On May 5, 2022 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the first quarter ended March 31, 2022 and reiterated full year 2022 product revenue guidance (Press release, Vertex Pharmaceuticals, MAY 5, 2022, View Source [SID1234613793]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"Following upon our success in transforming the treatment of cystic fibrosis, Vertex’s unique and differentiated R&D strategy continues to deliver with positive Phase 2 proof-of-concept studies in multiple disease areas, and another wave of therapies set to enter the clinic in the second half of this year," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "With yet another quarter of strong revenue performance characterized by 22% year-over-year growth, we are well positioned for continued innovation and sustained growth as we work to bring additional transformative medicines to more patients around the globe."

Starting in the first quarter of 2022, Vertex no longer excludes research and development charges resulting from upfront or contingent milestone payments in connection with collaborations, asset acquisitions and/or licensing of third-party intellectual property rights from its Non-GAAP financial measures. Non-GAAP financial measures for the first quarter of 2021 have been recast to reflect this change.

Product revenues increased 22% to $2.10 billion compared to the first quarter of 2021, primarily driven by the strong launches of TRIKAFTA/KAFTRIO in multiple countries internationally and the strong performance of TRIKAFTA in the U.S., including the June 2021 launch of TRIKAFTA in children 6-11 years old in the U.S. Net product revenues in the first quarter of 2022 increased 9% to $1.37 billion in the U.S. and increased 55% to $729 million outside the U.S., compared to the first quarter of 2021.

GAAP and Non-GAAP net income increased compared to the first quarter of 2021, driven by strong product revenue growth.

Cash, cash equivalents and marketable securities as of March 31, 2022 were $8.2 billion, an increase of approximately $0.7 billion compared to December 31, 2021. The increase was primarily driven by strong revenue growth and operating cash flow.

Starting in the first quarter of 2022, Vertex no longer excludes research and development charges resulting from upfront or contingent milestone payments in connection with collaborations, asset acquisitions and/or licensing of third-party intellectual property rights from its Non-GAAP financial measures.

Key Business Highlights

Cystic Fibrosis (CF) Marketed Products

Vertex anticipates the number of CF patients treated with our medicines will continue to grow as the uptake of TRIKAFTA in the U.S. and the launches of KAFTRIO outside the U.S. continue, and as we enter into additional reimbursement agreements and achieve new approvals for the treatment of younger patients. Recent progress includes:

Health Canada granted marketing authorization for TRIKAFTA in children 6 to 11 years of age. With this approval, approximately 500 children with CF are newly eligible for treatment with a CFTR modulator.
Vertex signed a reimbursement agreement with the Australian Pharmaceutical Benefits Scheme for TRIKAFTA (ivacaftor/tezacaftor/elexacaftor) for the treatment of patients with cystic fibrosis 12 years and older with at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. With this agreement, approximately 700 people in Australia will have access to a CFTR modulator therapy for the first time.
Vertex completed enrollment in the Phase 3 study of TRIKAFTA/KAFTRIO in children 2 to 5 years old. The Company anticipates filing a supplementary new drug application (sNDA) with the FDA before the end of 2022.
In March, Vertex filed an sNDA with the FDA for ORKAMBI for the use of ORKAMBI in children 12 months to less than 24 months old. Vertex intends to submit regulatory filings in Europe in Q2 2022.
In January, the European Commission and the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) approved a label extension for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of CF in children ages 6 through 11 years old who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. With these approvals, approximately 1,900 children are newly eligible for KAFTRIO.
In 2021, Vertex presented the first long-term follow-up data for TRIKAFTA, demonstrating no loss in mean lung function at 96 weeks, a first for any CFTR modulator to date in people with F/F and F/MF mutations. Vertex has now completed a comparison of long-term data in TRIKAFTA patients to matched untreated controls, and will present these data at an upcoming medical congress.
TRIKAFTA/KAFTRIO is now approved and reimbursed or accessible in more than 25 countries.

R&D pipeline

Vertex is delivering on a diversified pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is summarized below.

Cystic Fibrosis

Vertex continues to pursue next-in-class, small molecule CFTR modulator therapies as well as new treatment options for the approximately 5,000 patients who cannot benefit from CFTR modulators alone.

