On February 25, 2021 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a development-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for 2020 (Press release, Y-mAbs Therapeutics, FEB 25, 2021, View Source [SID1234575667]).

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"We are pleased with our 2020 financial results, especially seen in conjunction with the approval of DANYELZA, and the sale of our Priority Review Voucher for $105 million. For omburtamab, we are working closely with the FDA to address the Agency’s request for additional data, and we believe we have made progress and continue to work very hard on resubmitting the BLA. We also entered into a licensing agreement for Greater China for DANYELZA and omburtamab including milestones of up to $120 million and distribution agreements for DANYELZA and omburtamab for Eastern Europe, Russia and Israel, and thereby secured expanded access for children around the world to this important immunotherapy," stated Thomas Gad, founder, Chairman and President.

Dr. Claus Moller, Chief Executive Officer, continued, "We successfully completed a follow-on offering with gross proceeds of approximately $115.0 million earlier this week, and in parallel, we are making good progress in the clinic and have continued to advance the earlier stage programs in our pipeline. Nivatrotamab, our leading bispecific antibody, recently received ODD and RPDD from the FDA and our INDs for nivatrotamab in Small Cell Lung Cancer (Phase 2) and 177Lu-omburtamab-DTPA for medulloblastoma (Phase 1/2) and B7-H3 positive CNS/leptomeningeal metastasis in adults (Phase 1/2) were recently cleared by the FDA."

Fourth Quarter 2020 and Recent Corporate Developments

Subsequent to the end of the fourth quarter, on February 17, 2021, Y-mAbs announced the pricing of a follow-on shelf public offering, resulting in gross proceeds to the Company of approximately $115.0 million.

On December 28, 2020 Y-mAbs announced that it entered into a definitive agreement to sell its DANYELZA Priority Review Voucher to United Therapeutics Corporation for $105 million. Under the terms of the license agreement with Memorial Sloan Kettering, Y-mAbs will retain 60% of the net proceeds received from the sale. The transaction closed in January 2021.

On December 18, 2020, Y-mAbs announced that it had entered into a license agreement with SciClone Pharmaceuticals International Ltd to be the exclusive development and commercialization partner of DANYELZA and omburtamab for the treatment of pediatric patients in China, including upfront, approval and sales milestones of up to $120 million.

On December 18, 2020, Y-mAbs announced that it had entered into a distribution agreement with Swixx BioPharma AG to be the exclusive distributor of DANYELZA and omburtamab for the treatment of pediatric patients in Eastern Europe and Russia.

On December 9, 2020, Y-mAbs announced an update on DANYELZA data from the Company’s Study 201, which was presented at the ESMO (Free ESMO Whitepaper) Immunocology-Oncology Virtual Congress 2020.

On December 4, 2020, Y-mAbs announced that it had entered into a license and distribution agreement with Takeda Israel for the registration and commercialization of DANYELZA and omburtamab for the treatment of pediatric patients in the State of Israel.

On November 25, 2020, Y-mAbs announced that the FDA had approved DANYELZA for the treatment relapsed or primary refractory high-risk neuroblastoma.

On November 19, 2020, Y-mAbs announced that a clinical update of omburtamab for the treatment of diffuse intrinsic pontine glioma was presented at the SNO Virtual Annual Meeting.

On October 26, 2020, Y-mAbs announced that the FDA had cleared the Company’s IND for 177Lu-omburtamab-DTPA for the treatment of B7-H3 positive CNS and Leptomeningeal Metastasis from tumors in adult patients.

On October 14, 2020, Y-mAbs announced that the FDA had cleared the Company’s Investigational New Drug application for 177Lu-omburtamab-DTPA for the treatment of medulloblastoma, which is the most common type of primary brain cancer in children.

On October 7, 2020, Y-mAbs announced that the FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation for its leading bispecific antibody product candidate, nivatrotamab, for the treatment of neuroblastoma.

On October 5, 2020, Y-mAbs announced that it had received a Refusal to File letter from the FDA for the omburtamab BLA for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma. Subsequently, Y-mAbs has been in close dialog with the Agency to amend the BLA with the goal of resubmitting by the end of the second quarter or in the third quarter 2021.
Financial Results

Y-mAbs reported a net loss of $119.3 million, or ($2.97) per basic and diluted share, for the year ended December 31, 2020, compared to a net loss of $81.0 million, or ($2.30) per basic and diluted share, reported for the year ended December 31, 2019.

