On August 6, 2020 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the second quarter 2020 and reviewed recent business highlights (Press release, Alnylam, AUG 6, 2020, View Source [SID1234563145]).

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"In the second quarter of 2020, the world experienced unprecedented challenges as it continued to confront the COVID-19 pandemic. While the pandemic continues in some countries and states, our global commercialization strategy is now enabling some customer-facing activities to resume in most markets as we enter the third quarter. We are very pleased with our ONPATTRO and GIVLAARI commercial performance in the second quarter, in the face of the ongoing pandemic, and believe it reflects strong demand for our products as well as our team’s unwavering commitment to assure access to these RNAi therapeutics for patients around the world," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "In the quarter, we also continued advancing our robust late-stage pipeline of investigational RNAi therapeutics, where, notably, we presented full results from the ILLUMINATE-A Phase 3 study and completed regulatory filings for lumasiran, and continued enrollment in our APOLLO-B and HELIOS-B Phase 3 studies of patisiran and vutrisiran, respectively. Further, a key business highlight in the second quarter was our completion of a landmark strategic financing collaboration with Blackstone, which we believe will secure our ability to achieve a self-sustainable financial profile without the need for future equity financing. These and other achievements position us to realize our Alnylam 2020 vision of building a multi-product, global biopharma company with a deep clinical pipeline to fuel continued growth and a robust, organic product engine to drive sustainable innovation and further value creation."

Second Quarter 2020 and Recent Significant Corporate Highlights

Commercial Performance

ONPATTRO

Achieved global net product revenues for the second quarter of 2020 of $66.5 million.
Attained over 1,050 patients worldwide on commercial ONPATTRO treatment as of June 30, 2020.
Continued strong market access in the U.S., with over 15 value-based agreements (VBAs) to date with commercial payers and confirmed access for over 98% of covered U.S. lives.
Continued progress with market access efforts across the CEMEA region (Canada, Europe, Middle East, and Africa), with recent launches in Spain and Italy.
The Company announces today that it has achieved agreement on pricing and reimbursement in France, completing patient access for ONPATTRO in all major European markets in under 2 years following approval.
GIVLAARI

Achieved global net product revenues for the second quarter of 2020 of $11.0 million.
Since launch, received over 85 Start Forms in the U.S. and attained over 100 patients globally on commercial GIVLAARI treatment from launch through June 30, 2020.
Received marketing authorization approval for GIVLAARI in Brazil for the treatment of acute hepatic porphyria in adults.
Continued strong progress with market access in the U.S., with seven completed VBAs with commercial payers, and confirmed access for over 75% of covered U.S. lives.
Continued progress with market access efforts across the CEMEA region, with a successful launch in Germany, commencement of cohort ATU supply in France, and named patient sales in other countries.
Received an Improvement of Medical Benefit (ASMR) score of II from Haute Autorité de Santé (HAS) in France, concluding that GIVLAARI offers significant additional therapeutic value. Only two new commercial medicines received a similar ASMR score in 2019.
Continued work with physicians in multiple regions to support requests for pre-approval access to GIVLAARI in an Expanded Access Program (EAP) in accordance with local requirements.
R&D Highlights

