On May 6, 2020 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the first quarter 2020 and reviewed recent business highlights (Press release, Alnylam, MAY 6, 2020, View Source [SID1234557089]).

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"The first quarter of 2020 was an unprecedented period, as the world confronted the beginnings of an ongoing public health crisis with the COVID-19 pandemic. It’s also a moment in time that underscores how particularly proud we are at Alnylam to be part of an industry advancing science and innovation to address the coronavirus crisis at hand, while also maintaining our focus on continuing to bring important medicines to patients in need. To that end, we are very pleased with our first full quarter as a multi-product, global commercial company where we delivered continued and steady worldwide growth of ONPATTRO and impressive early demand for GIVLAARI in the U.S.," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "In addition, we also continued to drive progress across our late-stage pipeline, including submission of NDA and MAA filings for lumasiran, completion of enrollment in the ILLUMINATE-B and HELIOS-A Phase 3 studies of lumasiran and vutrisiran, respectively, and continued enrollment in our APOLLO-B and HELIOS-B Phase 3 studies of patisiran and vutrisiran, respectively. We’re also thrilled to highlight today the achievement of initial human proof of concept for ALN-AGT in development for the treatment of hypertension, an area of prevalent unmet need that cries out for innovation."

Dr. Maraganore continued: "Notwithstanding these accomplishments and our strong underlying confidence for Alnylam in 2020 and beyond, the trajectory of the COVID-19 pandemic is dynamic and remains uncertain in its scope, timeline, and impact to healthcare systems. While we are implementing what we believe are appropriate mitigation steps across our operations, we are lowering our 2020 revenue guidance for ONPATTRO by 5 percent and taking thoughtful steps to reduce our expense profile. Notwithstanding the challenges and uncertainties caused by the pandemic, we remain confident that we are on track to exceed our Alnylam 2020 strategic plan announced in 2015, building a multi-product, global biopharma company with a deep clinical pipeline to fuel future growth and a robust, organic product engine for sustainable innovation, a profile rarely achieved in our industry."

First Quarter 2020 and Recent Significant Corporate Highlights

Commercial Performance

ONPATTRO

Achieved net product revenues for the first quarter of 2020 of $66.7 million.
Attained over 950 patients worldwide on commercial ONPATTRO treatment as of March 31, 2020.
Received marketing authorization approval for ONPATTRO in Brazil for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy, with patients now on commercial product.
Continued progress with market access efforts across the Canada, Europe, Middle East, and Africa (CEMEA) region, with recent launches in Italy, Sweden, Israel, Turkey, and Spain.
Maintained momentum in Asia with a strong launch in Japan and submission of a New Drug Application (NDA) in Taiwan.
In the U.S., Alnylam has now completed definitive value-based agreements (VBAs) covering ONPATTRO with over 15 commercial payers, including 9 of the top 10 commercial payers, with signed VBAs now covering over 135 million U.S. lives in the aggregate.
GIVLAARI

Achieved net product revenues for the first quarter of 2020 of $5.3 million, representing strong initial demand in the first full quarter of the GIVLAARI launch in the U.S.
Received over 60 Start Forms in the U.S. and attained over 50 patients on commercial GIVLAARI treatment from launch through March 31, 2020.
Made significant progress toward establishing VBAs, including a Prevalence-Based Adjustment feature, with multiple ongoing discussions with U.S. payers; the Company announces today finalization of the first VBA for GIVLAARI.
Continued progress with market access efforts across the CEMEA region, with initial launch underway in Germany.
Continued work with physicians in multiple regions to provide pre-approval access to GIVLAARI in an Expanded Access Program (EAP) in accordance with local requirements.
R&D Highlights

