On May 9, 2022 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the first quarter ended March 31, 2022, and shared recent operational progress (Press release, bluebird bio, MAY 9, 2022, View Source [SID1234613918]).

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"For more than a decade, bluebird bio has helped chart the path for the field of gene therapy and today, we are closer than ever before to realizing our mission of bringing potentially curative gene therapies to patients and their families," said Andrew Obenshain, chief executive officer, bluebird bio. "We have taken action to sharpen our focus and our finances, as the FDA completes its review of the beti-cel and eli-cel BLAs, and we prepare for the launch of two gene therapies in the second half of the year. In parallel, we are actively collecting the manufacturing comparability data required for lovo-cel and remain focused on submitting our BLA for sickle cell disease to the FDA in Q1 2023."

RECENT HIGHLIGHTS

BETI-CEL AND ELI-CEL

ADVISORY COMMITTEE MEETING ON JUNE 9-10, 2022 – On April 13, the U.S Food and Drug Administration (FDA) confirmed its plans to review betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel) in an FDA Cellular, Tissue and Gene Therapies Advisory Committee Meeting that will take place over two days on June 9-10, 2022. The advisory committee will discuss the efficacy and safety data supporting the benefit/risk of beti-cel and eli-cel respectively, as well as safety information relevant to both therapies. If approved, beti-cel and eli-cel would be the Company’s first FDA approved therapies as an independent severe genetic disease company. An overview of the agenda was included in the Federal Register announcement posted on April 13. The full agenda will be determined by the FDA and included in briefing materials that will be posted by the FDA closer to the date of the advisory committee meeting.
LOVO-CEL

CONTINUED PROGRESS TOWARD BLA SUBMISSION – bluebird bio has enrolled more than half of the patients in the HGB-210 study that will be needed to support manufacturing data requirements for the lovotibeglogene autotemcel (lovo-cel) biologics licensing application (BLA) and the Company is on track to submit a BLA to the FDA for sickle cell disease (SCD) in the first quarter of 2023. As previously communicated, the Company has treated all patients in HGB-206 Group C who will form the primary basis of efficacy for BLA submission, with the demonstration of analytical comparability and validation of the commercial manufacturing process as the key remaining actions prior to submission of the planned BLA. Enrollment and dosing for patients 18 and older are continuing in the HGB-210 study and the Company remains in active dialogue with the FDA about the resolution of the partial clinical hold for patients under 18.
COMPANY

RESTRUCTURING UPDATE – On April 5, 2022, bluebird bio announced a comprehensive restructuring intended to deliver up to $160 million in cost savings over the next two years, reduce its workforce by approximately 30% and extend its cash runway into the first half of 2023. Near-term cost savings were realized in April as bluebird began implementing the restructuring. The Company continues to evaluate additional financing options, including public or private equity financings and monetizing any priority review vouchers that may be issued upon approval of beti-cel or eli-cel.
NEW HEADQUARTERS MOVE –bluebird bio has begun transitioning to its new headquarters in Assembly Row. The Company’s new HQ is designed to reflect modern ways of working and estimated to result in more than $120 million in cost savings over the next six years. bluebird will maintain laboratory space and operations at 60 Binney St. in Cambridge through 2023.
UPCOMING INVESTOR EVENTS

Members of the management team will participate in the following upcoming investor conferences:

BofA Securities 2022 Health Care Conference, Wednesday, May 11, at 3:20 p.m. PT at the Encore Hotel, Las Vegas, NV
2022 RBC Capital Markets Global Healthcare Conference, Tuesday, May 17, at 8:00 a.m. ET at the Intercontinental NY Barclay, New York, NY
Goldman Sachs 43rd Annual Global Healthcare Conference, Wednesday, June 15, at 4:00 p.m. PT at the Terranea Resort, Rancho Palos Verdes, CA
To access the live webcast of bluebird bio’s presentations, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source A replay of the webcasts will be available on the bluebird bio website for 90 days following the event.

