On March 3, 2022 Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) reported financial results for the year ended December 31, 2021 (Press release, Concert Pharmaceuticals, MAR 3, 2022, View Source [SID1234609426]).

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"Our team is extremely proud and motivated by our success in enrolling more than 1,200 patients in our THRIVE-AA Phase 3 program in line with our projected timelines. This represents a significant milestone for Concert as we focus on commercializing CTP-543 to help meet the needs of the alopecia areata patient community," said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "We expect 2022 to be a data rich year, with the first key CTP-543 Phase 3 data readout next quarter."

Recent Business Highlights and Upcoming Milestones

CTP-543: An Investigational Treatment in Phase 3 Trials for Moderate to Severe Alopecia Areata

Topline Data in THRIVE-AA1 Expected in the Second Quarter of 2022. The Company expects to report topline results from the first CTP-543 Phase 3 trial, THRIVE-AA1, in the second quarter of 2022. THRIVE-AA1 is a randomized, double-blind, placebo-controlled Phase 3 clinical trial of CTP-543 to evaluate hair regrowth using the Severity of Alopecia Tool (SALT) after 24 weeks of dosing. The trial is evaluating 8 mg and 12 mg twice-daily doses of CTP-543 compared to placebo. The trial enrolled 708 adult patients with moderate to severe alopecia areata at sites in the U.S., Canada and Europe.

Enrollment Completed in the THRIVE-AA2 Phase 3 Trial. Similar to THRIVE-AA1, THRIVE-AA2 is a randomized, double-blind, placebo-controlled Phase 3 clinical trial of CTP-543 to evaluate hair regrowth using SALT after 24 weeks of dosing. The trial is evaluating 8 mg and 12 mg twice-daily doses of CTP-543 compared to placebo. The trial enrolled 517 adult patients with moderate to severe alopecia areata at sites in the U.S., Canada and Europe. The Company expects to report topline results from the THRIVE-AA2 trial in the third quarter of 2022.

New Drug Application (NDA) Filing Expected for Alopecia Areata in the First Half of 2023. If the CTP-543 clinical program is successful, the Company intends to file an NDA with the U.S. Food and Drug Administration (FDA) in the first half of 2023. The FDA has granted CTP-543 Breakthrough Therapy designation for the treatment of adult patients with moderate to severe alopecia areata and Fast Track designation for the treatment of alopecia areata. Alopecia areata is an autoimmune disease that may affect up to approximately 1.5 million Americans at any given time1.
Partnered Program: AVP-786 for Neurological Disorders

AVP-786 Late-Stage Trials to Complete in Third Quarter of 2022. Avanir Pharmaceuticals and Otsuka Pharmaceuticals are conducting a number of clinical trials evaluating AVP-786 in certain neurological disorders, including multiple Phase 3 studies in Alzheimer’s agitation. Avanir and Otsuka have full responsibility for the development, reporting of clinical results and commercialization of AVP-786. Concert is entitled to potential future milestones and royalties. Two trials are expected to complete in the third quarter of 2022:

AVP-786 Phase 3 trial in Alzheimer’s Agitation is projected to complete in July 2022.
AVP-786 Phase 2/3 trial in Negative Symptoms of Schizophrenia is projected to complete in August 2022.
Full Year 2021 Financial Results

Cash and Investment Position. Cash, cash equivalents and investments as of December 31, 2021 totaled $141.6 million, compared to $130.0 million as of December 31, 2020. In November 2021, the Company raised gross proceeds of $65.0 million under a financing arrangement with BVF Partners L.P. and RA Capital Management, L.P. The financing consisted of the sale of common and preferred stock, warrants and a portion of Concert’s right to receive potential future AVP-786 royalties under an existing licensing agreement with Avanir. Under its current operating plan, the Company expects its current cash and cash equivalents to fund the Company into the fourth quarter of 2022. In addition, Concert has the potential to receive an additional $103.1 million upon the full exercise of the warrants issued in connection with the November 2021 financing.

Revenue. Revenue was $32.6 million for the year ended December 31, 2021, compared to $7.9 million for the year ended December 31, 2020. Revenue recognized in 2021 was primarily attributable to the $32.0 million in cash proceeds received from Vertex Pharmaceuticals, Inc. for the purchase of potential future milestones under the companies’ 2017 asset purchase agreement related to VX-561. Revenue recognized in 2020 was the result of the expiration of licensing options under a previous collaboration with Celgene Corporation.

