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Aeglea BioTherapeutics Reports Third Quarter 2020 Financial Results and Corporate Highlights

On November 5, 2020 Aeglea BioTherapeutics, Inc. (NASDAQ: AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, reported its third quarter 2020 financial results, and provided recent corporate and program highlights (Press release, Aeglea BioTherapeutics, NOV 5, 2020, View Source [SID1234570020]).

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"Although it’s been a challenging environment for biotech with the global pandemic, we have continued to be productive in our Arginase 1 Deficiency patient identification and engagement efforts. I am pleased with the progress we are making overall as well as specifically at our PEACE trial sites," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "I am also excited by the momentum we are building in our Homocystinuria program with the recent granting of U.S. Orphan Drug Designation and a positive opinion for EU Orphan Drug Designation for ACN00177."

Recent Highlights and Updates

Pegzilarginase in Arginase 1 Deficiency

In September, Aeglea announced the PEACE Phase 3 pivotal trial was more than 50% enrolled with nearly twice the number of patients needed to complete enrollment identified at active trial sites. Enrollment for the trial is anticipated to be completed in January 2021.
As of September, Aeglea has identified over 250 Arginase 1 Deficiency patients in addressable markets, a 25% increase relative to the prior year. The number of currently identified patients represents more than 50% and 30% of the estimated genetic prevalence populations in the U.S. and EU5, respectively.
ACN00177 in Homocystinuria

In October, Aeglea announced the U.S. Food and Drug Administration granted Orphan Drug Designation to ACN00177 for the treatment of Homocystinuria.
Additionally, the European Medicines Agency Committee for Orphan Medicinal Products issued a positive opinion recommending Orphan Drug Designation for ACN00177 for the treatment of Homocystinuria in the European Union.
Corporate

In October, the Company strengthened its financial position with proceeds from shares of common stock sold under its ATM program, resulting in gross proceeds of $25 million, extending its cash runway into 2023.
Upcoming Events

Aeglea will be attending the following virtual investor conferences in the fourth quarter:

Piper Sandler 32nd Annual Virtual Healthcare Conference, December 1-3
3rd Annual Evercore ISI HealthCONx Conference, December 1-3
Third Quarter 2020 Financial Results

As of September 30, 2020, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $141.5 million. In addition, in October 2020 the Company raised approximately $24.6 million in net proceeds from shares of common stock sold under its ATM program. Based on Aeglea’s current operating plan, and taking into account the net offering proceeds, management believes it has sufficient capital resources to fund anticipated operations into 2023.

Research and development expenses totaled $12.5 million for the third quarter of 2020 and $17.8 million for the third quarter of 2019. The decrease was primarily associated with completing certain manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase, completing a Phase 1/2 clinical trial in patients with Arginase 1 Deficiency and closing cancer trials; offset by a ramp-up in manufacturing for ACN00177 in Homocystinuria and higher personnel-related expenses.

General and administrative expenses totaled $5.7 million for the third quarter of 2020 and $4.3 million for the third quarter of 2019. This increase was primarily due to ramping-up commercial capabilities and additional facilities to support company growth.

Net loss totaled $18.0 million and $21.6 million for the third quarter of 2020 and 2019, respectively, with non-cash stock compensation expense of $1.7 million and $1.4 million for the third quarter of 2020 and 2019, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating, progressive disease presenting in childhood with persistent hyperargininemia, spasticity, developmental delay, intellectual disability, seizures and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. 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 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.

About ACN00177 in Homocystinuria

Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. 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. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with Homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. The Company initiated a Phase 1/2 trial in the second quarter of 2020 and continues patient identification and administrative activities. The timing of first patient dosing in this Phase 1/2 trial will depend on determinations by individual sites as they adjust to impacts from COVID-19. ACN00177 has been granted Orphan Drug Designation by the U.S. FDA and received a positive opinion on Orphan Drug Designation from the European Medicines Agency.