Advanced the development of 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.
The Company announces today that due to the impact of the COVID-19 pandemic, enrollment delays in APOLLO-B will result in a shift in the enrollment completion date from late 2020 into 2021.
Received approval of GIVLAARI (givosiran) in the European Union for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older, with patients now on commercial product.
Advanced lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1).
Initiated and completed the rolling submission of an NDA to the Food and Drug Administration (FDA) and submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA), completing the filings in less than four months after announcing topline results from the ILLUMINATE-A Phase 3 study.
Completed enrollment in the ILLUMINATE-B Phase 3 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.
Completed enrollment in the HELIOS-A Phase 3 study in patients with hereditary ATTR amyloidosis with polyneuropathy, and remains 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.
Continued enrollment in the HELIOS-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy.
Inclisiran, potentially the first and only siRNA cholesterol-lowering treatment, continues to advance under Alnylam’s partner, Novartis.
Acceptance of NDA and MAA by FDA and EMA, respectively.
Findings published in The New England Journal of Medicine from the completed ORION Phase 3 pivotal trials showing durable and potent LDL-C reduction achieved with inclisiran, with a safety profile similar to placebo.
New analysis of pooled data from the completed ORION Phase 3 clinical trials presented at the American College of Cardiology’s Annual Scientific Session.
Alnylam’s partner, Sanofi, continues 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.
Advanced early- and mid-stage RNAi therapeutic pipeline programs.
The Company announces today positive initial topline results from the ongoing Phase 1 study (N=48) of ALN-AGT in hypertension, providing initial human proof of concept with over 90 percent mean knockdown of angiotensinogen (AGT) and an over 10 mmHg reduction of mean 24-hour systolic blood pressure at week 8 relative to placebo, with a durability that supports once quarterly or less frequent dose administration. In addition, ALN-AGT administration was associated with an encouraging safety and tolerability profile including no drug-related serious adverse events (SAEs).
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.
Under expanded collaboration with Vir, selected a Development Candidate (DC), ALN-COV (VIR-2703), for SARS-CoV-2 – the virus that causes COVID-19 – with potent and highly cross-reactive activity, with a plan for accelerated filing of an IND at or around year-end 2020.
The Company announces today that it will shift filing of its ALN-LEC IND into 2021 to focus on its COVID-19 investigational RNAi therapeutic program.
Additional Business Updates

Entered into a broad 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.
Expanded collaboration with Vir to include the development and commercialization of RNAi therapeutics targeting SARS-CoV-2 and up to three additional targets focused on host factors 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.
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.
Appointed Dr. Olivier Brandicourt, former CEO of Sanofi, to the Company’s Board of Directors, and announced the retirement of Dr. Paul Schimmel, an Alnylam co-founder, in May 2020 after nearly 18 years of service; Dr. Schimmel will remain a member of the Company’s Scientific Advisory Board.
Upcoming Events

In mid-2020, Alnylam intends to:

Continue global commercialization of ONPATTRO and GIVLAARI.
Present full results from the ILLUMINATE-A Phase 3 study of lumasiran at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) Congress, currently scheduled for June 7, 2020 as a virtual event.
Report topline results from the ILLUMINATE-B Phase 3 study of lumasiran.
File a Clinical Trial Application (CTA) for ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 for the treatment of non-alcoholic steatohepatitis (NASH), in collaboration with Regeneron.
Financial Results for the Quarter Ended March 31, 2020

"We believe our results for the first quarter demonstrate the strength of our commercial teams in challenging circumstances, delivering solid quarterly growth for ONPATTRO and robust uptake for GIVLAARI in the U.S. in its first full quarter of launch. While we have confidence in our business continuity plans, we expect an impact from the COVID-19 pandemic and are lowering our 2020 revenue guidance range for ONPATTRO by 5 percent from $285-$315 million to $270-$300 million. In parallel, we are implementing further discipline in our operations to moderate our spend and are lowering our operating expense guidance range for the year," said Jeff Poulton, Chief Financial Officer of Alnylam. "Of course, a business highlight for the recent period was the completion of our $2 billion strategic financing collaboration with Blackstone, the largest ever financing of a pre-profitable biotechnology company. We believe this new financing, together with our expectations for continued topline growth and disciplined expense management, bridges our transition toward a self-sustainable financial profile without the need for future equity offerings. In sum, we believe Alnylam is taking appropriate measures to operate through the pandemic and recovery phases in 2020, into the ‘new normal’ and beyond, continuing to deliver on the promise of RNAi therapeutics and our commitments to patients and our shareholders."

The $2 billion strategic financing collaboration with Blackstone is expected to enable Alnylam’s achievement of a self-sustainable financial profile without need for future equity financings.