Vertex is conducting two Phase 3 global, randomized, double-blind, active-controlled clinical trials (SKYLINE 102 and SKYLINE 103) evaluating Vertex’s new once-daily investigational triple combination of VX-121/tezacaftor/VX-561 in patients with CF. The SKYLINE 102 and SKYLINE 103 trials will compare the efficacy and safety of VX-121/tezacaftor/VX-561 to TRIKAFTA. More than 180 sites across both studies are open and enrolling, and enrollment in both trials is expected to be completed in late 2022 or early 2023.
In collaboration with Moderna, Vertex is developing CF mRNA therapeutics designed to treat the underlying cause of CF by programming cells in the lungs to produce functional CFTR protein for the treatment of the approximately 5,000 people with CF who do not produce any CFTR protein. IND-enabling studies have been completed, and Vertex is on track to submit an IND for this program in 2H 2022.
Beta Thalassemia and Sickle Cell Disease (SCD)

The CTX001 program employs a non-viral ex vivo CRISPR gene-editing therapy, which is being developed as a potential functional cure for transfusion-dependent thalassemia (TDT) and severe sickle cell disease (SCD). Vertex is developing CTX001 in collaboration with CRISPR Therapeutics.

Enrollment is complete in the ongoing Phase 3 clinical trials in TDT and SCD, with more than 75 patients dosed to date. Vertex anticipates presenting updated data from the clinical trials, with more patients and longer follow-up, at medical conferences in 2022.
Two new Phase 3 studies of CTX001 were initiated in pediatric patients with TDT and SCD.
Vertex plans to submit global regulatory filings for CTX001 in TDT and SCD in late 2022.
APOL1-Mediated Kidney Disease (AMKD)

Vertex has discovered multiple oral, small molecule inhibitors of APOL1 function, pioneering a new class of medicines that target an underlying genetic driver of kidney disease.

In March, Vertex initiated pivotal development of VX-147 in a single Phase 2/3 study in patients with AMKD with two APOL1 mutations and proteinuric kidney disease.
This Phase 2/3 adaptive study will first evaluate two doses of VX-147 to select a dose for Phase 3 and subsequently evaluate the efficacy and safety of the single, selected dose in the Phase 3 portion of the study. The primary efficacy endpoint for the final analysis is eGFR slope in patients receiving the VX-147 selected dose compared to placebo at two years. The study is designed to have a pre-planned interim analysis at Week 48 evaluating eGFR slope, supported by a percent change from baseline in proteinuria in the VX-147 arm versus placebo. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval of VX-147 in the U.S. for patients with AMKD.
Pain (NaV1.8)

Vertex has discovered multiple selective small molecule inhibitors of NaV1.8 with the objective of creating a new class of pain medicines that have the potential to provide effective pain relief, without the limitations of opioids.

In March, Vertex reported positive data from two Phase 2 dose ranging acute pain studies with VX-548, one following bunionectomy surgery and the other following abdominoplasty surgery. Both studies met their primary endpoint and established proof of concept for VX-548.
Vertex plans to advance VX-548 into pivotal development in acute pain in the second half of 2022, following discussions with regulators.
Type 1 Diabetes (T1D)

Vertex is evaluating cell therapies using stem cell-derived islets to replace the endogenous insulin-producing islet cells that are destroyed in people with T1D with the goal of developing a potential functional cure for this disease.

