On March 9, 2023 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the fourth quarter and full year ended Dec. 31, 2022 and provided an update on key strategic initiatives (Filing, 8-K, BioXcel Therapeutics, MAR 9, 2023, View Source [SID1234628408]).

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"Last year was a transformational period for the Company, highlighted by the launch of our first AI-discovered commercial product, IGALMI, in under four years since initiating human trials. A new treatment option is now available for patients suffering from agitation associated with schizophrenia or bipolar I or II disorder," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "We are building on these achievements in 2023 and look to accelerate our growth through commercial execution of IGALMI. We are also on track for significant data readouts for our overall neuropsychiatric program that has potential to address an estimated 139 million agitation episodes in the U.S.1-3* We have two pivotal study readouts for BXCL501 expected in the second quarter of 2023. Lastly, we plan to advance our lead immuno-oncology program, BXCL701, into a Phase 2b registrational trial, pending further discussion with the FDA, in conjunction with exploring strategic options for our OnkosXcel subsidiary. These upcoming milestones, along with our strong financial foundation and late-stage programs, position BioXcel Therapeutics to deliver significant value to our shareholders while helping treat millions of patients."

Company Highlights

Neuroscience Franchise

IGALMI (dexmedetomidine) sublingual film

IGALMI is approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults.4 Up to an estimated 16 million institutional episodes occur annually within these two patient populations in the U.S.1-3*

Commercial

· Enhanced Market Access: Continued to generate strong interest in IGALMI from key stakeholder groups to drive formulary access with initial 26-member field force.

o More than 600 hospital Pharmacy and Therapeutics (P&T) Committees decisions are scheduled, with over 65 total formulary wins to date.
o Targeting Integrated Delivery Network (IDN) universe of approximately 280,000 beds:
· More than 70,000 (~25%) target IDN beds are scheduled to vote; secured formulary approval for ~7,000 (2%) target beds.
o Three Group Purchasing Organization (GPO) contracts in place covering nearly 50% of target beds; in active discussions with other leading GPOs.

· Completed Deployment of IGALMI Institutional Sales Force: Integrated commercial team covers majority of U.S. agitation market.
o Expanded sales force to 70 representatives, extending coverage from 700 to more than 1,700 target hospitals.
o Current target reach is more than 1,100 hospitals and over 7,000 unique health care professionals to influence formulary access.

· Amplified Marketing Efforts: Increased IGALMI awareness and education planned.
o Launched new promotional campaign directed to health care providers (HCPs) through multiple channels.
o Established large-scale peer speaker program designed to educate thousands of target HCPs in 1H 2023.
o Planned promotional presence at leading national and regional conferences in 2023.

Medical Affairs

· Increased Field Team Engagement: Medical Science Liaison (MSL) and Medical Managed Care (MMC) teams continued to grow engagement with medical community and P&T committee members.
o Increased HCP interactions by 50% from Q3 to Q4 2022 and observed growing interest of medical community in obtaining clinical information.
o Established HCP relationships in top target academic medical centers.

· Clinical Data Dissemination:
o Published three manuscripts and two letters to the editor in 2022; the first publication from SERENITY II pivotal trial, published in the Journal of the American Medical Association (JAMA), has been viewed more than 32,000 times and cited by 15 other publications.
o Preparing four new manuscripts and 19 conference abstract submissions for 2023.

Development Pipeline

BXCL501, a proprietary, sublingual film formulation of dexmedetomidine, has received Breakthrough Therapy and Fast Track designation for the acute treatment of agitation associated with dementia.

Alzheimer’s Disease-related Agitation: TRANQUILITY program is designed to evaluate BXCL501 for the acute treatment of Alzheimer’s-related agitation, where up to 100 million agitation episodes are estimated to occur in the U.S. annually.1*
o TRANQUILITY II: Trial is fully enrolled; nearing completion of three-month observation period in a few patients in assisted living facilities (ALFs) and residential settings.
· Data cleaning and verification in progress.
· Top-line data from pivotal trial expected in Q2 2023.
o TRANQUILITY III: Continuing enrollment of patients with moderate to severe dementia in nursing homes.

· Bipolar or Schizophrenia-related Agitation (At-Home Use): SERENITY III program is designed to evaluate BXCL501 for use at home, where up to 23 million agitation episodes are estimated to occur in the U.S. annually.1-3*

SERENITY III consists of two parts:

o Part 1: Assessing the efficacy and safety of 60mcg dose in acutely agitated patients with bipolar I or II disorder or schizophrenia in a supervised setting, similar to SERENITY I and II.
· More than 90% of patients enrolled and complete enrollment is imminent.
· Top-line efficacy data from pivotal trial expected in Q2 2023.
o Part 2: Evaluating the safety of self-administration of 60mcg dose at-home.
· Expect to initiate trial in Q2 2023.