Advanced patisiran (the non-proprietary name for ONPATTRO), for the potential treatment of the cardiomyopathy of both hereditary and wild-type ATTR amyloidosis.
Continued enrollment in the APOLLO-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy.
Presented additional clinical data with patisiran – including 24-month results from the global open-label extension (OLE) study and results from an open-label study in post-orthotopic liver transplant hATTR amyloidosis patients – and published findings from an evaluation of patisiran with concomitant use of TTR stabilizers.
Presented new 12-month interim data from the ENVISION Phase 3 study of givosiran (the non-proprietary name for GIVLAARI) in acute hepatic porphyria (AHP).
In addition, published pivotal results from the ENVISION Phase 3 study in The New England Journal of Medicine.
Submitted a Marketing Authorization Application (MAA) for givosiran in Switzerland and Israel.
Advanced lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1).
Completed the rolling submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) and submitted an MAA to the European Medicines Agency (EMA), with both applications now accepted.
The FDA also granted Priority Review for the NDA and set an action date of December 3, 2020 under the Prescription Drug User Fee Act (PDUFA).
The EMA granted an accelerated assessment for the lumasiran MAA.
Received a positive scientific opinion from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) through the Early Access to Medicines Scheme (EAMS).
Presented complete results from the ILLUMINATE-A Phase 3 study.
Continued treating patients in ILLUMINATE-B, a global Phase 3 pediatric study of lumasiran in PH1 patients less than six years of age with preserved renal function, and remains on track to report topline results in mid-2020.
Continued enrollment in the ILLUMINATE-C Phase 3 study of lumasiran for the treatment of advanced PH1 in patients of all ages.
Advanced vutrisiran, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
Continued treating patients in the fully enrolled HELIOS-A Phase 3 study of vutrisiran in hATTR amyloidosis patients with polyneuropathy, and remain on track to report topline results in early 2021.
Received Fast Track Designation from the FDA for the treatment of the polyneuropathy of hATTR amyloidosis in adults.
Continued enrollment in the HELIOS-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy.
Alnylam’s partner, Novartis, continued advancing inclisiran, potentially the first and only siRNA cholesterol-lowering treatment, which is undergoing review for approval in the U.S. and EU.
Published results from the completed ORION Phase 3 pivotal trials in The New England Journal of Medicine, showing durable and potent LDL-C reduction achieved with inclisiran, with a safety profile similar to placebo.
Alnylam’s partner, Sanofi, continued enrollment in the ATLAS Phase 3 program for fitusiran in patients with hemophilia A or B with and without inhibitors, with topline results expected in H1 2021.
Presented updated interim results at the World Federation of Hemophilia meeting from a Phase 2 OLE study of fitusiran in patients with hemophilia A and B with and without inhibitors.
Advanced early- and mid-stage RNAi therapeutic pipeline programs.
In collaboration with Regeneron, advanced cemdisiran, an investigational RNAi therapeutic for the treatment of complement-mediated diseases.
Continued enrollment in a Phase 2 clinical trial of cemdisiran monotherapy in patients with IgA nephropathy, with topline results expected in 2021.
Regeneron filed a Clinical Trial Application (CTA) in The Netherlands to initiate a Phase 1 study of cemdisiran in combination with pozelimab, an anti-C5 monoclonal antibody, in normal healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH).
Alnylam’s partner, Vir Biotechnology, presented positive interim data from the ongoing Phase 2 trial in patients and results from the Phase 1 trial in healthy volunteers of ALN-HBV02 (VIR-2218), an investigational RNAi therapeutic for the treatment of chronic hepatitis B virus (HBV) infection.
Reported positive initial topline results from the ongoing Phase 1 study of ALN-AGT in hypertension, demonstrating acceptable safety, and clinically significant blood pressure lowering with durability enabling a quarterly or less frequent dosing regimen. Additional Phase 1 results are expected to be presented at a scientific meeting later in 2020, pending abstract acceptance.
Filed a Clinical Trial Application (CTA) for ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 in development for the treatment of nonalcoholic steatohepatitis (NASH). ALN-HSD is being advanced in collaboration with Regeneron.
Selected a Development Candidate (DC), ALN-COV (VIR-2703), for SARS-CoV-2 – the virus that causes COVID-19 – with a plan for accelerated filing of an IND around year-end 2020.
Continued progress advancing investigational RNAi therapeutics for CNS and ocular diseases, including ALN-APP, in development for the treatment of hereditary cerebral amyloid angiopathy (hCAA) and autosomal dominant Alzheimer’s Disease (ADAD), which remains on track for a CTA filing in 2021. The Company announces today that Regeneron has exercised its co-development/co-commercialization option on the ALN-APP program, which Alnylam will lead.
Additional Business Updates

Barry Greene, President of Alnylam, announced his intention to transition from Alnylam to pursue new opportunities at the end of the third quarter. Yvonne Greenstreet, Chief Operating Officer, will assume an expanded role as President and Chief Operating Officer on October 1, 2020.
Entered into a strategic financing collaboration with Blackstone under which Alnylam will receive up to $2 billion that is expected to enable Alnylam to achieve a self-sustainable financial profile without the need for future equity financing.
Entered into an agreement with Dicerna to develop and commercialize investigational RNAi therapeutics for the treatment of alpha-1 antitrypsin (A1AT) deficiency-associated liver disease, and completed a non-exclusive cross-licensing agreement with Dicerna regarding the companies’ respective intellectual property for Alnylam’s lumasiran and Dicerna’s nedosiran investigational programs for the treatment of primary hyperoxaluria.
Expanded infectious disease collaboration with Vir to include the development and commercialization of RNAi therapeutics targeting up to three host factor targets for SARS-CoV-2, including angiotensin converting enzyme-2 (ACE2), transmembrane protease, serine 2 (TMPRSS2), and potentially a third host factor target to emerge from Vir’s functional genomics work.
Formed a Distribution Agreement with taiba Middle East to commercialize Alnylam’s RNAi therapeutics in the Gulf states.
Upcoming Events

In mid- and late 2020, Alnylam intends to:

Achieve regulatory approval for ONPATTRO in Israel.
File a new drug application for GIVLAARI in Japan and achieve regulatory approval for GIVLAARI in Canada.
Report topline results from the ILLUMINATE-B Phase 3 study of lumasiran in PH1 patients less than six years of age with preserved renal function.
Present additional clinical results from the ongoing Phase 1 trial of ALN-AGT.
Initiate a Phase 1 trial of ALN-HSD.
Achieve regulatory approvals for lumasiran in U.S. and EU.
Alnylam’s partner Novartis expects an FDA action date for inclisiran in late 2020.
Alnylam’s partner Regeneron plans to initiate a Phase 1 study of cemdisiran in combination with pozelimab.
Financial Results for the Quarter Ended June 30, 2020

"We are extremely pleased with our financial results for the second quarter, particularly in the face of these challenging circumstances. Our team’s ability to swiftly adapt to virtual engagement with stakeholders and to support the transition of patients, where needed, to alternate sites of care, in addition to strong international results, led to commercial performance for both ONPATTRO and GIVLAARI that exceeded the initial expectations we communicated in May 2020. As a result, we are further revising our full-year revenue guidance for ONPATTRO, with an increase in the midpoint of our guidance as we narrow the range from $270-$300 million to $280-$300 million," said Jeff Poulton, Chief Financial Officer of Alnylam. "Outside of our commercial performance, a key business highlight in the second quarter was our achievement of year-over-year improvement in our non-GAAP operating loss versus 2019. Driven by strong top-line year-over-year growth and disciplined investment in our operations, we continue to believe 2019 represents our peak operating loss year as we begin our journey towards a self-sustainable financial profile. In summary, we believe that we’re well positioned to effectively navigate through the pandemic and continue to deliver on the promise of RNAi therapeutics, bringing transformative therapies to patients with serious diseases around the world."

Net Product Revenues

Net product revenues were $77.5 million in the second quarter 2020 representing 103% growth from the second quarter 2019 as a result of the addition of new patients on therapy and expansion into new markets for ONPATTRO, as well as the ongoing U.S. commercial launch and initial European launch of GIVLAARI.
Net Revenues from Collaborations

Net revenues from collaborations were $26.4 million in the second quarter 2020, an increase from $6.5 million in the second quarter 2019, primarily due to increases in revenues recognized from our Regeneron and Vir collaborations.
Research & Development (R&D) and Selling, General & Administrative (SG&A) Expenses

R&D expenses decreased in the second quarter 2020 compared to the same period in 2019 on a GAAP and non-GAAP basis primarily due to nonrecurring expenses in 2019 from license fees related to the execution of our collaboration agreement with Regeneron, as well as a decrease in expenses associated with material manufactured for clinical trials.
SG&A expenses increased in the second quarter 2020 compared to the same period in 2019 on a GAAP and non-GAAP basis primarily due to increased investment in commercial and medical affairs activity to support the ongoing launches of ONPATTRO and GIVLAARI and initial launch preparation activities for lumasiran.
Cash and Investments

Cash, cash equivalents, marketable debt and equity securities, and restricted investments were $1.95 billion at the end of the second quarter 2020 compared to $1.55 billion at the end of 2019. The increase was primarily due to $600.0 million in proceeds received in the second quarter of 2020 from the sale of future royalties and issuance of common stock to Blackstone and its affiliates, partially offset by cash used in our operations to support overall growth.
A reconciliation of our GAAP to non-GAAP results for the current quarter is included in the tables of this press release.

Conference Call Information

Management will provide an update on the Company and discuss second quarter 2020 results as well as expectations for the future via conference call on Thursday, August 6, 2020 at 8:30 am ET. To access the call, please dial 800-239-9838 (domestic) or +1-323-794-2551 (international) five minutes prior to the start time and refer to conference ID 6976021. A replay of the call will be available beginning at 11:30 am ET on the day of the call. To access the replay, please dial 888-203-1112 (domestic) or +1-719-457-0820 (international) and refer to conference ID 6976021.

A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events. An archived webcast will be available on the Alnylam website approximately two hours after the event.

About ONPATTRO (patisiran)

ONPATTRO is an RNAi therapeutic that was approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the polyneuropathy associated with the disease. For more information about ONPATTRO, visit ONPATTRO.com.

ONPATTRO Important Safety Information

Infusion-Related Reactions

Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO (patisiran). In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.

To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.

Reduced Serum Vitamin A Levels and Recommended Supplementation

ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

Adverse Reactions

The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory tract infections (29%) and infusion-related reactions (19%).

For additional information about ONPATTRO, please see the full Prescribing Information.

