On May 10, 2021 Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN; the "Company"), a biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases including rare disorders, reported financial results for the first quarter ended March 31, 2021, and provided a review of recent accomplishments and anticipated upcoming milestones (Press release, Biohaven Pharmaceutical, MAY 10, 2021, View Source [SID1234579545]).

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Vlad Coric, M.D., Chief Executive Officer of Biohaven commented, "NURTEC ODT’s performance, with its differentiated efficacy and safety profile, continues to be bolstered by overwhelmingly positive patient and provider sentiment to date. We are highly encouraged by the continued success of NURTEC ODT, with net revenues of $43.8 million in the first quarter exceeding expectations."

Dr. Coric added, "In addition to pursuing multiple life-cycle management and also non-migraine indications with our CGRP-antagonist franchise, we continue to make strides in advancing the many programs across our pipeline, recognizing the high unmet need for treatments within our MPO inhibitor and glutamate modulating platforms. The impressive enrollment of verdiperstat in both the MSA trial and the Healey ALS trial is a testament to our commitment to delivering solutions within the neurodegenerative space. We look forward to announcing topline results across multiple assets in our pipeline over the next year."

First Quarter and Recent Business Highlights

Continued strong uptake of NURTEC ODT – With significant market opportunity ahead, Biohaven remains focused on investing in the ongoing success of NURTEC ODT, defined by growth of oral CGRP class volume and increasing NURTEC ODT market share in the U.S. Despite first quarter prescription softness across the industry, we are pleased to see continued substantive growth quarter over quarter in product demand and net sales. NURTEC ODT retains its ~89% commercial coverage, while expanding coverage in Medicaid and Medicare throughout ’21.

NURTEC ODT Approved in Israel and United Arab Emirates for Acute Treatment of Migraine – In March, the Company announced that both regions obtained market authorizations by the Israeli Ministry of Health and the United Arab Emirates Ministry of Health, respectively. Distribution agreements are in place with Medison Pharma in Israel and Genpharm Services in the Middle East and Gulf Region.

Submitted EU MAA of first ever dual acting filing for the treatment of migraine, inclusive of both acute and preventive treatment – The Company submitted the MAA for rimegepant dual activity, inclusive of acute and prevention of migraine. The submission has been validated by the European Medicines Agency and the EU procedure has been initiated. If approved, Vydura will be the commercial name for Rimegepant in the EU.

Enrolled First Patient in Phase 2/3 of Oral Zavegepant – In March, the Company enrolled the first patient in a Phase 2/3 clinical trial for the preventive treatment of migraine. As a part of the previously announced funding agreement with Royalty Pharma to advance the development of zavegepant, Biohaven concurrently received a $100 million milestone payment. The randomized, double-blind, placebo-controlled trial will enroll approximately 2,900 people with migraine and will evaluate the efficacy and safety of 100 mg and 200 mg doses of oral zavegepant. The zavegepant program encompasses intranasal zavegepant as well as oral formulations of zavegepant for migraine and non-migraine indications.

Enrolled More Than 50% of Participants Ahead of Timeline in Verdiperstat Pivotal Healey ALS Platform Trial – In March, more than 50% of participants were enrolled in the verdiperstat regimen of HEALEY ALS Platform Trial, which is evaluating the efficacy of several treatments including verdiperstat in people with amyotrophic lateral sclerosis ("ALS"). The trial, conducted across over 50 sites, will study approximately 160 adults with ALS. Participants will be randomized in a 3-to-1 ratio to be treated with verdiperstat 600 mg oral tablet twice daily or placebo for 24 weeks. Verdiperstat is a potential first-in-class, brain-penetrant, selective inhibitor of myeloperoxidase in development by Biohaven for the treatment of neurodegenerative diseases, including ALS and multiple system atrophy.

Initiated pivotal Phase 3 trial of troriluzole in OCD – In January, the Company announced that it had commenced enrollment in a Phase 3 clinical trial of troriluzole in patients with OCD. Biohaven is advancing the 280 mg once daily dose of troriluzole into two double-blind, placebo-controlled Phase 3 clinical trials with identical study designs and plans to enroll approximately 600 patients in each of these adjunctive treatment trials across study sites in both the United States and Europe. Phase 3 trial enhancements include increased sample size to adequately power for previously observed treatment effects, a higher dose of troriluzole and optimized clinical trial design to minimize the placebo effect.

Acquired novel immune modulating platforms and target degrader platform – In January, the Company acquired Kleo Pharmaceuticals, Inc. (Kleo), including its MATE and ARM platforms, and exclusively licensed a target degrader platform from Yale University based on intellectual property derived from ground-breaking research in the laboratory of Professor David Spiegel. The Company assumed Kleo’s laboratory facilities located in Science Park in New Haven, Connecticut and formed Biohaven Labs to serve as the integrated chemistry and discovery research arm of Biohaven. Biohaven Labs will continue several existing Kleo discovery partnerships, including with the Bill and Melinda Gates Foundation for the development of a Hyperimmune Globulin Mimic for COVID-19 and PeptiDream for the development of immuno-oncology therapeutics.