Merrimack Reports Third Quarter 2020 Financial Results

On November 5, 2020 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) ("Merrimack" or the "Company") reported its third quarter 2020 financial results for the period ended September 30, 2020 (Press release, Merrimack, NOV 5, 2020, View Source [SID1234570019]).

"We are pleased that both Ipsen Pharmaceuticals and Elevation Oncology continue to enroll patients in separate clinical stage programs which could result in milestone payments to Merrimack." said Gary Crocker, Chairman of Merrimack’s Board of Directors. "We remain focused on conserving cash and reduction of our operating expenses to ensure that we have sufficient financial resources to capture future potential milestone payments from Ipsen and Elevation."

Second Quarter 2020 Financial Results

Merrimack reported net loss of $1.0 million for the third quarter ended September 30, 2020, or $0.08 per basic share, compared to a net loss of $0.7 million, or $0.05 per basic share, for the same period in 2019.

No gain on sale of assets was recognized for the third quarter ended September 30, 2020 compared to $3.5 million for the same period in 2019.

General and administrative expenses for the third quarter ended September 30, 2020 were $1.0 million, compared to $4.3 million for the same period in 2019.

As of September 30, 2020, Merrimack had cash and cash equivalents and investments of $15.8 million, compared to $16.6 million as of December 31, 2019.

As of September 30, 2020, Merrimack had 13.4 million shares of common stock outstanding.

Updates on Programs Underlying Potential Milestone Payments

Ipsen

On October 22, 2020 Ipsen released its third quarter financial results. As part of that update, Ipsen reported that it is continuing to study ONIVYDE in Phase III clinical trials in first line pancreatic ductal adenocarcinoma and in second line small cell lung cancer.

Elevation Oncology

On October 26, 2020 Elevation Oncology announced the presentation of preclinical data on its HER3 program. This announcement included confirmation that Elevation’s phase II CRESTONE study is currently enrolling patients.

Akebia Reports Third Quarter 2020 Financial Results and Recent Business Updates

On November 5, 2020 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose of bettering the lives of people impacted by kidney disease, eported financial results for the third quarter ended September 30, 2020 and provided business updates, including confirming completion of a pre-NDA meeting with the U.S. Food and Drug Administration (FDA) for vadadustat (Press release, Akebia, NOV 5, 2020, View Source [SID1234570018]). Vadadustat is Akebia’s investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) in development for the treatment of anemia due to chronic kidney disease (CKD) in adult patients on dialysis and not on dialysis.

"We recently completed our pre-NDA meeting with the FDA. This was an important milestone for our vadadustat development program, and we remain on track to submit an NDA to the FDA as early as possible next year. A key component of this NDA is the positive data from our global Phase 3 INNO2VATE program for the treatment of anemia due to CKD in adult patients on dialysis, which we shared most recently at ASN Kidney Week. These data were clear and consistent, and showed that vadadustat achieved both the primary and key secondary efficacy endpoints, as well as the primary and key secondary safety endpoints of the program for patients on dialysis. Based on our pre-NDA meeting, we remain confident that these results support the potential approval of vadadustat for the treatment of anemia due to CKD in adult patients on dialysis," said John P. Butler, President and Chief Executive Officer of Akebia Therapeutics. "Subject to regulatory review and approval, we believe vadadustat has the potential to be a new oral standard of care to help address the unmet needs of adult patients on dialysis, including both incident and prevalent dialysis patients. We believe this could translate into a potential $2 billion market opportunity in the U.S., alone. Together with our collaborator, Otsuka, we look forward to bringing this innovative therapy to patients on dialysis globally, if approved."

Butler continued, "The pre-NDA meeting also allowed us the opportunity to clarify key questions regarding data from PRO2TECT, our global Phase 3 program for the treatment of anemia due to CKD in adult patients not on dialysis, and we look forward to working with the FDA in their review of these data. While the PRO2TECT data showed that vadadustat achieved both the primary and key secondary efficacy endpoints, it did not meet the program’s primary safety endpoint for patients not on dialysis, and we remain appropriately cautious in our outlook for potential approval of vadadustat in patients not on dialysis. Importantly, we believe the PRO2TECT data will not adversely impact the potential approvability of vadadustat for the treatment of anemia due to CKD in adult patients on dialysis."

