On August 5, 2021 Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, reported that corporate and financial update for the second quarter ended June 30, 2021 (Press release, Athenex, AUG 5, 2021, View Source [SID1234585793]).

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"During the second quarter, we held a Type A meeting with the FDA to discuss the path forward for oral paclitaxel and encequidar in metastatic breast cancer and the deficiencies that were raised in the Complete Response Letter (CRL) we received in February," said Johnson Lau, Chief Executive Officer of Athenex. "The FDA has indicated that a well-designed and well-conducted trial may adequately address these deficiencies. We are now evaluating the optimal design for a new clinical study which we plan to present to the FDA in the fourth quarter of 2021. We are continuing to advance oral paclitaxel in angiosarcoma towards registration."

"Our acquisition of Kuur Therapeutics and its innovative allogeneic CAR-NKT technology was a strategically important development for Athenex and we believe it has the potential to unlock tremendous value," continued Dr. Lau. "Kuur’s technology, combined with our TCR program, could propel us into a leadership position in cell therapy. We are pleased with the smooth integration of the Kuur team and operations to date and look forward to providing further updates as the programs advance."

Second Quarter 2021 and Recent Business Highlights

Clinical Programs

Oral Paclitaxel and Encequidar

Athenex held a Type A meeting with the US Food & Drug Administration (FDA) regarding the New Drug Application (NDA) for oral paclitaxel in metastatic breast cancer during second quarter 2021. The Company is evaluating the optimal design for a new clinical study which it plans to present to the FDA in 4Q 2021.
An abstract "Phase 1 Study with Expansion Cohorts to Assess the Safety, Tolerability, and Activity of oral paclitaxel + encequidar in Combination with Pembrolizumab in Subjects with Advanced Solid Malignancies" has been accepted for e-Poster presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021.
Cell Therapy

Presented data from a Phase 1 study (GINAKIT2) of KUR-501 in neuroblastoma at the annual meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2021. The data showed one complete response and one partial response out of 11 evaluable and heavily pre-treated pediatric neuroblastoma patients. The therapy was well tolerated with only one Grade 2 CRS.
Continued to enroll patients in a Phase 1 study (ANCHOR) evaluating KUR-502 in patients with relapsed/refractory CD19 positive malignancies including B cell lymphomas, acute lymphoblastic leukemia (ALL), and chronic lymphocytic leukemia (CLL).
Received IRB approval to conduct a Phase 1 trial of TCRT-ESO-A2 (high-affinity TCR-T targeting NY-ESO-1 positive solid tumors).
Commercial Update

Klisyri (tirbanibulin)

Almirall, S.A. (BLM: ALM), Athenex’s partner, received marketing authorization of Klisyri from the European Commission in July 2021 for the topical treatment of actinic keratosis (AK) on the face or scalp in adults. The approval followed receipt of a positive opinion from the CHMP of the European Medicines Agency in May 2021.
Athenex entered into license agreements for tirbanibulin with Seqirus (a subsidiary of CSL Limited) and AVIR Pharma, expanding its international commercial partnerships for tirbanibulin in Australia and New Zealand, and Canada, respectively.
Specialty Pharmaceutical Business

Athenex Pharmaceutical Division (APD) currently markets a total of 34 products with 61 SKUs.
Athenex Pharma Solutions (APS) currently markets 4 products with 16 SKUs.
503B operations at the Dunkirk Facility expected to commence in Q4 2021.
Corporate Update

Athenex has accepted the resignation of its Chief Financial Officer, Randoll Sze, effective August 13, 2021. Mr. Steven Adams, the company’s current Corporate Controller, will take on the role of Interim Chief Accounting Officer until a permanent CFO is appointed.
Key Anticipated Milestones

Plan to discuss the design of a new clinical study for oral paclitaxel in metastatic breast cancer with the FDA in Q4 2021
Launch of Klisyri in a major European market in 2H 2021
Abstract on the ANCHOR Phase 1 study submitted to ASH (Free ASH Whitepaper) 2021
Phase 1 trial to evaluate TCRT-ESO-A2 (high-affinity TCR-T targeting NY-ESO-1 solid tumors) to initiate enrollment in 3Q 2021
Results from the I-SPY 2 trial of oral paclitaxel plus anti PD-1 expected in 2022
Second Quarter 2021 Financial Highlights

Revenues from product sales decreased to $21.4 million for the three months ended June 30, 2021, from $40.2 million for the three months ended June 30, 2020, a decrease of $18.8 million or 47%. This decrease was attributable to a decline in APD product sales of $20.4 million primarily as the result of a decrease in demand for COVID-19 related drugs from the prior year, including some significant non-recurring orders of approximately $14.1 million. In addition, in the first half of 2021, we experienced significant COVID-related challenges in our Indian supply chain and to a lesser extent in China. As a result, we were not able to receive some inventory from our partners located in these regions for a certain period of time. The Company also experienced a higher amount of product sales in 2020 as we started fulfilling demand for certain drugs used to treat patients hospitalized with COVID and demand for FDA shortage products. As a result, the product revenues in the second quarter of last year were particularly high, given the COVID pandemic had just started. Fluctuations in the infection rate and the spread of the global health pandemic and market demand may continue to significantly affect our product sales in the future. These were partially offset by an increase in 503B and contract manufacturing revenue of $1.5 million and $0.4 million, respectively.