UPCOMING ANTICIPATED MILESTONES

LOVO-CEL

The Company is in active communication with the FDA to resolve the partial clinical hold and resume enrollment and treatment of patients under the age of 18.
The Company plans to complete manufacturing of commercial drug product validation lots by mid-2022.
The Company expects to confirm vector and drug product analytical comparability by Q4 2022.
The Company plans to submit its BLA for lovo-cel in Q1 2023.
BETI-CEL

The FDA has set a PDUFA goal date of August 19, 2022, for a decision on the approval of beti-cel in patients with β-thalassemia with commercial launch expected to follow in the beginning of Q4 2022 if approved.
An FDA advisory committee meeting for beti-cel and eli-cel will be held over the course of two days on June 9-10, 2022.
ELI-CEL

The FDA has set a PDUFA goal date of September 16, 2022, for a decision on the approval of eli-cel in patients with cerebral adrenoleukodystrophy with therapy availability expected in Q4 2022 if approved.
An FDA advisory committee meeting for beti-cel and eli-cel will be held over the course of two days on June 9-10, 2022.
bluebird bio is in active communication with the FDA to resolve the clinical hold and anticipates the FDA’s questions may be resolved concurrent with the agency’s ongoing review of the Company’s BLA submission.
FIRST QUARTER 2022 FINANCIAL RESULTS

Cash Position: The Company’s restricted cash, cash and cash equivalents and marketable securities balance was approximately $312 million, including restricted cash of approximately $45 million, as of March 31, 2022. The full-year 2022 cash burn is expected to be less than $340 million with a 35 to 40 percent reduction in operating costs anticipated by year-end 2022.

The Company is exploring multiple financing opportunities, including public or private equity financings and monetizing any priority review vouchers that may be issued upon approval of beti-cel or eli-cel.
Revenues: Total revenue was $1.9 million for the three months ended March 31, 2022, compared to $0.9 million for the three months ended March 31, 2021.
R&D Expenses: Research and development expenses from continuing operations were $77.9 million for the three months ended March 31, 2022, compared to $82.8 million for the three months ended March 31, 2021. The decrease of $4.9 million was primarily due to decreased employee compensation, benefit, and other head-count related expenses, offset by increased manufacturing costs.
SG&A Expenses: Selling, general and administrative expenses from continuing operations were $36.1 million for the three months ended March 31, 2022, compared to $63.6 million for the three months ended March 31, 2021. The decrease of $27.5 million was primarily due to decreased employee compensation, benefit, and other head-count related expenses and decreased commercial readiness activities due to the Company’s decision to focus its efforts on the U.S. market for beti-cel, eli-cel, and lovo-cel.
Net Loss: Net loss from continuing operations was $122.2 million for the three months ended March 31, 2022, compared to $121.5 million for the three months ended March 31, 2021.

On May 9, 2022 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, reported financial results for the first quarter of 2022, including current and projected financial resources (Press release, Geron, MAY 9, 2022, View Source [SID1234613917]).

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"This quarter, we continued to build on the momentum from our clinical execution throughout 2021, with our first Phase 3 data readout expected in just eight months. We believe that if the Phase 3 IMerge trial in lower risk MDS confirms similar depth, breadth and durability of transfusion independence, and safety, observed in our Phase 2 study, imetelstat could represent a significant treatment advance for these heavily transfusion-burdened patients," said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. "Separately, we are advancing our IMpactMF Phase 3 refractory MF trial by continuing to open clinical sites around the world, which we expect to enable a potential interim analysis in 2024. We are also executing on our IMproveMF Phase 1 study, which has recently started. This study will allow us to potentially demonstrate safety and efficacy of combination therapy with imetelstat and ruxolitinib in the frontline MF setting."

