On August 5, 2022 Celyad Oncology SA (Euronext & Nasdaq: CYAD) (the "Company"), a clinical-stage biotechnology company focused on the discovery and development of chimeric antigen receptor T cell (CAR T) therapies for cancer, reported an update on its financial results and recent business developments for the first half ended June 30, 2022 (Press release, Celyad, AUG 5, 2022, View Source [SID1234617690]).

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!

"As the Company continues to evolve, we are excited about a renewed focus on additional value drivers for Celyad Oncology. Importantly, with our world-class intellectual property focused on allogeneic CAR T technology, we have multiple opportunities for partnerships with peers in the industry," commented Michel Lussier, interim Chief Executive Officer of Celyad Oncology. "We also were proud to recently announce that the FDA lifted the clinical hold on our CYAD-101 program. In addition, we look forward to the upcoming data read out for CYAD-211 in the second half of 2022. We are truly ushering in a new chapter for Celyad Oncology by unlocking the potential of not only our product candidates, but also our portfolio of IP, technology, and overall expertise in cell therapy."

Second Quarter 2022 and Recent Business Highlights

The Board of Directors named Hilde Windels as Chair of the Board of Directors
Michel Lussier named Interim Chief Executive Officer of the Company
Pipeline and Business Updates

CYAD-211 – Allogeneic shRNA-based, anti-BCMA CAR T for r/r MM

CYAD-211 is an investigational, short hairpin RNA (shRNA)-based allogeneic CAR T candidate for the treatment of r/r MM. CYAD-211 is engineered to co-express a B cell maturation antigen (BCMA) targeting CAR and a single shRNA, which interferes with the expression of the CD3ζ component of the T-cell receptor (TCR) complex.

Preliminary data reported in December 2021 from the dose-escalation segment of the IMMUNICY-1 Phase 1 trial evaluating CYAD-211 following cyclophosphamide/fludarabine (CyFlu) preconditioning chemotherapy in patients with r/r MM showed evidence of clinical activity with a good tolerability profile including no evidence of Graft versus Host Disease. In addition, all patients in the trial had detectable CYAD-211 cells in the peripheral blood.
Enrollment is currently ongoing in the IMMUNICY-1 Phase 1 trial to evaluate enhanced lymphodepletion (eLD) and increased CYAD-211 doses with the aim to improve cell persistence and potentially maximize the clinical benefit of CYAD-211. The IMMUNICY-1 protocol also allows for CYAD-211 redosing in certain patients.
Additional data updates from the eLD cohorts of the Phase 1 IMMUNICY-1 trial of CYAD-211 for r/r MM are expected during second half of 2022.
CYAD-101 – Allogeneic TIM-based NKG2D CAR T for mCRC

CYAD-101 is an investigational, non-gene edited, allogeneic CAR T candidate engineered to co-expresses the TIM peptide alongside a CAR based on NKG2D, a receptor expressed on natural killer (NK) and T cells, that binds to eight stress-induced ligands.

In June 2022 we submitted our complete response to the clinical hold of the CYAD-101-002 phase 1b trial to the FDA stating our intent to amend the eligibility criteria to exclude patients who have bilateral lung metastases and patients who have received treatment with epidermal growth factor receptor (EGFR) targeting monoclonal antibodies within the previous 9 months prior to trial recruitment. In July 2022, based on that complete response, we received notification that the FDA lifted the clinical hold on the CYAD-101-002 phase 1b trial
shARC Platform

Discovery research continues on programs focused on the co-expression of Interleukin-18 in conjunction with our short hairpin RNA shRNA technology platform, also known as our shARC (shRNA Armored CAR) franchise, with a focus on the development of next-generation, allogeneic CAR T candidates.

CYAD-02 – Autologous NKG2D CAR-T for r/r AML and MDS

CYAD-02 is an investigational, autologous CAR T therapy that co-expresses both the NKG2D CAR and a single shRNA targeting the NKG2D ligands MICA/MICB on the CAR T cells.

In December 2021, the Company presented clinical results from the dose-escalation CYCLE-1 Phase 1 trial evaluating CYAD-02 for the treatment of r/r acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Data from the trial showed that a single shRNA can target two independent genes (MICA/MICB) to enhance the phenotype of the CAR T cells. In addition, the dual knockdown showed a positive contribution to the initial clinical activity of CYAD-02 as well as a trend towards increased engraftment and persistence compared to the first-generation, autologous NKG2D receptor CAR T.
The Company continues to explore potential partnership opportunities for the future development of CYAD-02.
Strategic Focus on Intellectual Property

The Company maintains a robust intellectual property portfolio within the landscape of CAR T, including twelve foundational U.S.

patents associated with allogeneic CAR T for the treatment of cancer as well as patents for NKG2D receptor-based cell therapies. We believe these patents provide an avenue for the Company to develop its own programs as well as to seek potential partnership opportunities.

