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Immunic, Inc. Reports Third Quarter 2020 Financial Results and Provides Corporate Update

On November 5, 2020 Immunic, Inc. (Nasdaq: IMUX), a clinical-stage biopharmaceutical company developing a pipeline of selective oral immunology therapies aimed at treating chronic inflammatory and autoimmune diseases, reported financial results for the third quarter ended September 30, 2020 and provides corporate update (Press release, Immunic, NOV 5, 2020, View Source [SID1234570227]).

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"The third quarter was marked by a significant achievement for our lead asset, selective oral DHODH inhibitor, IMU-838, with the release of top-line data from our phase 2 EMPhASIS trial in patients with relapsing-remitting multiple sclerosis, and subsequent publication of the full unblinded data set. IMU-838 demonstrated robust efficacy, through the achievement of all primary and key secondary endpoints, combined with an outstanding safety and tolerability profile. We believe that the phase 2 results emphasize the game-changing potential of IMU-838 as a once-daily oral therapy for this indication," stated Daniel Vitt, Ph.D., Chief Executive Officer and President of Immunic. "On the heels of this overwhelmingly positive data, we completed a successful common stock offering, raising $103.5 million in gross proceeds, significantly bolstering our balance sheet. We are currently preparing our end-of-phase 2 submission for IMU-838 in relapsing-remitting multiple sclerosis to regulatory authorities, anticipated in the first quarter of next year, after which we look forward to scheduling our end-of-phase 2 meetings, during which we will have the opportunity to discuss our plans for a phase 3 program."

Dr. Vitt continued, "We have also made notable strides in our development of IMU-838 in patients with moderate COVID-19, most recently securing a financing agreement of up to €24.5 million (approximately $29 million) with the European Investment Bank for the ongoing development of our phase 2 CALVID-1 trial, a potential expansion into a confirmatory phase 3 trial and commercial-scale manufacturing of IMU-838. Additionally, we reported that an Independent Data Monitoring Committee recommended the continuation, without changes, of the CALVID-1 trial after an interim safety analysis. Recruitment has accelerated due to the spread of disease within the countries included in the trial and, at the beginning of November, we reached the enrollment goal of 200 patients, pre-specified in the protocol as sufficient to perform the main efficacy analysis of the phase 2 part of the CALVID-1 trial. The data of this unblinded main analysis of all available efficacy, biomarker and virus titer data is expected to be available in the first quarter of next year."

The company’s current cash position of approximately $133.2 million, at September 30, 2020, is expected to fund activities into the second half of 2022, and specifically through key value inflection points including those for the COVID-19 phase 2 trial with IMU-838; preparation for, and initiation of, a phase 3 trial of IMU-838 in relapsing-remitting multiple sclerosis (RRMS); the phase 2 readouts of IMU-838 in patients with ulcerative colitis (UC) and primary sclerosing cholangitis (PSC); as well as completion of the phase 1 trials of IMU-935 and IMU-856, respectively, including pharmacodynamic evaluations in patients for both of these trials.

Third Quarter 2020 and Subsequent Highlights

November 2020: Announced 200 patients enrolled and randomized in phase 2 CALVID-1 trial of IMU-838 for the treatment of moderate COVID-19, allowing for main phase 2 efficacy analysis to proceed.
October 2020: Signed financing agreement with the European Investment Bank for up to €24.5 million (approximately $29 million) to support the development of IMU-838 in patients with moderate COVID-19.
September 2020: Announced results of interim safety analysis from ongoing phase 2 CALVID-1 trial of IMU-838 in patients with moderate COVID-19. Independent Data Monitoring Committee recommended continuation of the trial without changes.
September 2020: Published full unblinded clinical data set from phase 2 EMPhASIS trial of IMU-838 in patients with RRMS and announced poster presentation at the MSVirtual2020/8th Joint ACTRIMS-ECTRIMS Meeting. Data confirmed and expanded on the previously announced top-line results, which showed achievement of all primary and secondary endpoints.
August 2020: Dosed first healthy volunteer in phase 1 clinical program of IMU-856, targeting restoration of intestinal barrier function.
August 2020: Completed a $103.5 million public offering of 5,750,000 shares of common stock, including the full exercise of underwriters’ option to purchase an additional 750,000 shares.
August 2020: Announced positive top-line data from phase 2 EMPhASIS trial of IMU-838 in patients with RRMS. Study met all primary and key secondary endpoints, indicating activity in RRMS patients.
July 2020: Enrolled first patients in investigator-sponsored phase 2 IONIC trial of IMU-838 in combination with Oseltamivir (Tamiflu), in collaboration with sponsor and lead site, University Hospitals Coventry and Warwickshire (UHCW) NHS Trust.
Update on Activities Relating to the Preparation of a Phase 3 Program for IMU-838 in RRMS

