On February 25, 2021 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided a business update (Press release, Insmed, FEB 25, 2021, View Source [SID1234575586]).

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"2020 was the most productive and significant year in Insmed’s history, as we evolved from a single-product company with a successful U.S. launch to a truly global organization advancing three distinct, value-creating programs. I am incredibly proud of our team’s performance, which is all the more exemplary against the backdrop of COVID-19," commented Will Lewis, Chair and Chief Executive Officer of Insmed. "In the fourth quarter, we initiated both the Phase 3 ASPEN study of brensocatib in patients with bronchiectasis and the ARIKAYCE frontline clinical trial program in patients with NTM lung disease caused by MAC; advanced our ARIKAYCE launch in Europe and prepared for a potential approval and launch in Japan while maintaining steady performance in the U.S.; and advanced the development of TPIP, for which we announced positive Phase 1 data just last week. We begin 2021 with incredible momentum and believe we have the capabilities and talent to achieve our ambitious vision."

Recent Corporate Developments & Program Highlights

ARIKAYCE

ARIKAYCE has now been launched in both Germany and the Netherlands following approval by the European Commission in October 2020 for the treatment of NTM lung infections caused by MAC in adults with limited treatment options who do not have cystic fibrosis (CF). Consideration should be given to official guidance on the appropriate use of antibacterial agents. Insmed will continue to work to secure reimbursement and launch in other European markets throughout 2021 and into 2022.
In Japan, Insmed continues to anticipate launching ARIKAYCE in mid-2021, pending approval by the Ministry of Health, Labour, and Welfare of our application for the treatment of patients with NTM lung disease caused by MAC who did not sufficiently respond to prior treatment.
In December 2020, the first patient was dosed in the post-approval confirmatory frontline clinical trial program of ARIKAYCE in patients with NTM lung disease caused by MAC. The program consists of ARISE, an interventional study designed to validate a patient-reported outcome (PRO) tool in MAC lung disease, and ENCORE, a pivotal trial designed to establish, using the PRO tool validated in the ARISE trial, the clinical benefits and evaluate the safety of ARIKAYCE in patients with newly diagnosed MAC lung disease. More information on these studies is available at clinicaltrials.gov (ARISE: NCT04677543; ENCORE: NCT04677569).
Brensocatib

In November 2020, the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to brensocatib for the treatment of non-cystic fibrosis bronchiectasis (NCFBE), recognizing the potential for brensocatib to offer a new treatment approach for patients with bronchiectasis.
In December 2020, the first patient was dosed in the Phase 3 ASPEN study of brensocatib in patients with bronchiectasis. ASPEN is a global, randomized, double-blind, placebo-controlled Phase 3 study to assess the efficacy, safety, and tolerability of brensocatib in patients with bronchiectasis. Patients with bronchiectasis due to CF may not be enrolled in the study. More information on this study is available at clinicaltrials.gov (NCT04594369).
Insmed plans to initiate a Phase 2 pharmacokinetic multiple-dose study of brensocatib in patients with CF by mid-2021.
Insmed anticipates that topline data from STOP-COVID19, the investigator-initiated trial of brensocatib in hospitalized patients with COVID-19, will be shared by early Q2 2021.
TPIP

Insmed has completed the Phase 1 healthy volunteer trial designed to assess the pharmacokinetics and tolerability profile of TPIP. As reported on February 19, 2021, data from the study demonstrated that TPIP was generally well tolerated, with a pharmacokinetic profile that supports continued development with once-daily dosing. Insmed plans to present full data from this study at an upcoming medical meeting.
Insmed plans to advance the development of TPIP with two parallel studies in patients with pulmonary arterial hypertension (PAH). One is an open-label, proof-of-mechanism study to understand the impact of TPIP on pulmonary vascular resistance (PVR) over a 24-hour period. The Company anticipates sharing topline data from this study in the second half of 2021. The other study will aim to investigate the effect of TPIP on PVR and 6-minute walk distance over a 16-week treatment period using an up-titration, once-daily dosing schedule. The Company plans to initiate this trial in the fourth quarter of 2021.
The Company also plans to initiate a study of TPIP in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD).
Fourth Quarter and Full-Year 2020 Financial Results