Revenues and Related Royalties Expense

Y-mAbs reported net revenues of $20.8 million for the year ended December 31, 2020 related to its licensing agreements in China and Israel. No revenues were reported for the year ended December 31, 2019.

Additionally, there were $2.2 million in royalties expense associated with the license revenue reported for the year ended December 31, 2020. No royalties expense was reported for the year ended December 31, 2019.

Operating Expenses

Research and Development
Research and development expenses were $93.7 million for the twelve months ended December 31, 2020, compared to $63.5 million for the twelve months ended December 31, 2019, an increase of $30.2 million. The increase in research and development expenses primarily reflects the following:

$13.4 million increase in personnel costs;
$13.2 million increase in milestones and license acquisition cost;
$1.1 million increase in professional and consulting fees; and
$1.6 million increase in outsourced services and supplies costs.
General and Administration
General and administrative expenses were $44.8 million for the twelve months ended December 31, 2020, compared to $19.5 million for the twelve months ended December 31, 2019, an increase of $25.3 million. The increase in general and administrative expenses primarily reflects the following:

$12.7 million increase in commercial infrastructure costs;
$8.9 million increase in personnel costs;
$2.1 million increase in business insurance; and
$2.1 million in professional fees.
Cash and Cash Equivalents

The Company had approximately $114.6 million in cash and cash equivalents as of December 31, 2020.

Webcast and Conference Call

The Company will host a conference call on Friday, February 26, 2021 at 9 a.m. Eastern Time. To participate in the call, please dial 877-407-0792 (domestic) or 201-689-8263 (international) and reference the access code 13716724. A webcast will be available at: View Source

On February 25, 2021 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported financial and operational results for the fourth quarter and year ended December 31, as well as guidance for anticipated 2021 milestones (Press release, Rocket Pharmaceuticals, FEB 25, 2021, View Source [SID1234575683]).

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"2021 is poised to be another exciting year for Rocket," said Gaurav Shah, M.D., Chief Executive Officer of Rocket. "We anticipate clinical data from all five of our gene therapy programs, initiation of our first in-house AAV cGMP production from our new manufacturing facility, and continued strengthening of our organization, manufacturing capabilities and infrastructure. We look forward to continued validation of our gene therapy platforms with updated results from registrational studies in FA and LAD-I, longer term findings in Danon Disease and PKD, and clinical proof-of-concept for IMO. Under strengthened leadership, we will complete buildout of Rocket’s new R&D and manufacturing facility leading to our first in-house cGMP production and work toward commercial readiness. Achieving these milestones will advance our mission to help improve the lives of patients facing rare and devastating childhood disorders."

"This new year comes on the heels of a seminal period in which we achieved transformational results across all four clinical programs including the near doubling of hemoglobin in our PKD trial, bone marrow normalization in our FA program, and reversal of disease phenotype in our Danon Disease study. These results demonstrated the significant potential of our platforms to change the lives of patients across many rare disease indications who currently have limited or no treatment options. We also expanded our organizational, R&D, and manufacturing capabilities toward commercial preparedness, and substantially strengthened our balance sheet. This work was achieved during a disruptive global pandemic, thanks to our employees, patients, partners, and the scientific community as a whole who met every challenge with determination and care. We look forward to continuing our steady progress by building on these results with hard work and perseverance as we move forward in 2021."