· Adjunctive Treatment for Major Depressive Disorder (MDD) for At-Home Use: Phase 1b Multiple Ascending Dose (MAD) trial is designed to test safety and tolerability of daily dosing of BXCL501 to inform proof-of-concept (POC) trial dose selection in combination with selective serotonin – or serotonin-norepinephrine reuptake inhibitors (SSRIs or SNRIs, respectively) in MDD patients. Over 300 million antidepressant prescriptions are filled annually in the U.S.,5* and current treatments are limited by slow onset of action and incomplete responses.
o Enrollment has been completed; cohorts have received either daily or twice-daily dosing regimens for seven days, including a final cohort in combination with an SNRI.
o Data cleaning and verification in progress.
o Top-line results are expected in Q2 2023.

Research and Development Pipeline

BXCL502: Novel serotonergic receptor antagonist being developed for chronic treatment of agitation in patients with dementia and other related neuro-psychiatric conditions.

· Protype formulation has been completed.
· Undergoing Investigational New Drug application-enabling studies.
· Development path leverages efficacy and safety data from prior Phase 3 pivotal studies conducted by other pharma companies.

Emerging Pipeline: Leveraging our proprietary AI platform and deep multidisciplinary neuroscience and development expertise to identify novel pipeline candidates for neuropsychiatric disorders and neuro-rare diseases.

· Large language models, knowledge graphs, deep learning and machine learning methodologies centered around neuro-circuitry and receptor targets that modulate them.
· This strategy has enabled discovery of numerous promising product concepts that have been rank-ordered using an index for transformative care and commercial opportunity.
· Company is prioritizing these opportunities to select development candidates.

OnkosXcel Therapeutics

OnkosXcel Therapeutics is a subsidiary of BioXcel Therapeutics focused on the sustained growth of the Company’s immuno-oncology (I-O) franchise, including BXCL701, its most advanced I-O program. BXCL701 is an investigational, oral innate immune activator in development for the treatment of aggressive forms of prostate cancer and other solid and liquid tumors.

· Small Cell Neuroendocrine Metastatic Castration-Resistant Prostate Cancer (SCNC) Program: Presented positive Phase 2a efficacy data for BXCL701 in combination with KEYTRUDA (pembrolizumab) at the 2023 ASCO (Free ASCO Whitepaper) GU Cancers Symposium, demonstrating clinical proof-of-concept. In 2023, there will be an estimated 288,3006 new prostate cancer patients in the United States, with approximately 11,000 patients7 progressing to SCNC.
o BXCL701 has demonstrated activity in two prostate cancer subtypes ("cold" tumors).
· Data from the SCNC cohort showed response rate, duration of response, and safety data consistent with previously presented data from the adenocarcinoma cohort.
o BXCL701 in combination with KEYTRUDA demonstrated manageable adverse events (AEs) with no evidence of potentiation of immune-related AEs.
o Planned Phase 2b potential pivotal study for BXCL701 monotherapy and in combination with KEYTRUDA in SCNC expected to initiate in 2H 2023, subject to further discussions with FDA.
o Expect to initiate Phase 1b/2 trial in small cell lung cancer (SCLC) in 2H 2023.

· Predictive Biomarker for BXCL701: DPP9 overexpression was identified as a potential actionable biomarker for BXCL701 response.

· Hosted BXCL701 Key Opinion Leader (KOL) Day: Esteemed oncology experts highlighted the development landscape, challenges with current immunotherapy in prostate cancer, BXCL701 mechanism of action, and positive results from Phase 2 trial of BXCL701 in rare forms of prostate cancer.
o Planning to pursue additional indications in partnership with Georgetown Lombardi Cancer Center and Dana-Farber Cancer Institute in pancreatic cancer and AML, respectively.

Strategic Advancements: Actively evaluating strategic options for OnkosXcel Therapeutics, including potential partnering or third-party investments.

Corporate Updates

Patent Portfolio: The company is developing a broad global intellectual property portfolio, with over 100 patent applications in prosecution and multiple patents issued as of January 31, 2023.

· Neuroscience Franchise (BXCL501 and pipeline): Company’s patent portfolio, includes five issued U.S. patents, with four listed in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (commonly known as the Orange Book), as well as three patents from Japan and four patents from other countries. Additionally, the portfolio has 13 utility patent applications and four provisional applications in the U.S. and 87 utility patent applications in other countries, as well as four Patent Cooperation Treaty (PCT) applications not yet in the national phase.