Dr. Scarlett continued: "The successful financing in April adds to our current financial resources, as well as provides access to additional capital upon the potential exercise of warrants in the future. We expect these current and projected financial resources will be sufficient for our projected operations through the end of 2023, including planned key regulatory milestones and pre-commercial activities. In addition, this extended cash runway provides us the opportunity to thoughtfully and broadly explore multiple business strategies, including partnering and financing options, to optimize the value of imetelstat for patients and shareholders."

Current and Projected Financial Resources

As of March 31, 2022, the Company had $178.0 million in cash and marketable securities. In April 2022, the Company completed an underwritten public offering of 53,333,334 shares of Geron common stock and a pre-funded warrant to purchase 18,095,238 shares of Geron common stock (pre-funded warrant), together with accompanying warrants to purchase 35,714,286 shares of Geron common stock (stock purchase warrants). The combined public offering price of the common stock and accompanying stock purchase warrants was $1.05 per share. The combined public offering price of the pre-funded warrant and accompanying stock purchase warrant was $1.049 per share. The net cash proceeds from this offering are approximately $70 million, after deducting the underwriting discount and other offering expenses payable by the Company and excludes any future proceeds from the exercise of the pre-funded warrant or the stock purchase warrants. The stock purchase warrants have an exercise price of $1.45 per share and expire five years from the date of issuance; however, such term will be shortened upon achievement of a regulatory milestone.

Under current planning assumptions, the Company projects its existing capital resources, including the net proceeds from the public offering completed in April 2022, and projected future proceeds of up to $124.3 million from the exercise of currently outstanding warrants will be sufficient to fund Geron’s projected level of operations, which includes preparatory activities for potential U.S. commercial launch of imetelstat in lower risk MDS, until the end of 2023.

First Quarter 2022 Results

For the first quarter of 2022, the Company reported a net loss of $30.1 million, or $0.09 per share, compared to $27.8 million, or $0.09 per share, for the same period in 2021.

Revenues for the first quarter of 2022 were $123,000 compared to $137,000 for the same period in 2021. Royalty revenues in 2022 and 2021 primarily reflect estimated royalties from sales of cell-based research products from the Company’s divested stem cell assets.

Total operating expenses for the first quarter of 2022 were $28.8 million compared to $28.6 million for the same period in 2021. Research and development expenses for the first quarter of 2022 were $22.1 million compared to $21.1 million for the same period in 2021. The increase in research and development expenses in the first quarter of 2022, compared to the same period in 2021, primarily reflects higher personnel-related costs for additional headcount. General and administrative expenses for the first quarter of 2022 were $6.7 million compared to $7.5 million for the same period in 2021. The decrease in general and administrative expenses in the first quarter of 2022, compared to the same period in 2021, primarily reflects the net result of reduced costs related to modernizing the internal infrastructure to support commercial launch and lower legal fees, partially offset by higher personnel-related expenses for additional headcount.

Interest income for the first quarter of 2022 was $112,000 compared to $173,000 for the same period in 2021. The decrease in interest income in the first quarter of 2022, compared to the same period in 2021, primarily reflects lower yields on the Company’s reduced marketable securities portfolio.

Interest expense for the first quarter of 2022 was $1.5 million compared to $743,000 for the same period in 2021. Currently, the Company has $50.0 million in principal debt outstanding.

Net other expense for the first quarter of 2022 was $56,000 compared to net other income of $1.2 million for the same period in 2021. During the first quarter of 2021, the Company sold all of its holdings in an equity investment resulting in a net realized gain of $1.2 million, including foreign currency translation adjustments.

Projected 2022 Financial Guidance Reaffirmed

For fiscal year 2022, under generally accepted accounting principles (GAAP), the Company continues to expect total operating expenses in the range of approximately $155 million to $165 million, which includes non-cash items such as: stock-based compensation expense, amortization of debt discounts and issuance costs and depreciation and amortization. The Company continues to expect non-GAAP total operating expenses for fiscal year 2022, which excludes estimated non-cash items such as: stock-based compensation expense, amortization of debt discounts and issuance costs and depreciation and amortization, in the range of approximately $140 million to $150 million.