R&D Expenses. Research and development expenses were $87.6 million for the year ended December 31, 2021, compared to $61.6 million for the year ended December 31, 2020. The increase in research and development expenses relates primarily to the clinical development for CTP-543.

G&A Expenses. General and administrative expenses were $22.5 million for the year ended December 31, 2021, compared to $18.9 million for the year ended December 31, 2020. The increase in general and administrative expenses relates primarily to increased external professional service expenses and non-cash stock-based compensation.

Net Loss. For the year ended December 31, 2021, net loss attributable to common stockholders was $80.1 million, or $2.33 per share, compared to net loss applicable to common stockholders of $74.8 million, or $2.40 per share, for the year ended December 31, 2020.
Conference Call and Webcast

The Company will host a conference call and webcast today at 8:30 a.m. ET to provide an update on the Company and discuss financial results for the year ended December 31, 2021. To access the conference call, please dial (855) 354-1855 (U.S. and Canada) or (484) 365-2865 (International) five minutes prior to the start time.

A live webcast of the financial results may be accessed in the Investors section of the Company’s website at www.concertpharma.com. Please log on to the Concert website approximately 15 minutes prior to the scheduled webcast to ensure adequate time for any software downloads that may be required. A replay of the webcast will be available on Concert’s website for three months.

On March 3, 2022 Rain Therapeutics Inc. (NasdaqGS: RAIN), (Rain), a late-stage company developing precision oncology therapeutics, reported its financial results for the fourth quarter and full year ended December 31, 2021, along with an update on the Company’s key developments, business operations and upcoming milestones (Press release, Rain Therapeutics, MAR 3, 2022, View Source [SID1234609455]).

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"Rain has achieved a number of important clinical milestones for milademetan including commencing two of the four planned trials in MDM2-dependent cancers. Rain dosed the first patient in the third quarter of last year and exceeded year-end 2021 targets for site activations for the pivotal, Phase 3 MANTRA trial in patients with liposarcoma and dosed the first patient in our Phase 2 MANTRA-2 basket trial in genetically selected patients with MDM2 gene amplification. We have also outlined two additional trials to start in the second half of this year, including the MANTRA-3 trial in patients with Merkel cell carcinoma, and our first combination trial MANTRA-4, with Roche’s anti-PD-L1 antibody, atezolizumab, in patients with advanced cancers exhibiting loss of the CDKN2A gene," said Avanish Vellanki, co-founder and chief executive officer of Rain.

Mr. Vellanki added, "We believe we have a strong capital position supporting completion of our ongoing and planned clinical trials, including an ample cash cushion for the Phase 3 MANTRA data timelines in liposarcoma. Further, considering the current capital markets picture in the biotech industry, we have taken steps to be even more prudent with capital to preserve the opportunity to generate data for milademetan across a diverse set of cancers."

Key Developments and Operational Updates

Phase 3 Dedifferentiated Liposarcoma Trial (MANTRA) is enrolling on schedule
Global site activations exceeded the target for year-end 2021, and all sites anticipated to be activated by the end of the first quarter of 2022. Enrollment in MANTRA trial on schedule.
Phase 2 Basket Trial (MANTRA-2) of Milademetan for MDM2-Amplified Advanced Solid Tumors
Rain dosed the first patient in its multicenter, open-label Phase 2 basket trial (MANTRA-2) in November 2021, evaluating milademetan in MDM2-amplified advanced solid tumors. Enrollment in MANTRA-2 trial on schedule.
Rain made significant progress in site activations for MANTRA-2 and anticipates all sites will be activated by the end of the first quarter of 2022.
Phase 2 Trial for Milademetan in Merkel Cell Carcinoma (MANTRA-3)
A Phase 2 clinical trial of milademetan as monotherapy in Merkel cell carcinoma (MCC) patients relapsed from first-line checkpoint inhibitors is anticipated to commence in the second half of 2022.
Rain Enters into Clinical Supply Agreement with Roche for the Supply of Atezolizumab in a Phase 1 Basket Trial in Advanced Solid Tumors Exhibiting Loss of the CDKN2A Gene (MANTRA-4)
In January 2022, Rain announced a clinical supply agreement with Roche for the supply of the anti-Programmed Death Ligand-1 (PD-L1) monoclonal antibody, atezolizumab.
A Phase 1 trial (MANTRA-4) is planned to evaluate the safety, tolerability and efficacy of milademetan in combination with Roche’s atezolizumab in patients with loss of CDKN2A and wild-type p53 advanced solid tumors and is anticipated to commence in the second half of 2022.
RAD52 Research Program
Continues to progress, with Rain-generated compounds exhibiting improved potency and selectivity versus the initial licensed portfolio.
Key Upcoming Milestones