License fees and other revenue increased by $0.5 million for the three months ended June 30, 2021. This increase was primarily due to $0.2 million grant revenue and $0.2 million in royalties received from Almirall for the sales of Klisyri after the product launch in the U.S. in February 2021.

Cost of sales for the three months ended June 30, 2021 totaled $19.7 million, a decrease of $13.3 million, or 40%, as compared to $33.0 million for the three months ended June 30, 2020. The decrease was primarily due to a decrease of $13.1 million in cost of APD product sales, generally in-line with the decrease in the product sales revenue. Additionally, cost of sales related to royalties for license income decreased by $1.2 million from the royalty payment incurred in 2020 on the license revenue from Xiangxue. Cost of 503B product sales increased by $1.3 million as production levels increased.

R&D expenses for the three months ended June 30, 2021 totaled $21.1 million, a decrease of $0.9 million, or 4%, as compared to $22.0 million for the three months ended June 30, 2020. This was primarily due to a decrease in regulatory costs, clinical operations, and preclinical operations. The decrease in these items was partially offset by increases in oral paclitaxel and encequidar API costs in preparation for product launch, together with increases in drug licensing costs, R&D related compensation expenses, and costs related to 503B and cell therapy development.

SG&A expenses for the three months ended June 30, 2021 totaled $21.2 million, an increase of $3.7 million, or 21%, as compared to $17.5 million for the three months ended June 30, 2020. This was primarily due to increase in professional fees and other expenses related to the acquisition of Kuur, increases in compensation related costs, change in fair value of contingent consideration and operating costs including insurance costs and IT costs. These increases were partially offset by a decrease in costs for preparing to commercialize Oral Paclitaxel as significant pre-launch activities occurred in 2020 and slowed upon receipt of the CRL in February 2021.

Interest expense totaled $5.7 million and $1.6 million for the three months ended June 30, 2021 and 2020, respectively. Interest expense in the current period was incurred from the Senior Credit Agreement with Oaktree, while interest expense in the prior period was primarily incurred from debt under a former credit agreement with Perceptive Advisors LLC and its affiliates.

Income tax benefit for the three months ended June 30, 2021 amounted to $11.0 million, compared to income tax expense of $0.1 million for the same period in 2020. The income tax benefit in the current year is primarily the result of taxable temporary difference due to the deferred tax liability recognized for the indefinite lived intangible assets acquired in connection with the acquisition of Kuur’s IPR&D. This taxable temporary difference is considered a source of taxable income to support the realization of deferred tax assets from the acquirer which resulted in a reversal of our valuation allowance.

Net loss attributable to Athenex for the three months ended June 30, 2021 was $34.3 million, or ($0.33) per diluted share, compared to a net loss of $40.5 million, or ($0.50) per diluted share, in the same period last year.

As of June 30, 2021, the Company had cash and cash equivalents of $76.9 million, restricted cash of $16.5 million, and short-term investments of $53.3 million.

For further details on the Company’s financial results, including the results for the six months ended June 30, 2021, refer to the Form 10Q to be filed with the SEC.

Financial Guidance

In terms of product sales guidance, the Company is limiting financial guidance to the existing Athenex product portfolio only. In 2020, the Company recorded a significant amount of revenues from international customers as a result of the global pandemic. However, it does not see these revenues as recurring in nature. The Company continues to expand its product portfolio. The Company is affirming the guidance it provided on May 6, 2021, as it currently expects its product sales in 2021, excluding any royalties from Klisyri, to be in line with 2020 levels.

Cash Conservation Update

As of June 30, 2021, the Company had cash and cash equivalents, restricted cash and short-term investments of $146.7 million. Given uncertainty stemming from the CRL for oral paclitaxel, the Company identified and adopted certain cash conservation measures. Considering these initial measures, and based on our current operating plan, the Company now expects that its cash and cash equivalents, restricted cash and short-term investments as of June 30, 2021, will enable it to meet its current operational liquidity needs and fund operations into 4Q 2022.