Key Pipeline and Operational Updates

Announced positive preliminary clinical data from RP-A501 trial for Danon Disease. The Company reported positive gene expression, clinical biomarker and preliminary functional data from the Phase 1 trial. Preliminary results from the three patients treated at the low dose demonstrated that RP-A501 was generally well tolerated with a manageable safety profile. In the higher-dose cohort, one patient experienced a drug-related severe adverse event related to complement activation. The patient’s risk was enhanced by high weight, vector dose, and pre-existing adeno-associated virus (AAV) immunity. All patients have fully recovered from immune-related sequelae. All three low dose participants demonstrated evidence of cardiac LAMP2B expression by Western blot and/or immunohistochemistry, and two who were compliant with the immunosuppressive regimen showed >50% expression and clinical biomarker improvements consistent with improved cardiac function. Brain natriuretic peptide and autophagic vacuoles improved in all three patients, while creatine kinase myocardial band either improved or stabilized in the immunosuppressive-compliant patients. Benefit observed in all three patients serves as clinical proof-of-concept. The Phase 1 trial continues to enroll patients with updated data anticipated in the second half of 2021.
Announced positive longer-term updates from the RP-L102 Fanconi Anemia (FA) and RP-L201 Leukocyte Adhesion Deficiency-I (LAD-I) programs supporting potential registration path using "Process B". The RP-L102 data presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper) are from seven of the nine patients treated in the U.S. Phase 1 and global Phase 2 studies. RP-L102 was generally well tolerated with no significant safety issues reported with infusion or post-treatment. Evidence of preliminary engraftment was observed in five out of seven patients. Two of three patients with greater than 12-months follow-up showed evidence of increasing engraftment, mitomycin-C resistance and stable blood counts. One patient’s course was complicated by Influenza B resulting in progressive bone marrow failure. The patient received a successful bone marrow transplant. The RP-L201 data presented are from three pediatric patients with severe LAD-I. RP-L201 was well tolerated with no safety issues reported with infusion or post-treatment. All patients achieved hematopoietic reconstitution within 5-weeks. The two patients with greater than 6-months follow-up demonstrated sustained CD18 expression of 23% to 40%, far exceeding the 4-10% threshold associated with survival into adulthood. Further updates from the RP-L102 and RP-L201 programs are anticipated in the second quarter of 2021.
Announced preliminary clinical data from the RP-L301 Pyruvate Kinase Deficiency (PKD) program demonstrating its potential to address the root cause of the disease for the first time at the genetic level. The data presented at ASH (Free ASH Whitepaper) are from two adult patients with significant anemia and transfusion requirement. RP-L301 was well tolerated in the first patient treated, with no serious safety issues or infusion-related complications observed 3-months post treatment. This patient nearly doubled hemoglobin levels to a normal range and normalized additional hemolysis markers. The second patient was recently treated with RP-L301. Additional Phase 1 data are anticipated in the second half of 2021.
Continued buildout of new Research and Development (R&D) and Manufacturing facility. In January, Rocket announced plans for its new R&D and Chemistry, Manufacturing and Controls (CMC) operation which will also serve as the Company’s new headquarters in Cranbury, New Jersey. This new 103,720 ft2 facility will support clinical development of Rocket’s growing pipeline of lentivirus and AAV gene therapies, with space scaled for AAV Current Good Manufacturing Practice (cGMP) production. The other half features state-of-the-art R&D labs to support the expanding pipeline and Quality Control laboratories to support CMC development for process and analytics. The first cGMP production at this facility will be initiated in 2021.
Announced FDA clearance of Investigational New Drug (IND) application for RP-L401 gene therapy for Infantile Malignant Osteopetrosis (IMO). RP-L401 is Rocket’s lentiviral vector-based gene therapy for the treatment of IMO, a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities, and bone marrow failure. Preliminary data are anticipated in the second half of 2021.
Expanded clinical sites for FA, Danon, LAD-I and IMO trials, adding to global centers of excellence. The newly added centers, which include some of the leading gene therapy research programs in the world, include: Great Ormond Street Hospital in London, Children’s Hospital of Philadelphia (CHOP), the University of Minnesota, the University of California, Los Angeles (UCLA), and the University of Colorado. These additional sites will expand patient access to Rocket’s clinical trials worldwide.
Received Fast Track Designation for IMO and Rare Pediatric Disease Designation for Danon Disease programs from FDA. The FDA’s Fast Track program facilitates the development of products intended to treat serious conditions that have the potential to address unmet medical needs and enables greater access to the FDA for the purpose of expediting the product’s development, review, and potential approval. The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S.
The California Institute for Regenerative Medicine (CIRM) awarded Rocket a $3.7 million CLIN2 grant award to support the clinical development of RP-L401 for IMO. Proceeds from the grant will help fund clinical trial costs, as well as provide manufactured drug product for Phase 1 patients enrolled at the U.S. clinical trial site at UCLA.
Strengthened organization with new Officer appointments. In January 2021, Carlos Garcia Parada joined Rocket as Chief Financial Officer (CFO). Prior to joining Rocket, Carlos held several roles of increasing responsibility over 30 years at Novartis where he led finance organizations in the US and other major pharmaceutical markets. He most recently served as the Vice President & Finance Head Oncology USA, a position he held since January 2011. In February 2021, Kinnari Patel, PharmD, MBA was appointed President and Chief Operating Officer (COO). Since joining Rocket in 2015, Dr. Patel has overseen pipeline development and execution, IT, HR, regulatory, clinical execution, alliance management, CMC and quality organizations.
Continued focus on supporting the patient community by serving as a pivotal resource for patient education during the COVID-19 pandemic, and by recognizing Rare Disease Day through "Lighting up for Rare." As part of Rocket’s mission to be a patient-centric gene therapy leader, throughout 2020 as patients faced significant difficulties presented by the pandemic, Rocket hosted a variety of programs and events to support patients through this critical time. Events included 1) virtual sessions for patients with Danon Disease and their families; 2) Rocket’s third PKD day; and 3) participation in the 2020 Fanconi Anemia Family Meeting, hosted by the Fanconi Anemia Research Fund. In continuation of Rocket’s commitment to Patients, Rocket is recognizing 2021 Rare Disease Day by leading the "Lighting up for Rare" campaign hosting a month of expert panel series driving awareness of rare diseases, culminating on February 28, 2021 as iconic landmarks across the U.S., including the Empire State Building, NASDAQ Tower, and Niagara Falls, are expected to be lit to drive awareness and foster solidarity among the global rare disease community.
Strengthened balance sheet, with ~$300 million equity offering and extension and partial conversion of existing convertible notes. On December 14, 2020, Rocket closed an upsized underwritten public offering of 5,339,286 shares of its common stock, inclusive of greenshoe, at the public offering price of $56.00 per share. This capital raise extends Rocket’s cash runway to the second half of 2023. In the first half of 2020, Rocket entered into two privately negotiated agreements with certain holders of its outstanding 5.75% Convertible Senior Notes due 2021. Rocket exchanged approximately $46.85 million aggregate principal amount of the Old Notes for new 6.25% Convertible Senior Notes due 2022. Approximately $5.15 million aggregate principal amount of the 2021 Notes will remain outstanding. Additionally, $8.5 million of the 2022 Notes have been converted into shares by certain holders. At year-end $38.35 million of the 2022 Notes remain outstanding.
Anticipated Milestones