Total revenue for the fourth quarter ended December 31, 2020 was $41.4 million, compared to total revenue of $45.7 million for the fourth quarter of 2019. Total revenue for the full year 2020 was $164.4 million, compared to total revenue of $136.5 million for the full year 2019.
Cost of product revenues (excluding amortization of intangible assets) was $10.9 million for the fourth quarter of 2020, compared to $8.7 million for the fourth quarter of 2019. For the full year 2020, cost of product revenues was $39.9 million compared to $24.2 million in 2019.
Research and development (R&D) expenses were $67.8 million for the fourth quarter of 2020, compared to $32.6 million for the fourth quarter of 2019. For the full year 2020, R&D expenses were $181.2 million compared to $131.7 million in 2019.
Selling, general and administrative (SG&A) expenses for the fourth quarter of 2020 were $56.0 million, compared to $50.2 million for the fourth quarter of 2019. For the full year 2020, SG&A expenses were $203.6 million, compared to $210.8 million in 2019.
For the fourth quarter of 2020, Insmed reported a GAAP net loss of $102.2 million, or $1.00 per share, compared to a GAAP net loss of $53.0 million, or $0.59 per share, for the fourth quarter of 2019. For the full year 2020, Insmed reported a GAAP net loss of $294.1 million, or $3.01 per share, compared to a GAAP net loss of $254.3 million, or $3.01 per share, in 2019.
Balance Sheet and Planned Investments

As of December 31, 2020, Insmed had cash and cash equivalents of $532.8 million. The Company’s total operating expenses for the fourth quarter of 2020 were $136.0 million and for the full year 2020 were $429.6 million. Adjusted R&D expenses for the fourth quarter of 2020 were $63.6 million and for the full year 2020 were $164.6 million. Adjusted SG&A expenses for the fourth quarter were $49.1 million and for the full year 2020 were $174.8 million. Adjusted R&D expenses and adjusted SG&A expenses are non-GAAP measures, which we describe further below.

The Company plans to invest in the following key activities in 2021:

(i)

U.S. commercialization of ARIKAYCE;

(ii)

clinical trial activities, including (a) advancement of the frontline clinical trial program for ARIKAYCE (ARISE and ENCORE), (b) advancement of the Phase 3 ASPEN study of brensocatib in patients with bronchiectasis, (c) advancement of clinical development of TPIP, and (d) the advancement of our earlier-stage research pipeline; and

(iii)

launch activities for ARIKAYCE in initial European countries and potential launch activities for ARIKAYCE in Japan.

Conference Call

Insmed will host a conference call beginning today at 8:30 AM Eastern Time. Shareholders and other interested parties may participate in the conference call by dialing (833) 340-0284 (domestic) or (236) 712-2425 (international) and referencing conference ID number 4261618. The call will also be webcast live on the company’s website at www.insmed.com.

A replay of the conference call will be accessible approximately two hours after its completion through March 27, 2021 by dialing (800) 585-8367 (domestic) or (416) 621-4642 (international) and referencing conference ID number 4261618. A webcast of the call will also be archived for 90 days under the Investor Relations section of the company’s website at www.insmed.com.

Non-GAAP Financial Measures

In addition to the U.S. generally accepted accounting principles (GAAP) results, this earnings release includes non-GAAP financial measures: adjusted R&D expenses, which Insmed defines as R&D expenses less stock-based compensation expense and depreciation; and adjusted SG&A expenses, which Insmed defines as SG&A expenses less stock-based compensation, depreciation, and certain milestones related to ARIKAYCE, which are payable under our amended agreements with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT). A reconciliation of these non-GAAP financial measures to their most directly comparable GAAP financial measure is presented in the table attached to this press release.

Management believes that these non-GAAP financial measures are useful to both management and investors in analyzing our ongoing business and operating performance. Management believes that providing this non-GAAP information to investors, in addition to the GAAP results, allows investors to view our financial results in the way that management views financial results. Management does not intend the presentation of these non-GAAP financial measures to be considered in isolation or as a substitute for results prepared in accordance with GAAP. In addition, these non-GAAP financial measures may differ from similarly named measures used by other companies.

About ARIKAYCE

ARIKAYCE is approved in the United States as ARIKAYCE (amikacin liposome inhalation suspension) and in the EU as ARIKAYCE Liposomal 590 mg Nebuliser Dispersion. Current international treatment guidelines recommend the use of ARIKAYCE for appropriate patients. ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney function. Insmed’s proprietary PULMOVANCE liposomal technology enables the delivery of amikacin directly to the lungs, where liposomal amikacin is taken up by lung macrophages where the infection resides, while limiting systemic exposure. ARIKAYCE is administered once daily using the Lamira Nebulizer System manufactured by PARI Pharma GmbH (PARI).

About PARI Pharma and the Lamira Nebulizer System

ARIKAYCE is delivered by a novel inhalation device, the Lamira Nebulizer System, developed by PARI. Lamira is a quiet, portable nebulizer that enables efficient aerosolization of ARIKAYCE via a vibrating, perforated membrane. Based on PARI’s 100-year history working with aerosols, PARI is dedicated to advancing inhalation therapies by developing innovative delivery platforms to improve patient care.