VX-880 is a stem cell-derived, fully differentiated islet replacement therapy, used in combination with standard immunosuppression to protect the implanted cells. VX-880 is being evaluated in a Phase 1/2 clinical trial for the treatment of T1D.
This program has been placed on clinical hold in the U.S. by the FDA, based on their determination of insufficient information for dose escalation with the product. Vertex is working collaboratively and with urgency to understand and address the FDA’s questions.
Vertex previously announced:
The first patient, who received a half dose of VX-880, is insulin-independent with an HbA1C of 5.2% at Day 270.
The second patient, who also received a half dose of VX-880, demonstrated restoration of glucose-responsive insulin production and significant improvement in glycemic control with reductions in exogenous insulin requirements.
Taken together, results in the first two patients establish proof-of-concept for VX-880 in the treatment of T1D. Per protocol, the Independent Data Monitoring Committee reviewed the totality of the safety data from the first two patients dosed and recommended advancement to Part B of the study, and treatment with the full target dose.
The third patient treated with VX-880 received the full target dose and has reached the Day 29 follow-up milestone.
Across the program, VX-880 has been generally well tolerated to date. There have been no serious adverse events (SAEs) considered related to VX-880. The majority of adverse events (AEs) were mild or moderate in all patients treated to date. The safety profile was generally consistent with the immunosuppressive regimen used in the study and the perioperative period.
Vertex is continuing to advance additional programs in T1D, in which these same stem cell-derived islets are encapsulated and implanted in an immunoprotective device or modified to produce hypoimmune stem cell islets with the goal of eliminating the need for immunosuppression.
Vertex is on track to submit an IND for the cells plus device program in 2022.
Vertex expects to share additional data for VX-880 at medical conferences this year.

Alpha-1 Antitrypsin (AAT) Deficiency

Vertex is working to address the underlying genetic cause of alpha-1 antitrypsin (AAT) deficiency by developing novel small molecule correctors of Z-AAT protein folding, with a goal of increasing the secretion of functional AAT into the blood and addressing both the lung and the liver aspects of AAT deficiency.

Vertex is on track to advance one or more novel small molecule Z-AAT correctors into the clinic in 2022.
Duchenne Muscular Dystrophy (DMD)

Vertex is investigating a novel approach to treating DMD by delivering CRISPR/Cas9 gene-editing technology to muscle cells with the goal of restoring near-full length dystrophin protein expression by targeting specific mutations in the dystrophin gene that cause the disease.

Vertex has advanced its first in vivo gene editing therapy for DMD into IND-enabling studies.
Consistent with its overall strategy, Vertex takes a portfolio approach to all of its programs, with additional assets in CF, SCD, Beta Thalassemia, AMKD, T1D, Pain, and AATD in earlier stages of development.

Non-GAAP Financial Measures

In this press release, Vertex’s financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex’s pre-tax income (i) stock-based compensation expense, (ii) gains or losses related to the fair value of the company’s strategic investments, (iii) increases or decreases in the fair value of contingent consideration, (iv) acquisition-related costs and (v) other adjustments. The company’s non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company’s business, are important in comparing current results with prior period results and provide additional information regarding the company’s financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company’s business and to evaluate its performance. The company’s calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information.

The company provides guidance regarding combined R&D and SG&A expenses and effective tax rate on a non-GAAP basis. The guidance regarding combined GAAP and non-GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development transactions, including collaborations, asset acquisitions and/or licensing of third-party intellectual property rights. The company does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material.

On May 5, 2022 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a clinical-stage biopharmaceutical company dedicated to the development of innovative therapies designed to treat or reverse disease, reported financial results for the three months ended March 31, 2022 and provided a business update (Press release, Caladrius Biosciences, MAY 5, 2022, View Source [SID1234613840]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"During the quarter, we continued to advance our CD34+ cell therapy development pipeline with the initiation of the proof-of-concept study for CLBS201 in diabetic kidney disease. However, the most important achievement was the culmination, after the close of the quarter, of our efforts to diversify and expand our development portfolio, which resulted in the recently announced signing of a merger agreement with Cend Therapeutics," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "This transaction will be transformational for Caladrius, creating, upon closing, a financially sound Nasdaq-listed company with a diverse product development pipeline, strong existing partnerships and potential for future attractive collaborations. The merged company will operate under the name of Lisata Therapeutics ("Lisata") with a primary focus on exploiting the full potential of Cend’s CendR Platform technology in a range of solid tumor oncology indications. CEND-1, the lead product candidate from the CendR Platform, has the potential to be combined with a myriad of chemo and immunotherapeutic agents and nanoparticle technology that could become an integral part of a revised standard of care therapy for many difficult to treat cancers. The collaboration with Cend will allow the Caladrius team to leverage its broad development expertise and experience, specifically in oncology, with the goal of rapidly progressing Lisata’s product development candidates toward global registrations. We couldn’t be more excited and motivated about the prospects that this merger will bring for patients and shareholders."

