On May 5, 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 first quarter of 2021, and associated Company developments (Press release, Supernus, MAY 5, 2021, View Source [SID1234579181]).

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"The approval of Qelbree provides pediatric patients living with ADHD a therapy with proven efficacy and a tolerable safety profile, and that is not a controlled substance," said Jack Khattar, President and CEO of Supernus Pharmaceuticals. "Our Qelbree commercial launch activities are ongoing and include engagement with both physicians and patient groups who have expressed great interest in this unique new alternative for the treatment of ADHD."

Net Product Sales

First quarter 2021 net product sales were $128.4 million, 39% higher than the same period in 2020.

Qelbree Launch Update

In April 2021, the U.S. Food and Drug Administration (FDA) approved Qelbree for the treatment of attention-deficit hyperactivity disorder (ADHD) in pediatric patients 6 to 17 years of age. The Company plans to make Qelbree available in the U.S. during the second quarter of 2021.
Supernus will conduct post-marketing commitment studies, including a new study of Qelbree in preschool aged children with ADHD, 4 to 5 years of age. The completion of these studies responds to a written request from the FDA and should therefore result in the FDA granting an additional 6 months of market exclusivity.
Product Pipeline Update

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

In December 2020, the Company announced positive results from a Phase III trial in adult patients with ADHD and plans to submit a supplemental New Drug Application (sNDA) to the FDA for Qelbree in adults in the third quarter of 2021.
SPN-830 (apomorphine infusion pump) – Continuous treatment of motor fluctuations ("on-off" episodes) in PD

The company recently met with the FDA to discuss the path forward for resubmission of the SPN-830 NDA. The FDA provided additional clarity related to the contents of the November 2020 Refusal to File (RTF) letter and the requirements for resubmission. The Company now plans to resubmit the SPN-830 NDA in the second half of 2021.
SPN-820 – Novel first-in-class activator of mTORC1

SPN-820 has advanced to a Phase II clinical program in treatment-resistant depression following the successful completion of a multiple-ascending dose (MAD) study in healthy volunteers. In the MAD study, SPN-820 exhibited a favorable safety and tolerability profile across a broad range of potentially therapeutic doses.
The Company expects to initiate a randomized Phase II clinical study in treatment-resistant depression by the end of 2021.
Financial Highlights

First quarter 2021 operating earnings were $13.2 million, as compared to $29.0 million in the first quarter 2020. In the first quarter of 2021, the Company recorded non-cash research and development expense of $15 million related to the equity investment in Navitor as a result of the accounting impact of the March 2021 Navitor corporate restructuring and non-cash contingent consideration expense of $1 million associated with the 2020 USWM acquisition. Operating earnings for the first quarter of 2021 included amortization of intangible assets expense of $6.0 million, compared to $1.3 million in the first quarter of 2020.

First quarter 2021 net earnings and diluted earnings per share were $5.7 million and $0.11, respectively, as compared to $21.5 million, or $0.40 per diluted share, in the same period last year.

As of March 31, 2021, the Company had $807.7 million in cash, cash equivalents and marketable securities, compared to $772.9 million as of December 31, 2020.

Full Year 2021 Financial Guidance

For full year 2021, the Company reiterates its prior financial guidance and added full year 2021 effective tax rate guidance as set forth below:

1) Total revenues includes net product sales and royalty revenue. Includes $10 million for Qelbree net product sales.
2) Combined research and development and selling, general and administrative expenses.
3) Operating earnings include amortization of intangible assets and contingent consideration expense.
4) The full year 2021 effective tax rate guidance of 28% – 31% is above the normally expected range of 26% – 28% due to the effect of discrete tax items in the period.

Conference Call Details

The Company will hold a conference call hosted by Jack Khattar, President and Chief Executive Officer and Jim Kelly, Executive Vice President and Chief Financial Officer, to discuss these results at 4:30 p.m. Eastern Time, today, May 5, 2021.

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 May 5, 2021 OliX Pharmaceuticals Inc. (KOSDAQ: 226950) a leading developer of RNAi therapeutics, and PCI Biotech (OSE: PCIB), a cancer focused biopharmaceutical company with a unique intracellular delivery technology, reported an extensive research collaboration (Press release, PCI Biotech, MAY 5, 2021, View Source [SID1234585157]). OliX Pharmaceuticals and PCI Biotech will combine their know-how and technology platforms to explore synergies and further partnership on dermatology and other applications.

