On August 5, 2021 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the second quarter ended June 30, 2021, and provided a corporate update (Press release, BioCryst Pharmaceuticals, AUG 5, 2021, View Source [SID1234585909]).

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"BioCryst is in an outstanding position, both near-term and long-term, with growing revenue from a strong ORLADEYO launch in the U.S., more approvals and launches of ORLADEYO around the globe and a pipeline in a molecule with our oral Factor D inhibitor, BCX9930, entering pivotal trials this year in the first of many indications," said Jon Stonehouse, president and chief executive officer of BioCryst.

Program Updates and Key Milestones

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

U.S. Launch

"The ORLADEYO launch is off to an excellent start because HAE patients want a safe and effective oral medicine to control their attacks and reduce their burden of therapy, and switching to ORLADEYO meets these needs for them," said Charlie Gayer, chief commercial officer of BioCryst.

ORLADEYO net revenue in the second quarter of 2021 was $28.5 million.

Patient switches continue to drive the launch with 60 percent of patients who were new to ORLADEYO in the second quarter switching from other prophylactic medicine to ORLADEYO and the remainder from acute-only treatment.

The number of physicians prescribing ORLADEYO grew by approximately 50 percent in second quarter.

The majority (approximately 70 percent) of HAE patients in the U.S. now have access to ORLADEYO through insurance policies.

Through the launch thus far, patient retention on therapy remains consistent with the one-year patient retention rate observed in the APeX-2 clinical trial.
ORLADEYO: Global Updates

On July 10, 2021, the company announced data presented at the European Academy of Allergy and Clinical Immunology Hybrid Congress 2021. HAE patients who were randomized to receive 150 mg of oral, once-daily ORLADEYO at the start of the APeX-2 trial had an 80 percent average reduction in their mean attack rate per month during weeks 25-96 of the trial, compared to baseline. Median attack rates also decreased from 2.7 attacks/month at baseline to 0.0 attacks per month in 16 of 17 months through the same period. ORLADEYO was generally well-tolerated during the treatment period with fewer drug-related adverse events reported in part 3 (weeks 49-96) as compared to part 1 (weeks 0-24) and part 2 (weeks 25-48). Eighty-one percent of the patients who entered part 3 completed the trial.

On June 16, 2021, the company announced that the Israeli Ministry of Health has accepted the regulatory submission of ORLADEYO for the prevention of recurrent attacks in patients with hereditary angioedema (HAE) 12 years and older. In addition, BioCryst entered into a distribution and supply agreement granting Neopharm Ltd., the exclusive rights to commercialize ORLADEYO in Israel.

On June 3, 2021, the company announced the launch of ORLADEYO in Germany.

On May 12, 2021, the company announced that the United Kingdom’s Medicines and Healthcare products Regulatory Agency has granted marketing authorization for ORLADEYO for the routine prevention of HAE attacks in HAE patients 12 years and older.

On April 30, 2021, the company announced that the European Commission (EC) has approved ORLADEYO for the prevention of recurrent HAE attacks in HAE patients 12 years and older. The EC approval of ORLADEYO is applicable to all European Union member states plus Iceland, Norway and Liechtenstein.

On April 14, 2021, the company announced that the Japanese National Health Insurance System (NHI) approved the addition of ORLADEYO to the NHI drug price list on April 21, 2021. This triggered a $15 million milestone payment to BioCryst from Torii Pharmaceutical Co., Ltd., the company’s commercial partner in Japan, which BioCryst received and recognized in the second quarter.
Complement Oral Factor D Inhibitor Program – BCX9930

On June 15, 2021, the company announced the designs for REDEEM-1 and REDEEM-2, two upcoming pivotal trials with its oral Factor D inhibitor, BCX9930, in patients with paroxysmal nocturnal hemoglobinuria (PNH). REDEEM-1 is a randomized, open-label, active, comparator-controlled comparison of the efficacy and safety of BCX9930 (500 mg bid) monotherapy in approximately 81 PNH patients with an inadequate response to a C5 inhibitor. REDEEM-2 is a randomized, placebo-controlled trial to evaluate the efficacy and safety of BCX9930 (500 mg bid) as monotherapy versus placebo in approximately 57 PNH patients not currently receiving complement inhibitor therapy. The primary endpoint for both trials is the change from baseline in hemoglobin, assessed at weeks 12 to 24 in REDEEM-1 and at week 12 in REDEEM-2. Trial site start-up activities are now underway at sites around the world and both pivotal trials are expected to begin enrolling patients in the second half of 2021.