As previously announced, the full unblinded clinical data set from the company’s phase 2 trial of IMU-838 in patients with RRMS showed achievement of all primary and key secondary endpoints, with high statistical significance. Notably, both doses (30 and 45 mg/day) appeared to be equally safe and efficacious, reducing the number of combined unique active (CUA) magnetic resonance imaging (MRI) lesions up to week 24, as compared to placebo. Based on this phase 2 data as well as established regulatory guidance that the lowest effective dose should be considered for future clinical trials, Immunic may propose the dose of 30 mg/day of IMU-838 for investigation in a phase 3 program.

Given the relative equal performance of the two doses tested, however, and to allow for pharmacodynamic modeling of the dose-response relationship, data from a lower dose in the effective dose range would be beneficial to complete a dose-effect assessment of IMU-838 in RRMS. For this reason, Immunic has already started an additional, small Cohort 2 sub-trial to obtain exploratory data on the expanded dose response of IMU-838. This additional, double-blind assessment includes a cohort of 60 patients who receive 10 mg/day of IMU-838 or placebo for 24 weeks. The still-active sites of the phase 2 trial of IMU-838 in RRMS continue to be used and, as a result, the company expects that this assessment can be executed in an accelerated fashion. This additional sub-trial is not expected to change any conclusions for dosing of IMU-838 in a future phase 3 program. Rather, it is expected to provide additional data to address any potential regulatory requests in the context of the design of a phase 3 program. Management believes that this strategy will enable risk reduction for end-of-phase 2 discussions with regulatory agencies.

The company intends to separate formal end-of-phase 2 meeting preparations from regulatory advice for non-clinical phase 3 related topics to be submitted to regulatory authorities in the near future. Subsequently, the company intends to submit formal end-of-phase 2 meeting requests with a sole focus on the phase 2 data, including the Cohort 2 interim data, and a proposed phase 3 program to regulatory authorities at the end of the first quarter 2021. The end-of-phase 2 meetings are expected to be held in or about May 2021.

In parallel to the preparation and execution of the regulatory discussions, Immunic has begun performing formal feasibility activities for a phase 3 program of IMU-838 in RRMS, including country and site selection, as well as other preparatory activities. The company also believes that this feasibility assessment will help ensure an expeditious execution of the development strategy for IMU-838.

Additional Anticipated Clinical Milestones

IMU-838 in PSC: Due to the COVID-19 pandemic, recruitment for the phase 2, investigator-sponsored proof-of-concept clinical trial for IMU-838 in PSC, being conducted at the Mayo Clinic, is currently paused as patients with PSC are considered high risk for COVID-19 infections and were advised by the investigators to avoid travelling to the clinical sites. Together with the investigators, Immunic currently expects a potential data readout during the fourth quarter of 2020 using the 18 patients who were enrolled prior to the COVID-19 pandemic. The overall recruitment target for this open-label study is 30 patients.
IMU-838 in COVID-19: Top-line efficacy, biomarker and virus titer data from the phase 2 CALVID-1 trial of IMU-838 in patients with moderate COVID-19, based on approximately 200 patients treated, is expected to be available in the first quarter of 2021, after which the company will be able to evaluate whether the program may be expanded into a confirmatory phase 3 trial.
IMU-838 in UC: Although the recruitment of the phase 2 clinical trial of IMU-838 for the treatment of UC has continued during the COVID-19 pandemic, the pandemic has affected, among other things, access to endoscopy sites or hospitals in some countries, which has interfered with recruitment speed, new site activations and clinical site access. As a result, top-line data is now expected to be available in the first half of 2022, instead of in the fourth quarter of 2021 as previously announced.
IMU-935 phase 1 program: The current, single ascending dose part of the ongoing phase 1 trial of IMU-935 is planned to be followed by a multiple ascending dose portion in healthy volunteers and a safety evaluation in patients with mild-to-moderate psoriasis. The trial has been resumed after a temporary pause of trials in healthy volunteers imposed by ethics committees in Australia due to COVID-19. Unblinded safety data from the single and multiple ascending dose parts in healthy volunteers is expected to be available in the first half of 2021. Initiation of the third portion in patients with mild-to-moderate psoriasis is expected in the first half of 2021 and is expected to last approximately 12 months.
Potential IMU-935 phase 2 trial in an orphan autoimmune disorder: Upon completion of the single and multiple ascending dose portions of the ongoing phase 1 trial, Immunic anticipates that it may also begin a phase 2 proof-of-concept clinical trial of IMU-935 in an orphan autoimmune indication. This orphan approach may allow for an accelerated path to approval, in parallel to IMU-935’s previously planned development in psoriasis. After a thorough review of suitable autoimmune conditions, the company has targeted IMU-935 for further development in Guillain-Barré syndrome, an acute neurological disorder in which the body’s immune system attacks its peripheral nervous system, and for which very few therapies exist. The company plans to announce additional details as soon as design and timing of the envisaged trial are defined.
Financial and Operating Results