· Immuno-oncology Franchise (BXCL701 and pipeline): Company’s patent portfolio, includes one issued patent in the U.S., one in Japan, and eight in other countries, as well as five provisional applications and seven utility patent applications in the U.S., including one with a Notice of Allowance, and 35 utility patent applications in other countries.

Fourth Quarter and Full Year 2022 Financial Results

Net Revenue: Net revenue was approximately $238,000 for the quarter and $375,000 for the full year 2022, which resulted from early product trials and reflects limited market access. Due to the Company’s direct shipping model to hospitals, wholesaler stocking was neither expected nor occurred.

Research and Development (R&D) Expenses: R&D expenses were $32.5 million for the fourth quarter of 2022, compared to $12.5 million for the same period in 2021.

R&D expenses were $91.2 million for the full year 2022, compared to $52.7 million for the same period in 2021. The increased expenses for both the fourth quarter and the full year were primarily attributable to an increase in clinical trial costs related to multiple major pivotal BXCL501 clinical programs.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $20.7 million for the fourth quarter of 2022, as compared to $13.6 million for the same period in 2021.

SG&A expenses were $68.8 million for the full year 2022, as compared to $54.2 million for 2021. The increased costs for both the fourth quarter and full year were primarily due to personnel and costs related to the launch of IGALMI in the U.S.

Net Loss: BioXcel Therapeutics had a net loss of $54.8 million for the fourth quarter of 2022, compared to a net loss of $26.1 million for the same period in 2021. For the full year, BioXcel Therapeutics reported a net loss of $165.8 million, compared to a net loss of $106.9 million for the same period in 2021. The loss for the year includes approximately $17.3 million in non-cash stock-based compensation. Total cash expenditures for 2022 totaled approximately $135.3 million.

Cash and cash equivalents totaled $193.7 million at December 31, 2022, compared to $233.0 million at December 31, 2021. To date in the first quarter of 2023, the Company utilized its existing at-the-market equity facility and received net proceeds of approximately $24 million.

The Company believes that full execution of our strategic financing with Oaktree and Qatar Investment Authority would result in a cash runway into 2025 for the Company.

Anticipated Milestones

· Clinical Trial Readouts
o Top-line data from pivotal TRANQUILITY II trial in Q2 2023
o Top-line efficacy data from pivotal SERENITY III trial in Q2 2023
o Top-line results from Phase 1b MAD trial for MDD program in Q2 2023
· Clinical Trial Initiations
o Initiate SERENITY III Part 2 in Q2 2023
o Initiate Phase 2b potential pivotal study of BXCL701 in SCNC in 2H 2023

Conference Call

BioXcel Therapeutics will host a conference call and webcast at 8:30 a.m. ET on March 9 to discuss its fourth quarter and full-year 2022 financial results and provide an update on recent operational highlights. To access the call, please dial 877-407-5795 (domestic) and 201-689-8722 (international). A live webcast will be available on the Investors section of the corporate website, bioxceltherapeutics.com, and a replay will be available through June 9, 2023.

BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the "Email Alerts" option under the News/Events menu of the Investors & Media section of its website.

About IGALMI (dexmedetomidine) sublingual film

INDICATION

IGALMI is indicated for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. Limitations of Use: The safety and effectiveness of IGALMI have not been established beyond 24 hours from the first dose.

IMPORTANT SAFETY INFORMATION

IGALMI can cause serious side effects, including:

· Decreased blood pressure, low blood pressure upon standing, and slower than normal heart rate, which may be more likely in patients with low blood volume, diabetes, chronic high blood pressure, and older patients. IGALMI is taken under the supervision of a healthcare provider who will monitor vital signs (like blood pressure and heart rate) and alertness after IGALMI is administered to help prevent falling or fainting. Patients should be adequately hydrated and sit or lie down after taking IGALMI and instructed to tell their healthcare provider if they feel dizzy, lightheaded, or faint.

Heart rhythm changes (QT interval prolongation). IGALMI should not be given to patients with an abnormal heart rhythm, a history of an irregular heartbeat, slow heart rate, low potassium, low magnesium, or taking other drugs that could affect heart rhythm. Taking IGALMI with a history of abnormal heart rhythm can increase the risk of torsades de pointes and sudden death. Patients should be instructed to tell their healthcare provider immediately if they feel faint or have heart palpitations.
· Sleepiness/drowsiness. Patients should not perform activities requiring mental alertness, such as driving or operating hazardous machinery, for at least 8 hours after taking IGALMI.
· Withdrawal reactions, tolerance, and decreased response/efficacy. IGALMI was not studied for longer than 24 hours after the first dose. Physical dependence, withdrawal symptoms (e.g., nausea, vomiting, agitation), and decreased response to IGALMI may occur if IGALMI is used longer than 24 hours.