Akebia plans to submit a New Drug Application (NDA) to the FDA for vadadustat as early as possible in 2021 for two indications: (1) the treatment of anemia due to CKD in adult patients on dialysis, and (2) the treatment of anemia due to CKD in adult patients not on dialysis. In addition, Akebia and its collaborator, Otsuka Pharmaceutical Co. Ltd., are working in close collaboration to prepare a Marketing Authorization Application (MAA) for submission to the European Medicines Agency (EMA) next year.

Recent Business Highlights

In October, the Company presented data from its global Phase 3 program at American Society of Nephrology Kidney Week 2020 Reimagined (ASN Kidney Week). Akebia’s global Phase 3 program consists 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).
Highlights of INNO2VATE ASN Kidney Week Presentation: As previously reported in May 2020, vadadustat achieved the primary and key secondary efficacy endpoints and the primary safety endpoint of the INNO2VATE program, defined as non-inferiority of vadadustat versus darbepoetin alfa in time to first occurrence of a major adverse cardiovascular event (MACE), which is the composite of all-cause mortality, non-fatal myocardial infarction (MI), or non-fatal stroke, across both INNO2VATE studies. Newly presented INNO2VATE data showed that vadadustat also achieved non-inferiority to darbepoetin alfa on key secondary safety endpoints including expanded MACE, cardiovascular MACE, cardiovascular mortality, and all-cause mortality.
Highlights of PRO2TECT ASN Kidney Week Presentation: As previously reported in September 2020, vadadustat achieved the primary and key secondary efficacy endpoints of the PRO2TECT program, but did not meet the primary safety endpoint. Newly presented pre-specified regional analyses of the PRO2TECT program showed vadadustat demonstrated no clinically meaningful increase in cardiovascular risk compared to darbepoetin alfa in analyses of MACE, expanded MACE and all-cause mortality in U.S. patients treated to a target hemoglobin (Hb) range of 10 to 11 g/dL, consistent with U.S. treatment guidelines.
In October, Akebia and Otsuka launched Balancing Anemia Due to CKD, a campaign and website designed to increase awareness and education of anemia due to CKD among healthcare providers with the goal of improving the management of this disease for patients.
In August, the Company announced the launch of vadadustat in Japan by Mitsubishi Tanabe Pharma Corporation (MTPC), Akebia’s partner in Japan, as a treatment for anemia due to CKD in both adult patients on dialysis and not on dialysis under the trade name VAFSEO.
In July, the Company announced an investigator-sponsored research study by The University of Texas Health Science Center at Houston (UTHealth) in Houston, Texas, evaluating the use of vadadustat as a potential therapy to prevent and lessen the severity of acute respiratory distress syndrome (ARDS), a complication of COVID-19. The study is currently underway and actively enrolling patients.
Third Quarter Financial Results

