On August 4, 2022 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the second quarter ended June 30, 2022, and provided a corporate update (Press release, BioCryst Pharmaceuticals, AUG 4, 2022, View Source [SID1234617575]).

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"With ORLADEYO firmly established in the marketplace as it steadily grows each quarter towards $1 billion in peak sales, our pipeline of other oral drugs for rare diseases and a balance sheet of nearly $500 million alongside our product revenues, BioCryst is uniquely positioned to bring multiple oral medicines for rare diseases to patients," 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

ORLADEYO net revenue in the second quarter of 2022 was $65.2 million.

In the second quarter, more new physicians prescribed ORLADEYO than any quarter since the third quarter of 2021.

Prescribing from the top 500 HAE treaters increased in the second quarter with 60 percent of those physicians now having prescribed ORLADEYO. These top 500 physicians accounted for two-thirds of new patient prescriptions in the quarter.

The majority of patients continue to be well controlled on ORLADEYO. Even as the patient base has continued to grow steadily, discontinuations in the second quarter declined to their smallest number since the third quarter of 2021.

Patients continue to have broad and rapid access to ORLADEYO with the median time for a patient to receive reimbursed product following a prescription now under three weeks.

Based on the strong performance of ORLADEYO in the first half of 2022, and the steady quarterly ORLADEYO net revenue growth the company expects in the second half of 2022, the company now expects full year 2022 ORLADEYO net revenue to be between $255 million and $265 million. The company expects peak global net ORLADEYO sales of $1 billion.
"More than a year and a half into the launch of ORLADEYO, we see steady growth and strong momentum, which we expect to continue. The majority of HAE patients are very satisfied with their experience using oral, once-daily ORLADEYO and physicians continue to consistently express their strong intent for future prescribing, which we see translating directly to our growing patient numbers quarter after quarter," said Charlie Gayer, chief commercial officer of BioCryst.

ORLADEYO: Global Updates

In the second quarter, ORLADEYO was approved in Canada and Switzerland. The company expects approvals and launches in additional countries throughout the year.

Also in the second quarter, pricing was finalized in Germany, France and Switzerland.

On June 9, 2022, the company announced it had selected Pint Pharma as its commercial partner for ORLADEYO in Latin America.
Complement Oral Factor D Inhibitor Program – BCX9930

On August 4, 2022, the company announced that the U.S. Food and Drug Administration (FDA) has lifted its partial clinical hold on the BCX9930 program. The company will resume enrollment in global clinical trials under revised protocols at a reduced dose of 400 mg twice daily of BCX9930. This includes the REDEEM-1 and REDEEM-2 pivotal trials in patients with paroxysmal nocturnal hemoglobinuria (PNH) and the RENEW proof-of-concept trial in patients with C3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (PMN).

Clinical evidence and recent laboratory studies have informed the company’s hypothesis that crystals form in the kidneys of some patients. The company believes that lowering the dose to 400 mg and ensuring adequate hydration will dilute the concentration of drug in the urine below the threshold where crystals can form.

The company’s goal is to find a safe and effective dose for BCX9930. The company expects this can be accomplished in a reasonable time frame after resuming enrollment, in a relatively small number of patients given the rate and timing of the serum creatinine rises in patients prior to the enrollment pause.

If successful, the company plans to invest more significantly in BCX9930 to tap the full potential of reaching many patients suffering from a number of alternative pathway diseases, and, if not successful, the company will stop investment in BCX9930 and move on to other molecules in the pipeline.
Additional Updates

On April 27, 2022, the company announced the European Medicines Agency (EMA) had granted PRIME designation to BioCryst’s ALK-2 inhibitor, BCX9250, for the treatment of fibrodysplasia ossificans progressiva.

