On May 10, 2021 AVEO Oncology (Nasdaq: AVEO), a commercial and clinical development stage biopharmaceutical company, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, AVEO, MAY 10, 2021, View Source [SID1234579544]).

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"The first quarter of 2021 was a transformational time for AVEO marked by the U.S. Food and Drug Administration (FDA) approval and launch of FOTIVDA, our first commercial product, and the advancement of our pipeline, all of which is supported by a strong balance sheet," said Michael Bailey, president and chief executive officer of AVEO. "Our sales team is now fully deployed, and we are encouraged by the early commercial activity supporting the FOTIVDA launch. We look forward to continuing to execute on our goal of establishing FOTIVDA as a standard of care within its renal cell carcinoma (RCC) indication."

"In parallel, we remain focused on the continued advancement of the remainder of our clinical programs. This includes the evaluation of FOTIVDA in the immunotherapy combination setting, with patient enrollment in the pivotal Phase 3 TiNivo-2 study of FOTIVDA in combination with OPDIVO anticipated to commence mid-year and the Phase 2 hepatocellular carcinoma (HCC) DEDUCTIVE trial reporting encouraging Phase 1b results at ASCO (Free ASCO Whitepaper) GI earlier this year. Beyond FOTIVDA, we continue to expect several key inflection points with the balance of our clinical pipeline later this year, including a go/no go decision on progressing to a pivotal study for ficlatuzumab in head and neck squamous cell carcinoma (HNSCC), the advancement of our Phase 1 study of AV-380 and an update on potential clinical development plans for AV-203."

FOTIVDA U.S. Regulatory and Commercial Updates

U.S. Commercial Launch Underway Following FDA Approval of FOTIVDA for the Treatment of Adult Patients with Relapsed or Refractory Advanced RCC Following Two or More Prior Systemic Therapies.
Announced FDA approval of FOTIVDA for the treatment of adults with relapsed or refractory advanced RCC following two or more prior systemic therapies on March 10, 2021.
Net product revenue for the first quarter of 2021 was $1.1 million, which reflects inventory shipped to distributors and a 15.0% gross to net estimate in the last week of March as FOTIVDA became commercially available on March 22, 2021.
All distributors that made orders in the first quarter have placed reorders for the second quarter.
As of April 30, 2021, 49 prescriptions have been filled and 75 samples have been requested and delivered.
In March 2021, AVEO announced that the National Comprehensive Cancer Network updated its Clinical Practice Guidelines to include FOTIVDA as a recommended regimen for subsequent therapy.
Tivozanib Clinical Updates

Additional Data from the TIVO-3 Study to be Presented at 2021 ASCO (Free ASCO Whitepaper) Annual Meeting in June 2021. Additional data from the TIVO-3 study will be featured during two poster presentations at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)(ASCO) Annual Meeting in June 2021. The presentations, titled "TIVO-3: Durability of response and updated overall survival of tivozanib versus sorafenib in metastatic renal cell carcinoma (mRCC)" and "Temporal characteristics of treatment-emergent adverse events and dose modifications with tivozanib and sorafenib in the phase 3 TIVO-3 study of relapsed or refractory mRCC", will be featured on Friday, June 4.
Announced a Collaboration with Bristol Myers Squibb to Evaluate FOTIVDA in Combination with OPDIVO in a Planned Pivotal Phase 3 TiNivo-2 Trial in IO Relapsed or Refractory RCC. In March 2021, AVEO announced that it entered into a clinical trial collaboration and supply agreement with Bristol Myers Squibb (NYSE: BMS) to evaluate FOTIVDA in combination with OPDIVO, Bristol Myers Squibb’s anti-PD-1 therapy, in the planned pivotal Phase 3 TiNivo-2 trial in patients with advanced relapsed or refractory RCC following prior immunotherapy exposure. Bristol Myers Squibb will provide OPDIVO clinical drug supply for the study. AVEO will serve as the study sponsor and will be responsible for costs associated with the trial execution. AVEO recently received feedback from the FDA on the trial design and expects to commence enrollment in the trial in mid-2021.
Presented New Analyses from the Phase 3 TIVO-3 Study at ASCO (Free ASCO Whitepaper) 2021 GU Cancers Symposium. In February 2021, AVEO presented key subgroup and quality of life analyses from the Phase 3 TIVO-3 study at the ASCO (Free ASCO Whitepaper) 2021 Genitourinary (GU) Cancers Symposium. The results further demonstrate the potential tolerability benefits of tivozanib. A copy of each presentation is available in the Scientific Publications & Presentations section of AVEO’s website.
Results from Phase 1b Portion of DEDUCTIVE Study in HCC Presented at 2021 ASCO (Free ASCO Whitepaper) GI Cancer Symposium. In January 2021, results from the Phase 1b portion of the Phase 1b/2 DEDUCTIVE clinical trial of tivozanib in combination with IMFINZI (durvalumab), AstraZeneca’s (LSE/STO/Nasdaq: AZN) human monoclonal antibody directed against programmed death-ligand 1, in patients with HCC were presented at the 2021 ASCO (Free ASCO Whitepaper) Gastrointestinal (GI) Cancers Symposium. There were no dose-limiting toxicities with the combination recorded in the DEDUCTIVE trial. In addition, the combination demonstrated a 29% partial response (PR) rate and a 71% disease control rate (PR + stable disease), which is comparable to findings with bevacizumab and TECENTRIQ (atezolizumab), an emerging standard of care in the same setting. Completion of enrollment in the ongoing Phase 2 portion of the study, which is expected to enroll up to an additional 30 subjects, is anticipated later this year.
Ficlatuzumab Clinical Update