Revenues: Total revenue was $60.0 million for the third quarter of 2020 compared to $92.0 million for the third quarter of 2019. The decline versus the prior year period was driven by lower collaboration revenue consistent with the Company completing the INNO2VATE and PRO2TECT studies.
Collaboration revenue was $25.6 million for the third quarter of 2020 compared to $62.0 million in the third quarter of 2019, and included $0.4 million in royalty revenue related to the commercial sale of vadadustat (VAFSEO) in Japan from MTPC.
Net product revenue for Auryxia (ferric citrate) was $34.4 million for the third quarter of 2020 compared with $30.0 million in the third quarter of 2019, an increase of 14.6 percent.
COGS: Cost of goods sold was $30.3 million for the third quarter of 2020 compared to $38.3 million for the third quarter of 2019 and includes the impact of $9.9 million in non-cash inventory write-downs largely related to a previously disclosed manufacturing quality issue related to Auryxia.
R&D Expenses: Research and development expenses were $46.9 million for the third quarter of 2020 compared to $74.5 million for the third quarter of 2019. The decline versus the prior year period was primarily driven by a decrease in costs consistent with the Company completing the INNO2VATE and PRO2TECT studies.
SG&A Expenses: Selling, general and administrative expenses were $40.2 million for the third quarter of 2020 compared to $34.2 million for the third quarter of 2019. The increase was primarily a result of higher professional service fees related to marketing and pre-commercialization activities for vadadustat and legal fees.
Net Loss: Net loss was $60.0 million for the third quarter of 2020 compared to $54.6 million for the third quarter of 2019. The increase in net loss compared to the prior year period was due primarily to lower total revenue and more specifically, lower collaboration revenue, partially offset by lower operating expenses.
Cash Position: Cash, cash equivalents and available-for-sale securities as of September 30, 2020 were $269.3 million. The Company expects its cash resources to fund its current operating plan beyond the expected U.S. launch of vadadustat, assuming regulatory approval.
Conference Call
Akebia will host a conference call at 9:00 a.m. Eastern Time today, Thursday, November 5th, to discuss its third 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 2860285. 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 November 11, 2020. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 2860285. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

Halozyme Announces Upcoming Data Presentations at American Society of Hematology Annual Meeting For APOLLO and ANDROMEDA Phase 3 Studies By Janssen Evaluating Subcutaneous Daratumumab Utilizing ENHANZE®

On November 5, 2020 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that studies sponsored by Janssen Research & Development, LLC (Janssen) evaluating subcutaneous (SC) daratumumab utilizing ENHANZE will be the subject of presentations at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place virtually December 5-8, 2020 (Press release, Halozyme, NOV 5, 2020, View Source [SID1234570017]).

Primary data from the Phase 3 APOLLO study of SC daratumumab in combination with pomalidomide and dexamethasone (D-Pd) in patients with multiple myeloma who have received one or more prior lines of therapy (Abstract #412) will be the subject of an oral presentation and official ASH (Free ASH Whitepaper) Press Program (Sunday, December 6 3:00 p.m. ET).

Results from the Phase 3 ANDROMEDA study of SC daratumumab in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) in patients with light chain AL amyloidosis (Abstract #552) will be the subject of an oral presentation (Monday, December 7 11:15 a.m. ET). The ANDROMEDA study will also be the subject of four poster presentations (Abstracts #1392, #1640, #1409 and #2305).

X4 Pharmaceuticals Reports Third Quarter 2020 Financial Results and Provides Corporate Update

On November 5, 2020 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, reported financial results for the third quarter ended September 30, 2020 (Press release, X4 Pharmaceuticals, NOV 5, 2020, View Source [SID1234570016]). The company also provided an update on its lead investigational candidate, mavorixafor, a novel small molecule in a Phase 3 clinical trial for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b trials in patients with Waldenström’s macroglobulinemia and Severe Congenital Neutropenia (SCN), respectively.

"The third quarter was highlighted by the publication of our positive Phase 2 safety and efficacy data for mavorixafor in WHIM syndrome in the prestigious journal, Blood, which, we believe, continues to demonstrate the significant potential of our lead candidate to treat this patient population," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "Further, in early October, we announced that the U.S. Food and Drug Administration (FDA) granted mavorixafor Fast Track Designation for the treatment of WHIM, facilitating expedited review of mavorixafor as we proceed through clinical development. Both achievements enhance our confidence in mavorixafor’s potential to deliver the first disease-modifying therapy for this undertreated patient population. We were also thrilled to enhance our leadership team and expertise through the additions of Art Taveras, Ph.D., as Chief Scientific Officer and new board member, Alison Lawton."