Research and Development (R&D) Expenses were $11.0 million for the three months ended September 30, 2020, as compared to $7.1 million for the same period ended September 30, 2019. The $3.9 million increase was primarily attributable to (i) a $2.9 million increase in external development costs for IMU-838, related to the phase 2 clinical trial in patients with COVID-19, (ii) a $2.2 million increase in license fees, drug supply and phase 1 costs related to IMU-856, (iii) a $0.6 million increase in development costs for the IMU-935 program, and (iv) a $0.5 million increase in personnel costs. The increases were partially offset by a decrease of $2.3 million in costs related to the phase 2 clinical trial of IMU-838 in patients with RRMS.
For the nine months ended September 30, 2020, R&D expenses were $27.5 million, as compared to $16.5 million for the same period ended September 30, 2019. The $11.0 million increase was primarily attributable to (i) a $4.8 million increase in external development costs for IMU-838, related to the phase 2 clinical trial in patients with COVID-19, (ii) a $4.5 million increase in license fees, drug supply and phase 1 costs related to IMU-856, (iii) a $1.4 million increase in drug supply costs related to IMU-935, (iv) a $1.2 million increase in drug supply costs related to IMU-838, and (v) a $0.6 million increase in personnel costs. The increases were partially offset by a decrease of $1.5 million related to the phase 2 clinical trial of IMU-838 in patients with RRMS.
General and Administrative (G&A) Expenses were $2.5 million for the three months ended September 30, 2020, as compared to $2.1 million for the same period ended September 30, 2019. The $0.4 million increase was primarily due to increased personnel expenses.
For the nine months ended September 30, 2020, G&A expenses were $7.3 million, as compared to $12.4 million for the same period ended September 30, 2019. The $5.0 million decrease was primarily due to (i) non-recurring costs related to the transaction with Vital Therapies including $6.4 million of stock-based compensation for company executives, key employees and members of the board of directors, and (ii) $2.1 million of non-recurring investment banking and legal fees in the first nine months of 2019. The decrease was partially offset by (i) a $2.3 million increase in personnel expenses, (ii) $1.0 million of increased legal, accounting and consultancy costs, and (iii) $0.2 million of increased costs across numerous categories primarily due to becoming a public company and expanding operations in the United States.
Other Income was $0.6 million for the three months ended September 30, 2020, as compared to $0.9 million for the same period ended September 30, 2019. The $0.3 million decrease was primarily attributable to the $0.4 million difference between the face value and fair value of the promissory note collected in full in September 2019 in connection with the sale of certain assets of Vital Therapies (ELAD Assets), partially offset by the $0.1 million write-off of the investment in Vital Therapies (Beijing) Company Limited (VTL China) included in the ELAD Assets sale.
For the nine months ended September 30, 2020, other income was $1.9 million, as compared to $1.6 million for the same period ended September 30, 2019. The $0.3 million increase was primarily attributable to $0.7 million of R&D tax incentives for clinical trials in Australia as a result of increased spending on clinical trials in Australia, partially offset by a decrease attributable to (i) the $0.4 million difference between the face value and fair value of the promissory note collected in full in September 2019 in connection with the sale of ELAD Assets offset by the $0.1 million write-off of the investment in VTL China included in the ELAD Assets sale, and (ii) a $0.1 million decrease of recognized income attributable to reimbursements of R&D expenses in connection with the option agreement with Daiichi Sankyo Co., Ltd.
Net Loss for the three months ended September 30, 2020 was approximately $12.9 million, or $0.70 per basic and diluted share, based on 18,405,840 weighted average common shares outstanding, compared to a net loss of approximately $8.2 million, or $0.82 per basic and diluted share, based on 10,022,856 weighted average common shares outstanding for the same period ended September 30, 2019.
Net loss for the nine months ended September 30, 2020 was approximately $32.9 million, or $2.35 per basic and diluted share, based on 13,966,690 weighted average common shares outstanding, compared to a net loss of approximately $27.2 million, or $3.96 per basic and dilutes share, based on 6,880,057 weighted average common shares outstanding for the same period ended September 30, 2019.
Cash and Cash Equivalents, as of September 30, 2020, were $133.2 million, which management expects to be sufficient to fund operations into the second half of 2022.

