On November 3, 2021 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for the third quarter of 2021, and associated Company developments (Press release, Supernus, NOV 3, 2021, View Source [SID1234594211]).

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Net Product Sales

For the first nine months of 2021, net product sales were $412.5 million, a 12% increase over $368.6 million in the same period in 2020. The increase was primarily due to the acquisition of the CNS portfolio of US WorldMeds in June 2020.

Third quarter 2021 net product sales were $145.5 million, compared to $152.1 million in the same period in 2020. The decrease was primarily due to a decrease in net product sales of APOKYN and Trokendi XR, partially offset by an increase in net product sales from other products, including Qelbree, which was launched in the second quarter of 2021.

Qelbree Launch Update

Qelbree’s growth has accelerated with the arrival of the "back to school" season in the third quarter of 2021, reaching total monthly prescriptions in September of 7,132, an increase of 37% compared to August, and an increase of 118% compared to monthly average during the three months period prior to September. The latest weekly prescriptions data shows 2,248 prescriptions, an increase of 51% compared to the weekly average over the prior 12-week period.
In addition, Qelbree’s base of prescribers has increased by 340% during the third quarter of 2021 compared to the second quarter of 2021, with more than 3,470 physicians prescribing the product.
Proposed Acquisition of Adamas Pharmaceuticals, Inc. (Adamas)

On October 11, 2021, the Company announced it entered into a definitive agreement to acquire Adamas, strengthening its Parkinson’s disease portfolio with two marketed products, including GOCOVRI (amantadine) 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. The acquisition, if completed, would strengthen Supernus’ Parkinson’s disease portfolio with GOCOVRI and diversify and increase its revenue base and cash flow.
Per the agreement, Supernus has offered to acquire all of Adamas’ common stock through a tender offer for $8.10 per share in cash (or an aggregate of approximately $400 million) payable at closing, plus two non-transferrable and non-tradable contingent value rights collectively worth up to $1.00 per share in cash (or an aggregate of approximately $50 million), for a total consideration of up to $9.10 per share in cash (or an aggregate of approximately $450 million). The transaction is subject to customary closing conditions and is expected to close in late fourth quarter 2021 or in early first quarter 2022.
Product Pipeline Update

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

The U.S. Food and Drug Administration (FDA) acknowledged it has received the supplemental new drug application (sNDA) for Qelbree for the treatment of ADHD in adult patients. The sNDA has a user fee goal date (PDUFA date) of April 29, 2022.
SPN-830 (apomorphine infusion pump) – Continuous treatment of motor fluctuations ("on-off" episodes) in Parkinson’s disease (PD)

The Company expects to resubmit the SPN-830 NDA to the FDA in November 2021.
SPN-820 – Novel first-in-class activator of mTORC1

An Investigational New Drug (IND) application was submitted to the FDA in September 2021. Consequently, the randomized Phase II clinical study of SPN-820 in treatment-resistant depression is on track and expected to start by the end of 2021.
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.
SPN-443 and SPN-446 – Two novel CNS drug candidates nominated for development

The Company’s internal research and development discovery program generated several new chemical entities (NCEs) including SPN-443 and SPN-446 that were nominated for development for various CNS indications including ADHD.
Financial Highlights

For the three months ended September 30, 2021, operating earnings, net earnings and diluted earnings per share were $32.6 million, $21.6 million and $0.40, respectively, as compared to $56.1 million, $40.0 million and $0.74, for the same period in 2020.

For the nine months ended September 30, 2021, operating earnings, net earnings and diluted earnings per share were $79.9 million, $51.0 million and $0.94, respectively, as compared to $130.7 million, $96.2 million and $1.79, for the same period in 2020.

Amortization of intangible assets expense for the three and nine months ended September 30, 2021 was $6.0 million and $18.0 million, respectively, compared to $6.1 million and $9.8 million, for the same periods in 2020.

As of September 30, 2021, the Company had $849.3 million in cash, cash equivalents, current and long-term marketable securities, compared to $772.9 million as of December 31, 2020.

