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;2022.htm [SID1234613934]).

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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.

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(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:

Non-GAAP Financial Information

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.

Conference Call Name: Supernus Pharmaceuticals First Quarter 2022 Financial Results Conference Call
Following the live call, a replay will be available on the Company’s website, www.supernus.com, under "Investor Relations".

On May 9, 2022 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its Chief Medical Officer, Mark Berger, M.D., will be featured as an expert panelist at the 33rd Annual Cancer Progress Conference, taking place virtually May 10-12, 2022 (Press release, Genprex, MAY 9, 2022, View Source [SID1234613950]).

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The live panel discussion, titled, "Into the Unknown – Bringing New Modalities into Solid Tumors," will take place on Thursday, May 12 at 9 a.m. ET. This panel will explore considerations with respect to positioning of cell-based and other emerging immunotherapy platforms in solid tumors. Topics to be addressed by this panel include the current state of play, alignment of science with evolving unmet needs and perceived paths toward value inflection within different solid tumor markets.

Since 1989, Cancer Progress is the only oncology conference that facilitates discussions of scientific progress within the context of development, regulatory, clinical, commercial and investment perspectives over three days of provocative, informative panel discussions. The conference will feature pivotal topics, frank discussions, vigorous debate, opportunities for audience questions and comments, and a partnering platform to enable meaningful connections and meetings with innovators, developers and investors.

On May 9, 2022 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, reported financial results for the three months ended March 31, 2022 and highlighted recent progress (Press release, Verastem, MAY 9, 2022, View Source [SID1234613967]).

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"In the first quarter of this year, we made significant progress building on our breakthrough therapy designation in recurrent low-grade serous ovarian cancer and advancing our development programs and scientific platform to establish VS-6766 as the backbone therapy for RAS-driven solid tumors. This includes completing enrollment for the selection phase of both our RAMP 201 trial in low-grade serous ovarian cancer and our RAMP 202 trial in KRAS G12V-mutant non-small cell lung cancer, with topline results planned for the second quarter and the second half of this year, respectively. Further, we initiated enrollment in the Phase 1/2 trial with Amgen to evaluate VS-6766 in combination with LUMAKRASTM (sotorasib) in patients with KRAS G12C-mutant non-small lung cancer," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "At the same time, we strengthened our flexibility by entering into a term loan facility with Oxford, which combined with our financial resources will allow us to effectively advance our current development and commercial objectives, working to bring VS-6766 and defactinib to patients with high unmet needs."

First Quarter 2022 and Recent Highlights

Low Grade Serous Ovarian Cancer (LGSOC)

Planned enrollment is complete in the selection phase (Part A; n=64) of the registration-directed Phase 2 RAMP 201 study investigating VS-6766 alone or in combination with defactinib for the treatment of recurrent LGSOC. Verastem plans to report topline results from Part A during the second quarter of 2022, following discussions with regulatory authorities.
Enrollment has commenced in the expansion phase (Part B) of RAMP 201, with both treatment arms (VS-6766 alone and in combination with defactinib) currently advancing in all patients. Verastem expects to complete enrollment in Part B during the second half of 2022.
Translational data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) meeting in April provided mechanistic insights into the encouraging response rates and progression free survival observed in patients with LGSOC treated with VS-6766 with defactinib in the investigator-initiated FRAME study. These data support the ongoing registration-directed Phase 2 RAMP 201 study assessing VS-6766 with defactinib for patients with LGSOC regardless of KRAS status.
KRAS Mutant Non-Small Cell Lung Cancer (NSCLC)

Planned enrollment is now complete in the selection phase (Part A; n=32) of the registration-directed RAMP 202 study investigating VS-6766 alone and in combination with defactinib in patients with KRAS G12V-mutant NSCLC. Enrollment has also been completed in the non-G12Vmutant cohort in the expansion phase (Part B). The Company expects to report topline results from Part A and initiate Part B during the second half of 2022, following discussions with regulatory authorities.
Based on preclinical rationale, Verastem has added BRAF-mutant cohorts to the RAMP 202 study to efficiently evaluate VS-6766 with defactinib in BRAF-mutant NSCLC. In Part A of the study, the Company expects to enroll two cohorts comprised of 15 patients each to evaluate the combination in patients with V600E or non V600E BRAF mutations, respectively. These cohorts are open and enrolling.
The Phase 1/2 RAMP 203 study evaluating VS-6766 in combination with Amgen’s LUMAKRASTM (sotorasib) in G12C-mutant NSCLC opened and is enrolling. The initial results are expected to be reported during the second half of 2022.
Corporate Updates