Rigel Reports Third Quarter 2020 Financial Results and Provides Business Update

On November 5, 2020 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported financial results for the third quarter ended September 30, 2020, including sales of TAVALISSE (fostamatinib disodium hexahydrate) tablets, for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment (Press release, Rigel, NOV 5, 2020, View Source [SID1234570226]).

"Our team has done an excellent job advancing our key value drivers while adapting to the widespread changes in the current global environment," said Raul Rodriguez, Rigel’s president and CEO. "We have continued to grow our TAVALISSE franchise with third quarter sales increasing 39% year-over-year and our global Phase 3 clinical trial for warm AIHA having enrolled over 60% of our patient goal. Additionally, exploration of fostamatinib’s potential in COVID-19 is rapidly expanding with our Phase 3 clinical trial launching this quarter and enrollment ongoing in the Phase 2 trials sponsored by the NIH/NHLBI and Imperial College London."

Business Update
Rigel’s FORWARD study, a Phase 3 pivotal trial of TAVALISSE in warm autoimmune hemolytic anemia (AIHA), has enrolled 57 of the 90 patients targeted for enrollment. The trial currently has over 90 clinical trial sites established across 22 countries.

Rigel plans to launch a Phase 3 clinical trial to evaluate the safety and efficacy of fostamatinib in hospitalized COVID-19 patients without respiratory failure that have certain high-risk prognostic factors. This multi-center, double-blind, placebo-controlled, adaptive design study is expected to enroll over 300 evaluable patients that will be randomly assigned to either fostamatinib plus standard of care (SOC) or matched placebo plus SOC (1:1). Treatment will be administered orally twice daily for 14 days. There will be a follow-up period to day 60. The primary endpoint of this study is the proportion of subjects who progress to severe/critical disease within 29 days.

The Phase 2 clinical trial sponsored by the NIH/NHLBI, in collaboration with Inova Health System, to evaluate the safety of fostamatinib for the treatment of hospitalized COVID-19 patients has enrolled 9 patients. This multi-center, double-blind, placebo-controlled study will randomly assign fostamatinib plus SOC or matched placebo plus SOC (1:1) to approximately 60 evaluable patients. Treatment will be administered orally twice daily for 14 days. There will be a follow-up period to day 60. The primary endpoint of this study is cumulative incidence of serious adverse events (SAE) through day 29. The trial also includes multiple secondary endpoints designed to assess the early efficacy and clinically relevant endpoints of disease course.

The Imperial College London-sponsored Phase 2 clinical trial to evaluate the efficacy of fostamatinib for the treatment of COVID-19 pneumonia has begun enrolling patients. The study is a two-stage, open label, controlled clinical trial with patients randomized (1:1:1) to fostamatinib plus SOC, ruxolitinib plus SOC, or SOC. Treatment will be administered twice daily for 14 days and patients will receive a follow-up assessment at day 14 and day 28 after the first dose. The primary endpoint of this study is progression from mild to severe COVID-19 pneumonia within 14 days in hospitalized patients.

Rigel recently launched FORTE, an observational study to further evaluate TAVALISSE as a second-line treatment for adult chronic ITP. The study goal is to generate additional data on patient quality of life and financial expenditures relative to the healthcare of ITP patients. Along with the post-hoc data analysis of its Phase 3 clinical program, Rigel plans to use this study to further increase and enhance its database of TAVALISSE in early line treatment of adult chronic ITP patients.