First Half 2022 Financial Results

Key financial figures for the first half of 2022, compared with the first half of 2021 and full year 2021, are summarized below:

Research and Development expenses were €10.5 million for the first half of 2022, compared to €10.0 million for the first half of 2021. The €0.5 million increase was mainly driven by intellectual property filing and maintenance fees to strengthen intellectual property prosecution and the increase of employee expenses mainly related to the full expense impact of the employees recruited during 2021 to support the Group’s preclinical and clinical programs, employee turnover and management changes, both of which were partially offset by the decrease in clinical activities resulting from the Phase 1b CYAD-101-002 (KEYNOTE-B79) trial which was on clinical hold during the second quarter of 2022.

General and Administrative expenses were €6.2 million for the first half of 2022, compared to €4.8 million for the first half of 2021. This increase is primarily attributable to an increase in insurance costs for the period, combined with an increase in employee expenses mainly related to management changes through the six-month period ended June 30, 2022.

A fair value adjustment of €1.1 million (non-cash income) related to the reassessment of the contingent consideration and other financial liabilities associated with the advancement of the Company’s NKG2D-based CAR T candidates as of June 30, 2022, required by International Financial Reporting Standards (IFRS), was mainly driven by the updated assumptions on projected revenue associated with the Company’s CYAD-101 program, for which the timing of the potential commercialization has been delayed by one year. Additionally, the addressable patient population for CYAD-101 has been reduced based on recent safety findings from the CYAD-101-002 Phase 1b trial. The fair value adjustment was also driven by updated assumptions to discount rate and revaluation of the U.S. dollar foreign exchange rate.

The Company also posted €1.6 million in net other income for the first half of 2022, compared to a net other income of €1.8 million for the first half of 2021. Other income for the first half of 2022 is primarily due to grant income from the Walloon Region of €1.4 million.

Net loss for the first half of 2022 was €14.1 million, or € (0.62) per share, compared to a net loss of €14.9 million, or € (1.02) per share, for the first half of 2021.

Net cash used in operations was €16.3 million for the first half of 2022, compared to €12.2 million for the first half of 2021.

As of June 30, 2022, the Company had cash and cash equivalents of €14.4 million ($15.0 million).

As of June 30, 2022, the total number of basic shares outstanding were 22.6 million similar to December 31, 2021.

Celyad Oncology First Half 2022 Conference Call Details

The conference call will be webcast live and archived within the "Events" section of the Celyad Oncology website.

On August 5, 2022 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted treatments for cancer, reported financial results for the second quarter ended June 30, 2022 (Press release, Cellectar Biosciences, AUG 5, 2022, View Source [SID1234617689]).

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 second quarter, iopofosine passed an important milestone as an independent data monitoring committee completed a futility/efficacy assessment and unanimously recommended continuation of our pivotal Phase 2B trial in Waldenstrom’s macroglobulinemia (WM)," said James Caruso, president and CEO of Cellectar. "This global trial includes participation from leading institutions and world-renowned WM thought leadership, and we are excited by the active engagement of our investigators." Mr. Caruso continued, "We also look forward to providing data from our phase 2a multiple myeloma trial and our phase 1 pediatric trial for malignant brain tumors and sarcomas in the second half of 2022."

Second Quarter 2022 Financial Highlights

Cash and Cash Equivalents: As of June 30, 2022, the company had cash and cash equivalents of $24.8 million, compared to $35.7 million as of December 31, 2021. Net cash used in operating activities during the six months ended June 30, 2022 was approximately $10.8 million. The company believes its cash on hand is adequate to fund basic budgeted operations into the third quarter of 2023.

Research and Development Expense: R&D expense for the three months ended June 30, 2022 was approximately $4.5 million, which was relatively consistent when compared to approximately $4.6 million for the three months ended June 30, 2021. For the six months ended June 30, 2022, R&D expense was approximately $8.4 million, while the comparable period in 2021 was $9.3 million. The reduction in the six month period was due primarily to the timing of activities related to our ongoing WM pivotal trial as trial initiation costs were higher in the prior year.

General and Administrative Expense: G&A expense for the three months ended June 30, 2022 was $2.9 million, compared to $1.4 million for the same period in 2021. G&A expense in the six months ended June 30, 2022 was approximately $5.2 million, as compared to approximately $3.1 million in the prior year. These increases were driven largely by increased professional fees and personnel costs.