About Brensocatib

Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the treatment of patients with non-cystic fibrosis bronchiectasis (NCFBE) and other neutrophil-mediated diseases. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs. Brensocatib is an investigational drug product that has not been approved for any indication in any jurisdiction.

About TPIP

Treprostinil palmitil inhalation powder (TPIP) is a dry powder formulation of treprostinil palmitil, a treprostinil prodrug consisting of treprostinil linked by an ester bond to a 16-carbon chain. Developed entirely in Insmed’s laboratories, TPIP is a potentially highly differentiated prostanoid being evaluated for the treatment of patients with PAH and other rare and serious pulmonary disorders. TPIP is administered in a capsule-based inhalation device. TPIP is an investigational drug product that has not been approved for any indication in any jurisdiction.

IMPORTANT SAFETY INFORMATION FOR ARIKAYCE IN THE U.S.

WARNING: RISK OF INCREASED RESPIRATORY ADVERSE REACTIONS

ARIKAYCE has been associated with an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease that have led to hospitalizations in some cases.

Hypersensitivity Pneumonitis has been reported with the use of ARIKAYCE in the clinical trials. Hypersensitivity pneumonitis (reported as allergic alveolitis, pneumonitis, interstitial lung disease, allergic reaction to ARIKAYCE) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (3.1%) compared to patients treated with a background regimen alone (0%). Most patients with hypersensitivity pneumonitis discontinued treatment with ARIKAYCE and received treatment with corticosteroids. If hypersensitivity pneumonitis occurs, discontinue ARIKAYCE and manage patients as medically appropriate.

Hemoptysis has been reported with the use of ARIKAYCE in the clinical trials. Hemoptysis was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (17.9%) compared to patients treated with a background regimen alone (12.5%). If hemoptysis occurs, manage patients as medically appropriate.

Bronchospasm has been reported with the use of ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma, bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea exertional, prolonged expiration, throat tightness, wheezing) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (28.7%) compared to patients treated with a background regimen alone (10.7%). If bronchospasm occurs during the use of ARIKAYCE, treat patients as medically appropriate.

Exacerbations of underlying pulmonary disease has been reported with the use of ARIKAYCE in the clinical trials. Exacerbations of underlying pulmonary disease (reported as chronic obstructive pulmonary disease (COPD), infective exacerbation of COPD, infective exacerbation of bronchiectasis) have been reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (14.8%) compared to patients treated with background regimen alone (9.8%). If exacerbations of underlying pulmonary disease occur during the use of ARIKAYCE, treat patients as medically appropriate.

Anaphylaxis and Hypersensitivity Reactions: Serious and potentially life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients taking ARIKAYCE. Signs and symptoms include acute onset of skin and mucosal tissue hypersensitivity reactions (hives, itching, flushing, swollen lips/tongue/uvula), respiratory difficulty (shortness of breath, wheezing, stridor, cough), gastrointestinal symptoms (nausea, vomiting, diarrhea, crampy abdominal pain), and cardiovascular signs and symptoms of anaphylaxis (tachycardia, low blood pressure, syncope, incontinence, dizziness). Before therapy with ARIKAYCE is instituted, evaluate for previous hypersensitivity reactions to aminoglycosides. If anaphylaxis or a hypersensitivity reaction occurs, discontinue ARIKAYCE and institute appropriate supportive measures.

Ototoxicity has been reported with the use of ARIKAYCE in the clinical trials. Ototoxicity (including deafness, dizziness, presyncope, tinnitus, and vertigo) were reported with a higher frequency in patients treated with ARIKAYCE plus background regimen (17%) compared to patients treated with background regimen alone (9.8%). This was primarily driven by tinnitus (7.6% in ARIKAYCE plus background regimen vs 0.9% in the background regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background regimen vs 2.7% in the background regimen alone arm). Closely monitor patients with known or suspected auditory or vestibular dysfunction during treatment with ARIKAYCE. If ototoxicity occurs, manage patients as medically appropriate, including potentially discontinuing ARIKAYCE.

Nephrotoxicity was observed during the clinical trials of ARIKAYCE in patients with MAC lung disease but not at a higher frequency than background regimen alone. Nephrotoxicity has been associated with the aminoglycosides. Close monitoring of patients with known or suspected renal dysfunction may be needed when prescribing ARIKAYCE.

Neuromuscular Blockade: Patients with neuromuscular disorders were not enrolled in ARIKAYCE clinical trials. Patients with known or suspected neuromuscular disorders, such as myasthenia gravis, should be closely monitored since aminoglycosides may aggravate muscle weakness by blocking the release of acetylcholine at neuromuscular junctions.