The most common side effects of IGALMI in clinical studies were sleepiness or drowsiness, a prickling or tingling sensation or numbness of the mouth, dizziness, dry mouth, low blood pressure, and low blood pressure upon standing.

These are not all the possible side effects of IGALMI. Patients should speak with their healthcare provider for medical advice about side effects.

Patients should tell their healthcare provider about their medical history, including if they suffer from any known heart problems, low potassium, low magnesium, low blood pressure, low heart rate, diabetes, high blood pressure, history of fainting, or liver impairment. They should also tell their healthcare provider if they are pregnant or breastfeeding or take any medicines, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Patients should especially tell their healthcare provider if they take any drugs that lower blood pressure, change heart rate, or take anesthetics, sedatives, hypnotics, and opioids.

Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201-1088 or [email protected].

On March 9, 2023 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for severe unmet medical needs, reported the first patient was dosed in a Phase 1b/2a trial evaluating bemcentinib in combination with the current standard of care, immune checkpoint inhibitor pembrolizumab and doublet chemotherapy, for the treatment of 1st line (1L) Non-Small Cell Lung Cancer (NSCLC) patients harboring STK11 mutations (STK11m) (Press release, BerGenBio, MAR 9, 2023, View Source [SID1234628407]).

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"Approximately 20% of non-squamous NSCLC patients harbor STK11m and do not currently have effective treatment options," said Martin Olin, Chief Executive Officer of BerGenBio. "One specific attribute of this group is that they almost all demonstrate high levels of AXL activation. We are elated to have dosed the first patient in our trial and to continue our evaluation of bemcentinib and its ability to inhibit AXL to revive STK11m NSCLC patients’ response to checkpoint inhibitors and chemotherapy."

The trial’s lead investigator, Rajwanth Veluswamy, M.D., MSCR, Assistant Professor of Medicine, Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai (New York, NY), commented, "Today, STK11 mutations are correlated with a very poor prognosis for patients suffering from NSCLC. These mutations are widely recognized for their ability to impede the activity of anti-PD-1/L1 therapy. My colleagues and I are driven to find a better outcome for this large patient population and are eager to assess bemcentinib’s potential in achieving this goal."

The global, open-label Phase 1b/2a trial is designed to determine the safety, tolerability and efficacy of bemcentinib with standard of care treatments in untreated advanced/metastatic non-squamous NSCLC patients with STK11 mutations and no actionable mutations. The Phase 1b portion of the study will evaluate the safety and feasibility of bemcentinib in combination with pembrolizumab and doublet chemotherapy in 1L advanced/metastatic non-squamous NSCLC patients, regardless of STK11 status. The Phase 2a expansion part will assess the efficacy of bemcentinib in the same treatment combination in 1L advanced/metastatic non-squamous NSCLC patients with STK11 mutations.

On March 9, 2023 Aptose Biosciences Inc. (Nasdaq: APTO; TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, reported financial results for the fourth quarter and full year ended December 31, 2022, on Thursday, March 23, 2023, after the close of the market, and provide a corporate update (Press release, Aptose Biosciences, MAR 9, 2023, View Source [SID1234628406]).

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Conference Call & Webcast:

Date: Thursday, March 23, 2023
Time: 5:00 PM ET
Audio Webcast Only: link
Q&A Participant Registration Link*:

here

(https://register.vevent.com/register/BI9394078d0ea14714aca591ffe06992f1)
*Analysts interested in participating in the question-and-answer session will pre-register for the event from the participant registration link above to receive the dial-in numbers and a personal PIN, which are required to access the conference call. They also will have the option to take advantage of a Call Me button and the system will automatically dial out to connect to the Q&A session.

The audio webcast also can be accessed through a link on the Investor Relations section of Aptose’s website here. A replay of the webcast will be available on the company’s website for 30 days.

The press release, the financial statements and the management’s discussion and analysis for the quarter and year ended December 31, 2022 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml.

On March 9, 2023 Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in liver and viral diseases, reported recent business progress and financial results for the fourth quarter and full year 2022 (Press release, Aligos Therapeutics, MAR 9, 2023, View Source [SID1234628405]).