Milademetan Dedifferentiated Liposarcoma Phase 3 Trial (MANTRA)
Top-line data anticipated in 2023
Milademetan MDM2-Amplified Phase 2 Basket Trial (MANTRA-2)
Interim data anticipated in the second half of 2022
Milademetan MCC Phase 2 Trial (MANTRA-3)
Phase 2 trial anticipated to commence in the second half of 2022
Milademetan Phase 1 Combination Trial (MANTRA-4)
Phase 1 trial anticipated to commence in the second half of 2022
Fourth Quarter Financial Results

For the three months and year ended December 31, 2021, Rain reported a net loss of $18.0 million and $51.4 million, respectively, as compared to a net loss of $5.4 million and $21.1 million for the same periods in 2020, respectively. Net loss per share for the three months and year ended December 31, 2021, was $0.68 and $2.65, respectively, as compared to a net loss per share of $1.56 and $6.29 for the same periods in 2020, respectively.

R&D expenses were $14.7 million and $40.8 million for the three months and year ended December 31, 2021, respectively, as compared to $4.2 million and $15.4 million for the same periods in 2020, respectively. The increases were primarily driven by the ongoing Phase 3 pivotal trial in dedifferentiated liposarcoma (MANTRA) and Phase 2 tumor-agnostic basket trial (MANTRA-2), as well as personnel costs. Non-cash stock-based compensation expenses included in R&D expenses were approximately $1.1 million and $2.5 million in the three months and year ended December 31, 2021, respectively, as compared to $0.2 million and $0.6 million in the same periods in 2020, respectively.

General and administrative (G&A) expenses were $3.4 million and $10.7 million for the three months and year ended December 31, 2021, respectively, as compared to $1.3 million and $3.6 million for the same periods in 2020, respectively. The increases were primarily due to higher costs associated with Rain becoming a public company, including the costs of director and officer insurance, personnel, legal, outside consulting, and accounting and audit fees. Non-cash stock-based compensation expense included in G&A expenses were approximately $0.2 million and $0.6 million for the three months and year ended December 31, 2021, respectively, as compared to $0.1 million and $0.3 million for the same periods in 2020, respectively.

Total non-cash stock-based compensation expenses were approximately $1.3 million and $3.1 million for the three months and year ended December 31, 2021, respectively, as compared to $0.3 million and $0.9 million for the same periods in 2020, respectively.

As of December 31, 2021, Rain had $140.2 million in cash, cash equivalents and short-term investments. Rain anticipates that its year-end cash position will provide runway into the first half of 2024, including completion of all its ongoing and planned clinical trials with an ample cash cushion for the Phase 3 MANTRA trial in liposarcoma.

As of December 31, 2021, Rain had approximately 26.5 million shares of common stock outstanding.

Fourth Quarter 2021 Results Conference Call and Webcast Details
The management of Rain Therapeutics will host a conference call and webcast for the investment community today, March 3, 2022 at 1:30 pm PT (4:30 pm ET). A live webcast may be accessed here: View Source The conference call can be accessed by dialing (833) 562-0127. The passcode for the conference call is 4729972.

Replay of the call will be available by visiting the "Events" section of the Rain website after the conclusion of the presentation and will be archived on the Rain website for 30 days.