Dr. Ragan continued, "While the operating environment remains challenging due to the ongoing COVID-19 pandemic, we continue to advance our mavorixafor development programs and enroll patients into our clinical trials. We anticipate initial data from our Phase 1b Waldenström’s macroglobulinemia trial in the first half of 2021, along with initial data from our Phase 1b trial in patients with SCN in 2021, and top-line results from our Phase 3 trial of mavorixafor in WHIM syndrome in 2022. As we continue to achieve important clinical progress," Dr. Ragan concluded, "we are supported by a strong balance sheet that we expect will drive our current strategic and development plans into early 2022."

Recent Highlights
•Published comprehensive data from the Phase 2 and open label extension study of mavorixafor in WHIM syndrome in Blood, the official journal of the American Society of Hematology (ASH) (Free ASH Whitepaper), expanding on previously presented data. New results included long-term patient-level data regarding the effect on neutrophils and lymphocytes and the effect of increasing doses of mavorixafor on total white blood cell counts and monocytes. In addition, the manuscript provided the most up-to-date pharmacokinetic data and presented a detailed analysis of the clinical improvements in infection rates and warts demonstrated with extended mavorixafor treatment.

•Granted Fast Track Designation by the FDA for mavorixafor in WHIM syndrome. Through the Fast Track program, X4 will be eligible for more frequent meetings with the FDA to discuss mavorixafor’s development plan, protocols and clinical data that would support its potential approval for WHIM. Mavorixafor was previously granted Breakthrough Therapy Designation by the FDA, as well as Orphan Drug status by the FDA and the European Commission for the treatment of WHIM syndrome

•Appointed Art Taveras, Ph.D., as Chief Scientific Officer. Dr. Taveras is a seasoned Chief Scientific Officer with experience in the discovery of novel, next-generation CXCR2 antagonists for the treatment of cancer. Previously, Dr. Taveras was VP, Small Molecule Drug Discovery at Biogen, President and CSO of ShangPharma ChemPartners, and CSO of CoMET Therapeutics. With his expertise in chemokine-related chemistries, Dr. Taveras is well suited to lead X4’s R&D initiatives and its continued evolution into a global rare disease company.

•Expanded Board of Directors through the appointment of biopharmaceutical industry veteran, Alison Lawton. Ms. Lawton strengthens the board’s drug development, manufacturing and commercialization experience and brings a unique understanding of X4’s core scientific and corporate goals, having previously served as a consulting Chief Operating Officer to the company and as a member of X4’s corporate advisory board. Ms. Lawton had an over 20 year tenure at Genzyme Corporation (now Sanofi Genzyme) including Senior Vice President and General Manager of Sanofi Biosurgery, a $750 million business and led global functional organizations, including regulatory affairs and quality systems, public policy, health outcomes and strategic pricing, product safety and risk management. Ms. Lawton most recently served as CEO of Kaleido Biosciences and currently serves as an independent director on the Board of ProQR Therapeutics NV.
Third Quarter 2020 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $90.7 million in cash, cash equivalents and restricted cash as of September 30, 2020. X4 continues to expect that its cash and cash equivalents will fund company operations into early 2022. Note this guidance does not include the $25 million in potential and contingent proceeds from our Hercules Debt Facility or any cash exercise proceeds from investors holding our outstanding warrants.

•Research and Development Expenses were $11.4 million for the third quarter ended September 30, 2020, as compared to $8.6 million for the comparable period in 2019. R&D expenses include $1.0 million of certain non-cash expenses for the quarter ended September 30, 2020.

•General and Administrative Expenses were $5.6 million for the third quarter ended September 30, 2020, as compared to $4.4 million for the comparable period in 2019. G&A expenses include $1.4 million of certain non-cash expenses for the quarter ended September 30, 2020.
•Net Loss: X4 reported a net loss of $17.4 million for the third quarter ended September 30, 2020 as compared to a net loss of $17.7 million for the comparable period in 2019. Net loss includes $2.5 million of certain non-cash expenses for the quarter ended September 30, 2020. Net loss in the third quarter of 2019 included a $4.0 million loss on the sale of non-financial assets.

Conference Call and Webcast
The Company will host a conference call and webcast today at 8:30 a.m. ET to discuss these financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 1096977. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.