Fanconi Anemia (RP-L102)
Updated "Process B" data (2Q21)
Danon Disease (RP-A501)
Longer-term Phase 1 data (2H21)
LAD-I (RP-L201)
Initial Phase 2 data (2Q21)
PKD (RP-L301)
Longer-term Phase 1 data (2H21)
IMO (RP-L401)
Initial Phase 1 data (2H21)
Upcoming Investor Conferences

10th Annual Global SVB Leerink Healthcare Conference—February 25, 2021
Cowen’s 41st Annual Healthcare Conference—March 2, 2021
Oppenheimer’s 31st Annual Healthcare Conference—March 16, 2021
Fourth Quarter and Full Year 2020 Financial Results

Cash position. Cash, cash equivalents and investments as of December 31, 2020, were $482.7 million.
Debt. Our balance sheet includes $39.9 million of fully convertible notes.
R&D expenses. Research and development expenses were $51.0 million and $106.4 million for the three and twelve months ended December 31, 2020, compared to $14.7 million and $58.6 million for the three and twelve months ended December 31, 2019. The increase in research and development expenses for the three and twelve months ended December 31, 2020, was primarily driven by an increase in new research agreements totaling $26.5 million in non cash expenses, clinical trial expenses, manufacturing expenses, compensation expense due to increased headcount and amortization expense related to the Cranbury, NJ facility lease.
G&A expenses. General and administrative expenses were $8.2 million and $27.9 million for the three and twelve months ended December 31, 2020, compared to $5.0 million and $17.5 million for the three and twelve months ended December 31, 2019. The increase in general and administrative expenses for three and twelve months ended December 31, 2020, was primarily driven by fees incurred in connection with the convertible note exchange, an increase in compensation expense and non-cash stock-based compensation expense due to increased headcount, and an increase in office and administrative expenses.
Net loss. Net loss was $60.9 million and $139.7 million or $1.08 and $2.52 per share (basic and diluted) for the three and twelve months ended December 31, 2020, compared to $19.9 million and $77.3 million or $0.39 and $1.58 per share (basic and diluted) for the three and twelve months ended December 31, 2019.
Shares outstanding. 60,996,367 shares of common stock were outstanding as of December 31, 2020.
Financial Guidance