Net Loss: The net loss attributable to common stockholders for the quarter ended June 30, 2022 was ($7.4) million, or ($1.22) per share, compared to ($6.0) million, or ($1.14) per share, in the quarter ended June 30, 2021, while the loss attributable to common stockholders in the first half of 2022 was ($13.6) million, or ($2.22) per share, compared to ($12.4) million, or ($2.45) per share for the first half of 2021.

On August 5, 2022 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a late clinical stage biopharmaceutical company focused on helping patients fight cancer with oncolytic viral immunotherapies, reported financial results for the second quarter ended June 30, 2022 and provided a corporate update (Press release, Candel Therapeutics, AUG 5, 2022, View Source [SID1234617688]).

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!

"Candel remains on track to achieve several milestones in the second half of 2022," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "We are encouraged by the initial phase 2 clinical trial data for CAN-2409 in non-small cell lung cancer presented in June at ASCO (Free ASCO Whitepaper). The data presented at ASCO (Free ASCO Whitepaper) showed an 87.5 percent disease control rate in heavily pretreated patients whose cancer was progressing on PD-1 agents at clinical trial entry. In the second half of this year, we are planning for multiple inflection points, including updated clinical data from the phase 2 lung cancer clinical trial, initiation of a phase 3 clinical trial in high-grade glioma, and we will present initial data from our enLIGHTEN Discovery Platform. I am thrilled to see the rapid progress the Candel team continues to make on the discovery and development of our oncolytic viral immunotherapies for patients with cancer."

Second Quarter 2022 & Recent Highlights

Presented initial data on its open-label phase 2 clinical trial of CAN-2409 in combination with anti-PD-1 or PD-L1 targeting agents in patients with stage III/IV non-small cell lung cancer (NSCLC) in June 2022.
The data showed cytotoxic T cell response and a disease control rate of 87.5 percent achieved in patients whose cancer was progressing on anti-PD-1 therapy at clinical trial entry.
These findings were presented by co-principal investigator Charu Aggarwal, MD, MPH, at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
In addition to the ASCO (Free ASCO Whitepaper) poster session, the Company hosted a discussion with key experts Roy Herbst, MD, PhD, and Daniel Sterman, MD, to review these initial data.
The Company appointed three new members to its Board of Directors: Gary Nabel, MD, PhD, renowned virologist and immunologist; Joseph Papa, prominent business leader; and Renee Gaeta, strategic financial expert. Effective August 8, 2022, these new members will replace current Board members: Alan E. Smith, PhD, Shaan Ghandi, MD, D.Phil, and Udi Meirav, PhD, maintaining Candel’s nine-seat Board.
Key Upcoming Milestones

In the fourth quarter of 2022, the Company expects to present new data from three clinical trials:
A phase 1b clinical trial of CAN-2409 in combination with nivolumab (Opdivo) combined with standard of care in first line treatment in patients with high-grade glioma.
A phase 1 clinical trial of CAN-3110 in patients with recurrent high-grade glioma.
A phase 2 clinical trial of CAN-2409 in combination with anti-PD-1 or PD-L1 agents in patients with stage III/IV NSCLC.
The Company anticipates initiating a phase 3 clinical trial evaluating CAN-2409 in patients with high-grade glioma in the third quarter of 2022.
The Company also plans to debut initial data from its new discovery platform, enLIGHTEN, in the fourth quarter of 2022.
Financial Results for the Quarter Ended June 30, 2022

Cash Position: Cash and cash equivalents as of June 30, 2022 were $86.8 million compared to $82.6 million as of December 31, 2021. The net increase was due to receipt of $20.0 million from a term loan with Silicon Valley Bank in February 2022, offset by costs to fund operating activities and the purchase of fixed assets. Based on current plans and assumptions, the Company expects that its existing cash and cash equivalents will be sufficient to fund its operations into the first quarter of 2024.

Research and Development Expenses: Research and development expenses were $5.0 million and $10.4 million for the three- and six-month period ended June 30, 2022 compared to $3.3 million and $6.0 million for the comparable periods in 2021. The increase was primarily due to personnel-related costs for additional headcount, as well as operating expenses related to the conduct of five ongoing clinical studies, the expected initiation of a phase 3 clinical study, and the expansion of manufacturing capabilities. Excluding stock-based compensation expense of $57,000 for the three months ended June 30, 2022 and $199,000 for the six-month period ended June 30, 2022, research and development expenses for the three- and six-month period ended June 30, 2022 were $5.0 million and $10.2 million.

General and Administrative Expenses: General and administrative expenses were $3.8 million and $7.4 million for the three- and six-month period ended June 30, 2022 compared to $2.0 million and $4.0 million for the comparable periods in 2021. The increase was primarily due to higher insurance costs, personnel-related costs and professional consulting fees associated with operating as a public company. Excluding stock-based compensation expense of $381,000 for the three-month period ended June 30, 2022, and $730,000 for the six-month period ended June 30, 2022, general and administrative expenses for the three- and six-month period ended June 30, 2022 were $3.4 million and $6.6 million.

