On November 9, 2021 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, reported that is providing a company update and third quarter 2021 financial results (Press release, Cumberland Pharmaceuticals, NOV 9, 2021, View Source;company-update-301420388.html [SID1234594916]). Net revenues from continuing operations during the quarter were $8.1 million and totaled $27.7 million for the first nine months of 2021, up 1.8% compared to the same period in 2020. The company also recorded an additional $0.5 million in revenue during the third quarter and $1.5 million year to date, associated with divested product rights for two brands it is no longer distributing.

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The Company’s financial position included $87.6 million in total assets, with $25.8 million in cash, $41 million of total liabilities, and $46.8 million of shareholders’ equity at the end of the quarter.

"We delivered another steady quarterly performance when factoring in the challenges of operating during the pandemic," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "Our diversified portfolio has been critical in keeping our forward progress during this time, and we are seeing signs that things are slowly starting to return to a more normal operating environment. Our team remains committed to advancing our mission of improving patient care through the delivery of high-quality medicines through the end of the year and beyond."

RECENT COMPANY DEVELOPMENTS:

RediTrex Launch

In late 2019, Cumberland received approval from the U.S. Food and Drug Administration ("FDA") for its New Drug Application for RediTrex, the Company’s methotrexate product line. RediTrex is a new line of pre-filled syringes specifically designed for ease of handling and dosing accuracy for the subcutaneous administration of methotrexate in patients with arthritis and psoriasis.

In late 2020, the Company received initial product supplies and then provided shipments of RediTrex to select accounts. Due to the pandemic, Cumberland delayed the national launch of the product, which was implemented during the third quarter of 2021.

RediTrex treats patients with severe, active rheumatoid arthritis, and polyarticular juvenile idiopathic arthritis who have difficulty tolerating or responding to orally delivered methotrexate. It is also approved for symptomatic control of severe, recalcitrant, disabling psoriasis in adults who are not adequately responsive to other forms of therapy.

With more than 54 million Americans living with some form of arthritis, the disease is among the most common causes of work disability in the U.S., according to the CDC. The oral form of methotrexate is typically the first line of treatment for rheumatoid arthritis. As the disease progresses, the dose must be increased to stay effective, often causing intolerable gastrointestinal side effects. Injectable methotrexate has been proven to be more effective than oral delivery, with fewer gastrointestinal reactions. Because of the increased efficacy and tolerability, injectable methotrexate can delay the need to move to costly biologics, lowering overall patient treatment costs. Once disease progression requires the use of biologics, continuing the treatment of injectable methotrexate along with the biologic has been shown to increase overall efficacy.

Other injectable methotrexate options available may not optimally meet the needs of an arthritis patient. Patients are offered either a vial and syringe for self-injection, or the use of an expensive autoinjector. The vial and syringe method can be difficult for a patient to handle due to limited dexterity in their hands. Additionally, obtaining the exact dose needed while preventing skin exposure to the caustic methotrexate can be quite challenging for many patients. The autoinjectors provide a better alternative to the vial and syringe, but they remove injection control from the patient and can be painful to administer. They are also the most expensive methotrexate delivery.

ESG Report

In July 2021, Cumberland released its second annual Sustainability Report (the "2020 Sustainability Report"), which details the Company’s activities pertaining to its environmental, social and governance ("ESG") matters. After issuing its inaugural ESG report last year (the "2019 Sustainability Report"), Cumberland remains committed to sustainability and to maintaining transparency of its corporate operations. As the largest biopharmaceutical company founded and headquartered in the Mid-South, the Company holds itself to the highest standards of ethical practices and understand the importance of recognizing and addressing its impact on its constituents, the community and the environment.

The 2020 Sustainability Report notes that Cumberland provided nearly 2.5 million patient doses of its products, safely disposed of over 4,000 pounds of expired and damaged products and had no product recalls. Cumberland also had no Company brands listed on the FDA’s MedWatch Safety Alerts for Human Medical Products, no Company product issues identified by FDA from their Adverse Event Reporting System and no clinical trials terminated due to failure to practice good clinical standards.

The 2020 Sustainability Report also highlights several initiatives Cumberland implemented as part of its commitment to delivering high-quality pharmaceutical products to improve patient care. For example, the Company continued a program to serialize all commercial products sold in the United States, allowing it to track every unit distributed, which helps to prevent counterfeit drugs from entering the market under the Cumberland brand. In addition, through its coupon program, Cumberland can cover up to 90% of patient prescription costs for its gastrointestinal products.