The fiscal year 2022 financial guidance reflects costs to support: (a) preparatory activities for top-line results from the IMerge Phase 3 clinical trial and readiness for potential regulatory filings and commercialization of imetelstat in lower risk MDS; (b) continued conduct of IMerge and IMpactMF and commencement of new clinical studies associated with the imetelstat pipeline expansion strategy; (c) finalizing validation batches of imetelstat at contract manufacturers to enable future production of imetelstat for clinical and commercial purposes; (d) projected increases in headcount and (e) interest payments on outstanding debt.

As of March 31, 2022, the Company had 79 employees. The Company plans to grow to a total of approximately 90 to 100 employees by year-end 2022.

Conference Call

Geron will host a conference call at 4:30 p.m. ET on Monday, May 9, 2022 to review recent events and first quarter 2022 financial results.

A live webcast of the conference call and related presentation will be available on the Company’s website at www.geron.com/investors/events. An archive of the webcast will be available on the Company’s website for 30 days.

Participants may access the webcast by registering online using the following link: View Source Participants that are unable to register online can access the conference call via telephone by dialing domestically +1 (888) 330-2434 or internationally +1 (240) 789-2725. The conference ID is 67335.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic malignancies. Data from Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment.

About IMerge Phase 3

IMerge Phase 3 is a double-blind, randomized, placebo-controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 170 transfusion dependent patients with Low or Intermediate-1 risk myelodysplastic syndromes (MDS), also referred to as lower risk MDS, who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The primary endpoint is the rate of red blood cell (RBC) transfusion independence (TI) for any consecutive period of eight weeks or longer, or 8-week RBC-TI rate. Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks, or 24-week RBC-TI rate, duration of TI and the rate of hematologic improvement-erythroid (HI-E), defined as a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden.

IMerge Phase 3 is fully enrolled and patient enrollment has been closed. For additional information about IMerge Phase 3, visit ClinicalTrials.gov/NCT02598661.

About IMpactMF

IMpactMF is an open label, randomized, controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 320 patients with Intermediate-2 or High-risk myelofibrosis (MF) who are refractory to prior treatment with a JAK inhibitor, also referred to as refractory MF. Patients will be randomized to receive either imetelstat or best available therapy. The primary endpoint is overall survival (OS). Key secondary endpoints include symptom response, spleen response, progression free survival, complete remission, partial remission, clinical improvement, duration of response, safety, pharmacokinetics, and patient reported outcomes.

IMpactMF is currently enrolling patients. For further information about IMpactMF, including enrollment criteria, locations and current status, visit ClinicalTrials.gov/NCT04576156.

About IMproveMF

IMproveMF is a single arm, open label Phase 1 study to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of imetelstat in combination with ruxolitinib in patients with frontline myelofibrosis (MF), consisting of two parts. Part one will enroll up to 20 patients and is designed to identify a safe dose for the combination of imetelstat and ruxolitinib. Part two will also enroll approximately 20 patients and is designed to confirm the dose identified in part one.

On May 9, 2022 Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (Inhibikase), a clinical-stage pharmaceutical company developing therapeutics to modify the course of Parkinson’s disease and related disorders, reported that it will report financial results for the first quarter ended March 31, 2022 on Monday, May 16, 2022, after the close of U.S. markets (Press release, Inhibikase Therapeutics, MAY 9, 2022, View Source [SID1234613916]). Following the announcement, the Company will host a conference call and webcast at 8:00 a.m. ET on Tuesday, May 17, 2022 to provide a corporate update and review the financial results.

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The conference call can be accessed by dialing 877-407-4018 (United States) or 201-689-8471 (International) with the conference code 13729218. A live webcast may be accessed using the link here, or by visiting the investors section of the Company’s website at www.inhibikase.com. After the live webcast, the event will be archived on Inhibikase’s website for approximately 90 days after the call.