Embryo-Fetal Toxicity: Aminoglycosides can cause fetal harm when administered to a pregnant woman. Aminoglycosides, including ARIKAYCE, may be associated with total, irreversible, bilateral congenital deafness in pediatric patients exposed in utero. Patients who use ARIKAYCE during pregnancy, or become pregnant while taking ARIKAYCE should be apprised of the potential hazard to the fetus.

Contraindications: ARIKAYCE is contraindicated in patients with known hypersensitivity to any aminoglycoside.

Most Common Adverse Reactions: The most common adverse reactions in Trial 1 at an incidence ≥5% for patients using ARIKAYCE plus background regimen compared to patients treated with background regimen alone were dysphonia (47% vs 1%), cough (39% vs 17%), bronchospasm (29% vs 11%), hemoptysis (18% vs 13%), ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%), musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs 10%), exacerbation of underlying pulmonary disease (15% vs 10%), diarrhea (13% vs 5%), nausea (12% vs 4%), pneumonia (10% vs 8%), headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash (6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs 1%), and chest discomfort (5% vs 3%).

Drug Interactions: Avoid concomitant use of ARIKAYCE with medications associated with neurotoxicity, nephrotoxicity, and ototoxicity. Some diuretics can enhance aminoglycoside toxicity by altering aminoglycoside concentrations in serum and tissue. Avoid concomitant use of ARIKAYCE with ethacrynic acid, furosemide, urea, or intravenous mannitol.

Overdosage: Adverse reactions specifically associated with overdose of ARIKAYCE have not been identified. Acute toxicity should be treated with immediate withdrawal of ARIKAYCE, and baseline tests of renal function should be undertaken. Hemodialysis may be helpful in removing amikacin from the body. In all cases of suspected overdosage, physicians should contact the Regional Poison Control Center for information about effective treatment.

U.S. INDICATION

LIMITED POPULATION: ARIKAYCE is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients.

This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Limitation of Use: ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.

On February 25, 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 fourth quarter and full-year ended December 31, 2020 and provided business updates (Press release, Akebia, FEB 25, 2021, View Source [SID1234575585]). The Company will host a conference call today, Thursday, February 25, 2021, at 9:00 a.m. Eastern Time.

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Akebia also announced a $60 million 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). MTPC has the exclusive rights to commercialize vadadustat in Japan, where it is currently marketed under the trade name Vafseo (vadadustat), and certain other Asian countries. Under the terms of the agreement with HCR, Akebia receives an upfront cash payment of $45 million and is eligible to receive an additional $15 million if certain sales milestones are achieved. In exchange, HCR has the right to receive Vafseo royalties and sales milestones due to the Company under its collaboration agreement with MTPC, subject to an annual cap of $13 million and an aggregate cap of $150 million. After the annual cap is met in a given calendar year, Akebia will recognize 85 percent of Vafseo royalties and sales milestones from MTPC for that year. After the aggregate cap is met, Akebia will recognize 100 percent of Vafseo royalties and sales milestones until this revenue stream ends under the terms of the Company’s collaboration agreement with MTPC. The transaction does not include potential future regulatory milestones to be paid by MTPC.

"2020 was a year of focused execution for Akebia as we advanced vadadustat, our investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), and executed on our commercial priorities. Importantly, we achieved this while continuing to provide patients with access to our therapies and keeping with our goal of maintaining a strong balance sheet," stated John P. Butler, Chief Executive Officer of Akebia. "With this forward momentum, we have line of sight to significant milestones in 2021, including our NDA submission for vadadustat for the treatment of anemia due to chronic kidney disease (CKD) in both adult patients on dialysis and not on dialysis expected by the middle of the second quarter of this year."

Butler continued, "As we advance vadadustat toward commercialization, subject to approval, we continue to execute on key commercial, development, and financial priorities. As dialysis is a primary focus, we’re thrilled to have recently added LeAnne Zumwalt to our Board of Directors. LeAnne’s perspective and extensive operating experience with one of the largest dialysis operators in the U.S. will help ensure that our market and commercial strategies are well aligned with the needs of dialysis providers and their patients. We’re also pleased to have announced a $60 million royalty monetization transaction with HCR that we believe strengthens our balance sheet and helps preserve both our strategic and financial flexibility while we continue investing for the successful launch of vadadustat, upon approval."

Akebia plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for vadadustat by the middle of the second quarter of this year for the treatment of anemia due to CKD in both adult patients on dialysis and adult patients not on dialysis. In addition, Akebia and its collaborator, Otsuka Pharmaceutical Co. Ltd. (Otsuka), are working in close collaboration to prepare a Marketing Authorization Application (MAA) for submission to the European Medicines Agency (EMA), expected this year.