The 2020 Sustainability Report also highlights Cumberland’s investment in its employees through its continuing education programs, employee development initiatives and employee recognition awards. Cumberland’s workforce is 46% women – and 18% of its employees are minorities.

Ifetroban Clinical Studies

Cumberland has been evaluating its ifetroban product candidate in a series of clinical studies. The Company is sponsoring Phase II clinical programs to evaluate its ifetroban product candidates in 1) patients with cardiomyopathy associated with Duchenne Muscular Dystrophy, a rare, fatal, genetic neuromuscular disease that results in deterioration of the skeletal, heart and lung muscles, 2) Systemic Sclerosis or scleroderma, a debilitating autoimmune disorder characterized by diffuse fibrosis of the skin and internal organs and 3) Aspirin-Exacerbated Respiratory Disease, a severe form of asthma.

Enrollment in these clinical studies was interrupted due to the COVID-19 pandemic. However, many of Cumberland’s clinical study sites have reopened and resumed screening of patients for potential participation into its studies. The Company is awaiting results from the studies underway before deciding on the best development path for the registration of ifetroban, its first new chemical entity.

In September 2021, Cumberland’s Board of Directors approved a new clinical program for the use of ifetroban to treat Progressive Fibrosing Interstitial Lung Diseases ("PF-ILDs"). Nonclinical studies are complete, and the resulting manuscript has been prepared and submitted for publication. A Phase II clinical study is planned and an application to the FDA is in preparation to support this new clinical program.

Additional pilot preclinical and clinical studies of ifetroban are underway, including several investigator-initiated trials.

Hyponatremia Publication

The Health Outcome Predictive Evaluation (HOPE) COVID-19 Registry Analysis, an international study of over 4,000 patients published in November 2020, found that patients hospitalized with COVID-19 had a high risk of developing hyponatremia. These COVID-19 patients also had a higher incidence of mortality due to their hyponatremia. The study results support the use of an intravenous vaptan to treat hyponatremia in critically ill patients afflicted with COVID-19.

Hyponatremia, an imbalance of serum sodium to body water, is the most common electrolyte disorder among hospitalized patients. Cumberland’s Vaprisol product is one of two branded prescription products indicated for the treatment of hyponatremia, and the only intravenously administered branded treatment. Vaprisol has a proven day-1 response rate to normalize serum sodium levels in hyponatremic patients and move them out of the Intensive Care Unit as efficiently as possible.

New Line of Credit

On October 28, 2021, Cumberland entered into a Fourth Amendment to the Revolving Credit Note and Fifth Amendment ("Fifth Amendment") to the Revolving Credit Loan Agreement with Pinnacle Bank (the "Pinnacle Agreement"). The original Pinnacle Agreement was dated July 31, 2017 and the Fifth Amendment extended the term of the loan agreement for a three-year period ending October 1, 2024. The facility provides for a principal available for borrowing of up to $15 million and an opportunity to request an increase in availability to $20 million, upon the satisfaction of certain conditions and approval by Pinnacle Bank. The interest rate on funds borrowed under the facility ranges from 30-day LIBOR plus 175 to 275 basis points depending on the funded debt ratio.

Vibativ International Agreements

On August 25, 2021, Cumberland signed an agreement with Verity Pharmaceuticals International Limited to license and commercialize Vibativ in Puerto Rico. Verity is a specialty pharmaceutical company with commercial operations in the U.S. and Canada.

Vibativ is a patented, FDA-approved injectable anti-infective for the treatment of certain serious bacterial infections, including hospital-acquired and ventilator-associated bacterial pneumonia, and complicated skin and skin structure infections. It addresses a range of Gram-positive bacterial pathogens, including those that are considered difficult-to-treat and multidrug-resistant. In November 2018, Cumberland reached an agreement to acquire Vibativ from Theravance Biopharma and assume global responsibility for the product.

SciClone Pharmaceuticals (Holdings) Limited has licensed Cumberland’s Vibativ product for sale and distribution in China. In February 2021, SciClone completed an initial public offering and listing of their shares on the Hong Kong stock exchange. During the third quarter of 2021, SciClone submitted an application to the Chinese regulatory authority for the approval of Vibativ in that country. In September 2021, the filing was accepted by the regulatory agency for review. SciClone expects a review period of up to twelve months for their application and believes that the potential for Vibativ in China may be significant.
FINANCIAL RESULTS:

Net Revenues: For the three months ended September 30, 2021, net revenues from ongoing operations were $8.1 million, compared to $9.3 million for the prior year period. The company also recorded an additional $0.5 million in revenue during the third quarter associated with divested rights to products that the company no longer distributes.

