On February 28, 2022 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that Jounce management will participate in a novel immuno-oncology panel discussion at the Cowen & Co. 42nd Annual Health Care Conference on Monday, March 7, 2022 at 10:30 a.m. ET (Press release, Jounce Therapeutics, FEB 28, 2022, View Source [SID1234609132]).

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A webcast of the panel will be available by visiting "Events and Presentations" in the Investors and Media section of Jounce’s website at www.jouncetx.com. A replay of the webcasts will be archived for 30 days following the presentation.

On February 28, 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 fourth quarter and full year of 2021, and associated Company developments (Press release, Supernus, FEB 28, 2022, View Source [SID1234609149]).

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Net Product Sales

For full year 2021, preliminary net product sales were $567.5 million, an 11% increase over $509.3 million for the full year 2020. The increase was primarily due to the acquisition of the CNS portfolio of US WorldMeds in June 2020, growth in net product sales of Oxtellar XR, the launch of Qelbree in the second quarter of 2021, and net product sales of GOCOVRI (amantadine) from the acquisition of Adamas Pharmaceuticals, Inc. (Adamas) in November 2021.

Preliminary fourth quarter 2021 net product sales were $155.0 million, a 10% increase over $140.7 million in the same period in 2020. The increase was primarily due to net product sales of GOCOVRI from the acquisition of Adamas in November 2021, the launch of Qelbree in the second quarter of 2021, and growth in net product sales of Oxtellar XR.

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

The fourth quarter and full year 2021 revenue results included herein are preliminary and are therefore subject to change.

Qelbree Launch Update

Total IQVIA prescriptions were 34,328 in the fourth quarter of 2021, an increase of 122% compared to total prescriptions of 15,453 in the third quarter of 2021. In January 2022, the most recent month available, total prescriptions reached 14,177.
Total prescriptions are showing a quarter-to-date (first seven weeks) sequential growth rate of 42% in the first quarter 2022 versus the corresponding same seven-week period in the fourth quarter of 2021.
Qelbree continues to expand its base of prescribers, with over 5,600 prescribers in the fourth quarter of 2021, up from 3,470 prescribers from the third quarter of 2021.
Continued progress in securing and improving managed care coverage.
Preparations for the potential launch in the adult market are well underway, assuming timely approval by the U.S. Food and Drug Administration (FDA) of the supplemental New Drug Application (sNDA) for the adult indication.
Acquisition of Adamas Pharmaceuticals, Inc.

The Company completed the acquisition of Adamas in late November 2021, strengthening its Parkinson’s disease portfolio with two marketed products, including GOCOVRI extended release capsules, the first and only FDA-approved medicine indicated for the treatment of both "off" episodes and dyskinesia in patients with Parkinson’s disease receiving levodopa-based therapy. In addition, the acquisition diversifies and increases the Company’s revenue base and cash flow.
Total prescriptions for GOCOVRI in January 2022 grew by 30% compared to January 2021.
Product Pipeline Update

Qelbree (viloxazine, extended-release capsules) – Novel non-stimulant for the treatment of ADHD in adults

In September 2021, the FDA acknowledged it received the sNDA for Qelbree for the treatment of ADHD in adult patients. The sNDA has a user fee target action date (PDUFA date) in late April 2022.
SPN-830 (apomorphine infusion device) – Continuous treatment of motor fluctuations ("on-off" episodes) in Parkinson’s disease (PD)

The Company received notice from the FDA that its New Drug Application (NDA) resubmission for SPN-830 for the continuous treatment of motor fluctuations ("off" episodes) in Parkinson’s disease is considered a Standard Review, thereby assigning a timeline of 10 months for review by the FDA and establishing a PDUFA target action date in early October 2022.
The Company will work closely with the FDA as it reviews the SPN-830 NDA. The Company is preparing for the commercial launch of SPN-830 in the first quarter of 2023, assuming timely approval by the FDA.
SPN-820 – Novel first-in-class activator of mTORC1

The Company has initiated a Phase II multicenter, 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 400 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

A randomized Phase II clinical study of SPN-817 for the treatment of focal seizures is expected to start in the second half of 2022.
Financial Highlights

Fourth Quarter

For the three months ended December 31, 2021, preliminary combined research and development (R&D) and selling, general and administrative (SG&A) expenses are expected to range between $105.0 million and $110.0 million, as compared to $74.4 million for the same period in 2020. The expected increase is primarily due to activities to support the launch of Qelbree and transactions costs associated with the Adamas acquisition.