Financial Update
For the third quarter of 2020, Rigel reported a net loss of $14.2 million, or $0.08 per share, compared to a net loss of $11.5 million, $0.07 per share, in the same period of 2019.

In the third quarter of 2020, total revenues were $18.4 million, consisting of $16.3 million in net product sales and $2.1 million in contract revenues from collaborations for the achievement of a milestone in accordance with the amended license and collaboration agreement with Daiichi-Sankyo. Net product sales increased by 39% from $11.7 million in the third quarter of 2019. The decrease in contract revenues from collaborations in the third quarter of 2020 from $9.1 million in the same period of 2019 was due to developmental and commercial milestones from its various collaborative partners in 2019, partially offset by the milestone in the third quarter of 2020 from Daiichi-Sankyo as noted above.

Rigel reported total costs and expenses of $32.2 million in the third quarter of 2020, compared to $32.9 million in the same period of 2019. The decrease in total costs and expenses was primarily due to decreases to the timing of certain commercial-related activities due to the COVID-19 pandemic, partially offset by the increases in personnel-related costs and increased use of consultants.

For the nine months ended September 30, 2020, Rigel reported a net loss of $10.5 million, or $0.06 per share, compared to a net loss of $49.7 million, or $0.30 per share, in the same period of 2019.

Rigel reported total revenues of $90.2 million for the nine months ended September 30, 2020, compared to $43.9 million in the same period of 2019. Total revenues for the nine months ended September 30, 2020 consisted of $43.9 million in net product sales and $46.2 million in contract revenues from collaborations. The increase in contract revenues from collaborations related to revenue from the upfront fee previously received in 2019, as well as the milestone payment received from Grifols in the first quarter of 2020 upon EC approval of the MAA for fostamatinib in Europe and the $2.1 million in contract revenues for achievement of a milestone in accordance with the amended license and collaboration agreement with Daiichi-Sankyo, partially offset by the developmental and commercial milestones from its various collaborative partners in 2019.

Total costs and expenses for the nine months ended September 30, 2020 were $100.3 million, compared to $95.6 million in the same period of 2019. The increase in total costs and expenses was primarily related to increases in research and development cost for the on-going Phase 3 trial in warm AIHA, Phase 1 trial in RIP 1 inhibitor program and Phase 1 trial in IRAK 1/4 inhibitor program, partially offset by decreases in stock-based compensation expense and various third-party costs.

As of September 30, 2020, Rigel had cash, cash equivalents and short-term investments of $72.8 million, compared to $98.1 million as of December 31, 2019.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time
Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing 1-800-954-0603 (domestic) or 1-415-226-5355 (international). The conference call and accompanying slides will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About ITP
In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP include fatigue, excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. In addition to fostamatinib, current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs) and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 45,000 adult patients in the U.S. and can be a severe, debilitating disease. Warm AIHA (wAIHA), the most common form of AIHA, is characterized by the presence of antibodies that react with the red blood cell surface at body temperature. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients.

About Coronavirus Disease 2019 (COVID-19) & SYK-Signaling
COVID-19 is the infectious disease caused by Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2). SARS-CoV-2 primarily infects the upper and lower respiratory tract and can lead to acute respiratory distress syndrome (ARDS). Additionally, some patients develop other organ dysfunction including myocardial injury, acute kidney injury, shock resulting in endothelial dysfunction and subsequently micro and macrovascular thrombosis.1 Much of the underlying pathology of SARS-CoV-2 is thought to be secondary to a hyperinflammatory immune response associated with increased risk of thrombosis.2

Spleen tyrosine kinase (SYK) is involved in the intracellular signaling pathways of many different immune cells. Therefore, SYK inhibition may improve outcomes in patients with COVID-19 via inhibition of key Fc gamma receptor (FcγR) and c-type lectin receptor (CLR) mediated drivers of pathology, such as inflammatory cytokine release by monocytes and macrophages, production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation.3,4,5 Furthermore, SYK inhibition in neutrophils and platelets may lead to decreased thromboinflammation, alleviating organ dysfunction in critically ill patients with COVID-19.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.

Nevro Announces Third Quarter 2020 Financial Results

On November 5, 2020 Nevro Corp. (NYSE: NVRO), a global medical device company that is providing innovative, evidence-based solutions for the treatment of chronic pain, reported its financial results for the third quarter ended September 30, 2020 (Press release, Nevro, NOV 5, 2020, View Source [SID1234570225]).