About GIVLAARI (givosiran)

GIVLAARI is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved in the United States and Brazil for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is also approved in the European Union for the treatment of AHP in adults and adolescents aged 12 years and older. In the pivotal study, givosiran was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of aminolevulinic acid synthase 1 (ALAS1) messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP. For more information about GIVLAARI, visit GIVLAARI.com.

GIVLAARI Important Safety Information

Contraindications

GIVLAARI is contraindicated in patients with known severe hypersensitivity to givosiran. Reactions have included anaphylaxis.

Anaphylactic Reaction

Anaphylaxis has occurred with GIVLAARI treatment (<1% of patients in clinical trials). Ensure that medical support is available to appropriately manage anaphylactic reactions when administering GIVLAARI. Monitor for signs and symptoms of anaphylaxis. If anaphylaxis occurs, immediately discontinue administration of GIVLAARI and institute appropriate medical treatment.

Hepatic Toxicity

Transaminase elevations (ALT) of at least 3 times the upper limit of normal (ULN) were observed in 15% of patients receiving GIVLAARI in the placebo-controlled trial. Transaminase elevations primarily occurred between 3 to 5 months following initiation of treatment.

Measure liver function tests prior to initiating treatment with GIVLAARI, repeat every month during the first 6 months of treatment, and as clinically indicated thereafter. Interrupt or discontinue treatment with GIVLAARI for severe or clinically significant transaminase elevations. In patients who have dose interruption and subsequent improvement, reduce the dose to 1.25 mg/kg once monthly. The dose may be increased to the recommended dose of 2.5 mg/kg once monthly if there is no recurrence of severe or clinically significant transaminase elevations at the 1.25 mg/kg dose.

Renal Toxicity

Increases in serum creatinine levels and decreases in estimated glomerular filtration rate (eGFR) have been reported during treatment with GIVLAARI. In the placebo-controlled study, 15% of patients receiving GIVLAARI experienced a renally-related adverse reaction. The median increase in creatinine at Month 3 was 0.07 mg/dL. Monitor renal function during treatment with GIVLAARI as clinically indicated.

Injection Site Reactions

Injection site reactions were reported in 25% of patients receiving GIVLAARI in the placebo-controlled trial. Symptoms included erythema, pain, pruritus, rash, discoloration, or swelling around the injection site. One (2%) patient experienced a single, transient, recall reaction of erythema at a prior injection site with a subsequent dose administration.

Drug Interactions

Concomitant use of GIVLAARI increases the concentration of CYP1A2 or CYP2D6 substrates, which may increase adverse reactions of these substrates. Avoid concomitant use of GIVLAARI with CYP1A2 or CYP2D6 substrates for which minimal concentration changes may lead to serious or life-threatening toxicities. If concomitant use is unavoidable, decrease the CYP1A2 or CYP2D6 substrate dosage in accordance with approved product labeling.

Adverse Reactions

The most common adverse reactions that occurred in patients receiving GIVLAARI were nausea (27%) and injection site reactions (25%).

For additional information about GIVLAARI, please see full Prescribing Information.

About LNP TechnologyAlnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases

On August 6, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs ("Regulus"), reported that Jay Hagan, President and Chief Executive Officer of Regulus, will present at the Wedbush PacGrow Healthcare Virtual Conference on Wednesday, August 12, 2020 at 2:20 PM EDT (Press release, Regulus, AUG 6, 2020, View Source [SID1234563144]).

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On August 6, 2020 Pacira BioSciences, Inc. (Nasdaq: PCRX), a leading provider of innovative non-opioid pain management options, reported financial results for the second quarter of 2020 (Press release, Pacira Pharmaceuticals, AUG 6, 2020, View Source;991.htm [SID1234563143]).

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"We are very pleased to report that since the peak of the impact from COVID-19 in April, we’ve seen a steady and continued increase in EXPAREL sales. This is a testimony to the resolve of our physician partners who are dedicated to ensuring that patients receive their much-needed surgeries, particularly in the ambulatory setting," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "This pandemic has accelerated the shift of inpatient procedures to the 23-hour stay environment and we are well positioned to lead the way with our innovative opioid alternatives."
"Today, we are in a stronger position than ever as we wind down our partnership with DePuy Synthes and take full ownership of the EXPAREL franchise. Our recent financing strengthens our financial foundation and supports our strategy to expand our footprint in non-opioid pain management and regenerative health solutions while simultaneously ramping the top and bottom lines," continued Mr. Stack.