*Excludes $155 million – $175 million of stock-based compensation from estimated GAAP R&D and SG&A expenses.

Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies.

The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in the press release are stock-based compensation expenses and unrealized gain on marketable equity securities. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impact the fair value of these awards. The Company has excluded the impact of the unrealized gain on marketable equity securities because the Company believes this item is a one-time event occurring outside the ordinary course of the Company’s business.

The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release.

Conference Call Information
Management will provide an update on the Company and discuss first quarter 2020 results as well as expectations for the future via conference call on Wednesday, May 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%).

Indication
ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

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) for the treatment of adults and adolescents with acute hepatic porphyria (AHP). 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 Technology
Alnylam 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 May 6, 2020 Applied DNA Sciences Inc. (NASDAQ: APDN) ("Applied DNA" or the "Company") a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing that enables diagnostics, pre-clinical nucleic acid-based therapeutic drug candidates, supply chain security, anti-counterfeiting and anti-theft technology, reported that it has received approximately $5.3 million in net proceeds calendar year to date as a result of the exercise of equity warrants (Press release, Applied DNA Sciences, MAY 6, 2020, View Source [SID1234557088]).

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Applied DNA issued approximately 2.6 million warrants in connection with an underwritten public offering that closed on November 15, 2019. Each warrant entitles its owner to purchase one share of common stock in the Company at a per share price of $5.25 until November 15, 2024. Approximately 1.5 million warrants associated with the November 15, 2019 offering remain outstanding.

On May 8, 2020 EyePoint Pharmaceuticals, Inc. (NASDAQ: EYPT), a pharmaceutical company committed to developing and commercializing innovative ophthalmic products, reported financial results for the first quarter ended March 31, 2020 and highlighted recent corporate developments (Press release, pSivida, MAY 6, 2020, View Source [SID1234557087]).

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"We are pleased with product revenue performance in the first quarter, despite the negative impact on customer demand caused by COVID-19 pandemic-related closures of customer facilities beginning in March. We are encouraged that certain regions across the country are now starting to reopen for business, allowing us to begin resupplying physicians and ambulatory surgery centers with our innovative products," said Nancy Lurker, President and Chief Executive Officer of EyePoint Pharmaceuticals. "We believe that both YUTIQ and DEXYCU are well-positioned to support physicians and patients in this COVID-19 era, as both products deliver extended duration therapeutic treatment from a single injection, which may reduce the frequency of in-person follow-up visits and contact with the patient’s face and eyes."

Ms. Lurker continued, "We remain committed to our mission of delivering innovative ophthalmic products to patients in need and continue to advance our lead development asset EYP-1901 toward clinical trials. EYP-1901 is an anti-VEGF, tyrosine kinase inhibitor (TKI) six-month sustained release potential therapy using our bioerodible Durasert technology initially targeting wet age-related macular degeneration. Good laboratory practice (GLP) toxicology studies were initiated in March and we remain on schedule to file an Investigational New Drug (IND) application later this year with a Phase 1 clinical trial to follow."

Commercial Performance in First Quarter 2020

Customer demand trended strong for both products during the quarter prior to the emergence of the COVID-19 pandemic in the U.S. causing demand deterioration beginning in March.

During the quarter, public health authorities and government agencies including the Centers for Medicare & Medicaid Services (CMS), recommended the postponement of all

non-essential elective surgeries, including cataract surgery, for an extended period of time during the COVID-19 pandemic. As a result, ambulatory surgery centers (ASCs) closed or limited operations, decreasing DEXYCU product demand and orders. Our sales organization has maintained contact with customers during the pandemic by providing virtual support and education with regard to DEXYCU.

Uveitis and retinal specialist office visits continued to be conducted for YUTIQ, though at reduced frequency, as chronic non-infectious uveitis affecting the posterior segment of the eye can lead to blindness if left untreated.

There have been no disruptions to the supply chains for YUTIQ and DEXYCU and the Company continues to produce finished product for commercial sale.

In April, the Company announced a reorganization of its commercial operations including cancellation or deferral of planned spending to conserve cash due to the COVID-19 pandemic impact on expected revenue. This reorganization was primarily focused on a reduction in the external contract sales organization for DEXYCU. The Company plans to allocate its remaining DEXYCU commercial resources to high-volume ASCs in key U.S. regions.

