On May 4, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported that the European Commission (EC) has approved an additional indication for the oral once-daily therapy XTANDITM (enzalutamide) for adult men with metastatic hormone-sensitive prostate cancer (mHSPC, also known as metastatic castration-sensitive prostate cancer or mCSPC) (Press release, Astellas, MAY 4, 2021, View Source [SID1234579258]). Men diagnosed with mHSPC tend to have a poor prognosis, with a median survival of approximately 3-4 years,1 underscoring the need for new treatment options.

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With this indication, enzalutamide is now the only oral treatment approved by the EC to treat three distinct types of advanced prostate cancer — non-metastatic and metastatic castration-resistant prostate cancer (CRPC) and mHSPC.2 The EC approval is based on results from the pivotal Phase 3 ARCHES trial which evaluated enzalutamide in men with mHSPC.3

"Metastatic hormone-sensitive prostate cancer patients have limited options and, unfortunately, there is a poor prognosis for many men," said Andrew Armstrong, M.D., Professor of Medicine, Surgery, Pharmacology and Cancer Biology, Director of Research in the Duke Cancer Institute’s Center for Prostate and Urologic Cancers and lead investigator of ARCHES. "The research supporting this approval provides clinical evidence showing how enzalutamide can help improve outcomes for men with mHSPC, which gives healthcare professionals in Europe the option to offer the treatment across the advanced prostate cancer disease continuum."

Data from the ARCHES trial showed enzalutamide plus androgen deprivation therapy (ADT) significantly reduced the risk of radiographic progression or death by 61% versus placebo plus ADT in men with mHSPC (n=1,150; hazard ratio [HR]=0.39 [95% confidence interval (CI): 0.30-0.50]; P<0.0001).3

"Enzalutamide has been an established standard of care for men with advanced prostate cancer and has been prescribed to more than 610,000 patients worldwide since it was first approved in 2012," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development, Astellas. "This new indication for enzalutamide provides men with mHSPC a much-needed, additional therapy option earlier in their treatment journey. We look forward to working with health authorities across Europe to ensure men with mHSPC have access to enzalutamide as soon as possible."

The safety analyses of the ARCHES trial appear consistent with the safety profile of enzalutamide in previous clinical trials in CRPC. In ARCHES, Grade 3 or greater adverse events (AEs) (defined as severe/disabling or life-threatening) were similar for patients receiving both enzalutamide plus ADT and those who received placebo plus ADT (24.3% vs. 25.6%).3

The EC marketing authorization for enzalutamide in men with mHSPC is applicable to European Union (EU) member countries, and is also valid in Iceland, Norway and Liechtenstein.4

Astellas has already reflected the impact from this approval in its financial forecast of the current fiscal year ending March 31, 2022.

On May 4, 2021 Navidea Biopharmaceuticals, Inc. (NYSE American: NAVB) ("Navidea" or the "Company"), a company focused on the development of precision immunodiagnostic agents and immunotherapeutics, reported it will host a conference call and webcast on Tuesday, May 11 2021 at 5:00 p.m. (EDT) to discuss financial results and corporate developments for the first quarter ended March 31, 2021 (Press release, Navidea Biopharmaceuticals, MAY 4, 2021, View Source [SID1234579107]).

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Jed Latkin, Chief Executive Officer, Dr. Michael Rosol, Chief Medical Officer, and Joel Kaufman, Chief Business Officer, will host the call and webcast to discuss the financial results and provide an update on recent developments and clinical progress. Management will be available to answer questions live immediately following the earnings announcement and prepared remarks portion of the call.

To participate in the call and webcast, please refer to the information below:

A live audio webcast of the conference call will also be available on the investor relations page of Navidea’s corporate website at www.navidea.com. In addition, the recorded conference call can be replayed and will be available for 90 days following the call on Navidea’s website.

On May 4, 2021 EDAP TMS SA (Nasdaq: EDAP), a global leader in robotic energy-based therapies, reported that it will release its financial results for the first quarter ended March 31, 2021 after the markets close on Tuesday, May 11, 2021 (Press release, EDAP TMS, MAY 4, 2021, View Source,EDAP%20TMS%20S.A.&text=LYON%2C%20France%2C%20May%204%2C,Tuesday%2C%20May%2011%2C%202021. [SID1234579123]).