Third Quarter 2020 Financial Overview
Worldwide revenue for the third quarter of 2020 was $108.5 million, an increase of 8% compared to $100.2 million in the prior year period. On a sequential basis, third quarter worldwide revenue increased 92% compared to the second quarter of 2020. U.S. revenue in the third quarter of 2020 was $90.9 million, an increase of 8% compared to $84.2 million in the prior year period. On a sequential basis, third quarter U.S. revenue increased 78% compared to the second quarter of 2020. In the third quarter of 2020, total U.S. permanent implants increased 9%, while trials were down 5% compared to the prior year period. U.S. revenue growth in the third quarter of 2020 was driven by an increase in patient and customer activity compared to the severely COVID-impacted second quarter of 2020. Daily patient trial activity increased 50% over the second quarter of 2020 and improved sequentially every month from July to October. The Company estimates a majority of the canceled permanent implant cases from the second quarter of 2020 were completed by the end of the third quarter of 2020. International revenue was $17.5 million, an increase of 10% on an as reported basis (5% on a constant currency basis), compared to $15.9 million in the prior year period. On a sequential basis, third quarter International revenue increased 226% compared to the second quarter of 2020 International revenue in the third quarter of 2020 was primarily driven by a successful Omnia launch, an increase in patient and customer activity, and a rebound in customer inventory levels compared to the second quarter of 2020.

"We are pleased by the rebound in sales after a difficult second quarter and continue to be encouraged by the month-over-month improvement in trial procedures," said D. Keith Grossman, Chairman, CEO and President of Nevro. "With trials recovering, we believe our earlier expectations for roughly flat revenues compared to the prior year is still reasonable. However, increases in COVID activity around the world and any further pressure on facility capacity and patient willingness to seek care could provide some downward pressure to that view."

Gross profit for the third quarter of 2020 was $76.1 million, an increase of 9% compared to $69.9 million in the prior year period. Gross margin was 70.1% in the third quarter compared to 69.8% in the prior year period. Compared to the prior year period, the increase in gross margin in the third quarter of 2020 was primarily attributable to product mix.

Operating expenses for the third quarter of 2020 were $79.6 million, a 7% decrease compared to $85.9 million in the prior year period. The year-over-year decrease in operating expenses was primarily related to reduced travel and training-related expenses, decreases in discretionary expenses during the COVID-19 pandemic, as well as continued management focus on driving leverage throughout the business which had begun well before COVID. This was partially offset by a one-time charge of $2.5 million in the quarter related to the Company’s CFO transition. Legal expenses associated with patent litigation were $2.3 million for the third quarter of 2020, compared to $1.9 million in the prior year period.

Net loss from operations for the third quarter of 2020 was $3.5 million, a 78% improvement compared to a loss of $16.0 million in the prior year period. Adjusted EBITDA for the third quarter of 2020 was $13.6 million, compared to a loss of $2.0 million in the prior year period. Adjusted EBITDA excludes certain litigation expenses, interest, taxes and non-cash items such as stock-based compensation and depreciation and amortization. Please see the financial table below for GAAP to Non-GAAP reconciliations.

Cash, cash equivalents and short-term investments totaled $572.9 million as of September 30, 2020, an increase of $10.5 million in the third quarter of 2020.

PDN and NSRBP Clinical Update
Nevro’s Painful Diabetic Neuropathy (PDN) study continues to move forward and remains on track to present the next round of complete six-month data, along with a preview of 12-month responder rate data, at NANS in January of 2021. The Company has been in early discussions with the FDA regarding the submission strategy and is now planning to submit its PMA Supplement to the FDA in the fourth quarter of 2020. The Company remains on track for a second half 2021 commercial launch.

The Company also expects to present its Non-Surgical Refractory Back Pain (NSRBP) study with three-month data at NANS 2021 in January, with journal publications thereafter. In total, Nevro is expected to have a very large presence with several data presentations at the conference, including PDN and NSRBP.

Webcast and Conference Call Information
Management will host a conference call today at 1:30 pm PT/ 4:30 pm ET. Investors interested in listening to the call may do so by dialing (866) 324-3683 in the U.S. or +1 (509) 844-0959 internationally, using Conference ID: 6693376. In addition, a live webcast, as well as an archived recording, will be available on the "Investors" section of the Company’s website at: www.nevro.com.