The Company expects product demand to continue at current decreased levels until COVID-19 related restrictions on elective surgeries and office visits are lifted.

R&D Highlights

In March, the Company initiated GLP toxicology studies for EYP-1901, an anti-VEGF, TKI six-month sustained release product candidate using our bioerodible Durasert technology. EYP-1901 is being developed as a potential treatment for wet age-related macular degeneration, with the potential for future indications in diabetic retinopathy and retinal vein occlusion, all of which are diseases representing attractive market opportunities in need of long-lasting treatments to improve treatment compliance. The Company expects to file an IND with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2020 with a Phase 1 clinical trial to commence shortly thereafter.

Operations Update

In April, the Company received a $2 million loan through the Small Business Administration’s Paycheck Protection Program (PPP) under the Coronavirus Aid, Relief and Economic Security Act of 2020 (the CARES Act). The PPP loan will enable the Company to retain key commercial infrastructure and employees and avoid furloughs as product demand and revenues remain significantly reduced due to ASC and physician office closures necessitated by the COVID-19 pandemic. The Company plans to use the proceeds of the PPP loan to cover payroll costs, rent and utilities in accordance with the CARES Act.

The reorganization announced in April is expected to result in annual savings of approximately $7 million and one-time savings of approximately $10 million from other planned expenditure cancellations and deferrals.

Review of First Quarter Results Ended March 31, 2020

For the three months ended March 31, 2020, total net revenue was $7.5 million compared to $2.0 million for the three months ended March 31, 2019. Net product revenue for the three months

ended March 31, 2020 was $4.7 million, with $3.6 million for YUTIQ and $1.1 million for DEXYCU, compared to net revenue for three months ended March 31, 2019 of $1.2 million, with $543,000 for YUTIQ and $684,000 for DEXYCU.

Net revenue from royalties and collaborations for the three months ended March 31, 2020 totaled $2.8 million compared to $785,000 in the corresponding quarter in 2019.

Operating expenses for the three months ended March 31, 2020 increased to $18.9 million from $16.7 million in the prior year period, due primarily to increased sales and marketing costs and research and development costs. Non-operating expense, net, for the three months ended March 31, 2020 totaled $1.7 million of net interest expense. Net loss for the three months ended March 31, 2020 was $13.2 million, or $0.11 per share, compared to a net loss of $19.2 million, or $0.20 per share, for the prior year quarter.

Cash and cash equivalents at March 31, 2020 totaled $26.3 million compared to $22.2 million at December 31, 2019.

Financial Outlook

We expect that the Company’s cash and cash equivalents combined with projected cash inflows from anticipated YUTIQ and DEXYCU product sales can fund the Company’s operating plan into 2021 under current assumptions for the duration of the COVID-19-related closures across the U.S.

The Company continues to assess additional cash conservation measures to support its operation through the COVID-19 pandemic.

Conference Call Information

EyePoint will host a conference call today, Wednesday, May 6, 2020, at 8:30 AM ET to discuss the results for the first quarter ended March 31 and recent operational developments. To access the conference call, please dial (877) 312-7507 from the U.S. and Canada or (631) 813-4828 (international) at least 10 minutes prior to the start time and refer to conference ID 7972168. A live webcast will be available on the Investor Relations section of the corporate website at View Source A replay of the webcast will also be available on the corporate website.

On May 6, 2020 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported financial results for the first quarter ended March 31, 2020 (Press release, Compugen, MAY 6, 2020, View Source [SID1234557086]).

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"We have entered 2020 well positioned and with significant momentum to advance our clinical programs that address novel, internally-discovered, drug targets for cancer immunotherapy," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "On the clinical front, we recently presented at AACR (Free AACR Whitepaper) updated data from our ongoing Phase 1 dose escalation study of COM701 as a monotherapy and in combination with Opdivo. These encouraging data, which include signals of durable disease control and confirmed partial responses both in the monotherapy and combination arms, further confirm our hypothesis that by blocking PVRIG, an important new checkpoint, COM701 has the potential to broaden the patient population that can benefit from cancer immunotherapy. We also dosed the first patient in the Phase 1 study for COM902, our anti-TIGIT therapeutic antibody, which would enable us to clinically evaluate the dual blockade of PVRIG and TIGIT inhibitory pathways in the DNAM axis."