For the three months ended December 31, 2021, preliminary amortization of intangible assets expense are expected to range between $11.0 million and $12.0 million, as compared to $5.9 million for the same period in 2020. The expected increase is primarily due to amortization for the acquired intangible assets associated with the Adamas acquisition.

For the three months ended December 31, 2021, preliminary operating earnings are expected to range between $20.0 million and $25.0 million, as compared to $43.0 million for the same period in 2020. The expected decrease is primarily attributable to higher expenses to support the launch of Qelbree and transaction and other costs associated with the Adamas acquisition.

Full Year

For the full year ended December 31, 2021, preliminary combined R&D and SG&A expenses are expected to range between $377.0 million and $382.0 million, as compared to $276.6 million for full year 2020. The expected increase is primarily due to activities to support the launch of Qelbree and timing of both the Adamas and US WorldMeds acquisitions.

For the full year ended December 31, 2021, preliminary amortization of intangible assets expense is expected to range between $29.0 million and $30.0 million, compared to $15.7 million, for the same period in 2020. The expected increase is primarily due to amortization for the acquired intangible assets associated with the Adamas acquisition and the timing of the US WorldMeds acquisition.

For the full year ended December 31, 2021, preliminary operating earnings are expected to range between $100.0 million and $105.0 million, as compared to $173.7 million for full year 2020. The expected decrease is primarily due to increased costs and expenses to support the launch of Qelbree and the timing of the US WorldMeds acquisition.

Cash, cash equivalents and marketable securities

At December 31, 2021, the Company’s cash, cash equivalents, current and long-term marketable securities are approximately $458.8 million, compared to $772.9 million as of December 31, 2020. This decrease is primarily due to funding of the acquisition of Adamas, partially offset by cash flow from operations.

The financial information for the fourth quarter and full year 2021 included herein are preliminary and are therefore subject to change.

Full Year 2021 Financial Information

The Company currently anticipates it will require additional time to finalize its financial statements for the year ended December 31, 2021. Accordingly, the Company currently anticipates it will be unable to file timely its Annual Report on Form 10-K for the year ended December 31, 2021 and that it will file a Form 12b-25 on March 1, 2022.

Full Year 2022 Financial Guidance (GAAP)

The Company expects to achieve the following financial objectives in 2022:

Amount ($ in millions)
Total revenues (1) $640 – $680
Combined R&D and SG&A expenses $460 – $490
Operating earnings (2) $20 – $40
Effective tax rate 25% – 28%
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(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 Outlook

In providing an outlook for non-GAAP operating earnings, we exclude certain items which are otherwise included in determining the comparable GAAP financial measures. In order to inform our outlook measures of non-GAAP operating earnings, a description of the 2022 adjustments which have been applicable in determining non-GAAP operating earnings for the period are reflected in the tables above. In providing an outlook for non-GAAP operating earnings, we adjust for non-cash share-based compensation expense, depreciation and amortization, and accretion of contingent consideration. We are providing such outlook on a non-GAAP basis because we believe it is useful supplemental information to investors and others in understanding and evaluating operating results and trends in our business that could otherwise be masked by the effect of the expenses that we exclude.

We use the outlook measure of non-GAAP operating earnings to understand and compare operating results across accounting periods, for internal budgeting and forecasting purposes and to evaluate our financial performance and the ability to generate cash from operations. We believe the outlook measure of non-GAAP operating earnings allows for meaningful period-to-period comparisons and analysis of trends in our business, as they exclude expenses that are not reflective of ongoing operating results.

There are limitations associated with the use of the non-GAAP financial measure. The Company’s method for calculating the non-GAAP financial measure may differ from those used by other companies, and therefore comparability may be limited. We mitigate the limitations by reconciling the non-GAAP financial measures to the most comparable GAAP financial measures. 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. Investors are encouraged to review the reconciliation.

Conference Call Details

Supernus will host a conference call and webcast today, February 28, 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.

On February 28, 2022 Thermo Fisher Scientific reported that it is calling for new proposals for its Oncomine Clinical Research Grant (Press release, Thermo Fisher Scientific, FEB 28, 2022, View Source [SID1234609168]). The latest request for submissions from the global scientific community will award funding for molecular profiling research that helps accelerate the use of genomic sequencing in oncology .