Recent Business Highlights:

In February, the Company 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.
In November, the study design and methodology for Akebia’s global Phase 3 INNO2VATE program was published in Nephrology Dialysis Transplantation (NDT).
In October, the study design and methodology for Akebia’s global Phase 3 PRO2TECT program was published in American Heart Journal.
In October, Akebia completed a pre-NDA meeting with the FDA for vadadustat.
In October, top-line data from both INNO2VATE and PRO2TECT were presented at American Society of Nephrology Kidney Week 2020 Reimagined.
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.
Fourth Quarter and Full-Year 2020 Financial Results

Revenues: Total revenue was $56.7 million for the fourth quarter of 2020 compared to $69.6 million for the fourth quarter of 2019, and $295.3 million for the full-year 2020 compared to $335.0 million for the full-year 2019. The decline in both periods was driven by lower collaboration revenue consistent with the Company successfully completing its global Phase 3 clinical development 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).
Collaboration revenue was $22.1 million for the fourth quarter of 2020 compared to $40.6 million for the fourth quarter of 2019, and $166.4 million for the full-year 2020 compared with $223.9 million for the full-year 2019. The change in both periods is due to the timing of when vadadustat development expenses are incurred and the associated revenue is recognized on a percentage-of-completion basis. Collaboration revenue for the full-year 2020 includes $0.4 million in royalty revenue related to the commercial sale of Vafseo in Japan from MTPC, which launched in August of 2020.
Net product revenue was $34.6 million for the fourth quarter of 2020 compared with $28.9 million for the fourth quarter of 2019, an increase of 20 percent. Net product revenue was $128.9 million for the full-year 2020 compared to $111.1 million for the full-year 2019, an increase of 16 percent. While Akebia did not experience a significant impact from COVID-19 on revenues during the first nine months of 2020, the Company believes its revenue growth was negatively impacted during the fourth quarter of 2020 primarily as the kidney patient populations that it serves continue to experience both higher hospitalization and mortality rates due to COVID-19.
COGS: Cost of goods sold was $63.2 million for the fourth quarter of 2020 compared to $38.1 million for the fourth quarter of 2019. Excluding non-cash purchase accounting adjustments as a result of the merger with Keryx, the increase in the fourth quarter of 2020 was primarily driven by a $14.8 million non-cash charge related to excess purchase commitments. Cost of goods sold was $295.9 million for the full-year 2020, compared with $145.3 million for the full-year 2019. Cost of goods sold for 2020 includes a $115.5 million non-cash charge related to the impairment of the Auryxia intangible asset in the second quarter of 2020, a $25.1 million non-cash charge related to excess purchase commitments, and a $20.1 million non-cash charge for inventory write-downs largely related to a previously disclosed manufacturing quality issue related to Auryxia.
R&D Expenses: Research and development expenses were $37.6 million for the fourth quarter of 2020 compared to $80.4 million for the fourth quarter of 2019, and $218.5 million for the full-year 2020 compared to $323.0 million for the full-year 2019. The decline in both periods 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 $40.3 million for the fourth quarter of 2020 compared to $44.9 million for the fourth quarter of 2019, and $153.9 million for the full-year 2020 compared to $149.5 million for the full-year 2019.
Net Loss: Net loss was $87.0 million for the fourth quarter of 2020 compared to $94.5 million for the fourth quarter of 2019, and $383.5 million for the full-year 2020 compared to $279.7 million for the full-year 2019. The increase in net loss for the full-year 2020 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 December 31, 2020 were $268.7 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.
"As COVID-19 continues to adversely and disproportionately impact our patient population with higher hospitalization and mortality rates, we expect this will have a negative impact on future Auryxia revenue growth. While we are unable to fully quantify the impact of the COVID-19 pandemic on future revenues and revenue growth, we continue to work to position the Company to navigate these challenges. As such, our financial priorities remain focused on improving our cost structure and maintaining a strong balance sheet, as evidenced by our recent $60 million non-dilutive royalty transaction with HCR," stated David A. Spellman, Chief Financial Officer of Akebia Therapeutics.

Conference Call
Akebia will host a conference call at 9:00 a.m. Eastern Time today, Thursday, February 25th, to discuss its fourth quarter and full-year 2020 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 5455117. 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 March 3, 2021. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 5455117. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

On February 25, 2021 MannKind Corporation (NASDAQ:MNKD) reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Mannkind, FEB 25, 2021, View Source [SID1234575584]).

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"Our fourth quarter produced exceptional results, with $10.1 million in Afrezza net revenue and positive clinical data for Tyvaso DPI from the BREEZE study conducted by United Therapeutics," said Michael Castagna, Chief Executive Officer of MannKind Corporation. "UT also announced their plan to submit a new drug application for Tyvaso DPI to the FDA in April 2021. During the fourth quarter, we solidified our new direction with the acquisition of QrumPharma, which added a nebulized version of clofazimine to our pipeline of therapies for orphan lung diseases, and we entered into a collaboration agreement with Vertice for the co-promotion of Thyquidity, which is indicated for hyperthyroidism and is expected to expand our reach into endocrine diseases."