Dr. Cohen-Dayag added, "Our continued progress and execution enabled us to successfully raise approximately $79 million through an underwritten public offering, which we believe is testament to the growing recognition of our science-driven approach backed by our computational discovery capabilities. With our strengthened balance sheet, we are well positioned to continue our strategic clinical development plans which will be expanded to also include a third clinical trial, a Phase 1/2 triple combination study testing COM701 with Bristol-Myers Squibb’s Opdivo and their investigational TIGIT inhibitor in the second half of this year, as well as advance our early-stage programs to propel our future therapeutic pipeline. We are fortunate that, to date, the COVID-19 pandemic has not significantly impacted our operations, including our clinical development timelines. For now, we are not changing the anticipated data readouts and milestones timelines we previously provided. We are grateful to our employees and clinical investigators for their continued hard work and dedication. In these unique circumstances we remain focused on advancing our clinical and early-stage programs to maintain positive momentum to achieve our goals."

First Quarter 2020 and Recent Highlights

Presented updated data from the dose escalation arms of the Phase 1 trial of COM701 in patients with advanced solid tumors at The 2020 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting I

COM701 was well-tolerated through 20 mg/kg IV Q4 weeks as a monotherapy and 10 mg/kg IV Q4 weeks in combination with Opdivo (480 mg IV Q4 weeks) with no dose-limiting toxicities reported.

No increased toxicity was observed in the combination arm.

No patients discontinued treatment due to toxicity of any study drug.

Preliminary COM701 pharmacokinetic data supports IV Q4 weeks dosing, allowing dosing schedule aligned with Opdivo.

Encouraging disease control rates of 69% (11/16) for monotherapy and 75% (9/12) for the combination arm.

50% of patients (6/12) in the combination arm remain on study, some with continued responses observed beyond 200 days of treatment.

Durable responses of stable disease for over six months in six of 28 patients (21%) across treatment arms.

The two patients previously reported with confirmed partial responses, one from the monotherapy arm (microsatellite stable primary peritoneal cancer) and one from the combination arm (microsatellite stable colorectal cancer), remain on treatment.

Enrollment in the COM701 monotherapy dose escalation arm is completed and enrollment in the combination dose escalation arm at 20 mg/kg IV Q4 weeks is ongoing. Enrollment in the monotherapy expansion cohorts is expected to begin in Q2 2020.

Dosed the first patient in a Phase 1 dose escalation clinical trial of COM902, an immuno-oncology therapeutic antibody targeting TIGIT, in patients with advanced malignancies.

Announced plans to expand the Bristol-Myers Squibb collaboration with a Phase 1/2 triple combination study to evaluate COM701 in combination with Opdivo and BMS-986207, Bristol-Myers Squibb’s investigational TIGIT inhibitor. This study is expected to begin in 2H 2020.

Strengthened intellectual property portfolio

Granted EPO Patent No. 3295951, covering the composition of matter for COM701 and backup antibodies including any anti-PVRIG antibody having the binding fragments of COM701 or backup antibodies for the treatment of cancer.

Granted U.S. Patent No. 10,550,173, covering methods of screening for anti-PVRIG antibodies that inhibit the binding of PVRIG with PVRL2.

Granted EPO Patent No. 3258951, covering the use of any anti-PVRIG antibody that activates T cells and/or NK cells, in the treatment of cancer.

Published a peer-reviewed paper in Cancer Immunology Research in collaboration with Bayer demonstrating in vitro T cell activation and in vivo anti-tumor activity of BAY 1905254, a first-in-class immuno-oncology antibody targeting ILDR2. ILDR2 is a novel immune checkpoint discovered computationally by Compugen which is currently being evaluated by Bayer in a Phase 1 study as monotherapy and in combination with Keytruda.

Completed an underwritten public offering of 8,816,339 ordinary shares (including the shares issued in Q2 upon exercise of the underwriters’ option) at $9.00 per share. The net proceeds from the offering were approximately $74 million, after deducting underwriting discounts and commissions and other offering expenses that were paid by the Company.