Total Operating Expenses: Total operating expenses were $8.8 million and $17.8 million for the three- and six-month period ended June 30, 2022 compared to $5.3 million and $10.0 million for the comparable periods in 2021. The increase was primarily due to increased personnel-related costs and research and development activities and resulting expenses as well as increased operating expenses associated with being a public company. Excluding stock-based compensation expense of $438,000 for the three-month period ended June 30, 2022, and $929,000 for the six-month period ended June 30, 2022, total operating expenses for the three- and six-month period ended June 30, 2022 were $8.3 million and $16.9 million.

Net Loss: Net loss was $4.1 million and $5.0 million for the three- and six-month period ended June 30, 2022, as compared to $17.1 million and $21.6 million for the comparable periods in 2021. The net loss for the three- and six-month period ended June 30, 2022 includes a non-cash credit of $5.0 million and $13.3 million, and the net loss for the three- and six-month period ended June 30, 2021 includes a non-cash charge of $12.4 million for the change in the fair value of the Company’s warrant liability, and includes stock-based compensation expense of $438,000 and $929,000 for the three- and six-month period ended June 30, 2022, and $1.1 million and $1.5 million for the three- and six-month period ended June 30, 2021. Excluding non-cash credits for the changes in the warrant liability and charges for stock-based compensation, the net loss for the three- and six-month period ended June 30, 2022 was $8.7 million and $17.4 million, as compared to $3.6 million and $7.7 million for the comparable periods in 2021.

On August 5, 2022 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a clinical-stage biopharmaceutical company developing innovative therapies designed to treat or reverse disease, reported financial results for the three and six months ended June 30, 2022 and provided a business update (Press release, Caladrius Biosciences, AUG 5, 2022, View Source [SID1234617687]).

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!

"The second quarter of 2022 was a transformative and energizing quarter for Caladrius with the announcement of our proposed merger with Cend Therapeutics ("Cend"). The merger process, which, when completed, will result in the change of our name to Lisata Therapeutics ("Lisata"), is progressing well and, subject to the approval by our stockholders, remains on track to close in the third quarter of 2022," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "Following the closing of the proposed merger, Lisata will focus on maximally exploiting the full potential of Cend’s CendR Platform technology in a range of solid tumor cancer settings while progressing Caladrius’ current product candidate development programs to their next development milestone. CEND-1, the lead product candidate from the CendR Platform, has the potential to be combined with a myriad of chemo and immunotherapeutic agents that could become an integral part of a revised standard-of-care therapy for many difficult to treat cancers."

"In June, Cend announced the first patient had been treated in the Phase 2b ASCEND study of CEND-1 in combination with gemcitabine and nab-paclitaxel for the treatment of first-line, metastatic pancreatic ductal adenocarcinoma ("mPDAC"). This 125-patient study is a double-blind, randomized, placebo-controlled clinical trial being conducted at up to 40 sites in Australia and New Zealand led by the Australasian Gastro-Intestinal Cancer Trials Group in collaboration with the NHMRC Clinical Trial Centre at the University of Sydney. In addition, The Lancet Gastroenterology and Hepatology recently published groundbreaking data from the Phase 1b study of CEND-1 in combination with gemcitabine and nab-paclitaxel for the treatment of first-line mPDAC."

Dr. Mazzo continued, "While we continue to make progress on our current Caladrius programs, a tremendous amount of work already has been conducted under our collaboration agreement with Cend. This is an exciting time for the Company and the future Lisata. We look forward to providing additional updates in the coming weeks and months."

Proposed Merger with Cend Therapeutics

As previously disclosed, the Company entered into a definitive merger agreement with Cend Therapeutics, Inc., 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 is expected to be renamed Lisata Therapeutics, Inc. 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 collaboration agreement to maintain development momentum of the Cend pipeline.

Ongoing Development Portfolio Update

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 which is now in the pre-consultation phase of the registration process with the Pharmaceuticals and Medical Devices Agency ("PMDA") in Japan. Data from the follow-up of all patients completed in the registration-eligible clinical trial in Japan has been compiled and will be reviewed by PMDA during the third quarter of 2022, after which the PMDA will provide important perspective to be considered in preparation for the formal consultation meetings which precede the Japanese new drug application. Concomitantly, the Company will focus its efforts to secure a Japanese partner to complete the remaining steps to produce registration in Japan.