Business, Product Development and Financing Highlights

Subsequent to the close of the first quarter of 2022, the Company announced that it has entered into a definitive agreement to merge with Cend Therapeutics, Inc. ("Cend"), a privately-held, clinical-stage biotechnology company focused on a novel approach to enable more effective treatments for solid tumor cancers, under which Cend will merge with a wholly owned subsidiary of Caladrius in an all-stock approximate "merger of equals" transaction unanimously approved by the Boards of Directors of each company. Following closing, the combined company will be renamed Lisata Therapeutics, Inc. ("Lisata") and is expected to trade on the Nasdaq Capital Market under the ticker symbol "LSTA". The merger is currently expected to close in the third quarter of 2022 subject to the approval of Caladrius and Cend stockholders as well as the satisfaction of certain other customary closing conditions and applicable approvals. In the interim, Caladrius has made an investment of $10 million in Cend in connection with a development collaboration agreement to maintain development momentum of the Cend pipeline.

HONEDRA (CLBS12) for the treatment of critical limb ischemia ("CLI")

HONEDRA is the Company’s SAKIGAKE-designated product candidate for the treatment of CLI and Buerger’s disease in Japan for which, as previously announced, the Company suspended enrollment of its registration eligible trial, CLBS12-P01, and turned its focus to securing a Japanese partner to either complete study enrollment of the four remaining patients, if necessary, and/or to explore the possibility of submitting the existing data to the Japanese Regulatory Authorities for registration review. This decision was motivated by the Company’s desire to minimize additional operational and financial burden caused by enrollment delays and the lack of visibility on time to completion of the current study. The Company expects to receive guidance from the Pharmaceuticals and Medical Devices Agency ("PMDA") in Japan during the second or third quarter of 2022 on the next steps of development. In an upcoming clinical pre-consultation meeting, topline results from the CLBS12-P01 study will be presented and discussed with the PMDA. The outcome of this meeting will provide important perspective to be considered in preparation for the formal consultation meetings which precede the Japanese new drug application.

XOWNA (CLBS16) for the treatment of coronary microvascular dysfunction ("CMD")

XOWNA is an experimental regenerative therapy for the treatment of CMD. It was the subject of a positive Phase 2a study (the "ESCaPE-CMD trial") reported in 2020 and is currently being evaluated in the U.S. Phase 2b FREEDOM Trial. The FREEDOM Trial is a double-blind, randomized, placebo-controlled study designed to assess the efficacy and safety of delivering autologous CD34+ cells to subjects with CMD and without obstructive coronary artery disease. As previously communicated, enrollment in the FREEDOM Trial initially proceeded as planned with the first patient treated in January 2021; however, the impact of the COVID-19 pandemic in the U.S., coupled with supply chain issues associated with the catheters used for diagnosis of CMD and/or administration of XOWNA have made and continue to make enrollment much slower than originally predicted and challenging to accelerate. Notwithstanding the obstacles, Caladrius has taken steps to accelerate enrollment by expanding the number of participating investigational sites as well as modifying the study protocol to make study inclusion criteria more flexible. Caladrius continues to monitor the progress of the study and will consider additional future protocol and/or execution changes, as appropriate.

CLBS201 for the treatment of diabetic kidney disease ("DKD")

Progressive kidney failure is associated with attrition of the microcirculation of the kidney. Preclinical studies in kidney disease and injury models have demonstrated that protection or replenishment of the microcirculation results in improved kidney function. Based on these observations, the Company recently initiated a Phase 1, open-label, proof-of-concept trial evaluating CLBS201, a CD34+ regenerative cell therapy investigational product for intra-renal artery administration in patients with DKD. Patients selected for the study will be in the pre-dialysis stage of kidney disease and will exhibit rapidly progressing stage 3b disease. The protocol provides for a staggered, sequentially dosed cohort of six patients overseen by an independent Data Safety Monitoring Board with the objective of determining the tolerance of intra-renal cell therapy injection in DKD patients as well as the ability of CLBS201 to regenerate kidney function. A key read-out of data will occur at the 6-month follow-up visit for all patients. As previously announced, the first patient was treated in this Phase 1b study of CLBS201 in April 2022 leading to top-line data from all subjects expected by the first quarter of 2023.