Conference Call and Webcast Information

Athenex will host a conference call and live audio webcast today, Thursday, August 5, 2021, at 8:00 am Eastern Time to discuss the financial results and provide a business update.

To participate in the call, dial either the domestic or international number fifteen minutes before the conference call begins:

The live conference call and replay can also be accessed by audio webcast here and also under "Events and Presentations" at the Investor Relations section of the Company’s website, located at View Source

On August 5, 2021 Arbutus Biopharma Corporation (Nasdaq: ABUS), a clinical-stage biopharmaceutical company primarily focused on discovering, developing and commercializing a cure for people with chronic hepatitis B virus (HBV) infection, as well as therapies to treat coronaviruses (including COVID-19), reported its second quarter 2021 financial results and provides a corporate update (Press release, Arbutus Biopharma, AUG 5, 2021, View Source [SID1234585792]).

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William Collier, President and Chief Executive Officer of Arbutus, stated, "We had a productive second quarter, particularly in advancing our efforts to position AB-729 as a potential cornerstone therapy in future HBV combination regimens. Our recently announced proof-of-concept clinical collaborations with Vaccitech plc and Antios Therapeutics, Inc. to evaluate AB-729 with other agents reflects this objective as does our planned Phase 2a clinical trial to evaluate AB-729 in combination with Peg-IFNα-2a."

Mr. Collier added, "Looking ahead, we expect a productive second half of 2021 including: additional data from the ongoing Phase 1a/1b clinical trial with AB-729, specifically 90 mg multi-dose data (dosing interval every 12 weeks) in HBV DNA negative subjects and 90 mg multi-dose data (dosing interval every 8 weeks) in HBV DNA positive subjects, initiation of two Phase 2a proof-of-concept clinical trials for AB-729, and initial Phase 1a/1b data from our proprietary oral capsid inhibitor, AB-836."

Pipeline Update

AB-729

Arbutus is currently conducting a single- and multi-dose Phase 1a/1b clinical trial to determine the safety, tolerability, pharmacokinetics, and pharmacodynamics of AB-729 in healthy subjects and in subjects with chronic HBV infection. The Company presented three posters and a late breaker oral presentation at the 2021 EASL conference highlighting the most recent data from this clinical trial. AB-729 continues to demonstrate robust mean HBsAg reduction across all doses and dosing intervals with a favorable safety and tolerability profile, followed by a sustained plateau phase:

The efficacy and safety data for AB-729 derived from up to one year of dosing support our view that 60 mg every 8 weeks is an appropriate dose to move forward in the upcoming Phase 2a clinical trials.

Additionally, based on 3/5 evaluable subjects, long term dosing with AB-729 demonstrated increased HBV-specific immune responses, providing support for combination therapy including immunomodulatory agents.

Arbutus expects to provide additional data from ongoing cohorts of the Phase 1a/1b clinical trial in the second half of 2021, including initial data for a 90 mg every 12 weeks cohort in HBV DNA negative subjects and initial data in a 90 mg every 8 weeks cohort in HBV DNA positive subjects.

In July 2021, Arbutus received authorization from the U.S. Food and Drug Administration to proceed with its Investigational New Drug (IND) application for AB-729 in a Phase 2 proof-of-concept clinical trial to evaluate AB-729 in combination with ongoing NA therapy and short courses of Peg-IFNα-2a in subjects with chronic HBV infection. This clinical trial is expected to initiate in the second half of 2021.

To further support AB-729 as a potential cornerstone therapeutic in future HBV combination regimens, Arbutus has entered into several clinical collaborations to evaluate AB-729 in combination with other agents:

Through a collaboration with Assembly Biosciences, Inc. ("Assembly"), subjects are being enrolled in a Phase 2 proof-of-concept clinical trial with a triple combination of AB-729, Assembly’s lead HBV core inhibitor (capsid inhibitor) product candidate, vebicorvir ("VBR"), and nucleos(t)ide analog ("NA") therapy for the treatment of people with chronic HBV.

In July 2021, we entered into a clinical collaboration with Vaccitech plc ("Vaccitech") to evaluate a triple combination of AB-729 with Vaccitech’s proprietary immunotherapeutic, VTP-300, and standard-of-care NA therapy for the treatment of subjects with chronic HBV infection. We expect to file a Clinical Trial Application (CTA) in the second half of 2021 and initiate the clinical trial in early 2022.