The EMA also has recently granted orphan drug designation and the FDA has granted fast track status for BCX9250.
Second Quarter 2022 Financial Results

For the three months ended June 30, 2022, total revenues were $65.5 million, compared to $50.0 million in the second quarter of 2021 (+31 percent year-over-year (y-o-y)). The increase was primarily due to $65.2 million in ORLADEYO net revenue in the second quarter of 2022, compared to $28.5 million in ORLADEYO net revenue in the second quarter of 2021 (+129 percent y-o-y). The $65.2 million of ORLADEYO net revenue in the second quarter of 2022 included approximately $2.2 million of non-repeating reimbursement related accrual releases.

Research and development expenses for the second quarter of 2022 increased to $62.0 million from $52.9 million in the second quarter of 2021 (+17 percent y-o-y), primarily due to increased investment in the development of our Factor D program, including BCX9930, as well as other research, preclinical and development costs.

Selling, general and administrative expenses for the second quarter of 2022 increased to $38.0 million, compared to $26.3 million in the second quarter of 2021 (+44 percent y-o-y). The increase was primarily due to increased investment to support the commercial launch of ORLADEYO and expanded international operations.

Interest expense was $24.0 million in the second quarter of 2022, compared to $13.5 million in the second quarter of 2021 (+78 percent y-o-y). The increase was due to service on the royalty financings, which were completed in November 2021.

Net loss for the second quarter of 2022 was $58.9 million, or $0.32 per share, compared to a net loss of $43.2 million, or $0.24 per share, for the second quarter of 2021.

Cash, cash equivalents, restricted cash and investments totaled $418.9 million at June 30, 2022, compared to $222.8 million at June 30, 2021. Operating cash use for the second quarter of 2022 was $27.9 million.

Additionally, on July 29, 2022, having achieved all required revenue-based milestones, the company drew the available $75 million tranche under its existing credit facility from Athyrium Capital Management. On a pro-forma basis, net of fees, this results in pro-forma cash of approximately $492 million.

Financial Outlook for 2022

Based on the strength of the ORLADEYO launch through the first half of 2022, and the continued steady growth from new patient demand the company expects for the remainder of the year, the company expects full year 2022 net ORLADEYO revenue to be between $255 million and $265 million.

Based on the reduced spending on the BCX9930 program in the first half of the year, and lower than projected spending on the program for the remainder of the year, the company now expects operating expenses for full year 2022, not including non-cash stock compensation, to be between $390 million and $400 million.

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 866-374-5140 for domestic callers and 404-400-0571 for international callers and using conference ID 68509725#. 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 replay of the call will be available on the company website.

On August 4, 2022 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported financial results for the second quarter ended June 30, 2022 and provided a corporate update (Press release, Jounce Therapeutics, AUG 4, 2022, View Source [SID1234617574]).

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"Jounce has made significant progress advancing our pipeline, and we look forward to sharing more details on our two ongoing clinical trials, INNATE and SELECT, later this year. Today, we are pleased to announce that we’ve completed enrollment in our randomized Phase 2 biomarker-selected trial SELECT, and plan to submit a clinical data abstract later this year to ESMO (Free ESMO Whitepaper)-IO being held in December. In addition, we expect to provide preliminary clinical and biomarker data across multiple cohorts from the Phase 2 portion of INNATE with an abstract submission to ESMO (Free ESMO Whitepaper)-IO in December. Our current financial position enables our continued growth and execution beyond the proof-of-concept inflection points of INNATE and SELECT, while continuing our robust, novel discovery efforts," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "We continue to focus on our mission of delivering meaningful and long-lasting benefit to cancer patients through the discovery and pursuit of therapies that target new mechanisms of immune suppression across different types of immune cells, and bringing the right immunotherapies to the right patients."