Enrollment Complete in Phase 2 Open Label Randomized Study of Ficlatuzumab in HNSCC; Results Expected to Be Presented at ASCO (Free ASCO Whitepaper) in June 2021; Phase 3 Go/No Go Decision Anticipated for Mid-2021. In January 2021, AVEO announced completion of enrollment in its randomized confirmatory Phase 2 study of ficlatuzumab as a single agent or in combination with cetuximab (ERBITUX), an EGFR-targeted antibody, in metastatic HNSCC patients who have failed prior immunotherapy, chemotherapy and cetuximab. Ficlatuzumab is AVEO’s potent humanized immunoglobulin G1 (IgG1) monoclonal antibody that targets hepatocyte growth factor (HGF). The study was designed to confirm findings from a Phase 1/2 study of ficlatuzumab and cetuximab where the combination was well-tolerated and resulted in a disease control rate of 67%, as well as prolonged progression-free survival and overall survival compared to historical controls.

Results from the Phase 2 study are expected to be presented in a poster discussion at ASCO (Free ASCO Whitepaper) in June 2021. Following the ASCO (Free ASCO Whitepaper) data presentation, AVEO plans to announce a go/no go Phase 3 decision for ficlatuzumab in HNSCC.

In September 2020, AVEO regained full global rights to ficlatuzumab and has initiated clinical manufacture of ficlatuzumab to supply a potential Phase 3 clinical trial in HNSCC, as well as additional potential Phase 2 studies in pancreatic cancer and acute myeloid leukemia.
AV-380 Clinical Update

Phase 1 Clinical Study Initiated Following FDA Acceptance of IND Filing. Recently, AVEO initiated enrollment for a Phase 1 study of AV-380, a potent humanized IgG1 monoclonal antibody that targets growth differentiation factor 15 (GDF15), for the potential treatment of cancer cachexia.
AV-203 Clinical Update

To Regain Ex-North American Rights to AV-203. In March 2021, AVEO announced it will regain rights to AV-203 outside of North America, its clinical-stage potent humanized IgG1 monoclonal antibody that targets ErbB3 (also known as HER3), following the voluntary termination of its collaboration and license agreement by CANbridge Life Sciences. AVEO will regain rights to AV-203 in all territories globally, and CANbridge has initiated the process to transfer all preclinical data and materials to AVEO. The Company expects to provide an update on the AV-203 clinical development plan in the second half of 2021.
Corporate Updates

Strong Balance Sheet to Support U.S. Launch of FOTIVDA. In the first quarter of 2021, AVEO added approximately $78.1 million to its balance sheet, consisting of a $20.0 million drawdown under its previously announced $45.0 million loan and security agreement with Hercules Capital. Inc. (NYSE: HTGC, Hercules), $3.1 million from warrant exercises as of March 31, 2021, $3.4 million in stock sales under its at-the-market sales agreement with SVB Leerink LLC and $51.6 million in net proceeds from a public offering of its common stock.
Strengthened Board of Directors with Two New Appointments. AVEO has recently announced the appointments of Kevin Cullen, M.D., and Corinne D. Epperly, M.D., MPH, to its Board of Directors.