In June 2021, we entered into a clinical collaboration with Antios Therapeutics, Inc. ("Antios") to evaluate a triple combination of AB-729, Antios’ proprietary active site polymerase inhibitor nucleotide (ASPIN), ATI-2173, and Viread (tenofovir disoproxil fumarate), for the treatment of subjects with chronic HBV infection. This clinical trial is expected to initiate in the second half 2021.
AB-836: Oral Capsid Inhibitor

In January 2020, Arbutus selected AB-836, from a novel chemical series, as its next-generation oral capsid inhibitor. At EASL, Arbutus presented pre-clinical data suggesting the potential for increased efficacy and an enhanced resistance profile relative to previous generation capsid inhibitors. Arbutus completed CTA/IND-enabling studies in the fourth quarter of 2020 and initiated a Phase 1a/1b clinical trial for AB-836 in the first quarter of 2021. Initial data from healthy volunteers and HBV subjects from this clinical trial is expected in second half of 2021.
HBV Discovery Programs

Arbutus’ drug discovery efforts are focused on follow-on compounds for its current HBV pipeline. Arbutus expects to continue to advance its research in its oral PD-L1 inhibitor and RNA-destabilizer programs.
Research Efforts to Combat COVID-19 and Future Coronavirus Outbreaks

Based on its extensive antiviral drug discovery experience, Arbutus has established an internal research program to identify new small molecule antiviral medicines to treat COVID-19 and future coronavirus outbreaks. This effort, led by Dr. Michael Sofia, Arbutus’ Chief Scientific Officer, is focused on the discovery and development of new molecular entities that address specific viral targets including the nsp12 viral polymerase and the nsp5 viral protease. These targets are essential viral proteins which Arbutus has experience in targeting. Arbutus recently entered into a discovery research and license agreement with X-Chem, Inc. and Proteros biostructures GmbH focused on the discovery of novel inhibitors targeting the SARS-CoV-2 nsp5 main protease (Mpro). The agreement is designed to accelerate the development of pan-coronavirus agents to treat COVID-19 and potential future coronavirus outbreaks.
Financial Results

Cash, Cash Equivalents and Investments

Arbutus had cash, cash equivalents and investments totaling $121.3 million as of June 30, 2021, as compared to $123.3 million as of December 31, 2020. During the six months ended June 30, 2021, Arbutus used $31.9 million in operating activities, which was offset by $30.7 million of net proceeds from the issuance of common shares under Arbutus’s "at-the-market" offering program. The Company believes its cash, cash equivalents and investments of $121.3 million as of June 30, 2021 are sufficient to fund the Company’s operations through the third quarter of 2022.

Net Loss

Net loss attributable to common shares for the three months ended June 30, 2021 was $22.7 million ($0.23 basic and diluted loss per common share) as compared to $17.1 million ($0.25 basic and diluted loss per common share) for the three months ended June 30, 2020. Net loss attributable to common shares for the three months ended June 30, 2021 and 2020 included non-cash expense for the accrual of coupon on the Company’s convertible preferred shares of $3.3 million and $3.0 million, respectively.

Operating Expenses

Research and development expenses were $15.4 million for the three months ended June 30, 2021 compared to $10.5 million in the same period in 2020. The increase in research and development expenses for the three months ended June 30, 2021 versus the same period in 2020 was due primarily to higher expenses for the Company’s clinical development and discovery programs, including activities under our collaboration with Assembly and internal research efforts to treat COVID-19 and future coronavirus outbreaks, both of which initiated in mid-2020. General and administrative expenses were $4.4 million for the three months ended June 30, 2021 compared to $3.6 million for the same period in 2020. This increase was due primarily to increases in non-cash stock-based compensation expense and professional fees.

Outstanding Shares

The Company had approximately 97.7 million common shares issued and outstanding as of June 30, 2021. In addition, the Company had approximately 13.3 million stock options outstanding and 1.164 million convertible preferred shares outstanding, which (including the annual 8.75% coupon) will be mandatorily convertible into approximately 23 million common shares on October 18, 2021.

COVID-19 Impact

In December 2019 an outbreak of a novel strain of coronavirus (COVID-19) was identified in Wuhan, China. This virus continues to spread globally, has been declared a pandemic by the World Health Organization and has spread to nearly every country in the world. The impact of this pandemic has been, and will likely continue to be, extensive in many aspects of society. The pandemic has resulted in and will likely continue to result in significant disruptions to businesses. A number of countries and other jurisdictions around the world have implemented extreme measures to try and slow the spread of the virus. These measures include the closing of businesses and requiring people to stay in their homes, the latter of which raises uncertainty regarding the ability to travel to hospitals in order to participate in clinical trials. Additional measures that have had, and will likely continue to have, a major impact on clinical development, at least in the near-term, include shortages and delays in the supply chain, and prohibitions in certain countries on enrolling subjects in new clinical trials. While we have been able to progress with our clinical and pre-clinical activities to date, it is not possible to predict if the COVID-19 pandemic will materially impact our plans and timelines in the future.