Fourth Quarter 2020 Results

Total revenues were $18.4 million for the fourth quarter of 2020, reflecting Afrezza net revenue of $10.1 million and collaborations and services revenue of $8.4 million. Afrezza net revenue increased 30% compared to $7.8 million in the fourth quarter of 2019, primarily driven by higher product demand with a more favorable mix of Afrezza cartridges and more favorable gross-to-net deductions. Collaborations and services revenue increased $0.2 million compared to the fourth quarter of 2019.

Afrezza gross profit for the fourth quarter of 2020 was $6.4 million compared to $3.1 million in the same period of 2019, an increase of $3.3 million, or 105%, that was driven by a combination of increased Afrezza revenue and a reduction in cost of goods sold.

In-process research and development expense for the fourth quarter of 2020 was $13.2 million, reflecting the acquisition of QrumPharma for approximately $12.8 million in total consideration and approximately $0.4 million in transaction costs. The acquisition of QrumPharma was accounted for as an asset acquisition and expensed on the date of acquisition as substantially all of the fair value of the assets acquired was concentrated in a single asset that consisted of in-process research and development in a pre-clinical development state.

Research and development expenses for the fourth quarter of 2020 were $1.5 million compared to $2.0 million for the fourth quarter of 2019. This decrease was mainly related to lower clinical trial spending.

Selling, general and administrative expenses for the fourth quarter of 2020 were $17.1 million compared to $15.7 million for the fourth quarter of 2019. This increase of $1.4 million, or 9%, was primarily attributable to a $1.2 million increase in personnel costs related to the expansion of our sales and medical field force.

During the fourth quarter of 2020, loss on foreign currency translation for insulin purchase commitments, which are denominated in Euros, was $4.0 million compared to $2.6 million for the fourth quarter of 2019. The fluctuation was due to a change in the U.S. dollar to Euro foreign exchange rate.

Interest expense on debt for the fourth quarter of 2020 was $2.4 million compared to $2.3 million for the fourth quarter of 2019.

The net loss for the fourth quarter of 2020 was $26.4 million, or $0.11 per share, compared to $14.3 million in the fourth quarter of 2019, or $0.07 per share. The increase in the net loss of $12.1 million was primarily due to the write-off of in-process research and development related to the acquisition of QrumPharma. On a non-GAAP basis, excluding the expense incurred for the acquisition of QrumPharma, the net loss for the fourth quarter of 2020 was $13.2 million, or $0.06 per share.

Twelve Months Ended December 31, 2020

Total revenues were $65.1 million for the year ended December 31, 2020, reflecting Afrezza net revenue of $32.3 million and collaborations and services revenue of $32.8 million. Afrezza net revenue increased 28% compared to $25.3 million for the year ended December 31, 2019, primarily driven by higher product demand with a more favorable mix of Afrezza cartridges, a price increase and more favorable gross-to-net deductions, all of which was partially offset by a reduction in sales to Biomm (Brazil). Collaborations and services revenue decreased $4.9 million compared to the full year ended December 31, 2019, primarily driven by a $5.8 million decrease in revenue recognized from the UT Research Agreement, which was substantially completed in the second quarter of 2019.

Afrezza gross profit was $17.2 million for the year ended December 31, 2020, an increase of $12.0 million, or 230%, compared to a gross profit of $5.2 million in the same period in 2019, primarily due higher commercial product sales combined with a reduction in cost of goods sold.

In-process research and development expense for the year ended December 31, 2020 was $13.2 million, reflecting the research and development acquired and expensed from the acquisition of QrumPharma for approximately $12.8 million in total consideration and approximately $0.4 million in transaction costs.

Research and development expenses for the year ended December 31, 2020 were $6.2 million compared to $6.9 million for the year ended December 31, 2019. This decrease of $0.7 million, or 9%, was primarily attributable to lower clinical trial spending.

Selling, general and administrative expenses for the year ended December 31, 2020 were $59.0 million compared to $74.7 million for the year ended December 31, 2019. This decrease of $15.6 million, or 21%, was primarily attributable a $9.3 million decrease in costs for television advertising for Afrezza, a $4.1 million decrease in promotional and marketing activities in response to the COVID-19 pandemic and a $2.5 million decrease in professional fees.

An impairment of $1.9 million was recognized for the year ended December 31, 2020 on a commitment asset and debt issuance costs related to future funding commitments of the MidCap Credit Facility. There were no asset impairments for the year ended December 31, 2019.