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"Next-generation sequencing is vital to match eligible patients with lung carcinomas with targeted therapies, but often this testing takes too long to inform their care"

The recipients of the grants will each be awarded up to $200,000 in reagents and general funding for independent clinical research proposals that demonstrate excellence in cancer molecular profiling. Project proposals harnessing the value of next generation sequencing (NGS) in either the context of solid or hematological cancers will be reviewed by independent and internationally recognized experts for scientific rigor and merit. Grant proposals are now being accepted through April 7, 2022.

"The Oncomine Clinical Research Grant program aims to fund research furthering our understanding of cancer at the genomic level. The identification of specific cancer biomarkers associated with response or resistance to treatment can inform clinical care and potentially help predict patient outcomes," said José Luis Costa, Ph.D., director of medical affairs for clinical next-generation sequencing and oncology, Thermo Fisher Scientific. "Thermo Fisher is committed to supporting scientists and researchers globally as we look to advance cutting-edge genetic research."

Since launching the program in 2020, Thermo Fisher has awarded Oncomine Clinical Research Grants to 16 projects worldwide in support of research in hematology-oncology, immuno-oncology, liquid biopsy, and fusion gene detection. Recipients of the last funding round, which supported clinical research programs using NGS technology in solid tumors and hematology-oncology applications, included: Myung-shin Kim, The Catholic University of Korea Seoul St. Mary’s Hospital, South Korea; Wolfram Jochum, Kantonsspital St. Gallen, Switzerland; Fernando Lopez-Rios, Hospital Universitario 12 de Octubre, Spain; Tarek Bismar, University of Calgary, Canada.

"Next-generation sequencing is vital to match eligible patients with lung carcinomas with targeted therapies, but often this testing takes too long to inform their care," said grant recipient Fernando Lopez-Rios, Hospital Universitario 12 de Octubre. "With the support from the Oncomine Clinical Research Grant, we hope to demonstrate that comprehensive genomic sequencing with very rapid turnaround times can increase patient access and advance the idea of universal testing of all patients with lung carcinomas, including those with early-stage disease."

On February 28, 2022 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, reported that a phase II study of Kazia’s investigational new drug, paxalisib, in combination with metformin and a ketogenic diet for the treatment of newly diagnosed and recurrent glioblastoma, has been initiated at Weill Cornell Medicine, with the first patient successfully initiated in the study and a second patient already in screening (Press release, Kazia Therapeutics, FEB 28, 2022, View Source [SID1234609185]).

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Key Points

Research by Professor Lew Cantley, who discovered the PI3K pathway, suggests that a low-insulin state may enhance the activity of PI3K inhibitors such as paxalisib. Inducing a ‘ketogenic’ state, in which the body is fueled by fats and proteins rather than by glucose, is a very effective way to reduce insulin levels.
Patients on the study will also be treated with metformin, a common anti-diabetic drug, which is intended in this case to further reduce insulin levels.
The Cornell study will explore this approach. One arm of the study will examine patients with recurrent disease, and the other will enroll newly diagnosed patients.
Dr Howard Fine, founding Director of the Brain Tumor Center at New York-Presbyterian Weill Cornell Medical Center, will serve as Principal Investigator.
The study is expected to recruit between 30 and 60 patients, and to take approximately two years to complete.
Kazia will provide support, including study drug and a financial grant.
Dr Fine, Principal Investigator on the study, commented, "We have extensive and very convincing preclinical data to support this approach. My colleagues and I believe that administering paxalisib to patients in a ketogenic state may significantly enhance its efficacy. That in turn offers the potential to make a very significant difference in the treatment of glioblastoma, which remains one of the most challenging cancers in modern medicine."

Kazia CEO, Dr James Garner, commented, "We are delighted to have this study now underway and look forward to following its progress. This important milestone kicks off an exciting year for Kazia, with at least six potential data read-outs anticipated over the course of 2022. The GBM AGILE study continues to progress well, and additional studies, such as this one, have the potential to substantially extend and enhance the use of paxalisib in clinical practice."

Ketogenesis and Glioblastoma

Cells in the human body generally rely on glucose as ‘fuel’ for their energy requirements. However, when glucose is not readily available, cells can metabolise fats and proteins to provide energy. The fats and proteins are broken down to an intermediate form known as ketones, and so this biochemical pathway is referred to as ‘ketogenesis’.