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"Last year was very productive for our team," said Lawrence Blatt, PhD, MBA, Chairman & CEO of Aligos. "We made great progress advancing our NASH, COVID-19, and CHB drug candidates. In particular, our drug candidate for NASH, ALG-055009, continued to generate positive data in the clinic that differentiates it from other THR-beta agonists in development. Additionally, important nonclinical advances were made for ALG-097558, our protease inhibitor for COVID-19, which is anticipated to enter the clinic in the first half of this year. Finally, our CAM-E, ALG-000184, continues to generate potentially best-in-class antiviral activity data, including HBsAg reductions that are significantly more substantial than competitor CAM-E drugs, suggesting that it may be an important component in future treatments for CHB. Taken together, we expect these programs to have multiple important data readouts as we continue to advance them throughout 2023. These data have the potential to be important drivers of shareholder value and we look forward to sharing them as they emerge."

Recent Business Progress

Aligos Portfolio of Drug Candidates

NASH Program

ALG-055009 – Evaluation of single and multiple ascending doses (SAD, MAD) in healthy volunteers and subjects with hyperlipidemia was completed and MAD data were presented in November at The Liver Meeting (AASLD). Data from this Phase 1 study continue to be favorable and are positively differentiated compared to competitor drugs in the same class. We remain on track to submit a Phase 2 clinical trial filing by the end of 2023.
COVID-19

ALG-097558 – First-in-human-enabling nonclinical studies are ongoing. We anticipate initiating the clinical evaluation of single and multiple ascending doses of this drug candidate in healthy volunteers in Q2 2023, with top line safety and PK data available by Q4 2023.
HBV Programs

ALG-000184 (CAM-E) – Evaluation of a range of doses given over 28 days to both HBeAg positive and HBeAg negative CHB subjects was completed. Additionally, new interim data from cohorts evaluating dosing durations of up to 48 weeks in HBeAg positive subjects were presented by Professor Hou, Nanfang Medical University, China at the Asian Pacific Association for the Study of the Liver meeting (APASL) in February 2023. This presentation demonstrated favorable safety and antiviral activity for up to 12 weeks of dosing, including notable reductions in HBsAg levels. We anticipate sharing additional safety and antiviral activity data for up to 48 weeks of dosing from these and other ongoing cohorts at scientific conferences throughout 2023.

ALG-125755 (siRNA) – The SAD evaluation of ALG-125755 in healthy volunteers was completed and doses up to 200 mg were found to have a favorable safety and PK profile (Gane et al., APASL 2023). As a result, SAD cohorts in CHB subjects are now being evaluated. We anticipate sharing emerging data from these cohorts at scientific conferences throughout 2023.
Financial Results for the Fourth Quarter and Full Year 2022

Cash, cash equivalents and investments totaled $125.8 million as of December 31, 2022, compared with $205.8 million as of December 31, 2021. We continue to believe our cash balance provides sufficient cash to fund planned operations through the end of 2024.

Net losses for the three months ended December 31, 2022, were $21.9 million or basic and diluted net loss per common share of $(0.51), compared to net losses of $37.7 million or basic and diluted net loss per common share of $(0.89) for the three months ended December 31, 2021.

Net losses for the year ended December 31, 2022, were $96.0 million or basic and diluted net loss per common share of $(2.25), compared to net losses of $128.3 million or basic and diluted net loss per common share of $(3.22) for the year ended December 31, 2021.

Research and development (R&D) expenses for the three months ended December 31, 2022, were $19.1 million compared with $28.6 million for the same period of 2021. The decrease in R&D expenses for this comparative period is primarily attributable to a decrease in third-party expenses due to our continued wind down related to the discontinuation of our STOPS and ASO programs, and the manufacturing of drug supply in advance of our clinical and nonclinical activities. Total R&D stock-based compensation expense incurred for the three months ended December 31, 2022, was $1.9 million compared with $1.9 million for the same period of 2021.

R&D expenses for the year ended December 31, 2022 were $85.1 million, compared with $104.2 million for the same period of 2021. The decrease in R&D expenses for this comparative period is primarily attributable to a decrease in third-party expenses due to our continued wind down related to the discontinuation of our STOPS and ASO programs, and the manufacturing of drug supply in advance of our clinical and nonclinical activities. Total R&D stock-based compensation expense incurred in the year ended December 31, 2022, was $8.0 million, compared with $7.6 million for the same period of 2021.

General and administrative (G&A) expenses for the three months ended December 31, 2022, were $7.1 million compared with $9.7 million for the same period of 2021. The decrease in G&A expenses for this comparative period is primarily attributable to facility costs and outside services costs. Total G&A stock-based compensation expense incurred for the three months ended December 31, 2022, was $1.6 million compared with $1.7 million for the same period of 2021.