Secured debt facility with Oxford Finance LLC for up to $150 Million. Under the terms of the credit facility with Oxford Finance LLC, Verastem drew an initial $25 million term loan at closing. The Company has the ability to access up to an additional $125 million in a series of tranches, $75 million of which is based on certain pre-determined milestones and $50 million of which is available at the lender’s discretion.
With the credit facility and expected milestones related to the sale of COPIKTRA (duvelisb) to Secura Bio Inc. (Secura) in 2020, the Company expects to have a cash runway through 2025 to support the continued development and potential commercial launches of VS-6766 and defactinib.
Secura sublicensee, CSPC Pharmaceutical Group Limited (CSPC), obtained drug registration approval for duvelisib granted by the National Medical Products Administration of the People’s Republic of China for the treatment of adult patients with relapsed or refractory follicular lymphoma after at least two prior systematic therapies, which entitles Verastem to a $2.5 million milestone payment.
Preclinical data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) meeting in April continued to support the versatility of VS-6766 in RAS-driven tumors, including KRAS G12C-mutant NSCLC, low-grade serous ovarian cancer and cutaneous melanoma.
First Quarter 2022 Financial Results

Verastem Oncology ended the first quarter 2022 with cash, cash equivalents and investments of $106.3 million.

Total revenue for the three months ending March 31, 2022 (2022 Quarter) was $2.6 million, compared to $1.0 million for the three months ended March 31, 2021 (2021 Quarter). Revenue for the 2022 Quarter was primarily comprised of one regulatory milestone for $2.5 million achieved by Secura’s sublicensee, CSPC. Revenue for the 2021 Quarter was primarily comprised of one regulatory milestone for $0.8 million achieved by Secura’s sublicensee, Sanofi.

Total operating expenses for the 2022 Quarter were $19.6 million, compared to $15.1 million for the 2021 Quarter.

Research & development expenses for the 2022 Quarter were $13.6 million, compared to $8.9 million for the 2021 Quarter. The increase of $4.7 million, or 52.8%, primarily resulted from an increase in drug product and drug substance costs, contract research organization costs and investigator fees.

Selling, general & administrative expenses for the 2022 Quarter were $5.9 million, compared to $6.2 million for the 2021 Quarter. The decrease of $0.3 million, or 4.8%, primarily resulted from lower consulting and professional fees.

Net loss for the 2022 Quarter was $17.0 million, or $0.09 per share (basic and diluted), compared to net loss of $15.0 million, or $0.09 per share (basic and diluted), for the 2021 Quarter.

For the 2022 Quarter, non-GAAP adjusted net loss was $15.3 million, or $0.08 per share (diluted), compared to non-GAAP adjusted net loss of $12.4 million, or $0.07 per share (diluted), for the 2021 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation attached to this press release.

Use of Non-GAAP Financial Measures

To supplement Verastem Oncology’s condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net (loss) income and non-GAAP net (loss) income per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended March 31, 2022 and 2021 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

About VS-6766

VS-6766 (formerly known as CH5126766 and RO5126766) is a RAF/MEK clamp that induces inactive complexes of MEK with ARAF, BRAF and CRAF potentially creating a more complete and durable anti-tumor response through maximal RAS pathway inhibition. VS-6766 is currently in late-stage development.

In contrast to other MEK inhibitors, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors. The U.S. Food and Drug Administration granted Breakthrough Therapy designation for the combination of Verastem Oncology’s investigational RAF/MEK inhibitor VS-6766, with defactinib, its FAK inhibitor, for the treatment of all patients with recurrent low-grade serous ovarian cancer (LGSOC) regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy.1

Verastem Oncology is conducting Phase 2 registration-directed trials of VS-6766 alone and with defactinib in patients with recurrent LGSOC and in patients with recurrent KRAS G12V-mutant NSCLC as part of its RAMP (Raf And Mek Program) clinical trials, RAMP 201 and RAMP 202, respectively. Verastem Oncology has also established clinical collaborations with Amgen and Mirati to evaluate LUMAKRAS (sotorasib) and adagrasib in combination with VS-6766 in KRAS G12C-mutant NSCLC as part of the RAMP 203 and RAMP 204 trials, respectively.