Financial Results
Research and development expenses for the first quarter ended March 31, 2020 were $4.7 million, compared with $6.3 million in the comparable quarter in 2019. The decrease is attributed mostly to the restructuring process announced at the end of the first quarter of 2019 offset by an increase in expenses associated with clinical-related activities.

Net loss for the first quarter of 2020 was $7.1 million, or $0.10 per basic and diluted share, compared with a net loss of $8.4 million, or $0.14 per basic and diluted share, in the comparable quarter of 2019.

As of March 31, 2020, cash, cash related accounts and short-term and long-term bank deposits totaled approximately $121.2 million, compared with approximately $43.9 million as of December 31, 2019. The increase in cash balances during the first quarter is attributed to approximately $70 million of net proceeds from the underwritten public offering (excluding the shares issued upon exercise of the underwriters’ option in the second quarter), $5.2 million from the exercise of warrants and $7.2 million from the exercise of employee options, offset by operating expenses and working capital. The Company has no debt.

Conference Call and Webcast Information
The Company will hold a conference call today, May 6, 2020, at 8:30 AM ET to review its first quarter 2020 results. To access the conference call by telephone, please dial 1-888-407-2553 from the United States, or +972-3-918-0610 internationally. The call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.

On May 6, 2020 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2020, and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 6, 2020, View Source [SID1234557085]).

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"This is a transformational year for BioCryst as we prepare to launch berotralstat in multiple territories to bring our oral, once-daily prophylactic medicine to HAE patients, and begin generating significant revenue," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We are excited to see the clinical response in treatment-naïve PNH patents at the 50 mg and 100 mg twice-daily dose levels, and more than 98 percent suppression of complement in both alternative pathway assays at the 200 mg and 400 mg twice-daily levels in healthy volunteers," said Dr. William Sheridan, chief medical officer of BioCryst.

"This profile provides strong support for BCX9930 as an oral monotherapy. We look forward to studying 200 mg and 400 mg twice-daily in PNH patients and advancing this program to treat multiple complement-mediated diseases," Sheridan added.

Program Updates and Key Milestones

Hereditary Angioedema (HAE) Program – Berotralstat (BCX7353): Oral, once-daily treatment for prevention of HAE attacks

BioCryst expects three regulatory approvals for berotralstat in 2020 and early 2021. These timelines remain on track.

The U.S. Food and Drug Administration (FDA) is currently reviewing a new drug application for berotralstat and has set an action date of December 3, 2020, under the Prescription Drug User Fee Act (PDUFA).

In Japan, the Pharmaceuticals and Medical Devices Agency (PMDA) is reviewing a new drug application (JNDA) for berotralstat under the Sakigake timeline, and the company expects approval in Japan in the second half of 2020.

On March 30, 2020, the company announced that the European Medicines Agency (EMA) had validated its marketing authorization application (MAA) submission for berotralstat and begun their formal review of the MAA under the centralized procedure. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected within approximately 12 months from MAA validation.

Ongoing commercial launch preparations are on track in the U.S., EU and Japan. The company does not expect delays due to COVID-19.

On May 5, 2020, the company announced that the United States Patent and Trademark Office issued a notice of allowance for a new composition of matter patent which extends patent protection for berotralstat in the U.S. market by four years through October 2039.
Complement Oral Factor D Inhibitor Program – BCX9930

Low dose cohort (50 mg and 100 mg twice-daily) data in three treatment-naïve paroxysmal nocturnal hemoglobinuria (PNH) patients who completed 28 days of therapy shows BCX9930 inhibited complement and was safe and generally well tolerated.

Patients were severely ill with pre-treatment LDH from 3.7 to 11× the upper limit of normal (ULN) and low hemoglobin of 6.0 to 8.2 g/dL.

All patients had dose-dependent reductions in LDH and increases in hemoglobin.

No drug-related serious adverse events were observed.

No PNH patients experienced rash.

Based on the investigators’ assessment of clinical benefit, all three patients continued on therapy with BCX9930 (100 mg twice-daily) following the 28-day study window.

With the recent enrollment of a fourth patient with PNH, enrollment is now complete in treatment-naïve cohort 1 (50 mg and 100 mg twice-daily). Treatment-naïve cohort 2 (200 mg and 400 mg twice-daily) is expected to begin enrollment upon completion of cohort 1, with data expected in Q3 2020.

