On August 4, 2020 Idera Pharmaceuticals, Inc. ("Idera" or the "Company") (Nasdaq: IDRA) reported its financial and operational results for the second quarter ended June 30, 2020 (Press release, Idera Pharmaceuticals, AUG 4, 2020, View Source [SID1234562807]).

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"Idera meaningfully advanced its clinical pipeline and strengthened its financial resources in the first part of 2020. Further encouraged by data from ILLUMINATE-204, which we reported in the second quarter, we continue to work diligently against our timelines for ILLUMINATE-301. Those timelines currently remain on track for data in the first quarter of 2021, despite disruptions from the global impact of COVID-19," stated Vincent Milano, Idera’s Chief Executive Officer. "In addition, as part of our ‘beyond melanoma’ strategy, early data from ILLUMINATE-206 reinforces our optimism in the potential of tilsotolimod in patients with micro-satellite stable colorectal cancer. Lastly, our team’s outstanding perseverance and dedication to our patients combined with the further financing we recently secured will help us continue to execute these key objectives and potentially beyond."

Corporate Update

Since March 31, 2020, the Company entered into two private placement financings of up to $40.7 million, with $5.0 million received in April 2020 and $5.1 million received in July 2020. The Company anticipates that its current cash, cash equivalents, and short-term investments will fund our operations into the second quarter of 2021. With the Company’s current financing vehicles, there exists the possibility to extend that runway through subsequent proceeds to fund the potential NDA filing and commercial launch of tilsotolimod.

ILLUMINATE (tilsotolimod) Clinical Development Updates

ILLUMINATE-301: Randomized phase 3 trial of tilsotolimod in combination with Yervoy* (ipilimumab) versus Yervoy alone in patients with anti-PD-1 refractory advanced melanoma:

•Primary endpoint family of overall response rate (ORR) by blinded independent review using RECIST v1.1 and overall survival (OS);
•Trial initiated in March 2018;
•Enrollment completed in March 2020; and
•ORR and other preliminary data expected in the first quarter of 2021.

ILLUMINATE-206: Phase 2, open-label, multicohort, multicenter study to test the safety and effectiveness of tilsotolimod in combination with Yervoy and Opdivo* (nivolumab) for the treatment of solid tumors:
·Trial initiated in September 2019 with the microsatellite stable colorectal cancer (MSS-CRC) cohort;
·Initial safety run-in of 10 patients, which included Yervoy at 1 mg/kg every 8 weeks and Opdivo at 3 mg/kg every 2 weeks, showed that the regimen was generally well tolerated;
·Planned changes in the study design intended to improve potential outcomes in this patient population include increasing Yervoy dosing frequency to every 3 weeks and limiting the number of allowed prior lines of treatment to 2; and
·Enrollment of the next 10 patients is targeted to begin in the fourth quarter of 2020, with data anticipated in the second quarter of 2021.

ILLUMINATE-204: Phase 1/2 trial of tilsotolimod in combination with Yervoy or Keytruda± (pembrolizumab) in patients with anti-PD-1 refractory advanced melanoma:

•Final topline results released in April 2020 from the recommended phase 2 dose (RP2D) of 8 mg of tilsotolimod in combination with Yervoy, which is the treatment regimen being evaluated in the Company’s registrational trial, ILLUMINATE-301; and
•Final data from the trial will be shared in a Mini Oral presentation at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020, to be held September 19-21, 2020.

Second Quarter Financial Results

Research and development expenses for the three months ended June 30, 2020 totaled $5.4 million compared to $10.0 million for the same period in 2019. General and administrative expense for the three months ended June 30, 2020 totaled $2.6 million compared to $2.9 million for the same period in 2019. Additionally, during the three months ended June 30, 2020, we recorded $0.9 million and $15.3 million non-cash warrant revaluation loss and non-cash future tranche right revaluation loss, respectively, related to securities issued in connection with our December 2019 private placement transaction.

As a result of the factors above, net loss applicable to common stockholders for the three months ended June 30, 2020 was $24.2 million, or $0.72 per basic and diluted share, compared to net loss applicable to common stockholders of $11.2 million, or $0.39 per basic and diluted share, for the same period in 2019. Excluding the non-cash loss of approximately $16.3 million for the three months ended June 30, 2020 related to the securities issued in connection with the December 2019 private placement transaction, net loss applicable to common stockholders was $8.0 million, or $0.24 per basic and diluted share (calculated based upon the basic weighted-average number of common shares, due to the antidilutive effect of net loss).