Conference Call and Webcast Today

Arbutus will hold a conference call and webcast today, Thursday, August 5, 2021 at 8:45 AM Eastern Time to provide a corporate update. You can access a live webcast of the call, which will include presentation slides, through the Investors section of Arbutus’ website at www.arbutusbio.com or directly at Live Webcast. Alternatively, you can dial (866) 393-1607 or (914) 495-8556 and reference conference ID 2719108.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling (855) 859-2056 or (404) 537-3406, and reference conference ID 2719108.

About AB-729

AB-729 is an RNA interference (RNAi) therapeutic targeted to hepatocytes using Arbutus’ novel covalently conjugated N-acetylgalactosamine (GalNAc) delivery technology that enables subcutaneous delivery. AB-729 inhibits viral replication and reduces all HBV antigens, including hepatitis B surface antigen in preclinical models. Reducing hepatitis B surface antigen is thought to be a key prerequisite to enable reawakening of a patient’s immune system to respond to the virus. Based upon clinical data generated thus far in an ongoing single- and multi-dose Phase 1a/1b clinical trial, AB-729 has demonstrated positive safety and tolerability data and meaningful reductions in hepatitis B surface antigen.

About AB-836

AB-836 is an oral HBV capsid inhibitor. HBV core protein assembles into a capsid structure, which is required for viral replication. The current standard-of-care therapy for HBV, primarily nucleos(t)ide analogues that work by inhibiting the viral polymerase, significantly reduce virus replication, but not completely. Capsid inhibitors inhibit replication by preventing the assembly of functional viral capsids. They also have been shown to inhibit the uncoating step of the viral life cycle thus reducing the formation of new covalently closed circular DNA (cccDNA), the genetic reservoir which the virus uses to replicate itself.

About HBV

Hepatitis B is a potentially life-threatening liver infection caused by HBV. HBV can cause chronic infection which leads to a higher risk of death from cirrhosis and liver cancer. Chronic HBV infection represents a significant unmet medical need. The World Health Organization estimates that over 250 million people worldwide suffer from chronic HBV infection, while other estimates indicate that approximately 2 million people in the United States suffer from chronic HBV infection. Approximately 900,000 people die every year from complications related to chronic HBV infection despite the availability of effective vaccines and current treatment options.

On August 5, 2021 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company developing novel immune-modulating therapies to treat ocular and systemic diseases, reported financial results for the quarter ended June 30, 2021 and recent corporate highlights (Press release, Aldeyra Therapeutics, AUG 5, 2021, View Source [SID1234585791]).

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"We continue to make meaningful strides toward our goal of developing effective and highly differentiated new therapies to treat ocular and systemic diseases with significant unmet medical need," stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. "We remain on schedule to report top-line results from the Phase 3 TRANQUILITY and TRANQUILITY-2 dry eye disease clinical trials in the fourth quarter of this year, and we look forward to releasing results of our Phase 2 clinical trials of ADX-629 in systemic immunological diseases by the end of the year or early 2022."

Recent Corporate Highlights and Program Updates

Phase 3 TRANQUILITY and TRANQUILITY-2 Dry Eye Disease Clinical Trial Results Expected in the Fourth Quarter of 2021: Enrollment is ongoing in the dry eye chamber Phase 3 TRANQUILITY Trial of reproxalap. An identical trial, TRANQUILITY-2, is expected to begin enrollment this quarter. Ocular redness is the primary endpoint of the TRANQUILITY trials, which include tear RASP levels, Schirmer’s test, and dry-eye symptoms as secondary endpoints. Top-line results are expected in the fourth quarter of this year. In addition, the company has initiated a multi-center, double-masked, randomized, vehicle-controlled, parallel-group Phase 2 clinical trial of reproxalap in dry eye disease. The Phase 2 trial is designed to optimize the measurement of tear RASP levels, with approximately 75 patients expected to be enrolled per arm. Similar to TRANQUILITY, ocular redness is the primary endpoint in the trial.
Orphan Drug Designation Granted for ADX-2191 in Two Additional Retinal Disease Indications: The U.S. Food and Drug Administration (FDA) granted orphan drug designation to ADX-2191 (methotrexate for intravitreal injection) for the treatment of two rare retinal diseases: primary vitreoretinal lymphoma, an aggressive, high-grade cancer; and retinitis pigmentosa (RP), a clinical group of rare genetic eye diseases characterized by retinal cell death and loss of vision. Aldeyra plans to initiate a Phase 2 clinical trial of ADX-2191 in patients with RP this year. The FDA’s orphan drug designation program is designed to provide financial incentives to sponsors for developing drugs and biologics for rare diseases and conditions, in part defined as affecting fewer than 200,000 people in the United States. Sponsors of designated orphan drugs are eligible for partial tax credits for clinical trial costs, waiver of the user fee for marketing applications and, upon approval, consideration for seven years of marketing exclusivity.
Phase 2 Clinical Trial Results from ADX-629, an Orally Available, Systems-Based RASP Inhibitor, Expected in the Fourth Quarter of 2021 or First Quarter of 2022: Initial Phase 2 clinical trial results from ADX-629, a novel orally available RASP inhibitor currently undergoing testing in asthma, psoriasis, and COVID-19, are expected in the fourth quarter of 2021 or first quarter of 2022. ADX-629 represents a first-in-class systems-based therapeutic approach for the potential treatment of a myriad of immune-mediated diseases that today are treated with single-target drugs that can lead to serious toxicity.
Second-Quarter 2021 Financial Results