Full Year 2021 Financial Guidance

For full year 2021, the Company increases its financial guidance for operating earnings, lowers its financial guidance for total combined R&D and SG&A expenses, and lowers the top end of its financial guidance range for total revenues as set forth below:

(2) Operating earnings include amortization of intangible assets and contingent consideration expense (gain).
(3) The full year 2021 effective tax rate guidance of 28% – 31% is above the normally expected range of 26% – 28% primarily due to the effect of a one-time tax item in the period.

Conference Call Details

Supernus will host a conference call and webcast today, November 3, 2021, 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".

On November 3, 2021 Navidea Biopharmaceuticals, Inc. (NYSE American: NAVB) ("Navidea" or the "Company"), a company focused on the development of precision immunodiagnostic agents and immunotherapeutics, reported it will host a conference call and webcast on Wednesday, November 10, 2021 at 5:00 p.m. (EST) to discuss financial results and corporate developments for the third quarter ended September 30, 2021 (Press release, Navidea Biopharmaceuticals, NOV 3, 2021, View Source [SID1234594210]).

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Dr. Michael Rosol, Chief Medical Officer, and Erika Eves, Vice President of Finance and Administration, will host the call and webcast to discuss the financial results and provide an update on recent developments and clinical progress.

Questions will not be taken during the conference call, however you may submit questions up to 24 hours in advance of the meeting by sending an email to [email protected]. We ask that you limit your questions to those that are relevant to our business. Questions may not be addressed if they are, among other things, profane, irrelevant to our business, related to pending or threatened litigation, disorderly, or repetitious of statements already made. In addition, questions may be grouped by topic by our management with a representative question read aloud and answered. Questions will be addressed in the Q&A portion of the conference call, and we may also respond to questions on an individual basis or by posting answers on our website after the call.

To participate in the call and webcast, please refer to the information below:

A live audio webcast of the conference call will also be available on the investor relations page of Navidea’s corporate website at www.navidea.com. In addition, the recorded conference call can be replayed and will be available for 90 days following the call on Navidea’s website.

On November 3, 2021 The Parker Institute for Cancer Immunotherapy (PICI) and the Cancer Research Institute (CRI) reported the dosing of the first patients in the REVOLUTION trial, a phase I, open label platform study of novel immunotherapy combinations for first-line treatment of metastatic pancreatic cancer (Press release, Cancer Research Institute, NOV 3, 2021, View Source [SID1234594209]).

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"PICI is built on the promise of bringing together the brightest minds to come up with innovative solutions to cancer’s toughest problems, and this trial is a direct result," said Ute Dugan, MD, PhD, PICI’s Chief Medical Officer. "The innovative design also represents a new model of collaborative, end-to-end drug development that allows us to more rapidly advance promising therapeutic combinations from bench to bedside to market, and then to patients."

REVOLUTION uses a platform trial design that allows for more rapid testing of several therapies with more adaptability than a standard clinical trial. In a traditional clinical trial, all elements of the study must be approved prior to beginning. When the study meets its endpoint, researchers must decide what study to do next based on the findings.

In contrast, a platform design allows researchers to interpret the data during the trial and to make changes, such as testing additional treatments, expanding the number of people receiving a therapy that shows promise, or discontinuing a therapy that does not look promising. This allows investigators to follow the science with flexibility and efficiency, avoiding some of the administrative and logistical hurdles involved in starting up individual trials in a safe way and decreasing the likelihood that patients will receive ineffective treatments.

"Platform studies offer a science-informed flexibility that is better able to keep pace with the accelerating advances in clinical cancer immunology, especially those that involve promising drug combinations like those brought together through the Cancer Research Institute’s collaborations with PICI and our academic, industry, and other nonprofit partners," said Jill O’Donnell-Tormey, PhD, CEO and Director of Scientific Affairs at CRI.

REVOLUTION currently consists of two groups of patients. Cohort A will test a combination of standard-of-care chemotherapy, the CTLA-4 inhibitor ipilimumab and the PD-1 inhibitor nivolumab. In addition to providing valuable information on the efficacy of the combination itself, this cohort builds on the findings from PICI’s and CRI’s PRINCE trial, which suggested that the combination of chemotherapy and anti-PD-1 treatment may improve survival compared with chemotherapy alone.