On May 9, 2022 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the first quarter ended March 31, 2022 (Press release, Syndax, MAY 9, 2022, View Source [SID1234613915]). In addition, the Company provided a clinical and business update.

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"Throughout the first quarter, we continued to realize significant progress advancing our two pivotal programs, for which we expect to report topline data starting in the first half of 2023," said Michael A. Metzger, Chief Executive Officer. "We firmly believe revumenib (SNDX-5613) is positioned to serve as a first-to-market and best-in-class menin inhibitor for patients with NPM1 and MLLr acute leukemias. We have also begun exploring its activity beyond use as a monotherapy agent through initiation of two new trials, including in combination with venetoclax-azacitidine for use as a frontline treatment, and in combination with chemotherapy for relapsed/refractory (R/R) disease. Beyond acute leukemias, we intend to assess revumenib’s (SNDX-5613) potential in additional areas where menin inhibition could have a strong therapeutic benefit, and we look forward to commencing a proof-of-concept clinical trial in patients with advanced colorectal cancer (CRC), a highly underserved area lacking effective therapeutic options, in the fourth quarter of the year."

"Additionally, enrollment continues in the ongoing global pivotal Phase 2 AGAVE-201 trial of axatilimab in chronic graft-versus-host disease (cGVHD), with topline data expected in the first half of 2023 and a potential Biologic License Application (BLA) filing in 2023. Supported by recent receipt of Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA), we believe axatilimab has the potential to play a meaningful role in the cGVHD treatment landscape. Furthermore, as previously announced, we are committed to unlocking axatilimab’s full potential in additional fibrotic diseases where the monocyte-macrophage lineage plays a vital role, and remain on track to commence a Phase 2 trial in idiopathic pulmonary fibrosis (IPF) in the fourth quarter of this year."

Recent Pipeline Progress and Anticipated Milestones

Revumenib (SNDX-5613)

The pivotal Phase 2 portion of AUGMENT-101 is ongoing and the Company continues to expect completion of enrollment in at least one of the three pivotal cohorts later this year. The trials are expected to enroll a total of 64 adult and up to 10 pediatric patients across each of three distinct trial populations: patients with NPM1 mutant acute myeloid leukemia (AML), patients with MLLr AML, and patients with MLLr acute lymphocytic leukemia (ALL). Based on discussions with the U.S. FDA, AUGMENT-101 may serve as the basis for regulatory filings in each of the three distinct populations. The Company expects to receive initial topline data from the trials starting in the first half of 2023, with the potential for the first New Drug Application filing in 2023.
Two additional trials, BEAT-AML and AUGMENT-102, are now underway to assess the safety, tolerability, and preliminary anti-leukemic efficacy of revumenib (SNDX-5613) and establish an appropriate Phase 2 dose. BEAT-AML is a front-line combination trial of revumenib (SNDX-5613) with venetoclax and azacitidine being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial. The primary endpoint of the Phase 1 portion of the BEAT-AML trial is to determine the recommended Phase 2 dose of the combination. The Company also initiated a trial in combination with chemotherapy in patients with R/R NPM1 or MLLr acute leukemias, known as AUGMENT-102. The primary endpoint of AUGMENT-102 will assess the safety, tolerability, and recommended Phase 2 dose criteria. Topline data from the trials are expected beginning in 2023.
The Company reported it intends to initiate a proof-of-concept clinical trial to evaluate revumenib (SNDX-5613) in patients with unresectable metastatic microsatellite stable CRC, which represents the second leading cause of cancer death in the U.S. with an estimated incidence of over 55,000 patients per year1,2. Activation of the Wnt/b-catenin signaling pathway is believed to be a key initiating step and growth driver for the majority of CRC tumors. The menin-MLL1 protein complex has recently been shown to regulate b-catenin activity and in preclinical models, disrupting this complex through menin inhibition blocks growth of Wnt/b-catenin driven CRC tumors. The Company is expected to commence the trial in the fourth quarter of 2022.
Axatilimab