First Quarter 2022 Financial Highlights

Research and development expenses for the three months ended March 31, 2022, were $3.3 million, compared to $5.1 million for the three months ended March 31, 2021. Research and development activities in the current year period focused on the advancement of our ischemic repair platform and related to:

Expenses associated with efforts to continue execution and acceleration of enrollment of the FREEDOM Trial;

Expenses associated with the planning, preparation and initiation of the Phase 1b proof-of-concept trial for CLBS201 as a treatment for DKD; and

Ongoing expenses for HONEDRA in CLI and Buerger’s disease in Japan associated with study close out activities and preparation for the pre-consultation meetings with the PMDA.
General and administrative expenses, which focus on general corporate related activities, were $3.3 million for the three months ended March 31, 2022, compared to $3.0 million for the three months ended March 31, 2021, representing an increase of 11%. This increase was primarily due to an increase in fees associated with the review of potential strategic transactions.

Overall, net losses were $4.2 million and $8.1 million for the three months ended March 31, 2022 and March 31, 2021, respectively.

Balance Sheet Highlights

As of March 31, 2022, the Company had cash, cash equivalents and marketable securities of approximately $88.5 million, which positions us well relative to the projected capital obligations for our existing development programs as well as our cash and investments balance target at the time of the closing of the merger with Cend.

Conference Call

Caladrius will hold a live conference call today May 5, 2022, at 4:30 p.m. (ET) to discuss financial results, provide a business update and answer questions. To join the conference call, please refer to the dial-in information provided below. A live webcast of the call will also be available under the Investors & News section of the Caladrius website (View Source) and will be available for replay for 90 days after the conclusion of the call.

Please dial-in 10 minutes before the start of the conference call.

For those unable to participate on the live conference call, an audio replay will be available that day starting at 7:30 p.m. (ET) until May 12, 2022, by dialing 855-859-2056 (U.S. Toll-Free) or 404-537-3406 (International) and by entering the replay passcode: 7129748.

On May 5, 2022 Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) reported financial results for the first quarter of 2022 (Press release, Concert Pharmaceuticals, MAY 5, 2022, View Source [SID1234613614]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We are at an exciting point in our progress with CTP-543, our drug candidate for alopecia areata, a serious autoimmune disorder with significant unmet medical need. Our pivotal trials, THRIVE-AA1 and THRIVE-AA2, have collectively enrolled over 1,200 patients in the U.S., Canada and Europe. We’re grateful to the many individuals who chose to participate in our studies to advance research for this important autoimmune disorder and, in the event of positive data, provide us with the information we need to support filing our New Drug Application with the FDA, which is planned for the first half of next year," stated Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "We continue to expect to report topline results from the THRIVE‑AA1 trial this quarter. The THRIVE-AA2 topline results are expected to follow soon thereafter, in the third quarter of 2022."

Recent Business Highlights and Upcoming Milestones

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

Topline Data for 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 oral 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.
Topline Data for THRIVE-AA2 Expected in the Third Quarter of 2022. The Company expects to report topline results from the THRIVE-AA2 trial in the third quarter of 2022. 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 oral 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.
New Drug Application (NDA) Filing for Alopecia Areata Expected 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. If approved, the Company expects that CTP-543 would be one of the first FDA-approved treatments for alopecia areata. 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 time.1
First Quarter 2022 Financial Results