As of June 30, 2020, our cash, cash equivalents, and short-term investments totaled $31.0 million. Based on our current operating plan, we anticipate that our current cash, cash equivalents, and short-term investments, including $5.1 million gross proceeds in cash received in July 2020 pursuant to the July 2020 Securities Purchase Agreement, will fund our operations into the second quarter of 2021.

On August 4, 2020 Apollo Endosurgery, Inc. ("Apollo") (Nasdaq: APEN), a global leader in less invasive medical devices for gastrointestinal and bariatric procedures, reported financial results for the second quarter ended June 30, 2020.
Second Quarter 2020 and Recent Highlights (Press release, Apollo Endosurgery, AUG 4, 2020, View Source [SID1234562805]).

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•As pre-announced, second quarter revenue was $5.6 million, a decrease of approximately 60% compared to the second quarter of 2019, reflecting the sharp decline in healthcare utilization triggered from the second half of March through April due to the COVID-19 pandemic, followed by progressively recovering market conditions in May and June
•Implemented cost reduction initiatives in anticipation of a decline in second quarter sales due to the COVID-19 pandemic. As a result, second quarter cash used in operating and investing activities was consistent with pre-pandemic quarterly levels
•Submitted a 510k for the X-Tack Endoscopic HeliX Tacking System for the Treatment of Gastrointestinal Defect Closures
•Completed a $25 million equity financing which combined with reduced minimum liquidity requirements under the amended Loan and Security Agreement with Solar Capital Ltd. ("Solar") increased corporate liquidity by $32.5 million
Todd Newton, CEO of Apollo, said, "The COVID-19 pandemic had a significant impact on sales in the second quarter, but after the initial slowdown in April we have been on a recovery path. This recovery was particularly visible in the United States where June 2020 product sales were close to 90% of our June 2019 product sales level. In July, the recovery has continued with sales in our direct markets exceeding sales from July of last year.
"We took immediate actions to preserve the interests of our shareholders by aggressively reducing cash expenditures at the beginning of the second quarter, while still pursuing our most important development programs such as the X-Tack program. X-Tack is designed to enhance an endoscopist’s ability to address the closure challenges of large or irregularly shaped defects, whether encountered in the upper or lower GI tract, and meaningfully expands our addressable market. Additionally during the quarter, the Mayo Clinic published results from an IDE study using Orbera for the treatment of NASH. The Mayo study reported 65% of patients achieved resolution of NASH at 6 months. Lastly, the MERIT study continues to progress although planned activities at certain of the study sites were delayed due to COVID-19 factors. We believe that these and other efforts will open new avenues for revenue growth in the future."
Stefanie Cavanaugh, Apollo’s Chief Financial Officer, said, "We set an aggressive goal to match the anticipated reduction in revenue this quarter with reduced operating expenses as part of our COVID-19 response plan and keep cash use at a level consistent with pre-pandemic quarters. We met this goal. Also, we further reduced our market risk by concurrently raising $25 million of equity and securing beneficial covenant amendments from our lender. We will remain diligent to calibrate our cash expenditures with the pace of our business recovery."
Second Quarter Results
Worldwide Endoscopy product sales were $5.4 million for the second quarter of 2020, a decrease of 56% compared to the second quarter of 2019. The decline reflects the significant disruption of healthcare resource utilization worldwide from the COVID-19 pandemic.
U.S. Endoscopy sales decreased 40% to $3.1 million for the second quarter of 2020 and outside the US ("OUS") Endoscopy sales decreased 68% to $2.2 million. Between our two OUS sales channels, direct market Endoscopy sales decreased 63% while sales to distributors declined 76% compared to the second quarter of 2019.
ESS product sales represented 67% of total Endoscopy sales for the second quarter of 2020, compared with 63% for the second quarter of 2019.
Total revenue for the second quarter of 2020 was $5.6 million, a decline of 60% from $14.3 million in the prior year second quarter. Revenue reported in the second quarter of 2019 included $1.9 million related to transition services rendered in that period for the Surgical product line which was divested in December of 2018. In the second quarter of 2020, revenue from transition services were only $0.2 million.