Net revenue by product for the third quarter 2021, included $4.0 million for Kristalose, $1.9 million for Vibativ, $1.3 million for Caldolor, and $0.3 million for Vaprisol.

Year-to-date 2021 net revenues were $27.7 million, up 1.8% from $27.2 million during the first nine months of 2020. There were additional revenues of $1.5 million in 2021 associated with the divested product rights.

Year-to-date 2021 net revenues by product were $12.3 million for Kristalose, $8.8 million for Vibativ, $3.7 million for Caldolor and $1.9 million for Vaprisol.

Operating Expenses: Total operating expenses for the three months ended September 30, 2021, were $9.6 million, compared to $10.5 million during the prior year period.

Earnings: Net income (loss) for the third quarter 2021 was $(1.1) million or $(0.07) a share, compared to $(0.5) million or $(0.03) a share for the prior year period. Adjusted earnings (loss) for the third quarter was $(0.3) million, compared to $0.2 million for the prior year period.

Year-to-date net income (loss) in 2021 was $0.3 million, compared to $(2.5) million for the same period last year. Adjusted earnings for the first nine months of 2021 were $0.7 million, compared to an adjusted loss of $(0.3) million during the same period in 2020.

Balance Sheet: At September 30, 2021, Cumberland had $87.6 million in total assets including $25.8 million in cash and cash equivalents. Total liabilities were $41 million, including $15 million outstanding on the Company’s revolving line of credit, resulting in total shareholders’ equity of $46.8 million.

CONFERENCE CALL & WEBCAST:

A conference call and live internet webcast will be held on Tuesday, November 9, at 4:30 p.m. Eastern Time to discuss the results. To participate in the call, please dial 877-303-1298 (for U.S. callers) or 253-237-1032 (for international callers). A rebroadcast of the teleconference will be available for one week and can be accessed by dialing 855-859-2056 (for U.S. callers) or 404-537-3406 (for international callers). The Conference ID for the rebroadcast is 9476299. The live webcast and rebroadcast can be accessed via Cumberland’s website at View Source

On November 9, 2021 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported program updates and financial results for the third quarter ended September 30, 2021 (Press release, Poseida Therapeutics, NOV 9, 2021, View Source [SID1234594915]).

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"The last quarter saw continued progress as Poseida reached multiple key strategic milestones including FDA clearance of our first fully allogeneic CAR-T IND for P-BCMA-ALLO1, the presentation of strong CAR-T data in a solid tumor indication with our P-PSMA-101 program and, shortly after the quarter, the announcement of a strategic collaboration with Takeda focused on non-viral in vivo gene therapy programs utilizing our platform technologies," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida.

"The initiation of our P-BCMA-ALLO1 clinical trial represents the beginning of a long-planned strategic transition to what we believe is the ‘holy grail’ of cell therapy for oncology, a fully allogeneic CAR-T with a fully humanized heavy chain BCMA binder and a high percentage of TSCM cells which we believe are the key to success," continued Ostertag. "While we believe the P-BCMA-101 autologous program has competitive advantages and would be approvable, one long-term strategic benefit of that program has been to inform our highly-differentiated allogeneic approach. With the P-BCMA-ALLO1 clinical program now underway and with very high confidence in our allogeneic platform, we will begin a planned wind down of P-BCMA-101."

Program Updates

BCMA Program
P-BCMA-ALLO1, the Company’s first fully allogeneic CAR-T product candidate, is in development for the treatment of relapsed/refractory multiple myeloma. In August of 2021, the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for P-BCMA-ALLO1. The IND clearance and the start-up of the Phase 1 clinical trial mark the beginning of the Company’s strategic shift toward focusing on P-BCMA-ALLO1 rather than the autologous P-BCMA-101 program.

While data in the autologous trial showed meaningful responses and a favorable safety profile, the Company’s strategic focus has long been on allogeneic CAR-T therapies, leveraging the learnings of the autologous CAR-T program to provide benefits beyond those of autologous CAR-T, including a more desirable off-the-shelf product profile for future commercialization while maintaining the tolerability advantage of our autologous product candidate. P-BCMA-ALLO1 has the potential to deliver up to hundreds of doses per manufacturing run, thereby dramatically reducing both clinical trial costs and ultimately commercial product cost compared to the autologous P-BCMA-101 program.

PSMA Program
P-PSMA-101 is a solid tumor autologous CAR-T product candidate being developed to treat patients with metastatic castrate-resistant prostate cancer (mCRPC) currently in an ongoing Phase 1 dose escalation trial.