General and administrative (G&A) expenses for the year ended December 31, 2022, were $26.4 million compared with $28.5 million for the same period of 2021. The decrease in G&A expenses for this comparative period is primarily attributable to facility costs and outside services costs. Total G&A stock-based compensation expense incurred for the year ended December 31, 2022, was $6.7 million compared with $5.9 million for the same period of 2021.

On March 9, 2023 Acorda Therapeutics, Inc. (Nasdaq: ACOR) reported its financial results for the fourth quarter and full year ended December 31, 2022 (Press release, Acorda Therapeutics, MAR 9, 2023, View Source [SID1234628404]).

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"Acorda’s operating and financial performance improved throughout the year, meeting our financial guidance for 2022 AMPYRA net revenue, INBRIJA U.S. net revenue, cash, and adjusted OPEX. We also delivered a stream of business successes that have driven shareholder value," said Ron Cohen, M.D., Acorda’s President and Chief Executive Officer.

"These successes included a substantial AMPYRA arbitration award and markedly lower cost of goods for AMPYRA going forward, renegotiation of our agreements with Catalent for INBRIJA manufacturing on more favorable terms, the launches of INBRIJA in Germany and Spain, and obtaining an extension from Nasdaq to bring the company’s share price back into compliance with Nasdaq listing requirements," he continued.

"In 2023 we expect to make further progress in reducing operating expenses, increasing INBRIJA’s trajectory, and maintaining the strength of the AMPYRA brand. We are also in active discussions for additional agreements to commercialize INBRIJA in multiple ex-U.S. territories; and we also expect Biopas to launch in Latin America in early 2024."

Fourth Quarter 2022 Financial Results

For the quarter ended December 31, 2022, the Company reported INBRIJA U.S. net revenue of $9 million, a 13.1% decrease compared to the same quarter in 2021. The Company did not report any ex-U.S. INBRIJA sales in the fourth quarter for either period.

For the quarter ended December 31, 2022, the Company reported AMPYRA net revenue of $18.8 million, a 16.6% decrease compared to the same quarter in 2021. Additionally, for the quarter ended December 31, 2022, the Company reported FAMPYRA royalty revenues of $2.7 million, a 25.1% decrease compared to the same quarter in 2021. As previously disclosed, AMPYRA lost its exclusivity and generics entered the market in 2018, and the Company expects AMPYRA revenue to continue to decline. The decline in royalty revenues is largely attributed to the launch of generic competition in the German market in 2022.

Research and development (R&D) expenses for the quarter ended December 31, 2022 were $1.2 million, including negligible share-based compensation expenses, compared to $1.4 million, including $0.1 million of share-based compensation for the same quarter in 2021.

Sales, general and administrative (SG&A) expenses for the quarter ended December 31, 2022 were $26.3 million, including $0.2 million of share-based compensation, compared to $28.4 million, including $0.4 million of share-based compensation for the same quarter in 2021.

Provision for income taxes for the quarter ended December 31, 2022 was $2.4 million, compared to a provision for income taxes of $1.7 million for the same quarter in 2021.

The Company reported net income of $19.1 million for the quarter ended December 31, 2022, or $0.79 per basic share and $0.57 per diluted share. Net loss in the same quarter of 2021 was ($20.6) million, or a net loss of ($1.73) per share on both a basic and diluted basis. The increase in net income is primarily driven by recognition of a gain upon extinguishment of debt of a subsidiary of $27.1 million, and receipt of an arbitration award of $18.3 million, reduction in the change in fair value of contingent consideration of $3.1 million, and reduced R&D and SG&A expenses of $2.3 million, partially offset by a one-time contract termination fee of $4 million, and reduced net revenues of $5.5 million.

Full Year Ended December 31, 2022 Financial Results

For the full year ended December 31, 2022, the Company reported INBRIJA global net revenue of $30.9 million, $28 million of which was derived from sales in the U.S., and $2.9 million from ex-U.S. sales, compared to $29.6 million net revenue for the full year 2021, which was derived from U.S. sales only.

For the full year ended December 31, 2022, the Company reported AMPYRA net revenue of $72.9 million, compared to $84.6 million for the full year 2021. Additionally, for the full year ended December 31, 2022, the Company reported FAMPYRA royalty revenues of $11.7 million, compared to $13.8 million for the full year ended 2021. This decline in royalty revenues is largely attributed to the launch of generic competition in the German market in 2022.

Research and development (R&D) expenses in 2022 were $5.8 million, including $0.1 million of share-based compensation, compared to $10.4 million, including $0.7 million of share-based compensation for the full year 2021.