Pipeline Update & Highlights:

JTX-8064 (LILRB2/ILT4)

Continued to advance INNATE Phase 2 trial across 7 indications. Jounce is evaluating JTX-8064 in the ongoing Phase 2 portion of the INNATE clinical trial, which is comprised of indication specific expansion cohorts, including one monotherapy cohort and seven cohorts in combination with pimivalimab. Each combination cohort is a Simon’s 2-stage design, enrolling 10 patients for an initial analysis of response data, and then further expansion to a total of 29 patients if prespecified response criteria are met. Last quarter, Jounce announced the expansion of the first two combination cohorts. The expanded indications are 3rd/4th line PD-(L)1 inhibitor naive platinum-resistant ovarian cancer and 2nd/3rd line PD-(L)1 inhibitor resistant clear cell renal cell carcinoma. Having met the response criteria for expansion, both indications are continuing enrollment to 29 patients each. Jounce has seen an acceptable safety profile for both the monotherapy and combination therapy to date.

On track to report preliminary clinical data before year end. Jounce plans to submit a clinical abstract on preliminary clinical data including all 31 Phase 1 dose escalation patients and at least 80 Phase 2 combination treatment patients from INNATE, to the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Immuno-Oncology Congress 2022 being held from December 7-9 in Geneva, Switzerland. Phase 2 data will include safety, preliminary efficacy based on RECIST 1.1, pharmacodynamics, and potential predictive biomarker correlation with efficacy within each cohort, by prior PD-(L)1 inhibitor history, and in a cross-cohort analysis.
Vopratelimab (ICOS) and Pimivalimab (PD-1)

Patient enrollment complete in Phase 2 SELECT trial of vopratelimab. Patient enrollment is complete in the SELECT trial, a randomized Phase 2 trial evaluating vopratelimab, Jounce’s inducible costimulator (ICOS) agonist, in combination with pimivalimab versus pimivalimab alone in immunotherapy naïve, TISvopra biomarker-selected, second line non-small cell lung cancer (NSCLC) patients.

On track to report clinical data before year end. Jounce plans to submit a clinical data abstract on the SELECT trial, including additional single agent data for pimivalimab, to the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress 2022.

First-in-human manuscript from the ICONIC trial in Clinical Cancer Research. A manuscript was recently published in the journal Clinical Cancer Research from the Phase 1/2 ICONIC trial of vopratelimab alone and in combination with nivolumab in patients with advanced solid tumors. Data from ICONIC identified TISvopra as a potential predictive biomarker currently being investigated in SELECT.
JTX-1484 (LILRB4/ILT3)

Continued advancement of JTX-1484. JTX-1484 is the most recent product to emerge from Jounce’s Translational Science Platform and is a monoclonal antibody designed to block human LILRB4 (ILT3) expressed on myeloid cells in the tumor microenvironment with the potential to reduce immune suppression and enhance T cell functionality. JTX-1484 is currently in investigational new drug (IND) enabling activities, with the goal of filing an IND application in 2023. Jounce has submitted a preclinical abstract on JTX-1484 for consideration at this year’s annual Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting being held from November 9-11 in Boston, MA.
Discovery Pipeline

LILRB family preclinical data abstract submitted to SITC (Free SITC Whitepaper). Jounce has submitted a LILRB family preclinical data abstract for consideration at this year’s SITC (Free SITC Whitepaper) annual meeting.

Productive discovery engine with the goal of an IND every 12 to 18 months. Jounce continues to invest in and advance its growing immuno-oncology pipeline. Its discovery engine is built upon the capability to thoroughly interrogate different cell types in the tumor microenvironment, including T cells and myeloid cells. This approach has resulted in four clinical stage programs, with a fifth in IND enabling studies, over the last 6 years.
Corporate Update:

Updates regarding the Board of Directors. Jigar Raythatha has become the Chair of the Board, replacing Perry Karsen who has served as the Chair since April of 2016. Perry Karsen will remain as a member of the board.

Key promotions across leadership team. Jounce is announcing that Hugh Cole is being promoted from Chief Business Officer to Chief Operating Officer, and Dr. Haley Laken is being promoted from Senior Vice President of Program and Portfolio Strategy to Chief Development Officer. Both Mr. Cole and Dr. Laken are impactful leaders within Jounce and Jounce congratulates them on their respective promotions.
Second Quarter 2022 Financial Results:

Cash position: As of June 30, 2022, cash, cash equivalents and investments decreased to $162.3 million, compared to $220.2 million as of December 31, 2021. The decrease was due to operating expenses incurred during the period.