On May 9, 2022 Cardinal Health (NYSE:CAH) and URAC, the nation’s largest independent health care accreditation organization, reported an agreement to help customers of Cardinal Health pursue accreditation for their specialty pharmacies and practices (Press release, Cardinal Health, MAY 9, 2022, View Source [SID1234614064]).

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Leveraging insights from operating 70 outpatient pharmacies in 19 states, Cardinal Health offers pharmacies and medically integrated dispensaries a full range of solutions through its Specialty Pharmacy Services including consultative support, assessments and new facility openings, as well as providing leadership and day-to-day management. As part of the agreement with URAC, Cardinal Health customers will receive special pricing for new URAC accreditations, which validate that a pharmacy meets the most rigorous standards in the industry and demonstrates the value of the clinical services it provides.

"Hospitals across the country continue to face challenges in minimizing the costs and complexity of specialty medications. Many are searching for ways to better manage this growing area of outpatient pharmaceutical spend more effectively," said Peter J. Siavelis, senior vice president and general manager of Acute Care Distribution and Services at Cardinal Health. "Our agreement with URAC will further strengthen our commitment to providing the guidance and support needed to help hospitals and health systems determine the right path for their pharmacies."

URAC accreditation also serves as a framework for ensuring quality within an organization by identifying areas for improvement through performance analysis and education.

"Because of their role in serving patients with complex, chronic illnesses, pharmacies are a critical and growing resource within the health care landscape," said Jeffrey Carr, URAC’s vice president of business development. "We’re pleased to offer Cardinal Health preferred pricing for new accreditation to its customers nationwide, further validating the high-quality care they provide and their commitment to improving patient outcomes."

Cardinal Health helps hospitals and health systems determine the right outpatient pharmacy approach for their facilities based on their unique goals and needs. These services also extend to federally qualified health centers, independent physician practices, independent pharmacies and dispensing clinicians. Visit cardinalhealth.com/accreditedspecialtypharmacy to learn more.

On May 9, 2022 Nykode Therapeutics AS (Euronext Growth (Oslo): NYKD), a clinicalstage biopharmaceutical company dedicated to the discovery and development of vaccines and novel immunotherapies, reported positive interim results from its Phase 2 VB C-02 trial of VB10.16, its wholly-owned therapeutic cancer vaccine, in combination with the PD-L1 inhibitor atezolizumab in patients with HPV16-positive advanced cervical cancer (Press release, Nykode Therapeutics, MAY 9, 2022, View Source [SID1234613862]). Interim results from 39 patients with a median follow up of 6 months show an ORR of 21%—including two patients who achieved a complete response and six who achieved a partial response—and a very high disease control rate of 64%. The trial enrolled a heavily pre-treated patient population with more than two thirds of the patients having received at least two previous systemic lines of treatment.

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Interestingly, anti-tumor activity was observed in both PD-L1 positive (ORR of 27% and DCR of 77%) and PD-L1 negative patients (ORR of 17% and DCR of 58%) indicating a potential clinical benefit also in the PD-L1 negative population. In addition, a DCR of 71% was observed in patients with noninflamed tumors, including both immune desert and T cell excluded tumors. Together these findings suggest a differentiated anti-tumor response pattern of the combination treatment compared to checkpoint inhibitor monotherapy.

"We are thrilled to report these positive interim safety and efficacy results from our Phase 2 trial with VB10.16, which showed evidence of durable anti-tumor activity in a heavily pre-treated population of patients with late-stage cervical cancer," said Michael Engsig, Chief Executive Officer of Nykode Therapeutics. "These interim results support Nykode’s unique approach of targeting AntigenPresenting Cells (APCs), designed to produce a robust and long-lasting CD8 killer T cell response against cancer cells. We look forward to reporting updated efficacy data readouts from the Phase 2 trial during the first half of 2023 as we continue to advance our cervical cancer program."