The Company reported that the U.S. FDA has granted FTD to axatilimab for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy. FTD is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier.
Enrollment is ongoing in the Company’s global pivotal Phase 2 AGAVE-201 trial of axatilimab in patients with cGVHD. The trial is evaluating the safety and efficacy of three doses and schedules of axatilimab. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for cGVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score. The Company remains on track to report topline data in the first half of 2023, with the potential for a BLA filing in 2023.
First Quarter 2022 Financial Results

As of March 31, 2022, Syndax had cash, cash equivalents and short-term investments of $397.9 million and 59.0 million shares and share equivalents issued and outstanding. This includes 4.0 million pre-funded warrants.

First quarter 2022 research and development expenses increased to $30.0 million from $21.9 million for the prior year period. The increase was primarily due to increased clinical and manufacturing activities.

General and administrative expenses for the first quarter 2022 increased to $6.8 million from $5.7 million for the prior year period. The increase is primarily due to increased compensation and professional fees.

For the three months ended March 31, 2022, Syndax reported a net loss attributable to common stockholders of $37.2 million or $0.63 per share compared to a net loss attributable to common stockholder of $27.7 million or $0.54 per share for the prior year period.

Financial Update and Guidance

For the second quarter of 2022, research and development expenses are expected to be $30 to $35 million, and total operating expenses are expected to be $38 to $42 million. For the full year of 2022, research and development expenses are expected to be $130 to $140 million, and total operating expenses are expected to be $160 to $170 million.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, May 9, 2022.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

On May 9, 2022 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported preliminary financial results for the first quarter of 2022, and associated Company developments (Press release, Supernus, MAY 9, 2022, View Source [SID1234613914]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Qelbree Launch Update

Total IQVIA prescriptions were 47,324 in the first quarter of 2022, an increase of 38% compared to total prescriptions of 34,328 in the fourth quarter of 2021. In March 2022, the most recent month available, total prescriptions reached 18,380.
Qelbree continues to expand its base of prescribers, with approximately 6,900 prescribers in the first quarter of 2022, up from 5,600 prescribers from the fourth quarter of 2021.
Continued progress in securing and improving managed care coverage.
At the end of April 2022, the U.S. Food and Drug Administration (FDA) approved Qelbree for the treatment of attention-deficit hyperactivity disorder (ADHD) in adults. Supernus is expecting to launch Qelbree for adult patients by the end of May 2022. According to recent IQVIA Xponent 52-week data, prescriptions for the adult market represented approximately 68% of the total ADHD market.
Product Pipeline Update

SPN-830 (apomorphine infusion device) – Continuous treatment of motor fluctuations ("off" episodes) in Parkinson’s disease (PD)

The Company will continue to work closely with the FDA as it reviews the New Drug Application (NDA) resubmission for SPN-830 for the continuous treatment of motor fluctuations ("off" episodes) in Parkinson’s disease. The Company is preparing for the commercial launch of SPN-830 in the first quarter of 2023, assuming timely approval by the FDA. The FDA has established a PDUFA target action date in early October 2022.
SPN-820 – Novel first-in-class activator of mTORC1

The Company continues to enroll patients in a Phase II multi-center, randomized double-blind placebo-controlled parallel design study of SPN-820 in adults with treatment-resistant depression. The study will examine the efficacy and safety of SPN-820 over a course of five weeks of treatment in approximately 270 patients. The primary outcome measure is the change from baseline to end of treatment period on the Montgomery-Asberg Depression Rating Scale (MADRS) Total Score, a standard depression rating scale.
SPN-817 – A novel product candidate for the treatment of epilepsy

An open-label Phase II clinical study of SPN-817 in patients with treatment-resistant seizures is expected to start in the second half of 2022.
Financial Highlights

Net Product Sales

For the first quarter 2022, net product sales were $147.5 million, a 15% increase over $128.4 million for the same period in 2021. The increase was primarily due to net product sales of GOCOVRI from the Adamas acquisition in November 2021 (Adamas Acquisition) and growth in net product sales of Qelbree that was launched in the second quarter of 2021.