Second Quarter 2020 Financial Results
•Total revenues were $75.5 million in the second quarter of 2020, a 26% decrease versus the $102.6 million reported for the second quarter of 2019.
•EXPAREL net product sales were $73.0 million in the second quarter of 2020, a 26% decrease versus the $98.9 million reported for the second quarter of 2019.
•Second quarter 2020 iovera° net product sales were $1.4 million, a 31% decrease versus the $2.0 million reported for the second quarter of 2019.
•Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $0.8 million in the second quarter of 2020, compared to $0.9 million in the second quarter of 2019.
•Second quarter 2020 royalty revenues were $0.3 million, compared to $0.8 million in the second quarter of 2019.

•Total operating expenses were $82.7 million in the second quarter of 2020, compared to $97.3 million in the second quarter of 2019.
•Research and development (R&D) expenses were $13.6 million in the second quarter of 2020, compared to $17.8 million in the second quarter of 2019. R&D expenses include $6.1 million and $6.8 million of product development and manufacturing capacity expansion costs in the second quarters of 2020 and 2019, respectively.
•Selling, general and administrative (SG&A) expenses were $43.3 million in the second quarter of 2020, compared to $49.1 million in the second quarter of 2019.
•GAAP net loss was $7.3 million, or $0.17 per share (basic and diluted), in the second quarter of 2020, compared to GAAP net income of $2.7 million, or $0.07 per share (basic) and $0.06 per share (diluted), in the second quarter of 2019.
•Non-GAAP net income was $5.0 million, or $0.12 per share (basic and diluted), in the second quarter of 2020, compared to non-GAAP net income of $17.5 million, or $0.42 per share (basic) and $0.41 per share (diluted), in the second quarter of 2019.
•Adjusted EBITDA was $8.5 million in the second quarter of 2020, versus adjusted EBITDA of $21.9 million in the second quarter of 2019.
•Pacira ended the second quarter of 2020 with cash, cash equivalents, short-term and long-term investments ("cash") of $335.1 million. Cash used in operations was $15.6 million in the second quarter of 2020, compared to cash provided by operations of $22.8 million in the second quarter of 2019.
•Pacira had 42.2 million basic weighted average shares of common stock outstanding in the second quarter of 2020.
•For non-GAAP measures, Pacira had 42.9 million diluted weighted average shares of common stock outstanding in the second quarter of 2020.
See "Non-GAAP Financial Information" below.

Recent Highlights

•Preliminary net product sales for July 2020. Today the company also reported preliminary unaudited net product sales of EXPAREL and iovera° of $38.1 million and $0.8 million, respectively, for the month of July 2020. Given the rapidly changing variables related to the COVID-19 pandemic, the company does not have sufficient visibility to accurately forecast the impact of the pandemic and is currently not providing full-year 2020 financial guidance. In order to provide greater transparency, the company plans to report monthly intra-quarter unaudited net product sales until it has gained sufficient visibility around the impacts of COVID-19.

•FDA acceptance of sNDA for EXPAREL use in pediatric patients. In August 2020, Pacira announced the U.S. Food and Drug Administration (FDA) has accepted the submission of its supplemental new drug application (sNDA) seeking expansion of the EXPAREL label to include single-dose infiltration to provide postsurgical analgesia in children aged six and over. The expected action date by the FDA under the Prescription Drug User Fee Act (PDUFA) is March 22, 2021.
•Issuance of $402.5 million aggregate principal amount of 0.750% convertible senior notes due 2025. In July 2020, Pacira issued $402.5 million in aggregate principal amount of convertible senior notes due 2025 in a private placement. Pacira used approximately $211.1 million of the net proceeds to repurchase $185.0 million aggregate principal amount of its outstanding 2.375% Convertible Senior Notes due 2022. Pacira intends to use the remainder of the net proceeds from the offering for general corporate purposes, including working capital, research and development expenditures and the license or acquisition of complementary products and/or technologies.

•Conclusion of EXPAREL agreement with DePuy Synthes. In July 2020, Pacira announced the conclusion of its agreement with DePuy Synthes Sales Inc. to jointly market and promote the use of EXPAREL for orthopedic procedures in the United States. Under this collaboration, which began in January 2017, DePuy Synthes field representatives, specializing in joint reconstruction, spine, sports medicine, trauma and cranio-maxillofacial procedures, collaborate with the Pacira field teams to support EXPAREL use and education in orthopedic surgical settings. In addition to partnering with DePuy Synthes in support of orthopedic surgical procedures, Pacira field representatives have remained the overall EXPAREL account managers and commercial leads for soft tissue surgeons, anesthesiologists, and ambulatory surgery centers.

Today’s Conference Call and Webcast Reminder
The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Thursday, August 6, 2020, at 8:30 a.m. ET. To participate in the conference call, dial 1-877-845-0779 and provide the passcode 3276818. International callers may dial 1-720-545-0035 and use the same passcode. In addition, a live audio of the conference call will be available as a webcast. Interested parties can access the event through the "Events" page on the Pacira website at investor.pacira.com.