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 a U.S. Phase 2b study (the "FREEDOM Trial"). The FREEDOM Trial was originally designed as a 105-patient double-blind, randomized, placebo-controlled trial to further evaluate the efficacy and safety of intracoronary delivery of autologous CD34+ cells (XOWNA) in subjects with CMD and without obstructive coronary artery disease and was expected to complete enrollment in approximately 12 months. 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, as well as with a contrast agent typically used in many catheter laboratories, have made and continue to make enrollment much slower than originally predicted and challenging to accelerate. As a result, and as previously disclosed, the Company has suspended further enrollment activities and is conducting an interim analysis of the data during the third quarter of 2022 to determine the next steps for the program, which may require a discussion with and guidance from FDA. The Company expects to have a decision on next steps for the program by the end of 2022.

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 1b, 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 are in the pre-dialysis stage of kidney disease and exhibit rapidly progressing stage 3b disease. The protocol provides for a 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. The Company treated the first patient in April 2022 and completed treatment for all six subjects during the third quarter of 2022. Top-line data is anticipated from all subjects by the first quarter of 2023.

Second Quarter 2022 Financial Highlights

Research and development expenses for the three months ended June 30, 2022 were $3.2 million, compared to $4.3 million for the three months ended June 30, 2021, representing a decrease of $1.1 million or 25%. This decrease was primarily due to a decrease in expenses associated with HONEDRA in Japan, revenue received from the collaboration agreement and one-off recruiting expenses in the prior year. Research and development activities in the current year period focused on the advancement of our ischemic repair platform and related to:

execution of the FREEDOM Trial including preparation for an interim analysis;
execution of the Phase 1b proof-of-concept trial of CLBS201 as a treatment for DKD, which commenced in the first quarter of 2022 with the first patient in the study treated in April 2022; and
study close out activities and preparation for the pre-consultation meetings with the PMDA for HONEDRA in CLI and Buerger’s disease in Japan.
General and administrative expenses, which focus on general corporate related activities, were $3.5 million for the three months ended June 30, 2022, compared to $2.8 million for the three months ended June 30, 2021, representing an increase of 24%. This increase was primarily due to an increase in professional fees associated with the proposed merger with Cend Therapeutics, Inc.

Overall, net losses were $6.6 million and $5.7 million for the three months ended June 30, 2022 and June 30, 2021, respectively.

In order to provide Cend with capital for its development programs prior to the closing of the merger, the Company made an investment of $10 million in Cend in connection with a collaboration agreement to maintain development momentum of the Cend pipeline.

Balance Sheet Highlights

As of June 30, 2022, the Company had cash, cash equivalents and marketable securities of approximately $73 million, which is net of our $10 million investment in Cend and which we believe 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 Information

Caladrius will hold a live conference call today, August 4, 2022, at 4:30 p.m. (EDT) to discuss financial results, provide a business update and answer questions.

The Company is utilizing a new conference call service. Those wishing to participate must register for the conference call by way of the following link: CLICK HERE TO REGISTER. Registered participants will receive an email containing conference call details for dial-in options. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time.

A live webcast of the call will also be accessible under the Investors & News section of the Caladrius website and will be available for replay beginning two hours after the conclusion of the call for 12 months.

On August 5, 2022 Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), a biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases, including rare disorders, reported financial results for the second quarter ended June 30, 2022, and provided a review of recent accomplishments during and anticipated upcoming milestones (Press release, Biohaven Pharmaceutical, AUG 5, 2022, View Source [SID1234617686]).

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!

Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "As expected, we observed a strong rebound in NURTEC ODT net revenue as first quarter seasonal dynamics abated. Net product revenue of $194 million for the quarter represented a 57% sequential increase compared to the first quarter and reflects strong product demand. In addition, the sales mix of our 2-pack (16-tablet count) of Nurtec ODT continued to ramp in the second quarter. Investments made in first quarter copay programs drove volume and net revenue growth, and the benefits of the only ‘all-in-one’ therapy to treat and prevent migraine continued to resonate with and improve the lives of patients living with migraine. And now, with the intranasal zavegepant NDA filing acceptance, we are closer to bringing a new, critical medication to market that offers migraine patients ultra-rapid relief in as early as 15 minutes, and addresses the needs of patients experiencing nausea or vomiting who need non-oral treatment options."

Dr. Coric continued, "We are excited about the vast opportunities beyond CGRP and have made tremendous strides across our broader pipeline, starting with our program in focal epilepsy with what we believe is a best-in-class approach to Kv7 potassium channel activation. We are working hard to expedite enrollment in our Phase 1 trial and look forward to exploring its potential in other epilepsy types as well as in pain and affective disorders. Likewise, we recently dosed the first subject in our Phase 3 trial evaluating taldefgrobep alfa in subjects with Spinal Muscular Atrophy (SMA); we look forward to driving enrollment in this trial and exploring the potential of taldefgrobep alfa in additional indications. We also remain on track to report topline results from the verdiperstat arm of the platform trial in ALS, sponsored by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in the second half of the year. We are excited about the paradigm-shifting approaches we’re exploring across our expanding pipeline and will continue working determinedly to improve outcomes for patients living with neuroscience diseases."