Dr. Cullen, a widely recognized clinical oncologist with a specialty in head and neck cancer, is the Marlene and Stewart Greenebaum Distinguished Professor in Oncology and director of the Program in Oncology at the University of Maryland School of Medicine. He also serves as director of the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center.

Dr. Epperly brings over 15 years of experience in oncology as a physician and scientist, blending medicine and business with a proven track record in oncology drug development and launches, commercial and medical strategy, marketing, M&A and operations gained at Iovance Biotherapeutics, VBL Therapeutics, Bristol Myers Squibb, Goldman Sachs and the National Cancer Institute of the NIH.
Appointment of Mike Ferraresso to Chief Commercial Officer. In March 2021, AVEO announced the appointment of Mike Ferraresso to chief commercial officer. He is responsible for managing AVEO’s commercial strategy and operations, including the commercialization of FOTIVDA. Mr. Ferraresso, who joined AVEO in December 2017, most recently served as AVEO’s senior vice president, business analytics and commercial operations. He has over 20 years of commercial pharmaceutical and biotechnology experience, including 15 years developing and commercializing oncology products.
First Quarter 2021 Financial Highlights

AVEO ended Q1 2021 with $121.4 million in cash, cash equivalents and marketable securities as compared with $61.8 million at December 31, 2020.
Total revenue for Q1 2021 was approximately $1.9 million compared with $0.8 million for Q1 2020.
Research and development expense for Q1 2021 was $5.8 million compared with $7.8 million for Q1 2020.
Selling, general and administrative expense for Q1 2021 was $15.1 million compared with $3.7 million for Q1 2020.
Net loss for Q1 2021 was $22.1 million, or net loss of $0.81 per basic and diluted share, compared with a net loss of $8.4 million for Q1 2020, or net loss of $0.52 per basic and diluted share.
Net loss for Q1 2021 reflects an approximate $2.4 million non-cash loss attributable to the increase in the fair value of the 2016 private placement warrant liability that principally resulted from increases in the stock volatility rate that occurred within the first quarter, as well as a shorter remaining term as the warrants approach expiration. Net loss in Q1 2020 reflects an approximate $2.6 million non-cash gain attributable to the decrease in the fair value of the 2016 private placement warrant liability that principally resulted from the decrease in the stock price that occurred within the fiscal year.
Financial Guidance

AVEO believes that its $121.4 million in cash and cash equivalents as of March 31, 2021, along with net product revenues from the commercial launch of FOTIVDA in the United States, would enable AVEO to maintain its current operations for a period of at least 12 months following the filing of its Quarterly Report on Form 10-Q.

AVEO expects commercial spend will be approximately $40 million for the year. Gross margins are expected to be in the mid-to-high 80th percentile and research and development expense is expected to be approximately $40 million for existing pipeline plans during 2021. In addition, quarterly general and administrative expense should approximate the level seen during the first quarter of this year.

Conference Call and Webcast

In connection with this announcement, AVEO will host a conference call and audio webcast today, May 10, 2021, at 4:30 PM Eastern Time. The call can be accessed by dialing (844) 882-7841 (U.S. and Canada) or (574) 990-9828 (international). The passcode for the conference call is 3996993. To access the live webcast, or the subsequent archived recording, please visit the Investors section of the AVEO website at www.aveooncology.com.