The company plans to begin enrolling PNH patients resistant to C5 inhibitors in Q3 2020 and expects to report data from these treatment-resistant patients by the end of 2020.

Data from the 200 mg and 400 mg twice-daily multiple ascending dose (MAD) cohorts in healthy volunteers shows >98 percent suppression of the alternative pathway beyond 12 hours and no dose-limiting adverse events.
Given these data, the company expects to achieve its goal of monotherapy for PNH patients in cohort 2 (200 mg and 400 mg twice-daily).

Additional details can be found on slides, which can be accessed at the Investors’ section of BioCryst’s website at http://www.biocryst.com.

Coronavirus Antiviral Program – Galidesivir (BCX4430)

Patient dosing is underway in Brazil in a randomized, double-blind, placebo-controlled clinical trial to assess the safety, clinical impact and antiviral effects of galidesivir in patients with COVID-19. The trial (NCT03891420) is being funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.

Part 1 of the trial is enrolling 24 hospitalized adults diagnosed with moderate to severe COVID-19 confirmed by PCR. Three cohorts of eight patients will be randomized to receive intravenous (IV) galidesivir (n=6) or placebo (n=2) every 12 hours for seven days. Upon completion of part 1 of the trial, an optimized dosing regimen of galidesivir will be selected for part 2 of the trial, based on part 1 results. In part 2 of the trial, up to 42 hospitalized patients with COVID-19 will be randomized 2:1 to receive IV galidesivir or placebo.

In vitro testing of galidesivir against SARS-CoV-2, the virus that causes COVID-19, is underway. Galidesivir has been shown to be active against more than 20 RNA viruses in nine different families, including coronaviruses.

The company also is working closely with the government to increase the supply of galidesivir.
Additional Updates

On April 2, 2020, the company announced the appointment of Anthony Doyle as senior vice president and chief financial officer.

The company remains on track to report data in 2H 2020 from its ongoing Phase 1 clinical trial of BCX9250, an oral ALK-2 kinase inhibitor for treatment of fibrodysplasia ossificans progressiva (FOP), in healthy subjects.
First Quarter 2020 Financial Results

For the three months ended March 31, 2020, total revenues were $4.8 million, compared to $5.9 million in the first quarter of 2019. The decrease was primarily due to reduced peramivir product sales and lower royalty revenues, partially offset by amortization of deferred revenue from the Torii Pharmaceutical, Co. commercialization agreement.

Research and development (R&D) expenses for the first quarter of 2020 increased to $29.9 million from $27.5 million in the first quarter of 2019, primarily due to due to increased spending on the company’s complement-mediated diseases program and other preclinical development initiatives.

Selling, general and administrative (SG&A) expenses for the first quarter of 2020 increased to $15.9 million, compared to $6.2 million in the first quarter of 2019. The increase was primarily due to increased spending on commercial activities and medical affairs to support the U.S. commercial launch of berotralstat in 2020 and contingent legal costs associated with our Seqirus UK Limited (Seqirus) dispute.

Interest and other income were $6.4 million in the first quarter of 2020, compared to $0.6 million in the first quarter of 2019. The increase was primarily due to the partial arbitration award related to our Seqirus dispute.

Interest expense was $3.0 million in the first quarter of 2020, compared to $2.7 million in the first quarter of 2019 and was associated with an increase in the outstanding balance of the company’s secured credit facility in February 2019 and increased interest expense associated with the company’s non-recourse notes payable.

Net loss for the first quarter of 2020 was $37.6 million, or $0.24 per share, compared to a net loss of $31.1 million, or $0.28 per share, for the first quarter of 2019.

Cash, cash equivalents and investments totaled $114.6 million at March 31, 2020, and reflect a decrease from $137.8 million at December 31, 2019. Operating cash use for the first quarter of 2020 was $23.1 million.

Financial Outlook for 2020

BioCryst expects full year 2020 net operating cash use to be in the range of $125 to $150 million, and its operating expenses to be in the range of $135 to $160 million. The company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the company’s stock, as well as by the vesting of the company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 4679821. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 4679821.