Cohort B will investigate the combination of chemotherapy, ipilimumab and hydroxychloroquine (HCQ). HCQ is a drug that can stop a process called autophagy, which cells use to recycle their own materials to survive, particularly under stress. Previous research has indicated autophagy is a key mechanism by which metastatic pancreatic cancer cells evade the immune system.

The first patients have been dosed in each cohort.

2021-11-03-REVOLUTION_Cohorts_Final_800x400.gif

This trial will also further explore biomarker data generated by the PRINCE study, aiming to better understand which patients stand to benefit from immunotherapy.

"This is an exciting next step in PICI’s and CRI’s commitment to deliver bold, science-driven ideas, and this trial shows how those efforts build on each other as the research progresses," said PICI researcher and CRI Scientific Advisory Council member Robert H. Vonderheide, MD, DPhil, director of the Abramson Cancer Center of the University of Pennsylvania and the study’s overall principal investigator. "REVOLUTION will make use of the same capabilities as PRINCE while adding a framework that can test and advance potentially promising combination therapies more efficiently than standard clinical trials."

REVOLUTION is also designed with the greater field in mind, building off PICI’s and CRI’s shared goals of breaking silos and creating efficiencies through collaboration. PICI has partnered with the Pancreatic Cancer Action Network (PanCAN) and its Precision PromiseSM phase 2/3 platform study in metastatic pancreatic cancer for exactly this purpose. If a cohort of REVOLUTION proves successful, it can be considered for further development under the Precision Promise umbrella. Once again, this accelerates the clinical trial process, ensuring potentially lifesaving therapies make their way to patients as quickly as possible. This partnership also advances CRI’s commitment to discovering and developing immune-based treatments that improve outcomes for all cancer patients, including those who face pancreatic and other hard-to-treat cancers.

In addition to Penn, sites on the trial include the Dana-Farber Cancer Institute, Memorial Sloan Kettering Cancer Center, the University of California, San Francisco, the University of California, Los Angeles, the University of Texas MD Anderson Cancer Center, and Stanford Medicine.

Additional partners on the trial include the 1440 Foundation, Bristol Myers Squibb and PanCAN.

About Pancreatic Cancer
Pancreatic cancer is one of the deadliest types of tumors, and the number of diagnosed cases continues to rise each year. The disease is difficult to catch early, meaning by the time most people are diagnosed, their cancer is advanced and may have already spread. In addition, the tumors usually contain a variety of mutations, meaning one targeted therapy isn’t enough to stop the disease by itself. For patients who are diagnosed after cancer has spread to other parts of the body – a distinction that applies to more than half of all pancreatic patients – the 5-year survival rate is just three percent.

On November 3, 2021 Race Oncology Limited ("Race") reported that it has entered into a collaborative preclinical research program with The University of Newcastle to develop a companion diagnostic to support the use of Zantrene as a precision oncology treatment, targeting the FTO protein (Press release, Race Oncology, NOV 3, 2021, View Source [SID1234594208]). Eminent genomics researcher, Professor Murray Cairns of the University of Newcastle, will lead the project.

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Precision oncology utilises companion diagnostic tests to identify genetic changes in a patient’s cancer that render the cancer sensitive to a particular anti-cancer treatment1. By matching treatment to the cancer’s sensitivities, patients gain better treatment outcomes and avoid unnecessary side effects. Anti-cancer drugs with a companion diagnostic have a significantly increased probability of being approved by regulatory agencies like the US Food and Drug Administration, the European Medicines Agency and Australia’s Therapeutic Goods Administration2.

This project entitled "Genome-wide epitranscriptomic analysis of N6-methyl-adenosine modification at nucleotide resolution using RNA sequencing to identify biomarkers of aberrant tumour RNA methylation" builds on patentable IP developed by Race and will utilise the latest RNA genomics technologies to identify clinically relevant biomarkers of Zantrene sensitivity in human cancer cells and tissue samples.

"Race is extremely pleased to be working with Professor Cairns on this project. He is one of the leading researchers in the RNA genomics field and his background and expertise will maximise our chances of a successful outcome. For companies like Race, having a proprietary companion diagnostic is of increasing importance for our precision oncology clinical programs and will add significant IP protection around Zantrene."