About FOTIVDA (tivozanib)

FOTIVDA (tivozanib) is an oral, next-generation vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI). It is a potent, selective inhibitor of VEGFRs 1, 2, and 3 with a long half-life designed to improve efficacy and tolerability. AVEO received U.S. Food and Drug Administration (FDA) approval for FOTIVDA on March 10, 2021 for the treatment of adult patients with relapsed or refractory advanced renal cell carcinoma (RCC) following two or more prior systemic therapies. FOTIVDA was approved in August 2017 in the European Union and other countries in the territory of its partner EUSA Pharma (UK) Limited for the treatment of adult patients with advanced RCC. FOTIVDA has been shown to significantly reduce regulatory T-cell production in preclinical models.1 FOTIVDA was discovered by Kyowa Kirin.

INDICATIONS

FOTIVDA is indicated for the treatment of adult patients with relapsed or refractory advanced renal cell carcinoma (RCC) following two or more prior systemic therapies.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hypertension and Hypertensive Crisis: Control blood pressure prior to initiating FOTIVDA. Monitor for hypertension and treat as needed. For persistent hypertension despite use of anti-hypertensive medications, reduce the FOTIVDA dose.

Cardiac Failure: Monitor for signs or symptoms of cardiac failure throughout treatment with FOTIVDA.

Cardiac Ischemia and Arterial Thromboembolic Events: Closely monitor patients who are at increased risk for these events. Permanently discontinue FOTIVDA for severe arterial thromboembolic events, such as myocardial infarction and stroke.

Venous Thromboembolic Events: Closely monitor patients who are at increased risk for these events. Permanently discontinue FOTIVDA for severe venous thromboembolic events.

Hemorrhagic Events: Closely monitor patients who are at risk for or who have a history of bleeding.

Proteinuria: Monitor throughout treatment with FOTIVDA. For moderate to severe proteinuria, reduce the dose or temporarily interrupt treatment with FOTIVDA.

Thyroid Dysfunction: Monitor before initiation and throughout treatment with FOTIVDA.

Risk of Impaired Wound Healing: Withhold FOTIVDA for at least 24 days before elective surgery. Do not administer for at least 2 weeks following major surgery and adequate wound healing. The safety of resumption of FOTIVDA after resolution of wound healing complications has not been established.

Reversible Posterior Leukoencephalopathy Syndrome (RPLS): Discontinue FOTIVDA if signs or symptoms of RPLS occur.

Embryo-Fetal Toxicity: Can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.

Allergic Reactions to Tartrazine: The 0.89 mg capsule of FOTIVDA contains FD&C Yellow No.5 (tartrazine) which may cause allergic-type reactions (including bronchial asthma) in certain susceptible patients.

ADVERSE REACTIONS

The most common (≥20%) adverse reactions were fatigue, hypertension, diarrhea, decreased appetite, nausea, dysphonia, hypothyroidism, cough, and stomatitis, and the most common Grade 3 or 4 laboratory abnormalities (≥5%) were sodium decreased, lipase increased, and phosphate decreased.

DRUG INTERACTIONS

Strong CYP3A4 Inducers: Avoid coadministration of FOTIVDA with strong CYP3A4 inducers.

USE IN SPECIFIC POPULATIONS

Lactation: Advise not to breastfeed.

Females and Males of Reproductive Potential: Can impair fertility.

Hepatic Impairment: Adjust dosage in patients with moderate hepatic impairment. Avoid use in patients with severe hepatic impairment.

To report SUSPECTED ADVERSE REACTIONS, contact AVEO Pharmaceuticals, Inc. at 1-833-FOTIVDA (1-833-368-4832) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see FOTIVDA Full Prescribing Information which is available at www.FOTIVDA.com.

About Advanced Renal Cell Carcinoma

According to the American Cancer Society’s 2021 statistics, renal cell carcinoma (RCC) is the most common type of kidney cancer, which is among the ten most common cancers in both men and women. Approximately 73,750 new cases of kidney cancer will be diagnosed annually and about 14,830 people will die from this disease. In patients with late-stage disease, the five-year survival rate is 13%. Agents that target the vascular endothelial growth factor (VEGF) pathway have shown significant antitumor activity in RCC.2 According to a 2019 publication, 50% of the approximately 10,000 patients who progress following two or more lines of therapy choose not to receive further treatment,3 which may be attributable to tolerability concerns and a lack of data to support evidence-based treatment decisions in this highly relapsed or refractory patient population.