For those unable to participate in the live call, a replay will be available at 1-855-859-2056 (domestic) or 1-404-537-3406 (international) using the passcode 3276818. The replay of the call will be available for one week from the date of the live call. The webcast will be available on the Pacira website for approximately two weeks following the call.

On August 6, 2020 Lipocine Inc. (NASDAQ: LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported financial results for the second quarter ended June 30, 2020, and provided a corporate update (Press release, Lipocine, AUG 6, 2020, View Source [SID1234563142]).

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Second Quarter and Recent Corporate Highlights

·Presented clinical data on TLANDO at the American Urological Association ("AUA") Virtual Experience
oImpact of a New Oral Testosterone Undecanoate on Blood Pressure and Cardiovascular Risk
oA Novel Oral Testosterone Therapy Restores Testosterone to Eugonadal Levels Without Dose Titration
oEffects of a New Oral Testosterone Undecanoate ("TLANDO") on Liver
·Announced that the treatment potential of LPCN 1144 was demonstrated in a pre-clinical non-alcoholic steatohepatitis ("NASH") and hepatic fibrosis model
·Received FDA clearance on an Investigational New Drug ("IND") application for a Phase 2 clinical study with LPCN 1148 for the treatment of cirrhosis
·Received a decision from the U.S. Court of Appeals for the Federal Circuit affirming the decision of the United States Patent and Trademark Office ("USPTO") in the patent interference case between Lipocine and Clarus Therapeutics Inc. ("Clarus")
·Received decision from Chancery Court of Delaware dismissing the shareholder derivative action filed against Lipocine’s board of directors

Ongoing Activities and Upcoming Milestones

·TLANDO New Drug Application ("NDA") for use in adult males for conditions associated with hypogonadism under review by the U.S. Food and Drug Administration ("FDA") with a Prescription Drug User Fee Act ("PDUFA") target action date of August 28, 2020
·LPCN 1144 Phase 2 LiFT ("Liver Fat intervention with oral Testosterone") Phase 2 clinical study, a paired-biopsy study in confirmed pre-cirrhotic NASH subjects, has been initiated with enrollment continuing and top-line primary endpoint results expected by the end of 2020
·Intellectual property infringement lawsuit against Clarus is on-going, including fact discovery and expert testimony, with the jury trial scheduled for February 2021

Second Quarter Ended June 30, 2020 Financial Results

Lipocine reported a net loss of $6.4 million, or ($0.13) per diluted share, for the quarter ended June 30, 2020, compared with a net loss of $3.4 million, or ($0.14) per diluted share, in the quarter ended June 30, 2019.

Research and development expenses were $2.3 million for the quarter ended June 30, 2020, compared with $2.0 million for the quarter ended June 30, 2019. The increase in research and development expenses during the three months ended June 30, 2020 was primarily due to increased contract research organization and outside consulting and manufacturing costs related to the LPCN 1144 LiFT Phase 2 clinical study in NASH subjects of $965,000, as well as a $213,000 increase in personnel expense. These increases were offset by a $727,000 decrease in costs incurred in conjunction with TLANDO with the completion of the ABPM study in the first half of 2019, a $96,000 decrease in costs for TLANDO XR, a $25,000 decrease in contract manufacturing costs for LPCN 1107 and a $25,000 decrease in other research and development expenses.

General and administrative expenses were $2.0 million for the quarter ended June 30, 2020, compared with $1.4 million for the quarter ended June 30, 2019. The increase in general and administrative expenses during the three months ended June 30, 2020 was primarily due to a $613,000 increase in legal costs associated with the following activities: lawsuit filed against Clarus Therapeutics Inc. for patent infringement in April 2019, interference cases filed against Clarus and the on-going class action lawsuit defense. In addition, there was a $48,000 increase in personnel costs, offset by a $41,000 decrease marketing expense, a $34,000 decrease in administrative travel expenses and a $19,000 decrease in other general and administrative expenses.

As of June 30, 2020, we had $18.3 million of unrestricted cash, cash equivalents and marketable investment securities compared to $14.1 million at December 31, 2019. Additionally, as of June 30, 2020 and December 31, 2019 we had $5.0 million of restricted cash, which is required to be maintained as cash collateral under the SVB Loan and Security Agreement until TLANDO is approved by the FDA.

Six Months Ended June 30, 2020 Financial Results

Lipocine reported a net loss of $12.1 million, or ($0.27) per diluted share, for the six months ended June 30, 2020, compared with a net loss of $6.7 million, or ($0.28) per diluted share, for the six months ended June 30, 2019.