Cash and cash equivalents as of June 30, 2021 were $249.7 million. Based on Aldeyra’s current operating plan, the company believes that existing cash and cash equivalents will be sufficient to fund currently projected operating expenses through the end of 2023, including potential New Drug Application submissions for reproxalap; initial commercialization of reproxalap, if approved; and continued development of the company’s product candidates in ocular and systemic immune-mediated diseases.

For the quarter ended June 30, 2021, Aldeyra reported a net loss of $14.9 million, compared with a net loss of $7.5 million for the quarter ended June 30, 2020. Net loss per share was $0.28 for the quarter ended June 30, 2021, compared with $0.25 for the same period in 2020. Losses have resulted from the costs of clinical trials and research and development programs, as well as from general and administrative expenses.

Research and development expenses were $11.5 million for the quarter ended June 30, 2021, compared with $4.9 million for the same period in 2020. The increase of $6.6 million is primarily related to the increase in clinical research and development expenditures.

General and administrative expenses were $3.1 million for the quarter ended June 30, 2021, compared with $2.2 million for the quarter ended June 30, 2020. The increase of $0.9 million is primarily due to an increase in personnel-related costs and other miscellaneous administrative expenses.

For the quarter ended June 30, 2021, total operating expenses were $14.5 million, compared with total operating expenses of $7.1 million for the same period in 2020.

Conference Call & Webcast Information

Aldeyra will host a conference call at 8:00 a.m. ET today to discuss second-quarter 2021 financial results and recent corporate highlights. The dial-in numbers are (866) 211-4098 for domestic callers and (647) 689-6613 for international callers. The Conference ID number is 4968588. Due to expected high demand, please dial in at least 15 minutes prior to the start time.

A live webcast of the conference call will also be available on the Investor Relations page of the company’s website at View Source After the live webcast, the event will remain archived on the Aldeyra Therapeutics website for 90 days.

On August 5, 2021 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose of bettering the lives of people impacted by kidney disease, reported financial results for the second quarter ended June 30, 2021 and highlighted recent corporate milestones (Press release, Akebia, AUG 5, 2021, View Source [SID1234585790]). The Company will host a conference call today, Thursday, August 5, 2021, at 9:00 a.m. Eastern Time.

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"The first half of 2021 has been marked by significant milestones that have further strengthened Akebia’s position and the potential market opportunity for vadadustat, an investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), setting the stage for an exciting and catalyst-rich year ahead for us. During the quarter, these achievements included publication of our global Phase 3 results for vadadustat in the New England Journal of Medicine and, more recently, FDA acceptance of the vadadustat NDA for filing with a PDUFA target action date of March 29, 2022," stated John P. Butler, Chief Executive Officer of Akebia. "As there are currently no approved HIF-PHIs to treat anemia due to chronic kidney disease (CKD) in the U.S., we believe vadadustat is positioned as a potential first-in-class product with a broader opportunity in the dialysis market than originally anticipated. With vadadustat’s PDUFA date set for March, we are highly focused on pre-launch activities to ensure that we are well positioned for a successful U.S. launch in 2022, and are excited to be one step closer to having a novel, oral therapeutic available for people living with this disease, subject to regulatory approval."

Steven K. Burke, M.D., Senior Vice President, Research & Development and Chief Medical Officer of Akebia stated, "We remain confident in the clarity and quality of our data, and continue to be encouraged by the safety profile of vadadustat demonstrated in our global Phase 3 program for the treatment of anemia due to CKD. As seen in the recent New England Journal of Medicine publications of our global Phase 3 results, the data showed no significant safety signals on adverse events, including thromboembolic events, seizures and infections. More specifically, the data showed that these events were very similar for vadadustat as compared to darbepoetin alfa."