License and collaboration revenue: Jounce did not recognize any revenue during the second quarter of 2022. License and collaboration revenue of $25.4 million was recognized during the second quarter of 2021 and was comprised of a $25.0 million clinical development and regulatory milestone for FDA clearance of the IND for GS-1811 and $0.4 million related to non-cash revenue for the performance of research and transition services, both under the Gilead License Agreement.

Research and development expenses: Research and development expenses were $26.2 million for the second quarter of 2022, compared to $22.1 million for the same period in 2021. The increase in research and development expenses was primarily due to increased manufacturing activities performed for Jounce’s development programs, increased clinical and regulatory costs for INNATE, and increased payroll and lab supplies.

General and administrative expenses: General and administrative expenses were relatively flat at $7.5 million for the second quarter of 2022, compared to $7.3 million for the same period in 2021.

Net loss: Net loss was $33.5 million for the second quarter of 2022, resulting in basic and diluted net loss per share of $0.65. Net loss was $4.0 million for the same period in 2021, resulting in a basic and diluted net loss per share of $0.08. The increase in net loss is attributable to increased operating expenses and no revenue recognized under the Gilead License Agreement in the second quarter of 2022.
Financial Guidance:

Based on its current operating and development plans and cost containment efforts, Jounce is updating its financial guidance for 2022. Gross cash burn on operating expenses and capital expenditures for the full year 2022 is now expected to be at the lower end of the range of $115.0 million to $130.0 million. Jounce now expects its existing cash, cash equivalents and investments to be sufficient to enable the funding of its operating expenses and capital expenditure requirements into the first quarter of 2024.

Conference Call and Webcast Information:

Jounce Therapeutics will host a live conference call and webcast today at 8:00 a.m. ET. To access the conference call, participants may register here1.It is advised to register at least 10 minutes prior to joining the call. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of the company’s website at www.jouncetx.com. The webcast will be archived and made available for replay on the company’s website approximately two hours after the call and will be available for 30 days thereafter.

On August 4, 2022 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapies based on its proprietary arenavirus platform, reported that it will release its second quarter 2022 financial results and Company update before the market open on Thursday, August 11, 2022 (Press release, Hookipa Biotech, AUG 4, 2022, View Source [SID1234617573]).

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The Company will not be conducting a conference call in conjunction with this earnings release.

On August 4, 2022 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported financial results for the second quarter ended June 30, 2022 and provided a corporate update (Press release, Arvinas, AUG 4, 2022, View Source [SID1234617572]).

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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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"This is an exciting time at Arvinas as we remain on track to initiate two pivotal programs with ARV-471 in metastatic breast cancer in the second half of this year, have a clear development pathway for bavdegalutamide in molecularly defined metastatic castration-resistant prostate cancer, and are preparing to initiate our Phase 2 trial with ARV-766 by end of year," said John Houston, Ph.D., president and chief executive officer at Arvinas. "In June, we had a collaborative meeting with the U.S. Food and Drug Administration to gain alignment on the registrational trial design for bavdegalutamide. Based on our goal of advancing this important potential treatment for prostate cancer as quickly as possible, the promising clinical data we’ve seen to date, and a productive meeting with the Agency, we have decided to move directly into a pivotal Phase 3 trial. If successful, we think this approach may lead to the fastest potential path to availability for patients in need, both inside and outside the United States, and who are most likely to benefit from this innovative therapy."