Second Quarter and Recent Business Highlights

Q2 2022 net product revenue from sales of NURTEC ODT totaled $194 million, up sequentially 57% compared to Q1 2022 – NURTEC ODT has now achieved over 2,200,000 prescriptions, and over 79,000 unique prescribers through June 30, 2022, an increase of 11,500 prescribers from the first quarter, signaling continued traction across the prescribing community. The $194 million in net product revenues for the quarter represents a 109% increase over the second quarter of 2021 and a 57% increase from net product revenues over the first quarter of 2022. The increase of $70 million in net product revenues as compared to the first quarter of 2022 is due primarily to increased NURTEC ODT prescription sales volume and favorable pack mix per prescription. In addition, we continue to see improvements in patient affordability / copay support needs as patients satisfy annual deductible obligations. These are marginally offset by additional rebates during the second quarter of 2022 compared to the first quarter of 2022. The Company remains on track to meet full year 2022 net product sales guidance of $825 – $900 million.
Antitrust approval granted for proposed acquisition by Pfizer; other closing conditions – As previously disclosed, Biohaven entered into an agreement and plan of merger with Pfizer, Inc. ("Pfizer") on May 10, 2022, under which Pfizer will acquire Biohaven (the "Merger"). Under the terms of the agreement, Pfizer will acquire all outstanding shares of Biohaven not already owned by Pfizer for $148.50 per share in cash. Biohaven common shareholders, including Pfizer, will also receive 0.5 of a share of New Biohaven, a new publicly traded company that will retain Biohaven’s non-CGRP development stage pipeline compounds, per Biohaven common share. The boards of directors of both Biohaven and Pfizer have unanimously approved the transaction. Pfizer will pay transaction consideration totaling approximately $11.6 billion in cash. Pfizer will also make payments at closing to settle Biohaven’s third party debt and for the redemption of all outstanding shares of Biohaven’s redeemable preferred stock.
Consummation of the Merger is conditioned upon, among other things, expiration or termination of any applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended (the "HSR Act"). The applicable waiting period under the HSR Act expired at 11:59 p.m., ET, on June 27, 2022.
In addition to antitrust approval in the United States, the Merger was cleared by the German Federal Cartel Office on June 13, 2022, and was cleared by the Austrian Federal Competition Authority on June 23, 2022. On June 24, 2022, in response to a briefing paper filed by Pfizer on May 20, 2022, the UK Competition and Markets Authority confirmed that no further information is required on the Merger.
The Merger transaction is also subject to completion of the New Biohaven spin-off transaction and customary closing conditions, including the approval of Biohaven’s shareholders. The Company filed a preliminary proxy statement with the United States Securities and Exchange Commission ("SEC") on July 1, 2022.
U.S. FDA accepted Biohaven’s zavegepant NDA filing for the acute treatment of migraine in adults – In May, the Company announced that the FDA filed and accepted for review the Company’s recently submitted NDA for zavegepant, the only small molecule calcitonin gene-related peptide ("CGRP") receptor antagonist in an intranasal formulation, for the acute treatment of migraine in adults. The Prescription Drug User Fee Act ("PDUFA") goal date for completion of the FDA review of the NDA is set for the first quarter of 2023. The Company had announced the achievement of positive results in the pivotal trial of zavegepant for the acute treatment of migraine in adults in December, 2021.
Commenced enrollment in Phase 3 Spinal Muscular Atrophy ("SMA") study – In July, the Company commenced enrollment in a Phase 3 clinical trial assessing the efficacy and safety of taldefgrobep alfa in Spinal Muscular Atrophy (SMA). Taldefgrobep targets myostatin, a natural protein that limits skeletal muscle growth, through two mechanisms: lowering myostatin directly and blocking key downstream signaling mechanisms. The Company expects to enroll approximately 180 patients in this randomized, double-blind, placebo-controlled global trial.
Global Coalition for Adaptive Research ("GCAR") commenced enrollment in Glioblastoma Adaptive Global Innovative Learning Environment ("GBM Agile") Phase 2-3 adaptive platform trial for patients with glioblastoma – In July, GCAR announced the activation of Biohaven’s troriluzole and Vigeo Therapeutics’ VT1021 in GBM AGILE, a patient-centered, adaptive platform trial for registration that tests multiple therapies for patients with newly-diagnosed and recurrent glioblastoma ("GBM"). GBM AGILE is an international, innovative platform trial designed to more rapidly identify and confirm effective therapies for patients with glioblastoma through response adaptive randomization. The new interventions are opening first at Henry Ford Health Cancer in Detroit under Henry Ford site Principal Investigator Dr. Tom Mikkelsen and will subsequently open at more than 40 trial sites across the United States with additional global sites to follow.
Phase 3 clinical trial of troriluzole did not reach statistical significance in overall SCA population – In May, the Company announced top-line results from a Phase 3 clinical trial evaluating the efficacy and safety of its investigational therapy, troriluzole, in patients with spinocerebellar ataxia (SCA). The primary endpoint, change from baseline to Week 48 on the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA), did not reach statistical significance in the overall SCA population as there was less than expected disease progression over the course of the study. However, post hoc analysis of efficacy measures by genotype suggested a treatment effect in patients with the SCA Type 3 (SCA3) genotype, which represents the most common form of SCA and accounted for 41 percent of the study population. In the SCA3 subgroup, troriluzole showed a numerical treatment benefit on the change in f-SARA score from baseline to Week 48 compared to placebo. Given the debilitating nature of the disease and lack of approved therapies, the Company intends to share the SCA3 genotype data with regulators and work with the FDA to address a path forward for troriluzole in SCA.
Presented new Nurtec ODT, zavegepant, and migraine health economics and outcomes research (HEOR) data at the American Headache Society Annual Scientific Meeting – In June, the Company presented 31 new and encore presentations, including three late-breakers and three oral presentations, at the 64th annual scientific meeting of the American Headache Society. Key updates:
The Company presented full Phase 3 results for zavegepant nasal spray as an acute treatment of migraine as well as data from a 52-week open label extension study of Nurtec ODT (rimegepant) evaluating every other day preventive treatment of migraine plus as an as-needed acute treatment, and estimated reductions in monthly migraine days (MMDs) with rimegepant acute treatment.
Late-breaking submissions highlighted new data about patterns of medication utilization and migraine frequency for adults using Nurtec ODT as a preventive and acute treatment, results from a Phase 1 trial of Nurtec ODT in healthy Chinese adults, and outcomes of a Phase 3 study of Nurtec ODT conducted in China and Korea as an acute treatment of migraine.
Several HEOR posters notably highlighted (1) reductions in the prevalence of medication overuse headache following Nurtec ODT initiation, (2) reduced mean monthly quantity of dispensed Nurtec ODT which is potentially reflective of MMD reductions, (3) patient survey studies that illustrate preference for ODT oral formulations versus injectable or IV treatments in prevention, (4) improvements in health-related quality of life with long-term Nurtec ODT treatment, (5) the high interictal burden of migraine, and (6) patient reported attenuation of effect with biologic treatment in prevention.
Upcoming Milestones:

Biohaven is continuing to support the commercialization of NURTEC ODT for the acute and preventive treatment of migraine, as well as develop its product candidates through clinical and preclinical programs in a number of common and rare disorders. The Company expects to reach significant pipeline milestones across its platforms in the coming quarters.

Biohaven expects to:

Consummate the acquisition by Pfizer by early 2023, subject to the completion of the new Biohaven spin-off transaction and other customary closing conditions.
Advance Phase 1 study of BHV-7000 (Kv7) in focal epilepsy.
Continue to advance the commercialization of NURTEC ODT (rimegepant) for the acute and preventive treatment of migraine.
Continue to advance the zavegepant (intranasal spray) program towards commercialization for the acute treatment of migraine.
Submit an NDA for the acute treatment of migraine in China in the second half of 2022.
Report topline of verdiperstat in ALS in the second half of 2022.
Complete enrollment in Phase 3 study of troriluzole in Obsessive-Compulsive Disorder in 2023.
Capital Position:

Cash, cash equivalents, and marketable securities as of June 30, 2022, were $553.5 million, excluding $0.8 million of restricted cash, compared to $364.6 million as of December 31, 2021. On January 4, 2022, we received $500.0 million in upfront proceeds from Pfizer relating to our strategic collaboration arrangement, consisting of $150.0 million cash and $350.0 million in proceeds from the purchase of Biohaven common shares at a 25% market premium. In addition, during the second quarter of 2022 we drew $125.0 million in non-dilutive committed capital from our credit facility with Sixth Street.

Second Quarter 2022 Financial Highlights

Product Revenue, Net: Net product revenue was $194.0 million for the three months ended June 30, 2022, compared to $92.9 million for the three months ended June 30, 2021. The increase of $101.0 million in net product revenue was primarily due to increased NURTEC ODT sales volume as well as an increase in average pills per prescription during the three months ended June 30, 2022, compared to the three months ended June 30, 2021. The increase in the second quarter of 2022 compared to the second quarter of 2021 was also benefited from improved patient affordability support, partially offset by additional rebate related sales allowances.