Race Chief Scientific Officer, Dr Daniel Tillett
This work complements the recently announced FTO biomarker collaborative program with the Chaim Sheba Medical Center (ASX announcement: 27 October 2021) by focusing on understanding the m6A methylation status of RNA transcripts at the nucleotide level. This collaboration will provide critical mechanistic data on the effects of FTO inhibition by Zantrene in various cancer types.

"Zantrene’s potency as an FTO inhibitor provides an exciting new opportunity to target cancer by altering its RNA metabolism. I look forward to working with Race to develop a companion diagnostic for the drug’s precision usage in clinical oncology".

Professor Murray Cairns
The work is expected to support current and future clinical trials of Zantrene and provide the scientific basis for developing a clinically validated and Race-proprietary genetic companion diagnostic test for the targeted use of Zantrene in cancer treatment.

This preclinical research program is to start immediately with results to be reported over the coming 12 months.

1. Prasad, V., Fojo, T. & Brada, M. Precision oncology: origins, optimism, and potential. Lancet Oncol 17, e81–e86 (2016).

2. Valla, V. et al. Companion Diagnostics: State of the Art and New Regulations. Biomark Insights 16, (2021).

About Professor Murray Cairns
Professor Cairns is an NHMRC Senior Research Fellow, and Brawn Senior Fellow at the University of Newcastle’s College of Health, Medicine and Wellbeing. He heads the Precision Medicine Laboratory in the school of Biomedical Sciences and Pharmacy, which comprises a team of post-doctoral fellows, research assistants, bioinformaticians and research higher degree students. Professor Cairns has leveraged expertise in bioinformatics and high-throughput sequencing to establish an internationally recognised laboratory specialising in complex trait genomics. He is a leader in genetically informed precision medicine and is developing transformative approaches to address the problem of heterogeneity in the treatment of complex disorders.

Professor Cairns has attracted more than $14 million in funding to support his research programs, which have led to publications in the highest-ranking journals in the field, including Nature, Science, Cell, Nature Biotechnology, Nature Genetics, Nature Reviews Genetics, Nature Neuroscience and Molecular Psychiatry. Collectively, these publications have received more than 14,000 citations. His high-impact collaborative studies highlight an international reputation for research in complex disease genomics, posttranscriptional gene regulation, systems biology, and precision medicine.

On November 3, 2021 Micronoma, the first cancer detection biotech company to diagnose cancer at an early stage with microbiome-driven liquid biopsy technology, reported that added the Health Tech Challengers award in the diagnostics category to the list of accolades it has earned for its groundbreaking work this year (Press release, Micronoma, NOV 3, 2021, View Source [SID1234594207]). Health Tech Challengers identifies and brings together top global digital health tech startups that are innovators in one of six tracks fostering the digital health revolution. The award marks the fourth Micronoma has achieved this year.

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Micronoma Wins Fourth Innovation Award This Year

"It has been a wonderful year of recognition for our team, and the awards are important for bringing awareness and distinction to breakthrough technology like Micronoma’s Oncobiota liquid biopsy for true early-stage detection of cancer," said Sandrine Montgomery-Miller, CEO of Micronoma. "As we are preparing to make our innovations available to clinicians and their patients next year, it is invigorating to be recognized by distinguished experts who see the promise our work offers."

Other recognition Micronoma has received this year include:

-September 2021: Selection from among hundreds of companies as a finalist in the New Life Sciences Diagnostic category for the UCSF Health Award. The UCSF Health Awards honors outstanding health technologies and innovations dramatically transforming healthcare.

-July 2021: Winning the Bio-IT World Innovative Practices Award Grand Prize. This elite awards program highlights outstanding examples of how technology innovations and strategic initiatives can advance life sciences research, from basic biomedical research to drug development and beyond.

-February 2021: Winning the Unicorn Battle Silicon Valley, billed as the "World’s Largest Startup Pitch Contest" by geography and number of entries. This event recognizes startup companies that have accomplished impressive innovations.

The diagnostic implications of Micronoma’s work to use microbiome markers in liquid and tissue biopsies for early-stage cancer diagnosis are extensive. These distinctions show Micronoma is uniquely leading the way toward preventing unnecessary suffering caused by later-stage diagnoses and potentially enabling earlier, personalized, and less-invasive treatments.