Immunological analyses of the peripheral T cell responses demonstrated an increased HPV16-specific IFN T cell immune response post-vaccination in the majority of subjects and were associated with clinical efficacy indicating the induction of clinically relevant T cell responses. Clearance of circulating HPV16 DNA was significantly correlated with clinical response and progression free survival suggesting ctDNA may be an early marker of response to treatment in cervical cancer. VB10.16 is potentially a first-in-class therapeutic vaccine against HPV16-positive cervical cancer.

"The patients who were treated with VB10.16 in combination with atezolizumab in the C-02 trial were heavily pre-treated and are prone to progress quickly," said Professor Peter Hillemanns, Director of the Departments of Gynecology, Obstetrics and Breast Cancer at Hannover University Hospital, Germany and principal investigator of the C-02 trial. "It is very encouraging to see that a majority of patients experienced a clinical benefit and that many patients had durable responses. The combination of VB10.16 and atezolizumab was also well tolerated by patients."

"Treatment advances within the area of advanced cervical cancer have been limited though checkpoint inhibitors have demonstrated clinical efficacy in some patients. The unmet need is still high, and we are very pleased to see a high disease control rate and durable responses in these heavily pre-treated HPV16+ cervical cancer patients. The anti-tumor activity seen in both noninflamed and PD-L1 negative populations may potentially open up a new subset of patients for treatment. These findings indicate that VB10.16 may give a meaningful added clinical benefit compared to the existing standard of care treatment in this setting. We would like to thank all the investigators, patients and their relatives for participating in this trial. Without all of you the advances of VB10.16 would not have been possible," added Siri Torhaug, Chief Medical Officer of Nykode Therapeutics.

VB10.16 was generally safe and well tolerated, with 10% of patients experiencing Grade 3 or more treatment-related adverse events, indicating no increased toxicity compared with atezolizumab monotherapy and a favorable safety profile that is consistent with previously published data for the Nykode DNA vaccine technology platform. Nykode expects to report updated efficacy data read-outs from VB C-02 during the first half of 2023. Webcast Investors and analysts are invited to join a webcast presentation of the interim results conducted by CEO Michael . The slide presentation will be available in the Investors section of the Company’s website at View Source following the event. The live and archived webcast of the presentation can be accessed in the Investors section of the Company’s website at View Source/financial-reports-and-presentations.

About the VB C-02 trial VB C-02 is a multi-center, single arm, open-label Phase 2 trial of patients with advanced or recurrent, non-resectable HPV16-positive cervical cancer. Patients received treatment with VB10.16 in combination with the PD-L1 inhibitor atezolizumab for up to one year. The main aim of the trial was to assess the efficacy, immunogenicity and safety of VB10.16 when given in combination with atezolizumab. The trial is now fully enrolled with 52 patients enrolled at sites in Europe, and the preplanned second interim analysis was performed at the cut-off date of 14 February 2022. Nykode has previously reported positive interim safety data from the trial. Additional information about the VB C-02 trial is available at clinicaltrials.gov (NCT04405349).

About VB10.16
VB10.16 is a potentially first-in-class off-the-shelf therapeutic cancer vaccine candidate in development for the treatment of human papillomavirus type 16 (HPV16)-positive cancers. The cancer vaccine is designed based on Nykode’s Vaccibody technology platform of targeting antigens to antigen presenting cells. The candidate has demonstrated favorable clinical data in a Phase I/IIa study in pre-cancerous HPV16-induced high grade cervical intraepithelial neoplasia (HSIL; CIN 2/3) demonstrating a statistically significant correlation of immune responses and clinical responses. VB10.16 is currently being investigated in the VB C-02 trial in patients with advanced or recurrent, non-resectable HPV16-positive cervical cancer.

About cervical cancer
Cervical cancer is the fourth leading cause of cancer death in women worldwide and is most frequently diagnosed between the ages of 35 and 44. Each year around 600,000 women are diagnosed with cervical cancer worldwide. Almost all cases are caused by human papillomavirus (HPV) infection and HPV16 accounts for more than half of all cervical cancer cases. Approximately 80% of patients with cervical cancer have squamous cell carcinoma (arising from cells lining the bottom of the cervix) and most other patients have adenocarcinomas (arising from glandular cells in the upper cervix). Cervical cancer is often curable when detected early and effectively managed, but treatment options are more limited in advanced disease stages or when the cancer has spread.