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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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An accompanying conference call and webcast will be conducted by Marc Oczachowski, Chairman of the Board and Chief Executive Officer, and François Dietsch, Chief Financial Officer. The call will be held at 8:30am EDT on Wednesday, May 12, 2021. Please refer to the information below for conference call dial-in information and webcast registration.

On May 4, 2021 Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) reported financial results for the first quarter of 2021 (Press release, Concert Pharmaceuticals, MAY 4, 2021, View Source [SID1234584684]).

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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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"We are committed to advancing CTP-543, our breakthrough therapy candidate for moderate to severe alopecia areata. Patients currently lack effective, approved treatment options. We believe we have the potential to provide patients with a new, clinically meaningful and best-in-class treatment option," stated Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "We remain on track with our THRIVE-AA Phase 3 clinical program with CTP-543, in support of our New Drug Application that we anticipate filing in early 2023."

Recent Business Highlights and Upcoming Milestones

CTP-543: An Investigational Treatment for Moderate to Severe Alopecia Areata

THRIVE-AA1 Phase 3 Trial in Alopecia Areata On Track to Report Topline Data in 2022. The THRIVE-AA1 Phase 3 trial is a randomized, double-blind, placebo-controlled clinical trial of CTP-543 to evaluate hair regrowth after 24 weeks of dosing in approximately 700 adults with moderate to severe alopecia areata. The trial is evaluating 8 mg and 12 mg twice-daily doses of CTP-543 compared to placebo at sites in the U.S., Canada and Europe. The Company expects to report topline results from the THRIVE-AA1 trial in 2022. Additional information about the THRIVE-AA1 trial (NCT04518995) is available at clinicaltrials.gov.
Initiation of THRIVE-AA2 Phase 3 Trial in Alopecia Areata Expected in May 2021. The Company expects to initiate a second pivotal Phase 3 trial of CTP-543, THRIVE-AA2, later this month. The planned THRIVE-AA2 Phase 3 trial is a randomized, double-blind, placebo-controlled clinical trial of CTP-543 to evaluate hair regrowth after 24 weeks of dosing in approximately 440 adults with moderate to severe alopecia areata. The trial will evaluate 8 mg and 12 mg twice-daily doses of CTP-543 compared to placebo at sites in the U.S., Canada and Europe. Additional information about the THRIVE-AA2 trial (NCT04797650) is available at clinicaltrials.gov.
Update on CTP-543 Open Label, Long-Term Extension Study to be Presented at JAK Summit. Concert’s Chief Development Officer, James V. Cassella, Ph.D., will present an update on the ongoing open label, long-term extension study of CTP-543 at the 2nd JAK Inhibitors Drug Development Summit scheduled for July 1, 2021. The presentation will provide an update on the extension study, building on the data presented at the late-breaking news session at the European Academy of Dermatology and Venereology (EADV) in October 2020, during which the Company showed the maintenance of hair regrowth in patients on treatment for at least one year. Details about the upcoming meeting are available at https://jak-drugdevelopment.com/.
First Quarter 2021 Financial Results

Cash and Investment Position. Cash, cash equivalents and investments as of March 31, 2021 totaled $111.8 million as compared to $130.0 million as of December 31, 2020. Under its current operating plan, the Company expects its cash, cash equivalents and investments to fund the Company through 2021.
R&D Expenses. Research and development expenses were $18.5 million for the quarter ended March 31, 2021, compared to $14.0 million for the same period in 2020. The increase in research and development expenses relates primarily to the ongoing CTP-543 Phase 3 THRIVE-AA clinical program.
G&A Expenses. General and administrative expenses were $5.5 million for the quarter ended March 31, 2021, compared to $4.7 million for the same period in 2020, an increase of $0.8 million due to an increase in external professional service expenses and non-cash stock-based compensation expense.
Net Loss. Net loss applicable to common stockholders was $22.7 million, or $0.67 per share, for the quarter ended March 31, 2021, as compared to net loss applicable to common stockholders of $20.5 million, or $0.70 per share, for the quarter ended March 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 its first quarter 2021 financial results. 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 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 May 4, 2021 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported the launch of the preclinical development of its liquid tumor pipeline with the publication of results from its Phase 1 study evaluating AnktivaÔ (N-803), its IL-15 superagonist, in combination with RituxanÒ (rituximab), an anti-CD20 monoclonal antibody therapy, in patients with indolent non-Hodgkin lymphoma (iNHL), who had relapsed or were refractory after two lines of therapy (Press release, ImmunityBio, MAY 4, 2021, https://immunitybio.com/immunitybio-announces-78-percent-complete-response-following-chemotherapy-free-combination-of-il-15-superagonist-anktiva-with-rituxan-in-relapsed-non-hodgkin-lymphoma-patients/ [SID1234579039]).