Luminex Corporation Reports Strong Third Quarter 2020 Results

On November 5, 2020 Luminex Corporation (Nasdaq: LMNX) reported results for its third quarter ended September 30, 2020 (Press release, Luminex, NOV 5, 2020, View Source [SID1234570224]).

All amounts in this release are in conformity with U.S. generally accepted accounting principles ("GAAP").

CURRENT FINANCIAL HIGHLIGHTS

Total revenue for the third quarter of $106.1M, a 35% increase over Q3 2019, driven primarily by growth associated with the current COVID-19 global pandemic
Strong profitability in Q3 with 60% gross margins, an improvement of 7 percentage points over Q3 2019
Operating margin of $11.5M or 11% of revenue, a 300% improvement over Q3 2019
Net income of $1.8M, representing 2% of revenue, and $0.04 per share, up 134% over Q3 2019
Received two BARDA awards — $5,389,813 to support a future 510(k) filing for an updated NxTAG Respiratory Pathogen Panel that includes SARS-CoV-2, and $683,500 to support future enhancement of Luminex’s COVID-19 Multiplex Antibody Test
CEO COMMENTARY

"I’m very pleased with the performance of our diversified business during these very challenging times. Our team continues to execute on our key role in addressing the demands of this pandemic. With strong revenue growth, improved gross margins and continued control over operating expenses, we are seeing healthy profitability and cash flow," said Nachum "Homi" Shamir, Chairman, President and CEO of Luminex. "As a result of these unusual times; we thought it would be helpful to share with our investors some of our preliminary estimates of our 2021 revenue guidance. Currently, we estimate that in 2021, we will generate at least $475M of revenue with accelerated improvement in profitability and cash flow. We believe that this growth will be primarily attributable to our ARIES manufacturing expansion, as well as a number of additional new products in development, including those supporting our COVID-19-related efforts and the other pipeline products in our diverse portfolio. We plan to provide more precision regarding our expectations in early 2021. However, based on our current estimates, we anticipate surpassing our $500 million annual revenue target much sooner than we previously expected, while continuing to build an outstanding company."

ADDITIONAL HIGHLIGHTS OF THE QUARTER

Molecular Diagnostics revenue for the quarter of $59.9M, up 98% over Q3 2019
Licensed Technologies Group revenue of $34.7M, down 10% from Q3 2019
Flow Cytometry revenue of $9.9M, up 13% over Q3 2019, but with continued impact from the slowdown in academic research due to COVID-19
Operating cash flow of $14.9M and $39.0M for the three and nine months ended September 30, 2020
Ended the quarter with a total order book of more than $36M, of which $9M was COVID-19 related
Sold or contracted 87 sample-to-answer systems in the quarter, the majority of which were ARIES
Received FDA EUA for the xMAP SARS-CoV-2 Multi-Antigen IgG Assay on July 16, 2020

REVENUE GUIDANCE

As of the date hereof, Luminex is providing revenue guidance as follows:

Luminex expects revenue for the full year 2020 to be approximately $410M, with growth of 23% over 2019.
Luminex expects 2021 revenue to be at or above $475 million, reflecting more than 15% growth from Luminex’s full year 2020 guidance, with such revenue growth driven primarily by significant expansion of ARIES assay sales resulting from completion of the manufacturing line expansion and new product launches.
CONFERENCE CALL

Management will host a conference call at 4:00 p.m. Central Time / 5:00 p.m. EDT, Thursday, November 5, 2020 to discuss operating highlights and financial results for the third quarter 2020. The conference call will be webcast live and may be accessed at Luminex Corporation’s website at investor.luminexcorp.com. Analysts may participate on the conference call by dialing (877) 930-7053 (U.S.) or (253) 336-7290 (outside the U.S.). The access code is 4669812. The webcast will be archived for six months on our website using the ‘replay’ link.

AcelRx Pharmaceuticals Reports Third Quarter 2020 Financial Results

On November 5, 2020 AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings, reported its third quarter 2020 financial results (Press release, AcelRx Pharmaceuticals, NOV 5, 2020, View Source [SID1234570222]).