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The partnership is governed by a research collaboration agreement, under which the collaborators will perform an extensive evaluation of technology compatibility and synergy based on OLX104C (Androgenic Alopecia) preclinical studies. The companies will evaluate results achieved from this research collaboration to explore the potential for further development and partnership.

Commenting on the announcement, Dong Ki Lee, CEO of OliX Pharmaceuticals said: "We are very excited to collaborate with PCI Biotech. Combining our proprietary asiRNA platform with PCI’s leading fimaNAc delivery technology will significantly accelerate our efforts to bring our dermatological RNAi programs to the clinic. We also expect to expand the area of application to our cancer immunotherapy pipeline and mRNA vaccines developed by our subsidiary, mCureX, in the near future."

PCI Biotech’s CEO, Per Walday, added: "We are very pleased to initiate this exciting collaboration with OliX Pharmaceuticals. This collaboration builds on the strong potential of our intracellular delivery platform technology for nucleic acid based therapies, within a therapeutic field of good technological fit. Combining the leading technologies of OliX Pharmaceuticals and PCI Biotech may lead to new promising applications and we look forward to the collaboration."

On May 5, 2021 Quanterix Corporation (NASDAQ: QTRX), a company helping to transform healthcare with digital protein biomarker technology enabling precision health, reported financial results for the three months ending March 31, 2021 (Press release, Quanterix, MAY 5, 2021, View Source [SID1234579201]).

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"We made productive advances on our strategic and operational priorities during the first quarter 2021," said Chairman, Chief Executive Officer and President, Quanterix, Kevin Hrusovsky. "The precision health ecosystem continues to catalyze interest and adoption from healthcare payors, government agencies, academia, and pharmaceutical companies as they make tangible advances in developing drug therapies for Alzheimer’s, Parkinson’s, Multiple Sclerosis, traumatic brain injury, COVID-19, infectious diseases and cancers. We are rapidly innovating our technology and expanding our assay menu with a priority focus on early detection plasma biomarkers for Alzheimer’s and neuro-degeneration to keep pace with today’s research demands and deliver on the promise of non-invasive biomarkers to fuel breakthroughs in diagnostics and drug development."

First Quarter 2021 Financial Highlights

Key financial results for the first quarter of 2021 are shown below:

Q1 GAAP total revenue, which includes grant revenue of $2.3M, was $27.2M versus prior year Q1 of $15.7M;
Q1 non-GAAP total revenue was $24.9M versus prior year Q1 of $15.7M, an increase of 58%;
Q1 GAAP product revenue was $18.2M versus prior year Q1 of $9.8M, an increase of 86%;
Q1 GAAP service and other revenue was $6.4M versus prior year Q1 of $5.8M, an increase of 11%
Q1 GAAP gross margin was 60.1%; Q1 non-GAAP gross margin was 58.5% versus prior year Q1 of 48.5%, an increase of 1,000 bps.
For additional information on the non-GAAP financial measures included in this press release, please see "Use of Non-GAAP Financial Measures" and "Reconciliation of Non-GAAP Financials" below.

First Quarter Business Highlights

Received Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA) for Quanterix’ Simoa SARS-CoV-2 N Protein Antigen Test that can be run on the Simoa HD-X Analyzer.
Released the SARS-CoV-2 N Protein Antigen Advantage Assay tailored for Research Use, enabling scientists to measure viral load and immune response in a variety of sample matrices, including blood obtained by minimally invasive capillary (finger prick) sample.
Record Instrument placement revenue of $7.0M, an increase of 86%, and consumables revenue of $11.3m, an increase of 86%.
Simoa played a key role in research to detect the COVID-19 virus in serum/plasma, dried blood microsamples (DBS) and saliva samples, including a Nature Communications study validating non-invasive antigen detection methods as an important complement to current PCR-based nasal and nasopharyngeal (NP) swab samples.
Continued to experience significant demand for phosphorylated tau at threonine 181 (pTau181) assays for the study of Alzheimer’s Disease (AD) pathology and Neuro Multiplex assays associated with many neurodegenerative diseases.
Simoa neurology assays and technologies continue to support various Alzheimer’s drug development programs globally, focusing on detecting signs of precognitive impairment early in the disease cascade with blood biomarkers.
Established a joint Simoa laboratory with WuXi AppTec in China to improve access to Quanterix’ industry leading biomarker testing and development offerings across China and the APAC market.
Successfully raised approximately $270M in net proceeds through a follow-on offering in February, further strengthening our balance sheet with growth capital.
Participated in important industry events aimed at addressing public health challenges created by the COVID-19 pandemic, including the Precision Medicine World Conference (PMWC) 2021 COVID-19 Conference and the Precision Medicine Leadership Summit hosted in connection with Powering Precision Health.
Invited to discuss the Powering Precision Health movement, rebuilding public trust in science and leadership of the industry-wide proteomics revitalization disrupting healthcare on Health Care Rounds Podcast and Mendelspod.
Welcomed Laurie Olson, a seasoned industry executive and former Pfizer executive leadership team member, to our board as we continue to scale the business and capitalize on the market opportunity ahead.
Simoa technology was highlighted in 110 new publications in the first quarter 2021, bringing total Simoa-specific inclusions to more than 1,200 publications.
Conference Call