Business Highlights and Recent Developments

Arvinas met with the U.S. Food and Drug Administration (FDA) to discuss the potential for an accelerated approach for bavdegalutamide based on a single arm Phase 2 trial and a confirmatory Phase 3 trial for full approval. FDA provided advice on the proposed registrational trial design, including patient eligibility, study design, and follow-up expectations.
After considering multiple factors, including input provided by the FDA, Arvinas believes a randomized, pivotal Phase 3 trial provides the best opportunity to enable simultaneous global regulatory submissions.
The planned Phase 3 trial will evaluate the safety and efficacy of bavdegalutamide in patients with AR T878X/875Y tumor mutations and is expected to initiate in 2H 2023.
Final dose selection for the planned Phase 3 trial will be based on data from additional patients enrolled in the ongoing Phase 1/2 trial, which the company expects will confirm 400 mg as the Phase 3 dose. This further dose exploration will run concurrently with the company seeking regulatory guidance on final Phase 3 design.
Arvinas finalized an agreement with Foundation Medicine to develop the FoundationOneLiquid CDx as a companion diagnostic for use with bavdegalutamide.
Arvinas launched the Global Early Career Researcher Award, which seeks to recognize the efforts of up-and-coming researchers bringing innovation, new approaches, and creative thinking to advance the field of targeted protein degradation.
Arvinas appointed John Northcott to newly created position of Chief Commercial Officer, effective August 1, 2022.
Anticipated Upcoming Milestones and Expectations

ARV-471

Present data from the VERITAC Phase 2 expansion trial (200 mg and 500 mg) (4Q 2022)
Initiate two Phase 3 trials in patients with metastatic breast cancer (as monotherapy and in combination) (2H 2022)
Initiate a Phase 1b combination trial with cyclin dependent kinase (CDK) inhibitors or other targeted therapies (2H 2022)
Initiate a Phase 1b combination trial with everolimus (2H 2022)
Initiate a Phase 2 neoadjuvant trial in patients with early breast cancer (2H 2022)
Present safety data from the Phase 1b combination trial with palbociclib at a medical conference (1H 2023)
Bavdegalutamide (ARV-110)

Initiate a Phase 3 trial in metastatic castration-resistant prostate cancer, or mCRPC, for patients with AR T878/H875 tumor mutations (2H 2023)
ARV-766

Share Phase 1 dose escalation trial data in mCRPC (2H 2022)
Initiate Phase 2 expansion trial in mCRPC (2H 2022)
Financial Guidance
Based on its current operating plane, Arvinas believes its cash, cash equivalents, restricted cash and marketable securities as of June 30, 2022 is sufficient to fund planned operating expenses and capital expenditure requirements multiple years beyond 2024.

Second Quarter Financial Results

Cash, Cash Equivalents, Restricted Cash and Marketable Securities Position: As of June 30, 2022, cash, cash equivalents, restricted cash and marketable securities were $1,347.7 million as compared with $1,507.1 million as of December 31, 2021. The decrease in cash, cash equivalents, restricted cash and marketable securities of $159.4 million for the first six months of 2022 was primarily related to cash used in operating activities of $141.5 million (net of $6.5 million received from two collaborators), unrealized loss on marketable securities of $17.4 million, and the purchase of lab equipment and leasehold improvements of $3.5 million, partially offset by proceeds from the exercise of stock options of $3.0 million.

Research and Development Expenses: Research and development expenses were $75.3 million for the quarter ended June 30, 2022, as compared with $43.0 million for the quarter ended June 30, 2021. The increase in research and development expenses of $32.3 million for the quarter was primarily due to an increase in our continued investment in our platform and exploratory programs of $19.6 million, as well as an increase in expenses related to our ER program of $14.1 million, which is net of the cost sharing of ARV-471 under the global Pfizer collaboration agreement to develop and commercialize ARV-471 that was initiated in July 2021 (ARV-471 Collaboration Agreement), offset by a decrease in our AR program (which includes bavdegalutamide and ARV-766) of $1.4 million.

General and Administrative Expenses: General and administrative expenses were $24.3 million for the quarter ended June 30, 2022, as compared with $14.4 million for the quarter ended June 30, 2021. The increase of $9.9 million was primarily due to an increase in personnel and facility related costs of $6.9 million and insurance, taxes and professional fees of $2.9 million.