Research and development expenses were $4.8 million for the six months ended June 30, 2020, compared with $3.9 million for the six months ended June 30, 2019. The increase in research and development expenses during the six months ended June 30, 2020 was primarily due to increased contract research organization and outside consulting and manufacturing costs related to the LPCN 1144 LiFT Phase 2 clinical study in NASH subjects of $2.6 million and a $270,000 increase in personnel expense. These increases were offset by a $1.9 million decrease in costs incurred in conjunction with TLANDO with the completion of the ABPM study in the first half of 2019, a $46,000 decrease in costs for TLANDO XR, a $37,000 decrease in contract manufacturing costs for LPCN 1107 and a $20,000 decrease in other research and development expenses.

General and administrative expenses were $4.0 million for the six months ended June 30, 2020, compared with $2.6 million for the six months ended June 30, 2019. The increase in general and administrative expenses during the six months ended June 30, 2020 was primarily due to a $1.6 million increase in legal costs associated with the following activities: lawsuit filed against Clarus Therapeutics Inc. for patent infringement in April 2019, interference cases filed against Clarus and the on-going class action lawsuit defense, offset by a $11,000 decrease in personnel costs, a $60,000 decrease in administrative travel expense, a $41,000 decrease in marketing expense and a $112,000 decrease in other general and administrative expenses.

On August 6, 2020 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTOR, reported financial results for the second quarter ended June 30, 2020 and provided corporate updates (Press release, Selecta Biosciences, AUG 6, 2020, View Source [SID1234563141]).

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"This is a transformational time for Selecta, as we reinforce our position as a leader in immune tolerance. The strategic licensing agreement with Sobi puts us in a financial position that allows us to maximize efforts to unlock the full potential of the ImmTOR immune tolerance platform, by optimizing the efficacy and safety of biologics, enabling re-dosing of life saving gene therapies, and creating novel immunotherapies for autoimmune diseases," said Carsten Brunn, Ph.D., President and CEO of Selecta. "We remain committed to the development of SEL-212, and look forward to the initiation of the Phase 3 clinical program with Sobi in the third quarter of this year and the topline data readout from the Phase 2 COMPARE trial, also in the third quarter. We also look forward to advancing our gene therapy program in MMA to the clinic in the first half of 2021, and for the submission of an IND for our autoimmune diseases program, also in 2021."

Recent Highlights and Anticipated Upcoming Milestones:

Closed Strategic Licensing Agreement with Sobi for SEL-212, the Company’s Phase 3-ready Novel Treatment for Chronic Refractory Gout: The Company announced the closing of a strategic licensing agreement with Sobi for SEL-212, the Company’s lead product candidate

Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTOR, today reported financial results for the second quarter ended June 30, 2020 and provided corporate updates.
"This is a transformational time for Selecta, as we reinforce our position as a leader in immune tolerance. The strategic licensing agreement with Sobi puts us in a financial position that allows us to maximize efforts to unlock the full potential of the ImmTOR immune tolerance platform, by optimizing the efficacy and safety of biologics, enabling re-dosing of life saving gene therapies, and creating novel immunotherapies for autoimmune diseases," said Carsten Brunn, Ph.D., President and CEO of Selecta. "We remain committed to the development of SEL-212, and look forward to the initiation of the Phase 3 clinical program with Sobi in the third quarter of this year and the topline data readout from the Phase 2 COMPARE trial, also in the third quarter. We also look forward to advancing our gene therapy program in MMA to the clinic in the first half of 2021, and for the submission of an IND for our autoimmune diseases program, also in 2021."

Recent Highlights and Anticipated Upcoming Milestones:

Closed Strategic Licensing Agreement with Sobi for SEL-212, the Company’s Phase 3-ready Novel Treatment for Chronic Refractory Gout: The Company announced the closing of a strategic licensing agreement with Sobi for SEL-212, the Company’s lead product candidate

leveraging the ImmTOR immune tolerance platform. Sobi assumes responsibility for all development, regulatory, and commercial activities, and expenses in all markets outside China, while Selecta will run the Phase 3 study on behalf of Sobi, at Sobi’s expense. Selecta will also maintain manufacturing of ImmTOR, for which Sobi will reimburse Selecta for activities relating to SEL-212. The initial payments to Selecta total $100 million. Sobi must pay $75 million in cash as an upfront license fee within 45 days of the effective date and has paid $25 million in cash in a private placement of Selecta common stock at $4.62 per share. Selecta is also eligible to receive potential development, regulatory, and commercial milestone payments of up to $630 million, and tiered double-digit royalties on net sales.