Gross margin for the second quarter of 2020 was 43% as $0.5 million of unabsorbed overhead costs from reduced production volumes were charged to our cost of sales in the quarter. Gross margin in the second quarter of 2019 was 50%.
Total operating expenses decreased $7.6 million, or 53%, for the second quarter of 2020 due to the liquidity preservation program implemented in response to the COVID-19 pandemic.
Net loss for the second quarter of 2020 declined 29% to $6.3 million compared to $8.8 million for the second quarter of 2019.
Cash, cash equivalents and restricted cash were $19.7 million as of June 30, 2020.
Conference Call
Apollo will host a conference call on August 4, 2020 at 3:30 p.m. Central Time / 4:30 p.m. Eastern Time to discuss operating results for the second quarter ended June 30, 2020. To join the conference call by telephone, please dial +1-862-298-0970. A live webcast of the conference call will be made available on the "Events and Presentations" section of our Investor Relations website: ir.apolloendo.com.
A replay of the webcast will be made available on Apollo’s website, www.apolloendo.com following the event.
Non-GAAP Financial Measures
To supplement our financial results, we are providing a non-GAAP financial measure, Endoscopy product sales percentage change in constant currency, which removes the impact of changes in foreign currency exchange rates that affect the comparability and trend of our Endoscopy product sales compared to the same period of the prior year. Endoscopy product sales percentage change in constant currency is calculated by translating current foreign currency sales at last year’s exchange rate. This supplemental measure of our performance is not required by, and is not determined in accordance with GAAP.
We believe the non-GAAP financial measure included herein is helpful in understanding our current financial performance. We use this supplemental non-GAAP financial measure internally to understand, manage and evaluate our business, and make operating decisions. We believe that making non-GAAP financial information available to investors, in addition to GAAP financial information, may facilitate more consistent comparisons between the company’s performance over time with the performance of other companies in the medical device industry, which may use similar financial measures to supplement their GAAP financial information. However, our non-GAAP financial measure is not meant to be considered in isolation or as a substitute for the comparable GAAP metric.

On August 4, 2020 Invitae Corporation (NYSE: NVTA), a leading medical genetics company, reported financial and operating results for the second quarter ended June 30, 2020 (Press release, Invitae, AUG 4, 2020, View Source [SID1234562803]).

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"While we experienced significant disruptions in the healthcare system due to the pandemic, we quickly established a solid recovery during the quarter. Our results highlight the strength of our operations and the benefits of our diversified menu, investments in telehealth capabilities and longstanding customer relationships, all of which position us to adapt and meet the changing needs of our customers," said Sean George, Ph.D., co-founder and chief executive officer of Invitae. "We exited the quarter with a strong footing and increasing momentum. We remain confident in our ability to continue to deliver on our mission to bring genetic information into mainstream medicine."

Second Quarter 2020 Financial Results
•Accessioned more than 120,000 samples in the second quarter of 2020 compared to 111,000 samples in the second quarter of 2019. Billable volume exceeded 113,000 in the second quarter of 2020
•Generated revenue of $46.2 million in the second quarter of 2020 compared to $53.5 million in revenue in the second quarter of 2019
•Reported average cost per sample of $358 in the second quarter of 2020 compared to $252 average cost per sample in the second quarter of 2019. Non-GAAP average cost per sample was $318 in the second quarter of 2020
•Achieved gross profit of $3.2 million in the second quarter of 2020 compared to $25.5 million of gross profit in the second quarter of 2019. Non-GAAP gross profit was $8.0 million in the second quarter of 2020

Total operating expense, excluding cost of revenue, for the second quarter of 2020 was $145.3 million. Non-GAAP operating expense was $105.7 million in the second quarter of 2020.

Net loss for the second quarter of 2020 was $166.4 million, or $1.29 net loss per share, compared to a net loss of $48.7 million in the second quarter of 2019, or $0.54 net loss per share. Non-GAAP net loss was $99.2 million, or $0.77 non-GAAP net loss per share, in the second quarter of 2020.

At June 30, 2020 cash, cash equivalents, restricted cash, and marketable securities totaled $428.5 million. Net increase in cash, cash equivalents and restricted cash for the quarter was $78.0 million. Cash burn, including various acquisition-related expenses, was $89.2 million for the quarter and $63.8 million when excluding the $25.4 million cash paid to acquire YouScript and Genelex.

Early in the quarter, in response to impacts of the pandemic, the company took actions to significantly scale back operational expenditures. The result of these changes is expected to decrease the discretionary spend in cost of revenue and operating expense beginning in the third quarter.

"We continue to see a solid recovery in volume, improvement in our operating leverage and ability to improve revenue generation. As a result, we are well positioned with sufficient capital to execute our strategy in the coming years," continued Dr. George. "We have added or will be adding important capabilities to our platform through the acquisitions we announced this quarter and our ongoing product development efforts. With our mission clearly in focus, we can continue to navigate these unprecedented times."