Sales, general and administrative (SG&A) expenses were $106.3 million, including $1.4 million of share-based compensation, compared to $124.4 million, including $2.3 million of share-based compensation, for the full year 2021.

Provision for income taxes was $30.7 million, compared to a benefit from income taxes of $5.1 million for the full year 2021. This change is a result of the elimination of Net Operating Losses ("NOLs") due to a Section 382 change in control due to cumulative changes in the Company’s ownership over the preceding three years, driven by the Company’s interest payment of 2024 convertible senior secured notes in shares in June 2022, which required the write-off $57.9 million of NOLs.

The Company reported net loss of ($65.9) million, or a net loss of ($3.34) per basic and diluted share, compared to a net loss of ($104) million, or a net loss of ($9.79) per basic and diluted share for the full year ended 2021. The decrease in net loss is primarily driven by a gain upon extinguishment of debt of a subsidiary of $27.1 million, reduced R&D and SG&A expenses of $22.8 million, receipt of an arbitration award of $18.3 million, increase of the gain recognized in the change in fair value of contingent consideration of $9.6 million, and reduced cost of sales of $10.5 million, partially offset by an increased provision for income taxes of $35.8 million, lower net revenues of $10.5 million, and a one-time contract termination fee of $4 million.

At December 31, 2022, the Company had cash, cash equivalents, and restricted cash of $44.7 million compared to $65.2 million at year end 2021. Restricted cash includes $6.2 million in escrow related to the 6.00% semi-annual interest portion of the convertible notes.

Early 2023 / 2022 Highlights

In March 2023, Esteve announced that they had launched INBRIJA in Spain.
In February 2023, a Nasdaq Hearings Panel granted an extension until June 20, 2023 to comply with listing requirements and remain listed on the Nasdaq Global Select Market.
In January 2023, Acorda entered into a new long-term, global supply agreement with Catalent to significantly lower minimum purchase requirements for INBRIJA in 2023 and 2024; and beginning in 2025, Acorda will pay a fixed, per-capsule price for INBRIJA.
In December 2022, Acorda obtained waivers from the Finnish government of approximately $27.1 million in loans related to its Biotie subsidiary.
In December 2022, Acorda made a cash interest payment of approximately $6.2 million related to its Convertible Senior Secured Notes Indenture ("2024 Notes").
In November 2022, Acorda stockholders approved a reverse stock split, which can be utilized by the Company to regain compliance with the listing requirements for the Nasdaq Global Select Market.
In October 2022, Acorda was awarded a total of $18.3 million, including interest, through an arbitration involving a dispute with Alkermes over AMPYRA royalties. As a result, Acorda will no longer have to pay Alkermes any royalties on net sales for AMPYRA. In addition, Acorda is free to use alternative sources for supply of AMPYRA which it has secured. The Company estimates that as a result its cost of goods for Ampyra in 2023 will be lower by $10 million – $12 million.
In August 2022, Acorda announced a license agreement with Asieris Pharmaceuticals relating to its preclinical asset, Nepicastat. Acorda received an upfront payment of $0.5 million, and is eligible to receive up to an additional $7 million based on the achievement of regulatory milestones and royalties on future net sales of any product developed.
In June 2022, Acorda met its obligation to HealthCare Royalty Partners and received the full benefit of the royalty payments from Biogen on FAMPYRA sales.
In June 2022, Esteve launched INBRIJA in Germany and Acorda received $2.9 million in revenue related to this launch.
In May 2022, Acorda announced an agreement with Biopas Laboratories to commercialize INBRIJA in the nine largest markets in Latin America, including Brazil and Mexico. Acorda will receive a significant, double-digit, tiered percentage of the selling price of INBRIJA and will also receive sales-based milestones.
2022 Financial Guidance2

For the full year 2022, the Company achieved its guidance targets for INBRIJA U.S. net revenue of $28 million, AMPYRA net revenue of $72.9 million, adjusted OPEX of $112 million, and ending cash balance of $44.7 million.

For the full year 2022, adjusted EBITDA was a loss of ($2.4) million, which fell short of guidance of $5.6 – $5.8 million. The shortfall was primarily due to a non-cash adjustment to the change in fair value of contingent consideration identified through the Company’s year-end procedures. Additionally, the Company recorded inventory write-offs given estimates of future sales compared to inventory to be received under the new global supply agreement with Catalent. There was no impact to cash.

2023 Financial Guidance

For the full year 2023, the Company is targeting INBRIJA U.S. net revenue to be $38 – $42 million.