"The third quarter represented one of our most active and successful quarters since DSUVIA’s approval," said Vince Angotti, Chief Executive Officer of AcelRx. "We’ve made significant progress on our revenue growth strategy, highlighted by our oral and dental surgery collaboration with Zimmer Biomet, the addition of DSUVIA to the Department of Defense Joint Deployment Formulary and our new U.S. Army contract. With the recent publication of data supporting DSUVIA’s strong value proposition, we have gained momentum in DSUVIA’s use within existing hospital and ambulatory surgery center customers, and the rate of scheduled formulary reviews. Progress within the Department of Defense continues and we expect the initial ordering of DSUVIA for deployed troops in the fourth quarter."

Third Quarter Highlights

In July, AcelRx entered into a distribution agreement with Zimmer Biomet to market DSUVIA within the dental and oral surgery markets in the United States exclusively through Zimmer Biomet’s Dental division, expanding U.S. availability of DSUVIA. It is estimated that the applicable market in dental surgeries is over 7 million annual procedures.
In July, AcelRx completed a $10 million common stock offering priced at the market with two leading life science investors.
In August, AcelRx announced the publication of a study entitled "Reduced Opioid Use and Reduced Time in the Postanesthesia Care Unit Following Preoperative Administration of Sublingual Sufentanil in an Ambulatory Surgery Setting," by Christian Tvetenstrand, MD and Michael Wolff, MD, in the Journal of Clinical Anesthesia and Pain Management. Highlights of the publication included a greater than 50% overall reduction in opioids administered perioperatively and a 34% reduction in PACU time in the DSUVIA-treated patients compared to historical controls.
In August, AcelRx announced an investigator-initiated study with Cleveland Clinic that will assess the effects of DSUVIA on post-operative recovery from orthopedic surgery.
In September, AcelRx announced that the U.S. military’s access to DSUVIA had been expanded with the addition of DSUVIA to the Department of Defense Joint Deployment Formulary (JDF).
In September, the U.S. Army awarded AcelRx with an initial contract of up to $3.6 million over four years for the purchase of DSUVIA to support a DoD study to aid the development of clinical practice guidelines.
Financial Information

Cash, cash equivalents and short-term investments balance was $43.0 million as of September 30, 2020.
Net revenues for the third quarter 2020 were $1.4 million, of which approximately $1.3 million relates to product sales (compared to $0.3 million in product sales for the second quarter of 2020).
Combined R&D and SG&A expenses for the third quarter of 2020 totaled $8.6 million, a significant reduction compared to $12.0 million for the third quarter of 2019. Excluding stock-based compensation expense, these amounts were $7.5 million for the third quarter of 2020 compared to $10.7 million for the third quarter of 2019. The decrease in combined R&D and SG&A expenses in the third quarter of 2020 was primarily due to a reduction of $1.8 million in personnel costs and a $1.1 million reduction in DSUVIA-related commercialization expenses.
Net loss for the third quarter of 2020 was $8.9 million, or $0.10 per basic and diluted share, compared to $12.7 million, or $0.16 per basic and diluted share, for the third quarter of 2019.
Webcast and Conference Call Information
As previously announced, AcelRx will host a live webcast Thursday, November 5, 2020 at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss these financial results and provide other corporate updates. The webcast is accessible by visiting the Investors page of AcelRx’s website at www.acelrx.com and clicking on the webcast link. The webcast will be accompanied by a slide presentation. Investors who wish to participate in the conference call may do so by dialing (866) 361-2335 for domestic callers, (855) 669-9657 for Canadian callers or (412) 902-4204 for international callers. A webcast replay will be available on the AcelRx website for 90 days following the call by visiting the Investor page of AcelRx’s website at www.acelrx.com.

About DSUVIA (sufentanil sublingual tablet), 30 mcg
DSUVIA, known as DZUVEO in Europe, approved by the FDA in November 2018, is indicated for use in adults in certified medically supervised healthcare settings, such as hospitals, surgical centers, and emergency departments, for the management of acute pain severe enough to require an opioid analgesic, and for which alternative treatments are inadequate. DSUVIA was designed to provide rapid analgesia via a non-invasive route and to eliminate dosing errors associated with intravenous (IV) administration. DSUVIA is a single-strength solid dosage form administered sublingually via a single-dose applicator (SDA) by healthcare professionals. Sufentanil is an opioid analgesic previously only marketed for IV and epidural anesthesia and analgesia. The sufentanil pharmacokinetic profile when delivered sublingually avoids the high peak plasma levels and short duration of action observed with IV administration. The European Commission approved DZUVEO for marketing in Europe in June 2018 and AcelRx is currently in discussions with potential European marketing partners.