On May 10, 2021 Allakos Inc. (the "Company") (Nasdaq: ALLK), a biotechnology company developing lirentelimab (AK002) for the treatment of eosinophil and mast cell-related diseases, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Allakos, MAY 10, 2021, View Source [SID1234579543]).

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Recent Accomplishments

Announced the acceptance of two oral and five poster presentations at the Digestive Disease Week (DDW) Annual Meeting taking place May 21 to 23, 2021. ePosters and ePapers will be available from the DDW ePosters and ePapers website. Abstracts can now be found here.
Initiated a randomized, double-blind, placebo-controlled Phase 3 study of lirentelimab in patients with eosinophilic duodenitis.
Upcoming 2021 Milestones

Topline data from a randomized, double-blind, placebo-controlled Phase 3 study of lirentelimab in patients with eosinophilic gastritis (EG) and/or eosinophilic duodenitis (EoD) expected in the fourth quarter of 2021.
Topline data from a randomized, double-blind, placebo-controlled Phase 2/3 study of lirentelimab in patients with eosinophilic esophagitis (EoE) expected in the fourth quarter of 2021.
Initiation of a randomized, double-blind, placebo-controlled Phase 2/3 study of subcutaneous lirentelimab in patients with EG and/or EoD expected in the second half of 2021.
Initiation of a Phase 2 study in a non eosinophilic gastrointestinal disease in the second half of 2021.
First Quarter 2021 Financial Results

Research and development expenses were $38.9 million in the first quarter of 2021 as compared to $18.3 million in the same period in 2020, an increase of $20.6 million.

General and administrative expenses were $16.7 million in the first quarter of 2021 as compared to $11.6 million in the same period in 2020, an increase of $5.1 million.

Allakos reported a net loss of $55.6 million in the first quarter of 2021 as compared to $27.8 million in the same period in 2020, an increase of $27.8 million. Net loss per basic and diluted share was $1.04 for the first quarter of 2021 compared to $0.57 in the same period in 2020.

Allakos ended the first quarter of 2021 with $615.9 million in cash, cash equivalents and marketable securities.

On May 10, 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 first quarter ended March 31, 2021 and highlighted recent corporate milestones (Press release, Akebia, MAY 10, 2021, View Source [SID1234579542]). The Company will host a conference call today, Monday, May 10, 2021, at 9:00 a.m. Eastern Time.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Akebia is off to a great start in 2021, building positive momentum with solid execution on strategic priorities that set the stage for an exciting year. Most importantly, we submitted the vadadustat NDA to the FDA, a significant milestone for Akebia and our partner, Otsuka Pharmaceutical Co. Ltd. We are also very proud that the New England Journal of Medicine recently published the results from both INNO2VATE and PRO2TECT, the global Phase 3 programs for vadadustat. We believe these publications reinforce the scientific rigor and quality of the vadadustat clinical development program, and we are pleased with early feedback from the medical community," stated John P. Butler, Chief Executive Officer of Akebia. "We believe this progress positions us well to continue advancing vadadustat with the goal of bringing this novel therapeutic to patients as quickly as possible, subject to regulatory approval. We remain confident in the clarity and quality of our data, and we look forward to engaging with the FDA on our NDA. In addition, we are working with Otsuka on the preparation of a Marketing Authorization Application (MAA) for vadadustat for submission to the European Medicines Agency (EMA), expected this year."

Recent Business Highlights:

In April, the New England Journal of Medicine (NEJM) 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 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.
In February, Akebia completed a non-dilutive transaction with an entity managed by HealthCare Royalty Management, LLC (HCR), to monetize the Company’s rights to receive royalties and sales milestones on vadadustat net sales under its collaboration agreement with Mitsubishi Tanabe Pharma Corporation (MTPC), with an upfront payment of $45 million.
In February, Akebia announced that LeAnne M. Zumwalt joined Akebia’s Board of Directors. Ms. Zumwalt recently served as Group Vice President, Government Affairs at DaVita Inc.
In February, Akebia launched its Medical Engagement Hub, an online resource dedicated to scientific education and connecting U.S. healthcare professionals with Akebia Medical Affairs.
In January, the University of Texas Health Science Center at Houston (UTHealth) in Houston, Texas, announced that it had received $5.1 million in government funding for its study 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. This investigator-sponsored research study is currently underway and actively enrolling patients.
First Quarter Financial Results