About Milademetan

Milademetan (also known as RAIN-32) is a small molecule, oral inhibitor of MDM2, which is oncogenic in numerous cancers. Milademetan has already demonstrated antitumor activity in an MDM2-amplified subtype of liposarcoma (LPS) and other solid tumors in a Phase 1 clinical trial, supported by a rationally-designed dosing schedule to mitigate safety concerns and widen the therapeutic window of MDM2 inhibition. Milademetan is being evaluated in an ongoing Phase 3 clinical trial in patients with LPS (MANTRA), as well as a Phase 2 tumor-agnostic basket trial in certain solid tumors (MANTRA-2). Rain anticipates commencing a Phase 2 clinical trial of milademetan (MANTRA-3), for the treatment of patients with wildtype p53 Merkel cell carcinoma who are also refractory to immune checkpoint inhibition (ICI), in the second half of 2022, as well as a Phase 1 clinical trial to evaluate the safety, tolerability and efficacy of milademetan in combination with Roche’s atezolizumab in patients with loss of cyclin-dependent kinase inhibitor 2A (CDKN2A) and wildtype p53 advanced solid tumors (MANTRA-4), in the second half of 2022. Milademetan has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for patients with LPS.

On March 3, 2022 GT Biopharma, Inc. (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company’s proprietary tri-specific natural killer (NK) cell engager, TriKE protein biologic technology platform, reported that Dr. Greg Berk, President of R&D, Chief Medical Officer and Interim CEO, will participate in a fireside chat in-person at the 34th Annual ROTH Conference on Tuesday March 15, 2022, at 8:30 AM PT (Press release, GT Biopharma, MAR 3, 2022, View Source [SID1234609471]). Dr. Berk, will be available for one-on-one meetings throughout the event.

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The ROTH Conference will consist of 1-on-1 and small group meetings, analyst-selected fireside chats, thematic industry panels, and on-demand presentations by executive management from approximately 400 private and public companies in a variety of growth sectors.

To arrange a one-on-one with GT Biopharma please email [email protected] or contact your ROTH sales representative to arrange.

To submit a registration request or learn more please visit: View Source

On March 3, 2022 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines, reported that BRUKINSA (zanubrutinib) has been approved by Health Canada for the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy (Press release, BeiGene, MAR 3, 2022, View Source [SID1234609486]).

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"We will continue to work with physicians and their patients as part of our broad global clinical program for BRUKINSA, which includes 35 trials and more than 3,900 subjects across 28 markets."

"We are pleased that BRUKINSA is now approved in its third indication in Canada, R/R MZL, and this terrific milestone was made possible by the participating patients and investigators. This approval further supports our belief that BRUKINSA is a potentially best-in-class BTK inhibitor, with the MAGNOLIA trial results in R/R MZL providing additional evidence that the selective design of BRUKINSA can be translated into improved treatment outcomes," said Jane Huang, M.D., Chief Medical Officer of Hematology at BeiGene. "We will continue to work with physicians and their patients as part of our broad global clinical program for BRUKINSA, which includes 35 trials and more than 3,900 subjects across 28 markets."

"BRUKINSA is a highly selective next-generation BTK inhibitor designed to improve tolerability by minimizing off-target binding. In clinical trials, BRUKINSA achieved a high overall response rate among relapsed/refractory MZL patients and was generally well-tolerated. With this approval, Canadian patients with R/R MZL will have the option to receive BRUKINSA monotherapy as a treatment and a new hope for improved treatment outcomes," said Dr. Anthea Peters, a hematologist at the Cross Cancer Institute in Edmonton, Alberta.

"The approval of this medicine for the treatment of R/R MZL represents a new treatment option and is welcome news to Canadians living with this rare type of lymphoma," commented Antonella Rizza, Chief Executive Officer of Lymphoma Canada.

"We are delighted that BRUKINSA is now approved for patients with R/R MZL. Since our first approval 11 months ago, we have been expanding our organization in Canada and working to deliver on our commitment to create access to BRUKINSA for patients living with MCL, WM, and now MZL," added Peter Brenders, General Manager of Canada at BeiGene.

The Health Canada marketing approval for BRUKINSA in R/R MZL is based on efficacy results from two open-label, multicenter, single arm clinical trials. In the MAGNOLIA trial (NCT03846427) (n=68), which included previously treated patients with MZL who had received at least one prior anti-CD20-based therapy, 38% of patients had extranodal MZL, 38% had nodal MZL, 18% had splenic MZL and 6% patients had unknown subtype. In BGB-3111-AU-003 (NCT02343120) (n=20), which included patients with previously treated MZL, 45% had extranodal MZL, 25% had nodal MZL and 30% had splenic MZL. BRUKINSA Total Daily Dose was 320 mg daily, given as 160 mg twice daily or 320 mg once daily.