Revenues: Revenues were $31.3 million for the quarter ended June 30, 2022 as compared with $5.5 million for the quarter ended June 30, 2021. Revenue is related to the ARV-471 Collaboration Agreement, the license and rights to technology fees and research and development activities related to the collaboration and license agreement with Bayer that was initiated in July 2019, the collaboration and license agreement with Pfizer that was initiated in January 2018, and the amended and restated option, license and collaboration agreement with Genentech that was initiated in November 2017. The increase in revenues of $25.8 million was primarily due to revenue from the ARV-471 Collaboration Agreement.

Income Tax Expense: Income tax expense was $3.4 million for the quarter ended June 30, 2022, as compared with zero for the quarter ended June 30, 2021 due to taxable income projected for fiscal year 2022 primarily related to revenue recognized in 2022 for tax purposes from the ARV-471 Collaboration Agreement.

Net Loss: Net loss was $70.0 million for the quarter ended June 30, 2022, as compared with $50.3 million for the quarter ended June 30, 2021. The increase in net loss for the quarter was primarily due to increased research and development expenses, general and administrative expenses, and income tax expense, partially offset by increased revenue.

About bavdegalutamide (ARV-110)
Bavdegalutamide (ARV-110) is an investigational orally bioavailable PROTAC protein degrader designed to selectively target and degrade the androgen receptor (AR). Bavdegalutamide is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer.

Bavdegalutamide has demonstrated activity in preclinical models of AR mutation or overexpression, both common mechanisms of resistance to currently available AR-targeted therapies.

About ARV-471
ARV-471 is an investigational orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer.

In preclinical studies, ARV-471 demonstrated near-complete ER degradation in tumor cells, induced robust tumor shrinkage when dosed as a single agent in multiple ER-driven xenograft models, and showed superior anti-tumor activity when compared to a standard of care agent, fulvestrant, both as a single agent and in combination with a CDK4/6 inhibitor. In July 2021, Arvinas announced a global collaboration with Pfizer for the co-development and co-commercialization of ARV-471; Arvinas and Pfizer will equally share worldwide development costs, commercialization expenses, and profits.

About ARV-766
ARV-766 is an investigational orally bioavailable PROTAC protein degrader designed to selectively target and degrade AR. In preclinical studies, ARV-766 degraded all resistance-driving point mutations of AR, including L702H, a mutation associated with treatment with abiraterone and other AR-pathway therapies.

ARV-766 is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer, and ARV-766 may also have applicability in other AR-driven diseases both in and outside oncology. ARV-766 has demonstrated activity in preclinical models of resistance to currently available AR-targeted therapies.

On August 4, 2022 Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) reported financial results for the second quarter of 2022 (Press release, Concert Pharmaceuticals, AUG 4, 2022, View Source [SID1234617571]).

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"We are very happy to have seen consistent efficacy and safety results across our two Phase 3 clinical trials of CTP-543 in alopecia areata. The positive results that we reported from THRIVE-AA1 in May and from THRIVE-AA2 earlier this week, will form the basis of our New Drug Application, which we expect to submit to the FDA in the first half of 2023," said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "We have continued to execute well on a development program for CTP-543 that provides a robust data package in support of a potential FDA approval. We are proud that CTP‑543 could offer a near-term and potentially best-in-class treatment option for adults with moderate to severe alopecia areata."