Revenues: Total revenue was $52.3 million for the first quarter of 2021 compared to $88.5 million for the first 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 $21.9 million for the first quarter of 2021 compared to $59.3 million for the first quarter of 2020.
Net product revenue was $30.4 million for the first quarter of 2021 compared with $29.2 million for the first quarter of 2020, an increase of 4 percent.
COGS: Cost of goods sold was $34.6 million for the first quarter of 2021 compared to $27.7 million for the first quarter of 2020. The increase was driven by higher non-cash purchase accounting adjustments as a result of the merger with Keryx, and a $5.1 million non-cash charge to inventory reserves related to a previously disclosed manufacturing quality issue related to Auryxia (ferric citrate), partially offset by an $8.9 million non-cash gain due to a reduction to the liability for excess purchase commitments primarily as a result of the Company having successfully modified certain supply agreements.
R&D Expenses: Research and development expenses were $40.6 million for the first quarter of 2021 compared to $81.2 million for the first quarter of 2020. The decrease compared to the same period in 2020 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 $41.3 million for the first quarter of 2021 compared to $38.0 million for the first quarter of 2020.
Net Loss: Net loss was $69.6 million for the first quarter of 2021 compared to $60.7 million for the first quarter of 2020. The increase in net loss compared to the prior year period was due primarily to lower collaboration revenue and higher cost of goods sold, partially offset by lower operating expenses.
Cash Position: Cash, cash equivalents and available-for-sale securities as of March 31, 2021 were $272.8 million. The Company expects its cash resources to fund its current operating plan beyond the expected U.S. launch of vadadustat, assuming timely regulatory approval and the receipt of associated regulatory milestones.
"Despite the ongoing impact of COVID-19 on dialysis patients, we are encouraged by Auryxia’s revenue growth in the first quarter of 2021 when compared to the prior year’s period. We believe this performance highlights Auryxia’s favorable product profile and the critical nature of this therapy, as well as our team’s ability to execute at a high level," stated David A. Spellman, Chief Financial Officer of Akebia. "While we remain cautious due to COVID-19, together with our continued commercial efforts, we believe that Auryxia’s positioning will drive product revenue growth for the year."

Conference Call

Akebia will host a conference call at 9:00 a.m. Eastern Time today, Monday, May 10, to discuss its first 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 6250159. 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 May 16, 2021. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 6250159. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

On May 10, 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 its first quarter 2021 financial results, and provided recent corporate and program highlights (Press release, Aeglea BioTherapeutics, MAY 10, 2021, View Source [SID1234579541]).

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"We’ve had a strong start to 2021 and I am excited to see the significant momentum we have built for our pegzilarginase program from a clinical perspective as well as strengthening our commercial foundation in preparation for a potential FDA approval and launch. In just the last two months, we completed patient randomization for PEACE, secured a commercialization partner for pegzilarginase in Europe and the Middle East and launched THINK ARGININE, an Arginase 1 Deficiency disease education and diagnostic testing initiative in the United States," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "This substantial progress sets us up well for the rest of 2021. We expect to provide topline data from the PEACE study in the fourth quarter, continue progressing our commercial strategy for pegzilarginase and advance our AGLE-177 clinical program in Homocystinuria. These accomplishments are critical milestones as we move closer to our mission of bringing impactful therapies to patients with rare metabolic disorders who currently have limited treatment options."