(1) Includes net product sales of MYOBLOC, XADAGO and Osmolex ER.

Operating earnings (GAAP and non-GAAP)

First quarter 2022 operating earnings (GAAP) was $2.0 million, as compared to $13.2 million for the same period in 2021. The decrease is primarily due to amortization of acquired intangible assets from the Adamas Acquisition. First quarter 2022 adjusted operating earnings (non-GAAP) was $28.0 million, an increase of 11% compared to $25.2 million in the first quarter of 2021.

Reconciliation of GAAP to Non-GAAP Adjustments

An itemized reconciliation between operating earnings on a GAAP basis and operating earnings on a non-GAAP basis is as follows:

Non-GAAP operating earnings adjusts for non-cash items including amortization of intangible assets, share-based compensation expense, change in fair value of contingent consideration, and depreciation. Included in the amortization of intangible assets for the first quarter of 2022 is amortization of acquired intangible assets from the Adamas Acquisition in November 2021.

Net earnings (GAAP)

First quarter 2022 net earnings and diluted earnings per share (GAAP) were $25.6 million and $0.43, respectively, as compared to $5.7 million, or $0.11 per diluted share, in the same period in 2021.

Cash, cash equivalents and marketable securities

At March 31, 2022, the Company’s cash, cash equivalents, current and long-term marketable securities are approximately $437.5 million, compared to $458.8 million as of December 31, 2021. This decrease is primarily due to milestone payments associated with the 2020 USWM acquisition and transition expense payments related to the Adamas acquisition, partially offsetting cash generated from operations.

Full Year 2022 Financial Guidance (GAAP)

For full year 2022, the Company reiterates its prior financial guidance as set forth below:

(1) Includes net product sales and royalty revenue.
(2) Includes amortization of intangible assets and contingent consideration expense (gain).

Full Year 2022 Financial Guidance – GAAP to Non-GAAP Adjustments

An itemized reconciliation between projected operating earnings on a GAAP basis and projected operating earnings on a non-GAAP basis is as follows:

This press release contains a financial measure, non-GAAP operating earnings, which does not comply with United States generally accepted accounting principles (GAAP). The non-GAAP financial measure should be considered in addition to, not as a substitute for or in isolation from, or superior to measures prepared in accordance with GAAP. Non-GAAP operating earnings adjust for non-cash share-based compensation expense, depreciation and amortization, and accretion of contingent consideration, and for factors that are unusual, non-recurring or unpredictable, and exclude those costs, expenses, and other specified items presented in the reconciliation tables in this press release. We believe the use of non-GAAP operating earnings is useful supplemental information to investors regarding the Company’s results of operations and assist management, analysts, and investors in evaluating the performance of the business. There are limitations associated with the use of non-GAAP financial measures. Including such measures may not be entirely comparable to similarly titled measures used by other companies, may not reflect all items of income and expense, as applicable, that affect our operations, potential differences among calculation methodologies, may differ from the non-GAAP information used by other companies, including peer companies, and therefore comparability may be limited. We mitigate these limitations by reconciling the non-GAAP financial measure to the most comparable GAAP financial measure. Investors are encouraged to review the reconciliation. The Company’s 2022 financial guidance is also being provided on both a reported and a non-GAAP basis.

Conference Call Details

Supernus will host a conference call and webcast today, May 9, 2022, at 4:30 p.m. Eastern Time to discuss these results.

Please refer to the information below for conference call dial-in information and webcast registration. Callers should dial in approximately 10 minutes prior to the start of the call.

Following the live call, a replay will be available on the Company’s website, www.supernus.com, under "Investor Relations".