Cash position. As of December 31, 2020, we had cash, cash equivalents and investments of $482.7 million. Rocket expects such resources will be sufficient to fund its operations into the second half of 2023.

On February 25, 2021 ESSA Pharma Inc. (Nasdaq: EPIX) ("ESSA" or the "Company"), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, reported that the Company has entered into a clinical collaboration and supply agreement with Astellas Pharma Inc. ("Astellas") to evaluate ESSA’s lead product candidate, EPI-7386, a first-in-class N-terminal domain androgen receptor inhibitor, in combination with Astellas and Pfizer Inc.’s androgen receptor inhibitor, enzalutamide, in patients with metastatic castration-resistant prostate cancer ("mCRPC") (Press release, ESSA, FEB 25, 2021, View Source [SID1234575721]).

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Under the terms of the agreement, ESSA will sponsor and conduct a Phase 1/2 study to evaluate the safety, tolerability and preliminary efficacy of the combination of EPI-7386 and enzalutamide in mCRPC patients who have not yet been treated with second-generation antiandrogen therapies. Astellas will supply enzalutamide for the trial. ESSA will retain all rights to EPI-7386. The clinical study is expected to start in 2021.

"We are delighted to collaborate with Astellas to explore the potential clinical role of EPI-7386 in combination with Astellas’ enzalutamide in patients with metastatic castration-resistant prostate cancer, who have progressed on androgen deprivation therapy," said Dr. David R. Parkinson, Chief Executive Officer, ESSA Pharma Inc. "Combining our two therapies will simultaneously target both ends of the androgen receptor. In preclinical models, we have seen that combining EPI-7386 with current antiandrogens can lead to deeper and broader inhibition of androgen biology. We look forward to investigating the combination of these therapies and their potential role together in the treatment of prostate cancer."

About EPI-7386
EPI-7386 is an investigational, highly-selective, oral, small molecule inhibitor of the N-terminal domain of the androgen receptor. EPI-7386 is currently being studied in a Phase 1 clinical trial (NCT04421222) in men with mCRPC whose tumors have progressed on current standard-of-care therapies. The Phase I clinical trial of EPI-7386 began in Q3 of 2020 following FDA allowance of the IND and Health Canada acceptance. The U.S. FDA has granted Fast Track designation to EPI-7386 for the treatment of adult male patients with mCRPC resistant to standard-of-care treatment. ESSA retains all rights to EPI-7386 worldwide.

On February 25, 2021 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage biopharmaceutical company, reported financial results for the year ended December 31, 2020 and provided a corporate update (Press release, Altimmune, FEB 25, 2021, View Source [SID1234575742]).

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"The past year has been a transformative time for our Company as we made substantial progress in each of our five portfolio programs," said Vipin K. Garg, Ph.D., President and Chief Executive Officer. "During the year we initiated multiple clinical trials for several of our product candidates (T-COVID, ALT-801 and HepTcell) and completed preparations to begin a Phase 1 clinical trial of AdCOVID, which has begun enrolling volunteers. These achievements have set the stage for a busy and exciting year ahead, as we anticipate multiple data readouts from these programs over the coming months. With two promising technology platforms and five novel product candidates now advancing in clinical development, we believe 2021 has the potential to be a momentous year for Altimmune."