Cash and Investment Position. Cash, cash equivalents and investments as of March 31, 2022 totaled $109.0 million as compared to $141.6 million as of December 31, 2021. Under its current operating plan, the Company expects its current cash, cash equivalents and investments 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 its November 2021 financing.
R&D Expenses. Research and development expenses were $30.5 million for the quarter ended March 31, 2022, compared to $18.5 million for the same period in 2021. The increase in research and development expenses relates primarily to the clinical development for CTP-543.
G&A Expenses. General and administrative expenses were $5.5 million for each of the quarters ended March 31, 2022 and 2021. General and administrative expenses primarily consist of employee benefits and compensation and external professional services.
Net Loss. Net loss applicable to common stockholders was $37.7 million, or $1.03 per share, for the quarter ended March 31, 2022, as compared to net loss applicable to common stockholders of $22.7 million, or $0.67 per share, for the quarter ended March 31, 2021.
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 2022 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 5, 2022 MaaT Pharma (EURONEXT: MAAT – the "Company"), a French clinical-stage biotech and a pioneer in the development of microbiome-based ecosystem therapies dedicated to improving survival outcomes for patients with cancer reported its cash position as of March 31, 2022, and its revenues for the first quarter of 2022 (Press release, MaaT Pharma, MAY 5, 2022, View Source [SID1234613643]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Over the course of the first quarter 2022, the Company has continued its clinical development as previously announced in the context of its IPO in November 2021 such as:

In January 2022, announcement of positive interim and preliminary data of its Phase 1b trial for MaaT033 in preventing complications of allogeneic hematopoietic stem cell transplantation.
In March 2022, inclusion of the first patient in a Phase 3 trial "ARES" for drug-candidate MaaT013 in the treatment of Graft-versus-Host Disease.
In April 2022, initiation of a Phase 2a trial, sponsored by AP-HP, evaluating MaaT013 in combination with Immune Checkpoint Inhibitors for patients with melanoma.
Moreover, the Company entered a partnership agreement, in February 2022, with Skyepharma to build, to date, France’s largest cGMP manufacturing facility exclusively dedicated to the production of Microbiome Ecosystem Therapy (MET), thus increasing MaaT Pharma’s manufacturing capabilities ten-fold in line with supply needs required by 2030.

Cash position1

As of March 31, 2022, total cash and cash equivalents were EUR 41.1 million, as compared to EUR 43.3 million as of December 31, 2021. The Company believes it has sufficient cash to cover needs of the development programs presented during the IPO up until the end of the third quarter of 2023.

Revenues in Q1 20221

MaaT Pharma reported revenues of EUR 0.3 million for the quarter ended March 31, 2022, compared with 0.1 million for the same period of 2021. Revenues correspond to compensation invoiced in relation to the compassionate access program, as approved by the French National Drug Safety Agency (Agence Nationale de Sécurité du Médicament or ANSM). In 2022, the program was fully active whereas in 2021 the program began over the course of the first quarter, thus explaining the year-on-year increase.

Upcoming financial communication and investor conference participation

May 31, 2022 – Annual General Meeting
June 7, 2022 – Investor R&D Day
June 30, 2022 – 9th Portzamparc Annual Conference, Paris
July 28, 2022 – Revenues and Cash Position Quarter 2*
September 15 – 16, 2022 – KBCS Life Sciences Conference
September 29, 2022 – Half-year Results 2022*
*Indicative calendar that may be subject to change.

[1] Unaudited data

On May 5, 2022 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrine, metabolic, oncologic and neurological disorders by modulating the effects of the hormone cortisol, reported its results for the quarter ended March 31, 2022 (Press release, Corcept Therapeutics, MAY 5, 2022, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-first-quarter-financial-results [SID1234613658]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Financial Results

Revenue of $93.7 million, compared to $79.4 million in first quarter 2021
Reiterated 2022 revenue guidance of $400 – $430 million
Diluted net income per share of $0.20, compared to $0.18 in first quarter 2021
Cash and investments of $368.1 million, compared to $335.8 million at December 31, 2021
"As pandemic restrictions and fears recede, we expect our growth to continue and are reiterating our 2022 revenue guidance of $400 – $430 million," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "Korlym is an excellent treatment for patients with Cushing’s syndrome and there are many eligible patients who have yet to receive it."

Corcept’s first quarter 2022 revenue was $93.7 million, compared to $79.4 million in the first quarter of 2021. First quarter operating expenses were $66.9 million, compared to $59.8 million in the first quarter of 2021, due to increased expenses to support the expansion of our clinical development and commercial teams and legal fees. Diluted net income per share was $0.20 in the first quarter of 2022, compared to $0.18 in the first quarter of 2021.