Recent Highlights and Updates

Corporate

In March, Aeglea announced it has entered into a license and supply agreement with Immedica Pharma AB granting Immedica exclusive commercialization rights in Europe and several Middle Eastern countries. Aeglea will receive an upfront payment of $21.5 million and is eligible for commercial and regulatory milestones of up to approximately $130 million and mid-twenties percentage royalties on net sales.
Pegzilarginase in Arginase 1 Deficiency

In April, the Company completed patient screening and randomization for PEACE, its pivotal Phase 3 clinical trial. Trial enrollment of 32 patients exceeded the target of 30 patients, underscoring the high levels of interest seen from patients, caregivers and investigators. Topline data are expected in the fourth quarter of 2021.
In May, we announced the launch of THINK ARGININE, a disease education initiative to improve the awareness and diagnosis of Arginase 1 Deficiency (ARG1-D). The initiative consists of a comprehensive healthcare provider education campaign and a no-charge diagnostic testing program for adults and children with suspected ARG1-D in the United States.
The no-charge diagnostic testing program includes amino acid testing working with Mayo Clinic Laboratories and genetic testing partnered with Invitae.
Upcoming Events

Aeglea will participate in the upcoming virtual conferences and events:

ISPOR 2021, May 17-20
Virtual Rare Disease Week on Capitol Hill 2021, July 14-22
First Quarter 2021 Financial Results

As of March 31, 2021, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $128.5 million, which excludes the upfront license receivable from Immedica for $21.5 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2023.

Research and development expenses totaled $11.9 million for the first quarter of 2021 and $14.6 million for the first 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 $6.4 million for the first quarter of 2021 and $4.5 million for the first quarter of 2020. This increase was primarily due to ramping-up the Company’s commercial capabilities and infrastructure.

Net loss totaled $18.2 million and $18.7 million for the first quarter of 2021 and 2020, respectively, with non-cash stock compensation expense of $1.8 million and $1.3 million for the first 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 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. Aeglea is developing AGLE-177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as 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 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received U.S. Rare Pediatric Disease and Orphan Drug Designations as well as EU Orphan Drug Designation. Aeglea initiated a Phase 1/2 trial in 2020 and continues patient identification and administrative activities.

On May 10, 2021 BioXcel Therapeutics, Inc. ("BioXcel" or the "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its quarterly results for the first quarter ended March 31, 2021 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, MAY 10, 2021, View Source [SID1234579521]).

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"This quarter, we have remained committed to executing on our vision for our neuroscience franchise, as we continue to advance the development of BXCL501 for treatment of agitation associated with dementia and prepare for its potential approval in acute agitation associated with schizophrenia and bipolar disorders," stated Vimal Mehta, Chief Executive Officer of BioXcel. "We are pleased to have recently received Breakthrough Therapy designation for BXCL501 in agitation associated with dementia, highlighting the urgent need for new therapy options, as well as this candidate’s potential to provide a solution across treatment settings. While we solidify our plans for a pivotal program in this condition, we are committed to building the commercial infrastructure needed to successfully launch BXCL501 in the first two indications, in addition to follow-on indications in the future."

Dr. Mehta continued, "Importantly, we are moving forward with our strategic geographic expansion strategy, with plans to file a Marketing Authorization Application to the European Medicines Agency for BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders in the second half of 2021. As we continue to advance our innovative neuroscience program, we remain committed to delivering transformative medicines to patients, expanding our regulatory footprint and creating value for our stockholders."

First Quarter 2021 and Recent Highlights

BXCL501-Neuroscience Program

BXCL501 is an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist, designed for the treatment of agitation and opioid withdrawal symptoms. The Company believes BXCL501 may directly target a causal agitation mechanism.

Reported positive topline results from the Phase 1b/2 TRANQUILITY trial of BXCL501 for the acute treatment of agitation associated with dementia.
Granted Breakthrough Therapy designation by the U.S. Food and Drug Administration ("FDA") to BXCL501 for the acute treatment of agitation associated with dementia.
End of Phase 2 meeting with the FDA planned in 2Q 2021 to discuss registrational trial.
Recruitment in 40 mcg supplemental dose cohort of BXCL501 in TRANQUILITY expansion study is progressing well.
Completed its New Drug Application ("NDA") submission to the FDA for BXCL501 for the acute treatment of agitation associated with schizophrenia and the acute treatment of agitation associated with bipolar disorders I and II.
Plan to submit Marketing Authorization Application ("MAA") with European Medicines Agency ("EMA") using SERENITY I and II data for the acute treatment of agitation associated with schizophrenia and bipolar disorders I and II in 2H 2021.
Reported results from the Phase 1b/2 study of BXCL501 for the treatment of opioid withdrawal symptoms. Primary safety endpoints were met, and numerically improved retention rates, a secondary endpoint, were demonstrated in multiple BXCL501 dose cohorts.
PLACIDITY enrollment was voluntarily paused to assess challenges posed in opening relevant clinical sites and enrolling delirium patients in the ICU setting, including as a result of the burden COVID-19 has placed on ICUs.
Strengthened BXCL501 IP portfolio with issuance of two Japanese patents – Patent No. 6868698, directed to methods of treating agitation, which will expire no earlier than 2037, and Patent No. 1681960, directed to film design, which will expire no earlier than 2041.
Commercial Highlights