Announced BHV-1200 Demonstrates Effective Neutralization of Multiple Strains of COVID-19 – In February, the Company announced that a hyperimmune globulin mimic developed with Biohaven’s proprietary MATE platform has demonstrated functional binding and neutralization of the SARS-CoV-2 virus, including the strains known as the "English" and "South African" variants (also known as B.1.1.7 and B.1.351, respectively). The Company intends to advance BHV-1200 into a full clinical development program. Accelerated development of the COVID-19 MATE program has been supported by the Bill and Melinda Gates Foundation. In addition, the in vitro data indicate that BHV-1200 may activate important immune system components including antibody-dependent cellular phagocytosis and antibody dependent cellular cytotoxicity. Biohaven’s proprietary MATE-conjugation technology could also be used against other infectious diseases by changing the targeting moiety of its antibody binders.

Upcoming Milestones:
Biohaven is continuing to support the launch of NURTEC ODT and develop its product candidates through clinical and preclinical programs in a number of common and rare disorders. The Company expects to reach significant pipeline milestones with its CGRP receptor antagonists, glutamate modulators, and myeloperoxidase inhibitors.

Biohaven expects to:

•Continue to advance the commercialization of NURTEC ODT (rimegepant) for the acute treatment of migraine.
•Prepare rimegepant toward potential commercialization for the preventive treatment of migraine in the second quarter of 2021, if U.S. FDA approval is received.
•Report topline of intranasal zavegepant in the acute treatment of migraine in the second half of 2021.
•Report topline of verdiperstat for the treatment of MSA in the third quarter of 2021.
•Complete enrollment of verdiperstat for the treatment of ALS in the fourth quarter of 2021.
•Report topline of troriluzole in Spinocerebellar Ataxia in the fourth quarter of 2021 or early 2022.
•Report topline of troriluzole in OCD in 2022.

First Quarter Financial Results

Product Revenues, Net: Net product revenue was $43.8 million for the three months ended March 31, 2021, compared to $1.2 million for the three months ended March 31, 2020. The increase of $42.7 million in net product revenues is primarily due to a full quarter of NURTEC ODT sales during the three months ended March 31, 2021 compared to a partial quarter of NURTEC ODT sales during the three months ended March 31, 2020. The Company began selling NURTEC ODT in March 2020.

Cash and Marketable Securities: Cash, restricted cash, and marketable securities as of March 31, 2021, was $570.9 million, compared to $357.4 million as of December 31, 2020. The increase is primarily driven by proceeds from sales of common shares and receipt of the milestone payment from Royalty Pharma during the three months ended March 31, 2021.

Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $107.1 million for the three months ended March 31, 2021, compared to $56.1 million for the three months ended March 31, 2020. The increase of $51.0 million was primarily due to expenses from later stage trials in the zavegepant programs of $17.5 million (including clinical drug supply purchases), an increase of $13.8 million in non-cash share based compensation expense due to the Company’s annual equity incentive awards being granted in three months ended March 31, 2021 for performance year 2020 as compared to the 2019 annual equity incentive awards granted in the three months ended December 31, 2019, and one-time upfront license expenses of $7.9 million and development milestones of $5.0 million in three months ended March 31, 2021.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses, including non-cash share-based compensation costs, were $159.5 million for the three months ended March 31, 2021, compared to $95.6 million for the three months ended March 31, 2020. The increase of $64 million was primarily due to spending to support commercial sales of NURTEC ODT for a full fiscal quarter in 2021 vs a partial quarter in 2020 due to timing of the commercial launch in March 2020. Less than half of the SG&A expense was for commercial organization personnel costs, excluding non-cash share-based compensation expense. Non-cash share-based compensation expense was $28.7 million for the three months ended March 31, 2021, an increase of $18.0 million as compared to the same period in 2020. The increase in non-cash share based compensation expense was primarily due to the Company’s annual equity incentive awards timing, as noted above.

Net Loss: Biohaven reported a net loss attributable to common shareholders for the three months ended March 31, 2021 of $265.0 million, or $4.27 per share, compared to $172.9 million, or $3.07 per share for the same period in 2020. Non-GAAP adjusted net loss for the three months ended March 31, 2021 was $188.4 million, or $3.04 per share, compared to $134.9 million, or $2.39 per share for the same period in 2020. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges, non-cash interest expense related to the accounting for mandatorily redeemable preferred shares and liability related to sale of future royalties, changes in the fair value of derivatives, gains or losses from equity method investment, collaboration and license upfront expenses, and accrued development milestone payments. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

Conference Call Information
As previously announced, the Company will hold a conference call to discuss its first quarter 2021 results today at 8:30 a.m. EDT. To access the call, please dial 877-407-9120 (domestic) or 412-902-1009 (international). The conference call webcast, and accompanying slide presentation, can be accessed through the "Investors" section of Biohaven’s website at www.biohavenpharma.com. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. A replay of the call will be made available for two weeks following the conference call. To hear a replay of the call, dial 877-660-6853 (domestic) or 201-612-7415 (international) with conference ID 13718062. An archived webcast will be available on Biohaven’s website.

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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Discover why more than 1,500 members use 1stOncology™ to excel in:

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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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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"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.