As assessed by Independent Review Committee per 2014 Lugano Classification, BRUKINSA achieved an overall response rate (ORR) of 68% (95% CI; 56, 79) in the MAGNOLIA trial and 80% (95% CI; 56, 94) in BGB-311-AU-003. In the MAGNOLIA trial, the median response time was 2.8 months (range: 1.7 to 11.1 months).

Serious treatment-emergent adverse events were reported in 35 (40%) patients. The most common serious adverse events (≥ 2% of patients) were pyrexia (8%), pneumonia (7%), influenza (2%), anemia (2%), diarrhea (2%), atrial fibrillation and flutter (2%) and fall (2%).

Of the 88 patients with MZL treated with BRUKINSA, five (6%) patients discontinued treatment due to adverse event. The adverse events leading to treatment discontinuation included two cases of pneumonia (due to COVID-19 pneumonia), one case each of pyrexia, myocardial infarction, and diarrhea. Two (2%) patients had a dose reduction due to adverse events. Death due to adverse events within 30 days of last dose occurred in three (3%) patients. The adverse events leading to death were: COVID-19 pneumonia in two patients (2%) and myocardial infarction in one patient (1%).

The recommended dose of BRUKINSA is either 160 mg twice daily or 320 mg once daily, taken orally with or without food. The dose may be adjusted for adverse reactions and reduced for patients with severe hepatic impairment and certain drug interactions.

About myBeiGene Patient Support Program

The myBeiGene patient support program is designed to support patients, caregivers, and healthcare providers with easy access to BRUKINSA. It goes beyond financial assistance and provides practical and emotional support by connecting them to third-party resources that can address their individual needs. Oncology Nurse Advocates are available Monday through Friday from 8 a.m. to 5 p.m. Eastern Time at 1-833-234-4366.

About BRUKINSA

BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.

To date, BRUKINSA has received more than 20 approvals covering more than 40 countries and regions, including the United States, China, the EU and Great Britain, Canada, Australia, and additional international markets. Currently, more than 40 additional regulatory submissions are in review around the world.

BeiGene Oncology

BeiGene is committed to advancing best- and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R&D and medical affairs team of approximately 2,900 colleagues dedicated to advancing more than 100 clinical trials that have involved more than 14,500 subjects. Our expansive portfolio is directed predominantly by our internal colleagues supporting clinical trials in more than 45 countries and regions. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. BeiGene currently has three approved medicines discovered and developed in our own labs: BTK inhibitor BRUKINSA in the United States, China, the EU and Great Britain, Canada, Australia and additional international markets; and the non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab as well as the PARP inhibitor pamiparib in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen, Bristol Myers Squibb, EUSA Pharma and Bio-Thera. We also plan to address greater areas of unmet need globally through our other collaborations including with Mirati Therapeutics, Seagen, and Zymeworks.

In January 2021 BeiGene and Novartis announced a collaboration granting Novartis rights to co-develop, manufacture, and commercialize BeiGene’s anti-PD1 antibody tislelizumab in North America, Europe, and Japan. Building upon this productive collaboration, including a biologics license application (BLA) under FDA review, BeiGene and Novartis announced two new agreements in December 2021 granting Novartis an option to co-develop, manufacture, and commercialize BeiGene’s TIGIT inhibitor ociperlimab that is in Phase 3 development and adding five approved Novartis oncology products to the BeiGene product portfolio across designated regions of China.

On March 3, 2022 Corvus Pharmaceuticals, Inc. (Nasdaq: CRVS), a clinical-stage biopharmaceutical company, reported that the Company will host a conference call and webcast on March 10, 2022 at 4:30 pm ET (1:30 pm PT) to provide a business update and report fourth quarter and full year 2021 financial results (Press release, Corvus Pharmaceuticals, MAR 3, 2022, View Source [SID1234609505]).

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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

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The conference call can be accessed by dialing 1-877-407-0784 (toll-free domestic) or 1-201-689-8560 (international) and using the conference ID 13727689. The live webcast may be accessed via the investor relations section of the Corvus website. A replay of the webcast will be available on Corvus’ website for 90 days.