In conjunction with this announcement, Quanterix Corporation will host a conference call on May 5 at 4:30 p.m., EDT. Individuals interested in listening to the conference call may do so by dialing (800) 697-5978 for domestic callers, or (630) 691-2750 for international callers. Please reference the following conference ID: 9220 612#. A live webcast will also be available at: View Source

The webcast will be available on the Company’s website, View Source, for one year following completion of the call.

On May 5, 2021 Leidos (NYSE: LDOS), a FORTUNE 500 science and technology company, reported that it will participate in the Goldman Sachs Industrials and Materials Conference 2021 webcast (Press release, Leidos, MAY 5, 2021, View Source [SID1234579217]).

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Jim Reagan, Chief Financial Officer, will participate in a question and answer "fireside chat" on Tuesday, May 11, 2021 at 10:30 a.m. ET.

A live audio webcast of the event will be available on the Leidos Investor Relations website at View Source A replay of the webcast will be available following the presentation at the same link listed above for one year afterward.

On May 5, 2021 Ampio Pharmaceuticals (NYSE American: AMPE), a biopharmaceutical company focused on the advancement of immunology-based therapies for prevalent inflammatory conditions for which there are limited treatment options, reported quarterly financial results for the first quarter period ended March 31, 2021, and provided a corporate overview and business update (Press release, Ampio, MAY 5, 2021, View Source [SID1234579234]).

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Mr. Michael Macaluso, President and Chief Executive Officer, commented, "This has been an important quarter for Ampio, with noted progress across our therapeutic platform. For example, the FDA has recently responded to our plans for the AP-013 Phase III trial for the intra-articular injection of Ampion for patients suffering from severe osteoarthritis of the knee (OAK). The response not only provides us with flexibility for maintaining the Special Protocol Assessment (SPA) but, in addition, allows us to consider several alternative paths forward to an optimal solution that may include strategic discussions with potential partners for the commercialization, and expansion of osteoarthritis indications, of Ampion."

"We were also encouraged with the results of the AP-014 Phase I clinical trial of inhaled Ampion for COVID-19 patients. The study met its primary endpoint of safety and tolerability, and a 78% reduction in all-cause mortality was observed for patients treated with Ampion compared to Standard of Care (SOC). Approximately 24% of the patients receiving SOC alone died during the study, compared with 5% treated with Ampion."

"Our results from the AP-014 trial are strong and compelling," continued Macaluso, "and we look forward to promptly enrolling patients in a set of double-blind, placebo-controlled Phase II trials utilizing Ampion in COVID-19 patients with inhalation and intravenous routes of drug delivery. We will be looking for confirmation of the efficacy results seen in our Phase I trial in order to move quickly in applying for Emergency Use Authorization."

Mr. Michael Macaluso, President and Chief Executive Officer, Dr. David Bar-Or, Director and Founder, Ms. Holli Cherevka, Chief Operating Officer and Mr. Daniel Stokely, Chief Financial Officer will be hosting a Conference Call for the Investment Community this afternoon beginning at 4:30 PM ET (see details below).