Unlike healthy cells, most tumour cells are poorly able to metabolise ketones, and so depend on glucose for their energy needs. Consequently, many researchers have experimented with ‘ketogenic diets’ as a potential treatment for cancer.[1]

In addition, scientists in Professor Cantley’s lab have shown that insulin has the potential to counteract the anti-tumor effects of PI3K inhibitors[2]. In follow-up, Dr Fine’s lab has shown that insulin can reverse the anti-tumor effects of PI3K inhibitors specifically in glioblastoma cells. Insulin is a hormone produced by the body in response to high levels of glucose. When the body is in a state of ketosis, glucose is absent, and so insulin falls to very low levels.

For these reasons, there is a sound rationale to explore a combination of ketogenic diet and paxalisib in glioblastoma. In this study, patients will also receive metformin, a common anti-diabetic drug, which will help to further lower fasting insulin levels.

Clinical Trial Design

This study will comprise two arms. The first will contain patients with newly diagnosed glioblastoma who have unmethylated MGMT promotor status. These patients have historically responded poorly to the current standard of care, temozolomide. The second arm will contain patients with recurrent disease, regardless of the methylation status of their MGMT promotor, who have progressed after taking standard-of-care therapy.

In each arm, paxalisib will be combined with metformin and with a ketogenic diet. The diet will be overseen by expert clinical dieticians to ensure that it is scientifically appropriate and that patients are compliant.

An initial cohort of approximately 16 patients will be recruited to each of the two study arms. If there are signals of activity in a given arm, that arm will be expanded to approximately 30 patients. The primary endpoint will be progression-free survival at six months (PFS6). In addition to efficacy and safety, the study will examine a range of metabolic, pharmacodynamic, and novel radiographic imaging biomarkers to inform future research and clinical practice. The study is expected to take around two years to complete.

Principal Investigator

Dr Howard Fine will serve as Principal Investigator to the study. Dr Fine is the founding Director of the Brain Tumor Center at New York-Presbyterian Weill Cornell Medical Center, Feil Professor of Medicine a Professor of Neurology at Weill Cornell Medicine. He is an internationally recognized leader in the field of neuro-oncology, with more than 30 years of experience in both laboratory and clinical research as well as in the care of patients with brain tumors. Dr Fine has built large multidisciplinary brain tumor programs at top academic institutions such as the Dana Farber Cancer Institute / Harvard Medical School and the National Institutes of Health, has cared for nearly 20,000 patients with brain and spinal cord tumors in his career, has conducted over 100 clinical trials, published over 250 papers and book chapters on brain tumors, and for over two decades has run a continuously operating translational genetic / molecular laboratory devoted to a better understanding of, and better therapies for, brain tumors.

Weill Cornell Medical Center

The Joan and Sanford I. Weill Medical College of Cornell University, known generally as Weill Cornell Medicine, is the medical school of Cornell University, based in New York, NY, and is one of the leading medical research centers in the United States. Its notable alumni include Dr Anthony Fauci, director of the National Institute of Allergy and Infectious Disease.

Paxalisib Clinical Program

The initiation of this trial in glioblastoma brings the number of ongoing clinical studies of paxalisib in brain cancer to eight.

On February 28, 2022 Celsion Corporation (NASDAQ: CLSN), a clinical-stage company focused on DNA-based immunotherapy and next-generation vaccines, reported that, as previously authorized by its shareholders, the Company is implementing a consolidation (reverse stock split) of its outstanding Common Shares on the basis of one (1) new Common Share for every fifteen (15) currently outstanding Common Shares (Press release, Celsion, FEB 28, 2022, View Source [SID1234609091]).

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The new Common Shares will be effective for trading purposes as of the commencement of trading on Tuesday, March 1, 2022, and will trade under a new CUSIP number 15117N 602. The Company’s ticker symbol, CLSN, will remain unchanged. The Company has filed a Certificate of Amendment to its Certificate of Incorporation to effect the stock consolidation.

The new number of outstanding common shares will be approximately 5.8 million shares. The number of authorized shares and the par value per share will remain unchanged. No fractional shares will be issued in connection with the reverse stock split. Holders of fractional shares will be paid out in cash for the fractional portion. The number of outstanding options and warrants will be adjusted accordingly, with outstanding options being approximately 437,500 and outstanding warrants being approximately 168,500.

Celsion stockholders will receive instructions from the Company’s transfer agent, American Stock Transfer and Trust Company, relating to procedures for exchanging existing stock certificates for new certificates or book-entry shares and for the receipt of cash proceeds in lieu of fractional shares.