Program Highlights

AdCOVID:

Commenced enrollment in AdCOVID Phase 1 clinical trial evaluating a novel, needle-free intranasal delivery approach for COVID-19 vaccination
Altimmune has commenced enrollment in its Phase 1 clinical trial of AdCOVID, which is designed to evaluate a needle-free intranasal delivery approach for vaccination against COVID-19. Altimmune believes AdCOVID has the potential to become a leading candidate for COVID-19 vaccination based on its ease of administration, and the potential for reduced disease transmission, and cold chain-free vaccine distribution, if the product is demonstrated to have extended stability at room temperature. As demonstrated in the NasoShield and NasoVax clinical trials, the Company believes the expected attributes of AdCOVID make it ideally suited for use in a pediatric setting as the intranasal administration and expected tolerability profile are well suited to meet the needs of children.

The Phase 1 clinical trial will evaluate the safety and immunogenicity of AdCOVID in up to 180 healthy adult volunteers between the ages of 18 and 55. Subjects will receive AdCOVID at one of three dose levels administered as a nasal spray. In addition to the primary study endpoint of safety and tolerability, the immunogenicity of AdCOVID will be evaluated by serum IgG binding and neutralizing antibody titers, mucosal IgA antibody from nasal samples, and T cell responses. Altimmune anticipates having a full data readout from this Phase 1 study in Q2 2021.

Initiated development of additional AdCOVID vectors targeting emerging SARS-CoV-2 variants
The emergence of SARS-CoV-2 variants is raising concerns about the effectiveness of currently authorized vaccines and prompting vaccine developers to engineer new vaccine candidates to combat these viral mutations. Altimmune has initiated the development of vaccine candidates against several variants as one is likely to become dominant in the population in the coming months. Altimmune plans to have these new vaccine candidates ready for use in upcoming later-stage clinical trials.

Established a consortium of manufacturing partners for potential commercial supply of AdCOVID
Altimmune has executed agreements with three commercial manufacturing partners with significant experience in adenoviral vector production. The Company has also established relationships with leading drug product fill/finish partners with sufficient capacity to meet potential commercial demand. Together, the Company believes that this network of strategic manufacturing partners will ensure Altimmune’s commercial readiness to supply vaccine, assuming the clinical data support this advancement.

Furthered AdCOVID preclinical studies in collaboration with the University of Alabama at Birmingham (UAB) and Saint Louis University
Based on the promising preclinical data for AdCOVID published on the BioRxiv server, Altimmune continues preclinical studies of AdCOVID in collaboration with UAB and Saint Louis University to evaluate AdCOVID in additional animal models and to further evaluate heterologous prime boost regimens of AdCOVID in support of future clinical development activities. Data from these ongoing preclinical studies are expected in Q1 and Q2 2021.

ALT-801:

Commenced dosing in a Phase 1 clinical trial of ALT-801, a novel GLP-1/glucagon dual-agonist being evaluated for the treatment of NASH
Altimmune commenced dosing in a Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) clinical trial of ALT-801, a GLP-1/glucagon dual-agonist being developed for the treatment of NASH. This trial is being conducted in Australia and is expected to enroll approximately 100 volunteers. The primary pharmacodynamic endpoints in the trial are weight loss and reduction in liver fat, outcomes that have been associated with NASH resolution and fibrosis improvement in advanced clinical studies of other NASH therapeutics. The Company has successfully completed the initial phases of the study and anticipates a data read-out from the 6-week MAD study in Q2 2021, followed by 12-week data in Q3 2021.

Amended clinical trial protocol to extend MAD cohorts to incorporate 12-week Phase 1b study in Australia
Altimmune amended the clinical trial protocol for the ALT-801 Phase 1 clinical development program to incorporate its planned 12-week extension trial in patients with non-alcoholic fatty liver disease or NAFLD within the ongoing Phase 1 SAD/MAD trial in Australia. The Company believes that by incorporating the 12-week extension into this trial, it can avoid any potential impact of COVID-19 and maintain study timelines. Pending the results of this trial, Altimmune plans to transition rapidly to a 52-week, Phase 2, biopsy-trial based on NASH endpoints in early 2022. In parallel with these efforts, Altimmune continues to plan to file an Investigational New Drug (IND) application for ALT-801 in the United States in mid-2021.

Initiated chronic toxicology studies of ALT-801 to enable 52-week Phase 2 clinical study
Altimmune completed 6-week and 13-week GLP toxicology studies of ALT-801 with no significant toxicity or GI adverse events. The Company has initiated 6-month and 9-month GLP toxicology studies to support the planned 52-week biopsy-driven Phase 2 trial planned for early 2022.