The Company is targeting 2023 AMPYRA net revenue to be $65 – $70 million. As previously disclosed, AMPYRA lost its exclusivity and generics entered the market in 2018, and the Company expects AMPYRA revenue to continue to decline.

The Company is targeting adjusted OPEX to be $93 – $103 million and ending cash balance to be $43 – $47 million. The 2023 guidance includes the impact of the new global supply agreement with Catalent. Adjusted OPEX is described below under "Non-GAAP Financial Measures." As described below, we are unable to reconcile our adjusted OPEX guidance to GAAP due to the forward-looking nature of the adjustments that are needed to determine this information.

Updated Long-Term Financial Guidance

The financial guidance below includes non-GAAP financial measures. Adjusted OPEX for fiscal years 2024 – 2027 is described below under "Non-GAAP Financial Measures."

Long-term guidance for net revenue, 2024-2027, remains unchanged from previous guidance (other than rounding adjustments).

Long-term guidance for adjusted OPEX increased in 2024 from the previous guidance due to the expected payment of $1.0 million to support the completion of the PSD-7 for INBRIJA manufacture under the new global supply agreement with Catalent. Adjusted OPEX for 2025-2027 remains unchanged.

Guidance Ranges in
U.S.$M

2023

2024

2025

2026

2027

(unaudited)

INBRIJA U.S. net
revenue

$38 – $42

$50 – $56

$59 – $65

$63 – $70

$70 – $78

AMPYRA net revenue

$65 – $70

$62 – $68

$62 – $68

$64 – $71

$62 – $69

Adjusted OPEX

$93 – $103

$92 – $102

$93 – $103

$96 – $106

$99 – $109

Ending Cash Balance

$43 – $47

$51 – $56

$72 – $79

$97 – $107

$124 – $138

Webcast and Conference Call

The Company will host a webcast/conference call in conjunction with its fourth quarter and year end 2022 update and financial results today at 8:30 a.m. ET.

To participate in the Webcast, please use the following registration link:

View Source
If you register for the Webcast, you will have the opportunity to submit a written question for the Q&A portion of the presentation. After you have registered, you will receive a confirmation email with the Webcast details. On the day of the Webcast, you will receive an email 2 hours prior to the start of the Webcast with the link to join. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 11:30 a.m. ET on March 9, 2023 until 11:59 p.m. ET on April 8, 2023. To access the replay, please dial 1 866 813 9403 (domestic) or +44 204 525 0658 (international); access code 413769. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

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 historical and forward-looking non-GAAP financial measures. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP, and the calculation of the non-GAAP financial measures included herein may differ from similarly titled measures used by other companies. The Company believes that the presentation of these non-GAAP financial measures, when viewed in conjunction with actual GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because it excludes (i) expenses that pertain to corporate restructurings not routine to the operation of our business, (ii) non-cash charges that are substantially dependent on changes in the market price of our common stock, and (iii) other items as set forth above that are not ascertainable at the present time. We believe these non-GAAP financial measures help indicate underlying trends in the Company’s business and are important in comparing current results with prior period results and understanding expected operating performance. Also, management uses these non-GAAP financial measures to establish budgets and operational goals, and to manage the Company’s business and evaluate its performance. In addition, management believes that adjusted OPEX is important in evaluating the administrative costs of operating the Company’s business.

Adjusted OPEX includes (i) research and development expenses and (ii) selling, general, and administrative expenses and excludes (i) costs of goods sold, (ii) amortization of intangible assets, (iii) change in fair value of derivative liability, (iv) change in fair value of acquired contingent liability, and (v) the principal-only portion of an arbitration award less one-time contract termination expenses relating to the new global supply agreement with Catalent. Adjusted EBITDA is GAAP net income (loss) before income taxes less depreciation, amortization, and interest and excluding (i) non-cash compensation charges and benefits that are substantially dependent on changes in the market price of our common stock, (ii) changes in the fair value of acquired contingent consideration which do not correlate to our actual cash payment obligations in the relevant periods, (iii) expenses that pertain to corporate restructurings which are not routine to the operation of the business, (iv) changes in the fair value of derivative liability relating to the 2024 convertible senior secured notes, (v) one-time contract termination expenses relating to the new global supply agreement with Catalent, and (vi) gain on extinguishment of debt of a subsidiary which is a non-cash charge and not related to the operation of the business.

We are unable to reconcile our guidance for these non-GAAP measures to GAAP due to the forward-looking nature of the adjustments that are needed to determine this information, which includes information regarding future compensation charges, future changes in the market price of our common stock, and changes in the fair value of derivative and contingent liabilities, none of which are available at this time.