Corporate and Scientific Highlights
•Introduced expanded services and support for transition to telehealth across customer types
◦Launched new capabilities for Gia, the advanced clinical chatbot that became part of Invitae through the acquisition and rapid integration of Clear Genetics. New workflows added to Gia support obstetrician/gynecologists, oncologists, genetic counselors and other clinicians who order genetic testing
◦Increased support for the use of at-home testing using saliva kits, which do not require an in-person clinician visit
◦Provided professional education and support for clinicians transitioning to telehealth
•Acquired YouScript and Genelex to bring best-in-class pharmacogenetic testing, and robust, integrated clinical decision support to Invitae
•Further expanded international footprint, including the introduction of consumer-initiated telehealth genetic testing services in Canada for carrier testing in early pregnancy and cancer and cardiovascular risk testing
•Presented research that combined with new recommendations from a large, multidisciplinary consensus conference published in the Journal of Clinical Oncology, underscores the utility of increased access to genetic testing for men with prostate cancer across all stages of life
•Added 16 new biopharma partnerships, bringing the total number of partnership programs to more than 105, including nine new pharma partners in Invitae’s Detect programs providing no-charge genetic testing for conditions in which testing is underutilized and can improve diagnosis and treatment.
•Entered into a definitive agreement under which Invitae will combine with ArcherDX, Inc.
•Closed on a public offering with net proceeds of approximately $173.0 million and raised $44.5 million of net proceeds under the company’s ATM
•In July, added non-invasive prenatal screening (NIPS) based on whole genome sequencing (WGS) to the Invitae platform, providing patients with easier access to affordable genetic testing in early pregnancy to realize cost reductions, improve the company’s ability to scale services and pave the way for additional services based on WGS technology
•Partnered with a major health system to integrate clinical decision support software for use of pharmacogenetics in patient care

Webcast and Conference Call Details
Management will host a conference call and webcast today at 4:30 p.m. Eastern / 1:30 p.m. Pacific to discuss financial results and recent developments. To register for the conference call and webcast, please use one of the methods below. Upon registering, each participant will be provided with call details and a registrant ID.

Online registration: View Source

Phone registration: (888) 869-1189 or (706) 643-5902

The live webcast of the call and slide deck may be accessed here or by visiting the investors section of the company’s website at ir.invitae.com. A replay of the webcast and conference call will be available shortly after the conclusion of the call and will be archived on the company’s website.

On August 4, 2020 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the second quarter ended June 30, 2020 (Press release, BioMarin, AUG 4, 2020, View Source [SID1234562801]).

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Net Product Revenues for the second quarter of 2020 increased to $419.0 million, compared to $379.1 million in the second quarter of 2019. The increase in Net Product Revenues was attributed to the following:
•Aldurazyme Net Product Revenues increased by $26.5 million due to higher sales volume to Genzyme;
•Palynziq Net Product Revenues increased by $21.9 million driven by a combination of revenue from U.S. patients achieving maintenance dosing and new patients initiating therapy;
•Brineura Net Product Revenues increased by $11.0 million due in large part growth in the number of patients in all regions; and
•Kuvan Net Product Revenues increased by $9.3 million driven primarily by a U.S. price increase and Kuvan product mix; partially offset by
•Naglazyme and Vimizim Net Product Revenues combined decreased by $23.2 million primarily due to timing of orders as well as the impact of missed infusions resulting from the COVID-19 pandemic.
The decrease in GAAP Net Loss for the second quarter of 2020, compared to GAAP Net Loss for the same period in 2019 was primarily due to the following:
•an increase in gross profit (Total Revenues less Cost of Sales) of $21.2 million primarily driven by higher product sales; and
•an increase in the benefit from income taxes; partially offset by
•the effect of the one-time gain recognized in the second quarter of 2019 due to a third party’s achievement of a commercial milestone related to previously sold intangible asset; and
•higher selling, general and administrative (SG&A) expense related to pre-commercialization activities for valoctocogene roxaparvovec.
Non-GAAP Income for the second quarter of 2020 increased to $57.4 million, compared to Non-GAAP Income of $17.1 million for the same period in 2019. The increase in Non-GAAP Income for the quarter, compared to the same period in 2019, was attributed to decreased R&D expense and higher gross profit, partially offset by higher SG&A expense.
As of June 30, 2020, BioMarin had cash, cash equivalents and investments totaling approximately $1.7 billion, as compared to $1.2 billion on December 31, 2019.
Commenting on second quarter 2020 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "In the second quarter, BioMarin employees worked collaboratively to ensure access to our critically-important medicines to the people we serve, despite the global impact of COVID-19. In these challenging times, our strong financial results underscore both the essential nature of our products to patients and our ongoing efforts to maintain supply around the world."