T-COVID:

Completed Cohorts 1 and 2 in the Phase 1/2 trial of T-COVID in patients with early COVID-19
Altimmune, working with the Department of Defense, has completed the two safety cohorts in the EPIC (Efficacy and Safety of T-COVID in the Prevention of Clinical Worsening in COVID-19) study, a Phase 1/2 clinical trial of T-COVID, an investigational intranasally-administered therapeutic for the treatment of early COVID-19 infection. The trial is being overseen by an independent Data Safety Monitoring Committee, and no significant safety findings have been observed to date.

Cohort 3 is an efficacy and safety cohort that will include patients at higher-risk for severe COVID-19 infection, such as those 65 years or older, or those with one or more risk factors for severe COVID-19 complications. To ensure that a sufficient number of higher risk patients are enrolled, the study protocol was recently modified to require that a minimum number of patients meet one or more of these criteria in this final cohort. Additional enrichments of the study population are currently being evaluated to increase the event rates in the trial. While these modifications could extend the study timeline, the Company believes they could significantly enhance the probability of a meaningful trial outcome. Based on these changes, data from this trial is now expected in Q2 2021.

HepTcell:

Commenced dosing in a multinational Phase 2 clinical trial of HepTcell
In December, Altimmune began a multinational Phase 2 clinical trial of HepTcell, which is being conducted in the United States, Canada and Europe. The trial is a double-blind, randomized, placebo-controlled trial of 80 adult patients with HBeAg-negative inactive CHB and HBsAg ≤ 100 IU/mL.

HepTcell will be administered in 6 doses at 4-week intervals for 24 weeks, and patients will be followed for one year to evaluate safety and durability of response. The primary efficacy endpoint is virological response, defined as a 1-log reduction in HBsAg levels from baseline. Secondary efficacy endpoints include reactivation of anti-HBV T cell responses, HBsAg clearance, and other assessments of virologic response. Altimmune anticipates a data read-out from this trial in 1H 2022.

Financial Results for the Year Ended December 31, 2020

Altimmune had cash, cash equivalents and short-term investments of $216.0 million at December 31, 2020 compared to $37.3 million at December 31, 2019. The increase of $178.7 million is attributable to $213.5 million of net receipts during the year due primarily to its 2020 public offering, full utilization of the at-the-market offering program, and receipts from warrant exercises, offset by $34.4 million of cash used for operating activities.
Revenue was $8.2 million for the year ended December 31, 2020 compared to $5.8 million in the prior year period, an increase of $2.4 million. The change was primarily due to an increase in revenue under the Company’s U.S. government contracts due to timing of manufacturing and clinical trials for the NasoShield and T-COVID programs.
Research and development expenses were $49.8 million for the year ended December 31, 2020, compared to $17.8 million in the prior year period, representing an increase of $32.0 million. The increase was primarily attributable to increased costs related to development of AdCOVID, T-COVID and ALT-801 and an increase in the contingent liability for stock-based milestone payments associated with the acquisition of ALT-801.
General and administrative expenses were $13.2 million for the year ended December 31, 2020 compared to $8.5 million in the prior year period, an increase of $4.7 million. The increase is attributable to additional employee compensation as Altimmune’s workforce grew in 2020 along with an increase in professional costs.
Income tax benefit was $5.4 million for the year ended December 31, 2020, as compared to $59,000 for the same period in 2019. The increase is attributable to the Coronavirus Aid, Relief, and Economic Security Act (the "CARES Act") passed on March 27, 2020 which made temporary changes regarding the utilization and carry back of net operating losses.
Net loss attributed to common stockholders for the year ended December 31, 2020 was $49.0 million, or $1.91 net loss per share, compared to $21.0 million in the prior year, or $1.60 net loss per share. The difference in net loss is primarily attributable to higher research and development expenses and general and administrative expenses, offset by higher revenue and an increase in income tax benefit.
Conference Call Information

Following the conclusion of the call, the webcast will be available for replay on the Investor Relations page of the Company’s website at www.altimmune.com. The company has used, and intends to continue to use, the IR portion of its website as a means of disclosing material non-public information and for complying with disclosure obligations under Regulation FD.

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.