Collaboration and Other Revenue: Collaboration and other revenue was $21.1 million for the three months ended June 30, 2022. No collaboration and other revenue was recognized for the three months ended June 30, 2021. The collaboration and other revenue recognized during the three months ended June 30, 2022 was primarily due to $20.0 million of variable consideration recognized as part of our collaboration arrangement with Pfizer.

Research and Development ("R&D") Expenses: R&D expenses, including non-cash share-based compensation costs, were $218.5 million for the three months ended June 30, 2022, compared to $77.4 million for the three months ended June 30, 2021. The increase of $141.1 million was primarily due to the acquisition of our Kv7 Platform from Knopp Biosciences LLC in April 2022 for $93.7 million and a $25.0 million development milestone related to BHV-7000, increased program expenses for rimegepant, and increased personnel costs. Non-cash share-based compensation expense was $16.6 million for the three months ended June 30, 2022, an increase of $7.4 million as compared to the same period in 2021.

Selling, General and Administrative ("SG&A") Expenses: SG&A expenses, including non-cash share-based compensation costs, were $250.5 million for the three months ended June 30, 2022, compared to $170.1 million for the three months ended June 30, 2021. The increase of $80.4 million was primarily due to increases in spending to support increased commercial sales of NURTEC ODT, including the launch of NURTEC ODT for the preventative treatment of migraine which was approved by the FDA in May of 2021. Additionally, approximately $9.6 million of the increased SG&A expense related to fees incurred in connection with the Pfizer Merger, including increased legal and accounting costs. Less than half of the SG&A expense was for commercial organization personnel costs, excluding non-cash share-based compensation expense. Non-cash share-based compensation expense was $23.6 million for the three months ended June 30, 2022, an increase of $7.3 million as compared to the same period in 2021.

Other Expense, Net: Other expense, net was $152.2 million for the three months ended June 30, 2022, compared to $34.9 million for the three months ended June 30, 2021. The increase of $117.3 million in net expense was primarily due to a $109.7 million change in fair value of derivative liabilities, as well as $9.3 million of increased interest expense as a result of additional borrowings under our debt facility with Sixth Street Specialty Lending, Inc. ("Sixth Street"). The change in fair value of derivative liabilities was a result of derivative liabilities recorded in connection with change of control provisions associated with the pending acquisition of Biohaven by Pfizer for our series A and B preferred shares and term loans with Sixth Street.

Net Loss: Biohaven reported a net loss attributable to common shareholders for the three months ended June 30, 2022, of $441.4 million, or $6.21 per share, compared to $210.6 million, or $3.23 per share for the same period in 2021. Anticipated closing of the merger agreement necessitates accounting of derivative losses in the amount of $1.57 per share resulting in a total second quarter reported net loss of $6.21 per share. Non-GAAP adjusted net loss for the three months ended June 30, 2022 was $271.4 million, or $3.82 per share, compared to $170.9 million, or $2.62 per share for the same period in 2021. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges, non-cash interest expense related to the accounting for mandatorily redeemable preferred shares and liability related to sale of future royalties, changes in the fair value of derivatives and gains or losses from equity method investment. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain non-GAAP financial measures. In particular, Biohaven has provided non-GAAP adjusted net loss and adjusted net loss per share, adjusted to exclude the items below. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, Biohaven believes the presentation of non-GAAP adjusted net loss and adjusted net loss per share, when viewed in conjunction with GAAP results, provides investors with a more meaningful understanding of ongoing operating performance. These measures exclude (i) non-cash share-based compensation, which is substantially dependent on changes in the market price of common shares, (ii) interest expense related to the accounting for our mandatorily redeemable preferred shares and liability related to sale of future royalties, which are in excess of the actual interest owed, (iii) changes in the fair value of derivative liability, which does not correlate to actual cash payment obligations in the relevant periods, and (iv) gains or losses from equity method investment, which are non-cash and based on the financial results and valuation of another company that we did not manage or control.

Biohaven believes the presentation of these non-GAAP financial measures provides useful information to management and investors regarding Biohaven’s results of operations. When GAAP financial measures are viewed in conjunction with these non-GAAP financial measures, investors are provided with a more meaningful understanding of Biohaven’s ongoing operating performance and are better able to compare Biohaven’s performance between periods. In addition, these non-GAAP financial measures are among those indicators Biohaven uses as a basis for evaluating performance, and planning and forecasting future periods. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between these non-GAAP measures and the most directly comparable GAAP measures is provided later in this press release.