Akebia is also working in close collaboration with its partner, Otsuka Pharmaceutical Co. Ltd., to prepare a Marketing Authorization Application for vadadustat for submission to the European Medicines Agency, expected this year.

Recent Business Highlights:

In late May, the FDA accepted for filing the NDA for vadadustat for the treatment of anemia due to CKD in both adult patients on dialysis and adult patients not on dialysis. The FDA assigned the application a standard review and a Prescription Drug User Fee Act (PDUFA) target action date of March 29, 2022.
In April, the New England Journal of Medicine published the results of Akebia’s global Phase 3 program for vadadustat, which consisted of two programs that evaluated the efficacy and safety of vadadustat versus darbepoetin alfa for the treatment of anemia due to CKD in adult patients on dialysis (INNO2VATE) and not on dialysis (PRO2TECT).
In June, Akebia presented data regarding the hematologic efficacy of vadadustat for anemia in patients with kidney failure on dialysis and not on dialysis from the global Phase 3 program for vadadustat at the European Renal Association – European Dialysis and Transplant Association (ERA-EDTA) Virtual Congress 2021.
In March, Akebia submitted an NDA to the FDA for vadadustat for the treatment of anemia due to CKD in both adult patients on dialysis and adult patients not on dialysis.
Second Quarter Financial Results

Revenues: Total revenue was $52.9 million for the second quarter of 2021 compared to $90.1 million for the second quarter of 2020. The decrease compared to the same period in 2020 was primarily due to lower collaboration revenue consistent with the Company successfully completing the INNO2VATE and PRO2TECT global Phase 3 clinical programs.
Collaboration revenue was $20.0 million for the second quarter of 2021 compared to $59.4 million for the second quarter of 2020.
Net product revenue for Auryxia was $33.0 million for the second quarter of 2021 compared with $30.7 million for the second quarter of 2020, an increase of 7.4 percent.
COGS: Cost of goods sold was $52.5 million for the second quarter of 2021 and included a $30.3 million non-cash charge related to an increase to the liability for excess purchase commitments consistent with the Company’s long-term payor contract strategy, which remains focused on contract economics and net product revenue growth. Cost of goods sold was $174.6 million for the second quarter of 2020 and included a non-cash impairment charge of $115.5 million related to the Auryxia intangible asset, $19.9 million in non-cash charges related to the fair-value inventory step-up from the application of purchase accounting, $11.0 million in non-cash charges related to an increase to the liability for excess purchase commitments and $9.9 million primarily related to the write-down of inventory.
R&D Expenses: Research and development expenses were $37.2 million for the second quarter of 2021 compared to $52.8 million for the second quarter of 2020. The decrease compared to the prior year period was primarily due to the completion of the INNO2VATE and PRO2TECT global Phase 3 clinical programs.
SG&A Expenses: Selling, general and administrative expenses were $41.7 million for the second quarter of 2021 compared to $35.5 million for the second quarter of 2020. The increase compared to the prior year period was due primarily to higher marketing expenses.
Net Loss: Net loss was $83.0 million for the second quarter of 2021 compared to $175.8 million for the second quarter of 2020. The improvement in net loss compared to the prior year period was due primarily to the non-recurrence of a $115.5 million non-cash impairment charge in the prior year period, as well as lower operating expenses in the 2021 period, partially offset by lower collaboration revenue in the 2021 period.
Cash Position: Cash, cash equivalents and available-for-sale securities as of June 30, 2021 were $247.0 million. This balance includes net proceeds of $37.3 million from sales of common stock under the Company’s at-the-market offering program during the second quarter of 2021. The Company also received net cash proceeds of $16.1 million from sales of common stock under this program subsequent to quarter end through July 16, 2021. The Company believes that its cash resources will be sufficient to fund its current operating plan through at least the next twelve months. Additionally, the Company believes its cash runway would extend beyond the next twelve months assuming timely regulatory approval of vadadustat and the receipt of associated regulatory milestones.
"We continue to be encouraged by Auryxia’s revenue growth, which we believe is a great illustration of our commercial team’s execution in this ongoing COVID-19 environment," stated David A. Spellman, Chief Financial Officer of Akebia. "We believe this performance also highlights Auryxia’s favorable product profile and the critical nature of this therapy. While we remain cautious due to COVID-19, we believe the team’s focus and execution on marketing, sales, and payor strategies will continue to drive net product revenue growth."

Conference Call

Akebia will host a conference call today, Thursday, August 5, 2021, at 9:00 a.m. Eastern Time to discuss its second quarter financial results and recent business highlights. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 5529396. The call will also be webcast LIVE and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through August 11, 2021. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 5529396. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

On August 5, 2021 Aeglea BioTherapeutics, Inc. (Nasdaq:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, reported financial results for the second quarter ended June 30, 2021, and reviewed recent corporate updates and program highlights (Press release, Aeglea BioTherapeutics, AUG 5, 2021, View Source [SID1234585789]).