Mr. Bienaimé continued, "In the second quarter at the World Federation of Hemophilia Virtual Congress, we were pleased to share the four-year data update from our ongoing Phase 1/2 study, which demonstrated sustained clinical benefit following a single administration of valoctocogene roxaparvovec. All participants in the study received a single administration of valoctocogene roxaparvovec in 2016 and remained off exogenous factor prophylaxis through year four. These data strengthen our confidence in valoctocogene roxaparvovec and the opportunity to address the unmet therapeutic needs of people with severe hemophilia A. With our marketing applications under review in both the United States and Europe, we await the potential approval of valoctocogene roxaparvovec. We believe each of the submissions represent the first time a gene therapy product for any type of hemophilia indication is under review by health authorities. With the outcome of the Priority Review of our BLA anticipated August 21, 2020, our commercial team is preparing to launch what we believe is the most innovative product yet for people with bleeding disorders."
"Another key milestone in the third quarter of this year, representing the culmination of years of clinical study and development, was the July 23 submission of a MAA to the EMA for vosoritide to treat children with achondroplasia. The company remains on track to submit a NDA to the FDA later in the third quarter. Our multi-pronged dossier of data encompasses long-term clinical results in 5 to 18 year-olds from our Phase 2 study, natural history data, the ongoing study of newborns through 5 years, and highly statistically significant placebo-controlled Phase 3 results. The positive results from our vosoritide clinical programs bolster our confidence in the potential for this drug to be the first pharmacological treatment for the underlying cause of achondroplasia. Interest in our clinical studies with vosoritide has been extremely robust, demonstrating that families are keen to seek early treatment for their children."
Mr. Bienaimé concluded, "Despite impact from COVID-19 on our business in the short-term, we remain focused on working towards significant achievements that we believe will drive long-term value. Key milestones for the second half of 2020 include reaching GAAP profitability for a full year for the first time in our history, the potential approval of valoctocogene roxaparvovec, and the pursuit of vosoritide approval. With these exciting possibilities on the horizon, 2020 has the potential to be the most momentous year in our 20-year history."
2020 Full-Year Financial Guidance
GAAP Net Income guidance for 2020 has been updated to include the potential impact of intangible asset transfers between BioMarin entities. These intangible asset transfers are expected to occur in the second half of 2020, and are estimated to result in a one-time, non-cash income tax benefit of approximately $700 million to $900 million. The range acknowledges that the intangible asset transfers have not yet been completed and therefore the final value cannot yet be determined with certainty. The final valuation will be completed when the transactions occur. As a result, full year GAAP net income guidance has been updated to be in the range between $720 million and $980 million. The intangible asset transfers are not expected to impact Non-GAAP income.

On August 4, 2020 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported a poster presentation at the upcoming rescheduled International Symposium on Pediatric Neuro-Oncology (ISPNO) annual meeting taking place December 13-16 in Karuizawa, Japan (Press release, Cellectar Biosciences, AUG 4, 2020, View Source [SID1234562799]).

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One of the study investigators, Dr. Diane Puccetti, a faculty member of the University of Wisconsin School of Medicine and Public Health and Medical Director of the American Family Children’s Hospital will present the poster, entitled: "CLR 131 in patients with relapsed or refractory pediatric malignancies," which highlights Phase 1 study data including subjects with various brain tumors.

The Phase 1 study (NCT03478462) is an open-label dose escalation study of CLR 131 in children and adolescents with relapsed or refractory cancers, including malignant brain tumors, neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma, osteosarcoma and lymphomas (including Hodgkin’s lymphoma). Patients in the study have received infusions of CLR 131 in escalating dose levels. To date, all doses have been deemed safe and tolerated by the independent Data Monitoring Committee, including the 60mCi/m2 dose level.

A copy of the presentation materials can be accessed on the Events and Presentations section of the Cellectar website once the presentation concludes.

About CLR 131

CLR 131 is a small-molecule Phospholipid Drug Conjugate designed to provide targeted delivery of iodine-131 (radioisotope) directly to cancer cells, while limiting exposure to healthy cells unlike many traditional on-market treatment options. CLR 131 is the company’s lead product candidate and is currently being evaluated in a Phase 2 study in B-cell lymphomas, and a Phase 1 dose-escalating clinical study in pediatric solid tumors and lymphomas. The company recently completed a Phase 1 dose-escalation clinical study in relapsed/refractory (r/r) multiple myeloma. The FDA granted CLR 131 Fast Track Designation for both r/r multiple myeloma and r/r diffuse large b-cell lymphoma and Orphan Drug Designation (ODD) for the treatment of multiple myeloma, lymphoplasmacytic lymphoma/Waldenstrom’s macroglobulinemia, neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. CLR 131 was also granted Rare Pediatric Disease Designations for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. Most recently, the European Commission granted an ODD for r/r multiple myeloma.