In the second half of 2021, the company also plans to initiate a proof of concept trial of oral BCX9930 (500 mg bid) in renal complement-mediated diseases. The trial will be a basket study including cohorts of patients with C3 glomerulopathy, IgA nephropathy and primary membranous nephropathy.
Additional Updates

On July 28, 2021, the company announced the appointment of Vincent Milano to the BioCryst board of directors.
Second Quarter 2021 Financial Results

For the three months ended June 30, 2021, total revenues were $50.0 million, compared to $2.9 million in the second quarter of 2020. The increase was primarily due to $28.5 million in ORLADEYO net revenue in the second quarter of 2021, the recognition of a $15 million milestone payment to BioCryst from Torii Pharmaceutical Co., Ltd., the company’s commercial partner in Japan, following approval and successful pricing negotiations in Japan, and $4.6 million for RAPIVAB (peramivir injection) stockpile sales to the government, all realized in the second quarter of 2021.

Research and development expenses for the second quarter of 2021 increased to $52.9 million from $27.5 million in the second quarter of 2020, primarily due to increased investment in the development of BCX9930 as well as other research, preclinical and development costs, offset by a reduction in spend on the ORLADEYO program following our commercial launch in December 2020.

Selling, general and administrative expenses for the second quarter of 2021 increased to $26.3 million, compared to $13.9 million in the second quarter of 2020. The increase was primarily due to increased investment to support the U.S. commercial launch of ORLADEYO and expanded international operations.

Interest expense was $13.5 million in the second quarter of 2021, compared to $2.9 million in the second quarter of 2020. The increase was due to service on the royalty and debt financings which were completed in December 2020. The interest payment-in-kind (PIK) option on the Athyrium term loan has been exercised and $3.9 million has been added to the $125 million principal in the second quarter of 2021, and $7.5M since issuance.

Net loss for the second quarter of 2021 was $43.2 million, or $0.24 per share, compared to a net loss of $38.6 million, or $0.24 per share, for the second quarter of 2020.

Cash, cash equivalents, restricted cash and investments totaled $222.8 million at June 30, 2021, compared to $191.6 million at June 30, 2020. Operating cash use for the second quarter of 2021 was $22.0 million.

Financial Outlook for 2021

In the launch period for ORLADEYO, the company is not providing specific revenue or operating expense guidance. Based on our expectations for revenue, operating expenses, and our option to access an additional $75 million from our existing credit facility, we believe our current cash runway takes us into 2023.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 9886913. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 9886913.

On August 5, 2021 TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage cell therapy company with a pipeline of novel T cell therapies for patients suffering from cancer, reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, TCR2 Therapeutics, AUG 5, 2021, View Source [SID1234585908]).

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"As we enter the second half of the year, we are approaching the conclusion of the Phase 1 portion of our gavo-cel clinical trial and selection of an RP2D. We look forward to presenting safety, efficacy and translational data from at least 17 patients, up to dose level 5, in an oral presentation at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress on September 17," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "We believe that gavo-cel has the potential to significantly improve the standard of care for patients with treatment refractory mesothelin-expressing solid tumors. We are focused on identifying the RP2D before the end of 2021 to advance this program into Phase 2 where we can more definitively evaluate efficacy, including retreatment with gavo-cel and combinations with checkpoint inhibitors. We also remain committed to advancing our broad emerging pipeline, including new enhancements, allogeneic TRuC-T cells and new targets and look forward to showcasing these programs at our upcoming virtual R&D Day."

Recent Developments

Gavo-cel:

TCR2 announced today the Company plans to present new clinical data from the dose escalation portion of the Phase 1/2 clinical trial of gavo-cel in patients with treatment refractory mesothelin-expressing solid tumors as part of an oral presentation on September 17 at 14:20 CEST (8:20am EST) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 being held in-person from September 16-21, 2021. The presentation will include long-term follow-up from initial and new patients from the Phase 1 dose escalation, with data from additional non-mesothelioma patients, and will focus on safety, efficacy and translational data at dose levels 3 (1×108 cells/m2 with lymphodepletion), 4 (5×108 cells/m2 without lymphodepletion) and 5 (5×108 cells/m2 with lymphodepletion).
Corporate:

TCR2 announced today that it will host a virtual R&D Day on Wednesday, October 20, 2021 to showcase the broad emerging pipeline focusing on its enhancements, allogeneic strategies and new targets.
TCR2 announced the appointment of Peter Olagunju as its first Chief Technical Officer where he will oversee process development, manufacturing, quality control and technical operations for the Company’s TRuC-T cell programs and emerging pipeline. Previously, Mr. Olagunju was Senior Vice President of Technical Operations at FerGene, Inc. Before that, Mr. Olagunju was Vice President of Global Patient Operations at bluebird bio, Inc., where he held several roles of increasing responsibility and was the program lead and functional head of manufacturing supporting the European approval for ZYNTEGLO, a transformational gene therapy for Transfusion dependent Thalassemia.
Anticipated Milestones

TCR2 to highlight interim progress from the Phase 1 portion of the gavo-cel Phase 1/2 clinical trial for patients with mesothelin-expressing solid tumors in an abstract at the 2021 World Conference on Lung Cancer.
TCR2 to highlight additional safety, efficacy and translational data from all patients receiving therapy up to dose level 5 (second and third gavo-cel doses) in the Phase 1 portion of the gavo-cel Phase 1/2 clinical trial focused on mesothelin-expressing solid tumors in an oral presentation at the ESMO (Free ESMO Whitepaper) Congress 2021.
TCR2 to present an interim update from the Phase 1 portion of the TC-110 Phase 1/2 clinical trial for patients with CD19+ non-Hodgkin lymphoma or adult acute lymphoblastic leukemia in the second half of 2021.
TCR2 plans to file an IND for TC-510, the first enhanced TRuC-T cell (targeting mesothelin with a PD-1:CD28 switch), in the second half of 2021.
TCR2 plans to select a development candidate for its allogeneic program in the second half of 2021.
TCR2 to present preclinical data on its IL-15 enhancements program in the fourth quarter of 2021.
TCR2 to host virtual R&D Day focused on the broad emerging pipeline on October 20, 2021.
TCR2 anticipates production of clinical trial material from ElevateBio LLC and its manufacturing facility in Stevenage, UK, both in anticipation of demand from the Phase 2 expansion trial of gavo-cel, in 2022.
Financial Highlights

Cash Position: TCR2 ended the second quarter of 2021 with $317.3 million in cash, cash equivalents, and investments compared to $228.0 million as of December 31, 2020. Net cash used in operations was $15.0 million for the second quarter of 2021 compared to $16.0 million for the second quarter of 2020. TCR2 projects net cash use of $100-110 million for 2021, which includes tenant improvements to the Rockville facility. We expect cash on hand to support operations through 2023.

R&D Expenses: Research and development expenses were $18.6 million for the second quarter of 2021 compared to $12.9 million for the second quarter of 2020. The increase in R&D expenses was primarily due to an increase in headcount, additional lab facilities, and manufacturing facilities.

G&A Expenses: General and administrative expenses were $5.7 million for the second quarter of 2021 compared to $3.8 million for the second quarter of 2020. The increase in general and administrative expenses was primarily due to an increase in personnel costs and external professional fees.

Net Loss: Net loss was $24.3 million for the second quarter of 2021 compared to $16.2 million for the second quarter of 2020.
Adoption of New Lease Standard

During the second quarter of 2021, TCR2 adopted the new lease standard ASC 842 effective January 1, 2021. The lease standard requires companies to record right-of-use assets and lease liabilities for all leases. With the adoption of the new lease standard, the Company removed its facility in Rockville, MD as an asset under a built-to-suit lease in the amount of $41 million and removed the associated liabilities of $37 million. As of June 30, 2021, the Company’s right-of-use assets under operating leases, including the Rockville facility, were $30.6 million and operating lease liabilities were $27.6 million.

Upcoming Events

TCR2 Therapeutics management is scheduled to participate at the following upcoming conferences.

2021 Wedbush PacGrow Healthcare Conference: Robert Hofmeister, Ph.D., Chief Scientific Officer of TCR2 Therapeutics, will participate in a panel using a virtual platform on Tuesday, August 10, 2021 at 9:45am ET

On August 5, 2021 Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, reported that it will issue financial results for its fiscal quarter ended June 30, 2021 on Thursday, August 12, 2021 (Press release, Anavex Life Sciences, AUG 5, 2021, View Source [SID1234585907]).

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Management will host a conference call on Thursday, August 12, 2021, at 4:30 pm EDT to review financial results and describe the Company’s growth strategy. Following management’s remarks, there will be a question-and-answer session.