For the year ended December 31, 2020, foreign currency translation for insulin purchase commitments, which are denominated in Euros, resulted in a loss of $8.0 million compared to a gain of $1.9 million for the year ended December 31, 2019. The fluctuation was due to a change in the U.S. dollar to Euro foreign exchange rate.

Interest expense on debt for the year ended December 31, 2020 was $9.5 million compared to $10.9 million for the year ended December 31, 2019. This $1.4 million decrease was primarily attributable to a $3.4 million milestone obligation to Deerfield that was achieved in the third quarter of 2019 and a decrease of $0.8 million of interest expense related to the Deerfield Credit Facility, which was extinguished in the third quarter of 2019. This decrease was partially offset by an increase in interest expense from the MidCap Credit Facility of $2.3 million and an increase in interest expense from our Mann Group promissory notes of $0.6 million in 2020.

The net loss for the year ended December 31, 2020 was $57.2 million, or $0.26 per share, compared to $51.9 million net loss for the year ended December 31, 2019, or $0.27 per share. The higher net loss was mainly attributable to the write-off of in-process research and development related to the acquisition of QrumPharma and a loss on foreign currency translation related to insulin purchase commitments denominated in Euros, offset by a decrease in selling, general and administrative expenses. On a non-GAAP basis, excluding the expense incurred for the acquisition of QrumPharma, the net loss for the year ended December 31, 2020 was $44.0 million, or $0.20 per share.

Cash, cash equivalents, restricted cash, and short-term investments at December 31, 2020 was $67.2 million compared to $50.2 million at December 31, 2019.

Debt Reductions Subsequent to December 31, 2020

Pursuant to the terms of the senior convertible notes, the Company forced the conversion of all $5.0 million in principal of such notes into 1,666,667 shares of the Company’s common stock.

In addition, the Mann Group converted $9.6 million of principal and $0.4 million of accrued interest on its convertible promissory note into 4.0 million shares of the Company’s common stock. As of the date hereof, $53.4 million in principal remains outstanding under the promissory notes held by the Mann Group ($18.4 of which is convertible).

Sale-Leaseback of the Danbury Manufacturing Facility

Subsequent to December 31, 2020, the Company entered into a non-binding letter of intent ("LOI") with a third party to sell and lease back a portion of the Company’s Danbury manufacturing facility and administrative offices. The terms of the LOI include a sales price of approximately $95 million – $105 million, a lease term of 20 years with four 5-year renewal options, and annual rent of approximately $10 million – $11 million at the beginning of the lease. If the transaction is completed, the Company intends to use the proceeds for general corporate purposes, and may also pay down a portion of its senior secured debt. The completion of the transactions contemplated by the LOI is subject to certain conditions, including the negotiation of satisfactory definitive agreements and satisfactory results of the buyer’s inspections and other investigations, all of which are anticipated to be completed during the first quarter of 2021. However, there can be no assurances that this proposed transaction will be completed in the timeframe or on the principal terms set forth above, or at all.

Non-GAAP Measures

Certain financial information contained in this press release is presented on both a reported basis (GAAP) and a non-GAAP basis. Reported results were prepared in accordance with GAAP whereas non-GAAP measures exclude items described in the reconciliation tables below. Non-GAAP financial information is intended to portray the results of our baseline performance, supplement or enhance management, analysts and investors overall understanding of our underlying financial performance and facilitate comparisons among current and past periods. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP.

Conference Call

MannKind will host a conference call and presentation webcast to discuss these results today at 9:00 a.m. Eastern Time. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at View Source under News & Events. A replay will be available on MannKind’s website for 14 days.

On February 24, 2021 Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of fully engineered circular RNA therapeutics (oRNAs), reported the completion of its $80 million Series A financing (Press release, OrnaTherapeutics, FEB 24, 2021, View Source [SID1234626172]). Founded on groundbreaking research from the Massachusetts Institute of Technology (MIT) and built by MPM Capital, Orna utilizes elegant molecular design and delivery technologies to overcome the limitations of linear mRNA therapeutics and unlock RNA’s full therapeutic potential to treat a wide range of diseases including cancer, autoimmune, and genetic disorders. Led by Thomas M. Barnes, Ph.D., as Chief Executive Officer, Orna’s team of skilled scientists, molecular engineers, and industry veterans leverage their broadly applicable oRNA platform to dramatically change and improve current approaches to treatment with the potential to make a difference in the lives of patients. The financing was co-led by MPM Capital, Taiho Ventures, and F2 Ventures, with participation from leading strategic investors: Kite, a Gilead Company, Bristol Myers Squibb, Astellas Venture Management, Novartis Institutes for Biomedical Research, and the PAGS Group.