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"2021 is proving to be a pivotal year for Aeglea. In the second quarter, we achieved a significant milestone in our second clinical development program. With the initiation of dosing in our Phase 1/2 clinical trial of AGLE-177, Aeglea is making progress for patients who suffer from Homocystinuria, a rare and progressive disease which currently has limited treatment options," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "Additionally, with the strengthening of our leadership team, deepening relationship with the rare disease community and advancing our scientific communications platform, we continue to prepare for the data readout for our Phase 3 clinical trial of pegzilarginase in Arginase 1 Deficiency in the fourth quarter as well as lay the groundwork for potential approval and commercialization."

Second Quarter and Recent Highlights and Updates

AGLE-177 in Homocystinuria

Initiated dosing with AGLE-177 in a Phase 1/2 clinical trial in people with Homocystinuria. The trial is anticipated to enroll 16–20 patients at sites located in the United Kingdom and Australia. Aeglea expects to provide a clinical update prior to the end of 2021.
Pegzilarginase in Arginase 1 Deficiency

Published 20-week data from a Phase 1/2 and open-label extension studies of pegzilarginase in the Journal of Inherited Metabolic Disease. The article, titled "Clinical Effect and Safety Profile of Pegzilarginase In Patients with Arginase 1 Deficiency," is available in the July issue.
Corporate

Strengthened the leadership team with appointment of Jonathan Alspaugh as chief financial officer and Jim Kastenmayer as general counsel.
Mr. Alspaugh joined Aeglea from Evercore, where he most recently served as a managing director in the firm’s corporate advisory business.
Mr. Kastenmayer joined Aeglea from Viela Bio, where he provided strategic guidance and legal advice, including advising Viela in connection with the FDA approval and launch of its first commercial product.
Expanded the board of directors with the appointment of Marcio Souza. Mr. Souza serves as the president & chief executive officer of Praxis Precision Medicines.
Participated in Rare Disease Awareness Week on Capitol Hill 2021, meeting with patient advocates and law makers to raise awareness and discuss the specific needs facing the rare disease community.
Supported the launch of the Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, of which Aeglea is a founding member.
Upcoming Investor Events

Wells Fargo 2021 Virtual Healthcare Conference, September 9–10
HC Wainwright 23rd Annual Global Investment Conference, September 13–15
2021 Cantor Virtual Healthcare Conference, September 27–30
Second Quarter 2021 Financial Results

As of June 30, 2021, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $130.4 million inclusive of the $21.5 million upfront cash payment received from Immedica Pharma AB pursuant to the March 2021 license and supply agreement. The Company expects its cash, cash equivalents and investments will enable it to fund its operating expenses and capital expenditure requirements into 2023.

Aeglea recognized license and development revenues of $13.7 million in the second quarter of 2021, as a result of its license and supply agreement with Immedica for the commercial rights of pegzilarginase in certain territories outside the United States. The revenues recorded in the second quarter of 2021 are related to the transfer of the license and delivery of trial and regulatory services. Aeglea recognized no revenue for the corresponding period of 2020.

Research and development expenses totaled $13.6 million for the second quarter of 2021 and $16.9 million for the second quarter of 2020. The decrease was primarily associated with completing certain pre-commercial manufacturing activities for Aeglea’s lead product candidate, pegzilarginase.

General and administrative expenses totaled $6.8 million for the second quarter of 2021 and $4.7 million for the second quarter of 2020. This increase was primarily due to ramping-up the Company’s commercial capabilities and infrastructure.

Net loss totaled $6.8 million and $21.4 million for the second quarter of 2021 and 2020, respectively, with non-cash stock compensation expense of $2.1 million and $1.6 million for the second quarter of 2021 and 2020, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is a novel recombinant human enzyme, which has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality. Aeglea’s Phase 1/2 and Phase 2 open-label extension data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company’s ongoing single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough, Fast Track and Orphan Drug Designations from the FDA as well as Orphan Drug Designation from the European Medicines Agency.

About AGLE-177 in Homocystinuria

AGLE-177 is a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. AGLE-177 is currently being studied in a Phase 1/2 clinical trial for the treatment of patients with Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of homocysteine and homocystine. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis), developmental delay, intellectual disability, lens dislocation and severe near sightedness. Preclinical data demonstrated that AGLE-177, which is designed to lower abnormally high blood levels of homocysteine, improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received both U.S. and EU Orphan Drug Designation as well as U.S. Rare Pediatric Disease Designation.