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The peer-reviewed article titled "Phase 1 trial of N-803, an IL-15 receptor agonist, with rituximab in patients with indolent non-Hodgkin lymphoma" (see link HERE) published in the American Association for Cancer Research (AACR) (Free AACR Whitepaper) journal Clinical Cancer Research, highlights safety, efficacy, and translational data from the Company’s ongoing open-label, multi-center, dose-escalation Phase 1 study (NCT02384954).

The study was designed to evaluate Anktiva in combination with rituximab in patients with iNHL, to explore the potential of Anktiva to enhance tumor-targeting of anti-CD20 therapeutic antibodies, and to determine the safety and efficacy of subcutaneous (SQ) versus intravenous (IV) administration. Anktiva has been engineered to exhibit a longer half-life and more potent trans-presentation relative to endogenous IL-15 to promote natural killer (NK) cell and T cell expansion to control cancer.

Key study results include:

The combination regimen of Anktiva and Rituxan was well tolerated with a single reported grade 4 adverse event (AEs) and no reported grade 5 AEs
For patients with anti-CD20 mAb sensitive disease, the overall response rate (ORR) in the SQ cohort was 78% (7 of 9)
7 of 7 (100%) responses in the SQ cohorts were complete remissions (CR)
Prolonged stable disease (SD) and conversion of SD and/or partial response (PR) to CRs with a prolonged duration without progression were observed, with 8 of 12 patients without progression at 18-24 months
For the 5 patients with anti-CD20 mAb refractory disease in both IV and SQ cohorts, the ORR was 2 of 5 (40%) with 1 CR, 1 PR, 1 SD, and 2 progressive disease (PD) with the PR and SD are ongoing at over 18 months
In correlative immunology experiments, Anktiva in combination with Rituxan induced the expansion, activation and modulation of NK cells and CD8+ T cells, with minimal impact on CD4+ T cells and Tregs
Multi-dimensional mass cytometry studies demonstrated remodeling of iNHL patient immune landscapes by promotion of an activation signature in nearly all main immune cell lineages including NK cells, CD8+ TEM, gd T cells, and CD14 and CD16 monocytes
Todd Fehniger, M.D., Ph.D, Washington University School of Medicine in St Louis, said, "These encouraging data suggest that Anktiva, ImmunityBio’s IL-15 superagonist, has the potential to enhance the activity of an anti-CD20 therapeutic antibody. We believe the excellent safety profile seen in the SQ cohort, when combined with compelling efficacy which includes prolonged progression free survival and a 78% complete response rate in rituximab-sensitive patients, warrants the further exploration of this regimen in iNHL patients. The translational immunology data generated also provide important proof-of-mechanism, with activation of important cell population including NK Cells and CD8+ T cells, which are key in driving immunotherapy responses. Together, these results suggest Anktiva may have broad potential to enhance the activity of therapeutic monoclonal antibodies across a wide range of tumor types."

The study enrolled patients with iNHL (follicular lymphoma, marginal zone lymphoma, small lymphocytic lymphoma, lymphoplasmacytic lymphoma) that were relapsed or refractory after >2 prior lines of therapy. Patients were considered anti-CD20 mAb refractory if they progressed on anti-CD20 mAb therapy within 6 months of their last dose of anti-CD20 mAb. Treatment consisted of IV rituximab 375 mg/m2 and IV or SQ N-803 (in increasing dosing cohorts of 1, 3, 6, 10, 15, 20 µg/kg).