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Orna’s engineered circular RNAs have several key advantages, including superior protein expression, simpler and more cost-effective manufacturing, and improved delivery. Unlike other RNA therapies, oRNA does not require the addition of cap or tail structures and does not require the use of modified nucleotides to address innate immune responses. In addition, oRNAs are designed to drive unparalleled protein production while resisting degradation inside the body.

"Our elegant solution to the circular RNA engineering problem has allowed us to reveal that oRNA is simply the better format for long coding RNA," said Dr. Barnes. "Furthermore, our initial combination of oRNAs with technology to deliver them to immune cells has paved the way to create groundbreaking new therapies to fundamentally change the way we treat cancer and autoimmune diseases."

The company was founded by (Robert) Alex Wesselhoeft, Ph.D., Raffaella Squilloni, M.B.A. and Professor Dan Anderson, Ph.D. (MIT), and was seeded and incubated by MPM Capital, with Shinichiro Fuse, Ph.D. serving as the interim CEO, and Brian Goodman, Ph.D. as head of Business Development. "We believe that oRNA is the future, and Orna has built the right team to execute on this vision," said Ansbert Gadicke, M.D., co-founder and Managing Director, MPM Capital, Board Chair of Orna Therapeutics. "Orna’s elegant and meticulously calculated approach has the potential to forever change the field of mRNA therapeutics."

The potential applications of Orna’s technology are far reaching. In particular, Orna believes it can apply its oRNA technology to potentially address the limitations of current immunotherapies by delivering chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (in situ CAR therapy). The Series A financing will enable Orna to bring its initial programs to IND enablement, while continuing to advance its robust platform and build the capabilities to support Orna’s mission in its new office and laboratory space located in Cambridge, Massachusetts. Orna is supported by an experienced and seasoned Board of Directors and scientific advisors, including:

Daniel Anderson, Ph.D., Professor, MIT
Sakae Asanuma, CFA, MBA, President, Taiho Ventures
Thomas M. Barnes, Ph.D., CEO, Orna Therapeutics
Shinichiro Fuse, Ph.D., Managing Director, MPM Capital
Ansbert Gadicke, M.D. (chair), co-founder and Managing Director, MPM Capital
Morana Jovan-Embiricos, Managing Partner, F2 Ventures
Francesco Marincola, M.D., SVP and Global Head of Cell Therapy Research of Kite

On February 24, 2021 Verity Pharmaceuticals reported that it has received a notice of compliance with conditions (NOC/c) from Health Canada for the VERITY-BCG (Bacillus Calmette-Guérin [BCG]: Strain Russian BCG-I) (Press release, veritypharma, FEB 24, 2021, View Source [SID1234605586]). This approval is welcome news for patients and health care providers who have endured a Canadian and global shortage of this important bladder cancer drug.

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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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Bacillus Calmette-Guérin (BCG) is a proven immunotherapy of choice for bladder cancer. It is used as first-line intravesical therapy following tumor resection of non-muscle-invasive bladder cancer. Primary producers of BCG announced shortages within the last decade, leading to a worldwide scarcity of the treatment. This new source of BCG from Verity Pharma is expected to be available by mid 2021.

"Canada’s multi-year BCG drug shortage will now be a thing of the past with this approval," said Howard Glase, Chief Executive Officer, Verity Pharma. "We know these drug shortages were leading to rationing and dose sparing of this crucial bladder cancer drug and patients had delayed or even cancelled treatments. Canadian patients will now have full access to this life-saving therapy."

According to an article by the National Centre for Biotechnology Information, "physicians treating patients affected by non-muscle invasive bladder cancer (NMIBC) have been in shock during the last six years since manufacturing restrictions on the production of the first-option medicine, Mycobacterium bovis Bacillus Calmette-Guérin (BCG), have resulted in worldwide shortages."i

Bladder Cancer Canada states that bladder cancer is the 5th most common cancer in Canada and almost 12,000 people will be diagnosed with bladder cancer this year alone.

Verity has partnered with Serum Institute of India, which manufactures BCG. Serum Institute is the world’s largest manufacturer of vaccines.

"Verity is proud to continue bringing critical drugs to Canadians. VERITY-BCG is an example of our expertise in supplying life-saving therapeutic options to help fulfill unmet patient needs," said Glase.

About VERITY-BCG

VERITY-BCG is an adjuvant therapy after transurethral resection (TUR) of a primary or relapsing superficial papillary urothelial cell carcinoma of the bladder stage Ta (grade 2 or 3) or T1 (grade 1, 2, or 3), without concomitant carcinoma in situ. It is only recommended for stage Ta grade 1 papillary tumors, when there is judged to be a high risk (>50%) of tumor recurrence.

More information is available here: View Source