Launched "Boiling Point" educational campaign to raise awareness about agitation associated with schizophrenia and bipolar disorder and its impact on patients and clinicians.
Completed U.S. market assessment initiatives and design of sales force size and structure.
Medical Affairs Initiatives

Deployed the Medical Science Liaison and Medical Manage Care teams and have begun scientific and medical-to-medical exchange with healthcare professionals and payers, respectively.
Presented two posters covering data from the pivotal Phase 3 SERENITY trials in patients with acute agitation associated with schizophrenia and bipolar disorders I and II at the American Psychiatric Association ("APA") Annual Meeting.
BXCL701-Immuno-Oncology Program

BXCL701 is an orally-delivered small molecule, immunomodulator designed to inhibit dipeptidyl peptidase ("DPP") 8/9 and block immune evasion by targeting Fibroblast Activation Protein ("FAP"). It has shown single agent anti-tumor activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients.

The adenocarcinoma cohort of the ongoing Phase 1b/2 trial of BXCL701 and pembrolizumab (KEYTRUDA) in aggressive forms of prostate cancer has met its efficacy bar to move to stage two. The trial will now continue to full enrollment and more complete efficacy data are expected to be presented at a scientific conference later this year.
Granted orphan drug designation by the FDA to BXCL701 for the treatment of soft tissue sarcoma.
Corporate Highlights

Appointed June Bray to the Company’s Board of Directors. Ms. Bray brings over forty years of extensive U.S. and global regulatory experience across all therapeutics areas, including psychiatry and neurology.
Appointed Javier Rodriguez as Chief Legal Officer and Corporate Secretary. Mr. Rodriguez brings over 20 years of extensive strategic and legal experience within the biopharmaceutical industry.
First Quarter 2021 Financial Results

Research and Development Expenses: Research and development expenses were $14.7 million during the first quarter of 2021, as compared to $12.4 million for the same period in 2020. The higher expenses were primarily attributable to an increase in personnel and related costs necessary to enlarge our development and medical teams. In addition, we experienced increased professional fees in conjunction with higher consulting fees and CMC costs related to BXCL501, as well as increased costs related to our RELEASE clinical trial. These increases were offset in part by a decrease in SERENITY I and II clinical trial costs.

General and Administrative Expenses: General and administrative expenses were $11.6 million for the first quarter of 2021, as compared to $2.6 million for the same period in 2020. The increase was primarily due to higher personnel related costs as well as costs related to our expansion in preparation of the potential commercial launch of BXCL501 in the U.S., and increased legal, professional fees, and insurance costs.

Net Loss: BioXcel reported a net loss of $26.4 million for the first quarter of 2021, compared to a net loss of $14.9 million for the same period in 2020.

The first quarter 2021 results include approximately $5.6 million in non-cash stock-based compensation costs, compared to non-cash stock-based compensation of $776,000 for the same period in 2020.

As of March 31, 2021, cash and cash equivalents totaled approximately $194 million.

Conference Call

BioXcel will host a conference call and webcast today at 8:30 a.m. ET. To access the call, please dial 877-407-2985 (domestic) and 201-378-4915 (international). A live webcast of the call will be available on the Investors sections of the BioXcel website and a replay of the call will be available through at least May 24, 2021. BioXcel Therapeutics website is available at www.bioxceltherapeutics.com.

BioXcel may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the "Email Alerts" option under the News / Events menu of the Investors section of its website at www.bioxceltherapeutics.com.