The key areas of focus will be as follows:

COVID-19 Platform / Pipeline Overview and Update

AP-014 (inhaled) Phase I clinical trial of Ampion met its primary endpoint, and demonstrated improvement in all-cause mortality
Double-blind, placebo-controlled Phase II trials utilizing (i) inhaled Ampion for patients impacted from COVID-19 induced respiratory distress and (ii) intravenously delivered Ampion for COVID-19 patients, will begin enrollment in the U.S. during the second quarter of 2021
OAK Clinical Trial 2021 Timeline / Update

Positive FDA response provides guidance on multiple pathways forward on paused AP-013 Phase III trial in osteoarthritis of the knee (OAK)
Long-COVID and Other Clinical Trial 2021 Timeline / Update

Phase I Long-COVID trial is expected to commence enrolling patients in the U.S. during the second quarter of 2021
Update on Other Pre-Clinical Research Programs

Results of pre-clinical study demonstrated that Ampion inhibits pro-inflammatory pathway in types of immune cells implicated in COVID-19 and Lupus Nephritis
Ampion shown to suppress TRL7 signaling, and thereby, reducing the pro-inflammatory chemokine, CXCL10
Financial Results for the First Quarter Period Ended March 31, 2021

Cash and cash equivalents totaled $15.8 million at March 31, 2021, compared to $17.3 million at December 31, 2020. The decrease of $1.5 million is primarily attributable cash used to fund the operating activities for the period of $4.1 million; partially offset by net proceeds received from the utilization of our at-the-market (ATM) equity offering and warrant exercises totaling $2.6 million and $0.1 million, respectively.

Research and development expenditures for the first quarter period ended March 31, 2021 were $2.3 million, compared to $4.3 million for the same period in 2020. The decrease in research and development expenses of $2.0 million, or 46%, for the first quarter period ended March 31, 2021 compared to the amounts for the same period in 2020 was primarily due to the overall decrease in clinical trial and sponsor research related expenses related to the AP-013 study being temporarily paused in April 2020 and continuing through current as a result of the COVID-19 pandemic. The pause of this study resulted in a reduction of expenses totaling $2.4 million which was partially offset by $0.2 million of expenses associated with the AP-014 (inhaled Ampion) and AP-017 (intravenous Ampion) Phase I studies; both of which were initiated in periods subsequent to March 31, 2020.

General and administrative expenses for the first quarter period ended March 31, 2021 were $1.5 million, compared to $1.8 million for the same period in 2020. The decrease in general and administrative expenses for the first quarter period ended March 31, 2021 compared with the same period for 2020 is primarily due to the decrease in professional fees as a result of the decrease in legal costs which is primarily attributable to both the securities class action and derivative cases being closed in the third quarter of 2020.

Other income was $0.2 million for the first quarter period ended March 31, 2021 compared to other income of $0.8 million for the same period in 2020. For the first quarter period ended March 31, 2021 the other income relates directly to the warrant derivative gain recorded for the investor warrants as a result of the warrant exercises during the period which reduced the liability and was partially offset by the increase in stock price of 6.3% during the period. The derivative gain of $0.8 million recorded for the 2020 period related to the reduction of the derivative liability resulting from the 28.2% reduction of stock price during the period.

Net loss for the first quarter period ended March 31, 2021 was $3.7 million, or $0.02 on a fully diluted per share basis, compared to a net loss of $5.2 million, or $0.04 on a fully diluted per share basis, for the same period in 2020. The lower net loss reported for the first quarter period ended March 31, 2021 compared to the same period for 2020 is primarily attributable to a reduction in clinical trial and sponsor related research expenses of $2.4 million as a result of pausing the AP-013 study in early 2020 due to the COVID-19 pandemic; partially offset by the higher reported derivative gain resulting from the reduction of the derivative liability associated with the unexercised investor warrants.

The total shares of common stock outstanding were 195,689,128 at March 31, 2021, compared to 193,378,996 at December 31, 2020.

Financial Guidance

Based on its current operating plans and expected access to equity financing, Ampio expects to have cash and cash equivalents along with access to external sources of liquidity sufficient to fund research and development programs and business operations through the second quarter of 2022.

Conference Call and Webcast

Ampio will host a conference call today at 4:30pm EST (1:30 pm PST) to discuss these first quarter 2021 results and provide a corporate business update.

The live call / webcast may be accessed as follows:

A replay of the conference call will also be available from the Investors Relations section of the Company’s website at www.ampiopharma.com and will be archived there shortly after the live event.