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

Positive Phase 3 Results Reported for CTP-543 THRIVE-AA1 Study. In May 2022, the Company reported positive topline results for its first CTP-543 Phase 3 clinical trial. A statistically significant proportion of patients treated with either 8 mg twice-daily or 12 mg twice-daily of CTP-543 in the THRIVE-AA1 study experienced greater scalp regrowth compared to placebo. The proportion of patients achieving a Severity of Alopecia Tool (SALT) score of 20 or less (meaning 20 percent or less scalp hair loss) at Week 24 was 41.5 percent in the 12 mg twice-daily dose group and 29.6 percent in the 8 mg twice-daily dose group, compared to 0.8 percent of patients in the placebo group. The treatment difference for both dose groups of CTP-543 relative to placebo was statistically significant (p<0.0001). The safety profile seen with CTP-543 in THRIVE-AA1 was consistent with previous studies of CTP-543.
Positive Phase 3 Results Reported for CTP-543 THRIVE-AA2 Study. In August 2022, the Company reported positive topline results for its second CTP-543 Phase 3 clinical trial. A statistically significant proportion of patients treated with either 8 mg twice-daily or 12 mg twice-daily of CTP-543 in the THRIVE-AA2 study experienced greater scalp regrowth compared to placebo. The proportion of patients achieving a SALT score of 20 or less at Week 24 was 38.3 percent in the 12 mg twice-daily dose group and 33.0 percent in the 8 mg twice-daily dose group, compared to 0.8 percent of patients in the placebo group. The treatment difference for both dose groups of CTP-543 relative to placebo was statistically significant (p<0.0001). The safety profile seen with CTP-543 in THRIVE-AA2 was consistent with previous studies of CTP-543.
JAAD Publication Highlights Significant Reduction in Severity of Hair Loss with CTP-543. The Company recently published safety and efficacy data from its randomized, double-blind, placebo-controlled dose-ranging Phase 2 clinical trial of CTP‑543 in the Journal of the American Academy of Dermatology (JAAD). The publication reported clinically meaningful and statistically significant scalp hair regrowth after 24 weeks of treatment with CTP-543 in both the 8 mg twice-daily and 12 mg twice-daily dose groups in patients with alopecia areata, as well as safety data and patient-reported outcomes of improvement. This trial supported the advancement of CTP-543 into Phase 3 development.
Concert to Participate in Alopecia Areata Awareness Month in September. 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 up to approximately 1.5 million individuals in the U.S. and which often results in poor health-related quality of life as well as high incidence of anxiety and depression. Follow our #LightItUpBlue4AlopeciaAreata campaign on Twitter at @ConcertPharma.
Second Quarter 2022 Financial Results

Cash and Investment Position. Cash, cash equivalents and investments as of June 30, 2022 totaled $153.7 million as compared to $141.6 million as of December 31, 2021. Under its current operating plan, the Company expects its cash, cash equivalents and investments to fund the Company into the second quarter of 2023. In June 2022, Concert closed an equity offering raising gross proceeds of $54.6 million before underwriting discounts and offering expenses. Concurrent with the initial closing of the offering, Concert received $18.9 million from the partial exercise of the Tranche 1 warrants issued to BVF Partners L.P. and RA Capital Management in connection with its November 2021 financing. The Company has the potential to receive an additional $70.1 million in 2022 upon full exercise of the remaining warrants issued in connection with the November 2021 financing.
R&D Expenses. Research and development expenses were $20.9 million for the quarter ended June 30, 2022, compared to $20.2 million for the same period in 2021. 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 $4.8 million for the quarter ended June 30, 2022, compared to $5.6 million for the same period in 2021. The decrease in general and administrative expenses relates primarily to decreased non-cash stock-based compensation.
Net (Loss) Income. For the quarter ended June 30, 2022, net loss applicable to common stockholders was $24.0 million, or $0.59 per share, as compared to net income applicable to common stockholders of $5.4 million, or $0.16 per share, for the quarter ended June 30, 2021. Net income for the quarter ended June 30, 2021 included $32.0 million in revenue from proceeds received from Vertex Pharmaceuticals, Inc. for the purchase of potential future milestones under the companies’ 2017 asset purchase agreement related to VX-561.
Conference Call and Webcast: New System to Access Call Live and On Demand
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.

Please note that there is a new system to access the live call in order to ask questions. To join the live call, please register here. A dial in and unique PIN number will be provided to join the call.

An audio-only webcast of the call may be accessed in the Investors section of the Company’s website at www.concertpharma.com. A replay of the webcast will be available on Concert’s website for three months.