Webcast / Conference Call Information:

The live webcast of the conference call can be accessed online at View Source

To join the conference call, live via telephone, interested parties within the U.S. should dial, toll-free, 1 (866) 939-3921 and international callers should dial 1 (678) 302-3550. Please use confirmation number 50210582, followed by the pound sign (#).

A replay of the conference call will also be available on www.anavex.com.

On August 5, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies with its ARCUS genome editing platform, reported that it will publish financial results for the second quarter 2021 and provide a business update on August 12, 2021 (Press release, Precision Biosciences, AUG 5, 2021, View Source [SID1234585906]).

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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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On August 5, 2021 Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) reported financial results for the second quarter of 2021 (Press release, Concert Pharmaceuticals, AUG 5, 2021, View Source [SID1234585905]).

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"Our development priority is to advance CTP-543 through its pivotal trials for alopecia areata in support of a planned early 2023 New Drug Application. We remain on track with our clinical progression of CTP-543, a drug candidate with the potential to address an important unmet medical need and sizeable market opportunity," said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "Alopecia areata is a chronic autoimmune disease with no approved treatment that causes significant emotional and psychological distress in many patients. There’s an enormous need for an FDA-approved treatment, and we’re proud that Concert was one of the first in the industry to act on this important disease by advancing CTP-543, which has the potential to be a best in class treatment."

Recent Highlights and Upcoming Milestones
CTP-543: An Investigational Treatment for Moderate to Severe Alopecia Areata

Initiated CTP-543 THRIVE-AA2 Phase 3 Trial in May 2021. THRIVE-AA2, the second study in the CTP-543 pivotal program, 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 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. The Company expects to report topline results from the THRIVE-AA2 trial in the second half of 2022.
CTP-543 THRIVE-AA1 Phase 3 Trial Progressing. THRIVE-AA1, the first study in the CTP-543 pivotal program, 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 in approximately 700 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. The Company expects to report topline results from the THRIVE-AA1 trial in the first half of 2022.
CTP-543 Long-term Data Presented at JAK Inhibitors Drug Development Summit. Data from an ongoing open label, long-term extension study with CTP-543 were presented at the 2nd JAK Inhibitors Drug Development Summit on July 1, 2021. The presentation highlighted that in the extension study, treatment with CTP-543 showed continued maintenance of hair regrowth relative to the hair growth shown in the Company’s previously conducted Phase 2 trials in patients with alopecia areata. Approximately 57% of participants receiving 12 mg of CTP-543 twice-daily following 52 weeks of dosing achieved a clinically-meaningful SALT score of 20 or less. A SALT score ≤ 20 corresponds to 80% or more hair coverage on the scalp. CTP-543 has been generally well tolerated in the extension study, and adverse events are consistent with those reported in the Phase 2 trials.
September is Alopecia Areata Awareness Month. Throughout the month of September, Concert, along with the alopecia areata community, will raise awareness and recognize the importance of alopecia areata, a serious autoimmune disorder that affects approximately 1 million individuals in the U.S. and which often results in poor health-related quality of life as well as high levels of anxiety and depression. Follow our #LightItUpBlue4AlopeciaAreata campaign on Twitter at @ConcertPharma.
Second Quarter 2021 Financial Results

Cash and Investment Position. Cash, cash equivalents and investments as of June 30, 2021 totaled $122.4 million as compared to $130.0 million as of December 31, 2020. Under its current operating plan, the Company expects its cash and cash equivalents to fund the Company into the second quarter of 2022. In May 2021, Vertex purchased the potential future milestones under the companies’ 2017 asset purchase agreement relating to VX-561 for $32.0 million.
Revenues. For the quarter ended June 30, 2021, revenue was $32.0 million, compared to $6.4 million for the same period in 2020. Revenue recognized in 2021 was the result of the $32.0 million of proceeds received from Vertex. 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 $20.2 million for the quarter ended June 30, 2021, compared to $14.8 million for the same period in 2020. The increase in research and development expenses relates primarily to the clinical development program for CTP-543.
G&A Expenses. General and administrative expenses were $5.6 million for the quarter ended June 30, 2021, compared to $4.7 million for the same period in 2020. The increase in general and administrative expenses relates primarily to increased external professional service expenses and non-cash stock-based compensation.
Net Income (Loss). For the quarter ended June 30, 2021, net income applicable to common stockholders was $5.4 million, or $0.16 per share, as compared to net loss applicable to common stockholders of $13.0 million, or $0.41 per share, for the quarter ended June 30, 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 second quarter 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 of the second quarter 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.