clinical development program. SVB funded $15 million in the form of contingent convertible indebtedness and Achieve may borrow additional non-convertible term loans in an aggregate original principal amount of up to $10 million.

Virtual Smoking Cessation KOL Roundtable

Achieve hosted a Key Opinion Leader virtual roundtable on smoking and e-cigarette cessation in December 2021. Two esteemed smoking cessation experts discussed the current market for smoking and e-cigarette cessation, challenges with available treatments, and the potential of cytisinicline to be the first new FDA approved smoking cessation therapy in nearly two decades. Click here to view the event.

FDA Acceptance of IND for Cytisinicline in e-Cigarette and Vape Cessation

The FDA completed its review and accepted an IND application to investigate cytisinicline as a cessation treatment for nicotine e-cigarette users in November 2021. The Phase 2 ORCA-V1 study will enroll approximately 150 adult nicotine e-cigarette users in the United States and is expected to initiate in the second quarter of 2022. Grant funding to support the trial has been awarded in two phases from the National Institute on Drug Abuse of the National Institutes of Health. Completion of required milestones for the first phase of grant funding included the submission of the IND and clearance to proceed with the clinical trial by FDA. Achieve has submitted for completion of the first phase milestones and awaits clearance under the grant to proceed with ORCA-V1 study.

Financial Results

As of December 31, 2021, the company’s cash, cash equivalents, and restricted cash was $43.1 million. Total operating expenses for the fourth quarter and year ended December 31, 2021 were $7.1 million and $33.1 million, respectively. Total net loss for the fourth quarter and year ended December 31, 2021 was $7.2 million and $33.2 million, respectively. As of March 10, 2022, Achieve had 9,460,835 shares outstanding.

Conference Call Details

Achieve will host a conference call at 4:30 PM EST today, Thursday, March 10, 2022. To access the webcast, log on to the investor relations page of the Achieve website at View Source Alternatively, access to the live conference call is available by dialing (877) 472-9809 (U.S. & Canada) or (629) 228-0791 (International) and referencing conference ID 8066623. A webcast replay will be available approximately two hours after the call and will be archived on the website for 90 days.

On March 10, 2022 Chemomab Therapeutics Ltd. (Nasdaq: CMMB), (Chemomab), a clinical-stage biotechnology company focused on the discovery and development of innovative therapeutics for fibrotic and inflammatory diseases with high unmet need, reported that management will be presenting a corporate overview and participating in 1-on-1 meetings on March 15, 2022, at the 32nd Annual Oppenheimer Healthcare Conference (Press release, Chemomab, MAR 10, 2022, View Source [SID1234609849]).

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32nd Annual Oppenheimer Healthcare Conference (virtual)

Date: March 15, 2022
Time: 10:00am – 10:30am ET
Registration: Webcast Registration Link

To request a virtual 1-on-1 meeting with Chemomab management, investors should speak with an Oppenheimer representative.

An archived version of the presentation webcast will also be available at the Investor Relations section of the company’s website at investors.chemomab.com/events