Entered into Research License and Option Agreement with Sarepta for the Use of ImmTOR in Certain Neuromuscular Diseases: Selecta and Sarepta reached an agreement which provides Sarepta with the option to license the rights to develop and commercialize the ImmTOR platform for use in select neuromuscular diseases; Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMDs). Sarepta will evaluate its investigational gene therapies in combination with ImmTOR to mitigate the formation of neutralizing antibodies. Sarepta has made an initial payment to Selecta, and Selecta is eligible to receive certain pre-clinical milestone feesTwo Key Clinical Milestones for SEL-212: Selecta and Sobi anticipate initiating the Phase 3 clinical program in the third quarter of 2020. Selecta will take the lead running the Phase 3 program, at Sobi’s expense. The Phase 3 clinical program will consist of two double blinded, placebo-controlled trials of SEL-212. Each trial is expected to enroll 105 patients and have 35 patients receiving 0.1 mg/kg of ImmTOR and 0.2 mg/kg of pegadricase, 35 patients receiving 0.15 mg/kg of ImmTOR and 0.2 mg/kg of pegadricase, and 35 patients receiving placebo. The primary endpoint of the Phase 3 clinical trials is the maintenance of serum uric acid (SUA) levels of <6mg/dL at six months. One of the trials will have a six-month extension. In addition, the Company expects topline results from the Phase 2 COMPARE clinical trial in Q3 2020. This head-to-head study of a once-monthly dose of SEL-212 (ImmTOR + pegadricase) compared to biweekly doses of pegloticase is expected to read out on schedule. The primary endpoint of the COMPARE trial is the maintenance of serum uric acid (SUA) levels of <6mg/dL at three and six months.

Timeline for Gene Therapy and Autoimmune Diseases Programs Confirmed: The Company’s gene therapy program in methylmalonic acidemia, or MMA, in collaboration with AskBio, is expected to enter the clinic in the first half of 2021, with preliminary data expected in 2H 2021. In addition, Selecta intends to submit its Investigational New Drug Application (IND) for its autoimmune disease program in 2021. The first indication will be IgA nephropathy, a kidney disease that occurs when an antibody called immunoglobulin A (IgA) accumulates in the kidneys. The second indication is expected to be primary biliary cholangitis, or PBC, an autoimmune disease that causes progressive destruction of the bile ducts. Both diseases have well-defined target antigens, significant unmet medical need, and are well suited to the application of Selecta’s ImmTOR immune tolerance platform.

Appointed Peter G. Traber, MD, to the Position of Chief Medical Officer: Dr. Traber, who has been serving in the same position in an interim capacity, joined the Company full-time as of August 1, 2020. Dr. Traber has a broad range of experience in large pharma, biotech, and academia, and will oversee medical affairs, program management, and all aspects of clinical

development and strategy, as well as provide scientific and clinical guidance for potential business development initiatives.

Second Quarter 2020 Financial Results:

Cash Position: Selecta had $61.4 million in cash, cash equivalents, and restricted cash as of June 30, 2020, which compares to cash, cash equivalents, and restricted cash of $91.6 million as of December 31, 2019. Selecta believes its available cash, cash equivalents, and restricted cash as of June 30, 2020, together with the $25 million payment received from Sobi under the Sobi Private Placement in July and the expected payment from Sobi of $75 million under the Sobi License, which is due 45 days after the effective date, will enable Selecta to fund operating expenses and capital expenditure requirements into the first quarter of 2023.

Net cash used in operating activities was $23.5 million for the six months ended June 30, 2020, as compared to $27.4 million for the same period in 2019.

Research and Development Expenses: Research and development expenses for the second quarter 2020 were $10.7 million, which compares with $12.1 million for the same period in 2019. The decrease in costs was primarily the result of reduced expense for the Phase 2 COMPARE trial for SEL-212 offset by increases for the gene therapy program in collaboration with AskBio, and salaries and benefits.

General and Administrative Expenses: General and administrative expenses for the second quarter 2020 were $5.6 million, which compares with $4.1 million for the same period in 2019. The increase in costs was the result of expenses incurred for salaries, legal and professional fees offset by decreased travel expense.

Net Loss: For the second quarter 2020, Selecta reported a net loss of $24.1 million, or $0.25 per share, compared to a net loss of $16.4 million, or $0.37 per share, for the same period in 2019.

Conference Call and Webcast Reminder:
Selecta management will host a conference call at 8:30 a.m. ET today to provide a corporate update and review the company’s second quarter 2020 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10138605. Investors and the public can access the live and archived webcast of this call via the Investors & Media section of the company’s website, www.selectabio.com.