Cash and investments increased $32.3 million in the first quarter, to $368.1 million at March 31, 2022.

Clinical Development

"Corcept was founded on the premise that cortisol modulation has the potential to help treat many serious diseases," said Dr. Belanoff. "Our clinical development programs have produced increasing amounts of evidence validating this hypothesis and our programs in castration-resistant prostate cancer, antipsychotic-induced weight gain and non-alcoholic steatohepatitis will produce important data this year. We are especially excited about our advancing platinum-resistant ovarian cancer program. Based on the statistically significant and clinically meaningful results of our large, controlled Phase 2 study, we will soon initiate a pivotal trial."

Solid Tumors

Phase 3 trial in patients with recurrent platinum-resistant ovarian cancer planned
to start this quarter; Oral presentation of Phase 2 trial results at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting on June 6 in Chicago
Selection of the optimum dose of exicorilant or relacorilant plus enzalutamide in patients
with castration-resistant prostate cancer (CRPC) expected this quarter
Enrollment continues in 20-patient, open-label, Phase 1b trial of relacorilant plus
PD-1 checkpoint inhibitor pembrolizumab in patients with adrenal cancer with cortisol excess
"We are excited to start our Phase 3 trial of relacorilant in patients with recurrent platinum-resistant ovarian cancer," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "Our goal is to replicate the positive findings of our 178-patient Phase 2 trial, in which women who received relacorilant in addition to nab-paclitaxel exhibited meaningful improvements in progression-free survival, duration of response and overall survival, without increased side effects, when compared to women who received nab-paclitaxel alone. The 20,000 women in the United States and an equal number in Europe with platinum-resistant ovarian cancer have few good treatment options. If our Phase 3 trial is successful, relacorilant plus nab-paclitaxel could become the new standard of care for these patients. We plan to meet with the FDA in June regarding our proposed path forward."

Metabolic Diseases

Enrollment completed in GRATITUDE and GRATITUDE II – two double-blind, placebo-controlled Phase 2 trials of miricorilant to reverse recent and long-standing antipsychotic-induced weight gain (AIWG); data from both trials expected in fourth quarter 2022
Enrollment continues in Phase 1b dose-finding trial of miricorilant in patients
with presumed NASH
"We look forward to the results of GRATITUDE and GRATITUDE II," said Dr. Guyer. "Weight gain and other metabolic adverse effects caused by antipsychotic medications pose serious risks to the health of millions of patients, who have few treatment options. We initiated these double-blind, placebo-controlled trials to build on the positive data from our studies of both miricorilant and mifepristone in healthy volunteers."

Cushing’s Syndrome

Enrollment continues in Phase 3 GRACE trial of relacorilant as a treatment for patients with all etiologies of Cushing’s syndrome; new drug application (NDA) submission now expected
in second half 2023
Enrollment continues in Phase 3 GRADIENT trial of relacorilant as a treatment for patients
with Cushing’s syndrome caused by adrenal adenomas
"We advanced relacorilant to Phase 3 in Cushing’s syndrome based on its extremely promising Phase 2 efficacy and safety data. We expect our GRACE trial, which is accruing patients and generating data, to serve as the basis for relacorilant’s NDA in Cushing’s syndrome. The timing for the completion of this trial has been impacted by the pandemic, as clinical trial sites have experienced challenges in recruiting and managing patients. We are currently planning to submit this relacorilant NDA in the second half of 2023," said Dr. Guyer. "The Phase 3 GRADIENT trial will produce valuable data about an etiology of Cushing’s syndrome that has not been subject to rigorous, controlled study, but affects many patients."

Conference Call

We will hold a conference call on May 5, 2022, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, click this link (listen-only mode) or dial 1-833-693-0540 from the United States or 1-661-407-1581 internationally approximately 15 minutes before the start of the call. A replay will be available through May 12, 2022 at 1-855-859-2056 from the United States and 1-404-537-3406 internationally. The passcode will be 6942208. A replay will also be available on the Investors / Past Events tab of our website.

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system and can be lethal if not treated effectively. Corcept holds patents directed to the composition of relacorilant and the use of cortisol modulators, including Korlym, in the treatment of patients with hypercortisolism.