In August of 2021, the Company presented preliminary data at the 6th Annual CAR-TCR Summit virtual meeting that demonstrated meaningful patient responses while maintaining a favorable safety and tolerability profile with modest overall rates of CRS and no neurotoxicity observed at low doses. An additional update on the P-PSMA-101 program is expected in the first half of 2022.

MUC1-C Program
P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate in preclinical development with the potential to treat a wide range of solid tumors, including breast and ovarian cancers. P-MUC1C-ALLO1 is proceeding, with an anticipated IND filing and initiation of a Phase 1 clinical trial by the end of 2021.

Liver-Directed Gene Therapy Program
P-OTC-101 is the Company’s first liver-directed gene therapy program for the in vivo treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene, a condition characterized by high unmet medical need. The Company is currently evaluating whether to modify the P-OTC-101 program to move to the fully non-viral nanoparticle delivery system. The Company will update expected timing on program advancement once that evaluation is complete.

Other Operational Updates and Upcoming Events

Gene Therapy Research Collaboration with Takeda
In October of 2021, the Company entered into a collaboration and license agreement with Takeda Pharmaceuticals USA, Inc. to utilize Poseida’s proprietary genetic engineering platforms for the research and development of up to eight gene therapies.

The collaboration will focus on developing non-viral in vivo gene therapy programs, including Poseida’s Hemophilia A program. The Company will receive an upfront payment of $45.0 million, of which $5.0 million is for prepaid research. Per the agreement, Takeda will fund all ongoing partnered program research performed by Poseida. The collaboration may utilize all of Poseida’s novel genetic engineering platform technologies, including the piggyBac DNA Modification System for gene addition, the Cas-CLOVER Site-specific Gene Editing System for ultra-precise gene editing, biodegradable nanoparticle technologies for gene delivery and other emerging technologies. Poseida will lead research activities up to candidate selection, after which Takeda will assume responsibility for further development and commercialization.

GMP Facility
In the third quarter, the Company completed qualification and commenced GMP activity in its internal pilot manufacturing plant. The pilot plant is designed to support and speed the development of allogeneic product candidates including manufacturing clinical material for early-stage trials. The first product candidate to be produced out of the pilot plant will be P-MUC1C-ALLO1.

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting
The Company will give two virtual poster presentations at the upcoming SITC (Free SITC Whitepaper) Annual Meeting, being held in Washington, D.C., and virtually November 10-14, 2021. The full abstracts were made available on the SITC (Free SITC Whitepaper) website earlier today, with presentations taking place on November 12, 2021.

63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition
The Company will present data from the Company’s differentiated P-BCMA-101 autologous CAR-T program including how it has informed the P-BCMA-ALLO1 allogeneic CAR-T program in a poster publication at the ASH (Free ASH Whitepaper) Annual Meeting on Monday, December 13, 2021:

Session Name: 704. Cellular Immunotherapies: Clinical: Poster III
Presentation Time: 6:00 PM – 8:00 PM Eastern time
Location: Georgia World Congress Center, Hall B5

Financial Results for the Third Quarter 2021

Research and Development Expenses
Research and development expenses were $32.5 million for the third quarter ended September 30, 2021, compared to $27.0 million for the same period in 2020. For the nine months ended September 30, 2021, research and development expenses were $97.6 million, compared to $75.6 million for the same period in 2020. The increase was primarily due to increased stock-based compensation expense, headcount, external costs related to our preclinical programs and clinical stage programs, including the ongoing enrollment and manufacturing associated with our P-BCMA-101, P-BCMA-ALLO1 and P-PSMA-101 clinical trials, and internal costs related to facilities development.

General and Administrative Expenses
General and administrative expenses were $9.1 million for the third quarter ended September 30, 2021, compared to $6.5 million for the same period in 2020. General and administrative expenses were $26.3 million for the nine months ended September 30, 2021, compared to $15.6 million for the same period in 2020. The increase was primarily due to increased stock-based compensation expense, headcount, insurance costs and professional fees.

Net Loss
Net loss was $42.4 million and $126.4 million for the three and nine months ended September 30, 2021, respectively, and $34.4 million and $93.6 million for the three and nine months ended September 30, 2020, respectively.

Cash Position
As of September 30, 2021, our cash and cash equivalents balance was $197.8 million.

On November 9, 2021 Inhibrx, Inc. (Nasdaq: INBX), a biotechnology company with four clinical programs in development and a strong emerging pipeline, reported financial results for the third quarter of 2021 and provided an update on recent corporate highlights (Press release, Inhibrx, NOV 9, 2021, https://www.prnewswire.com/news-releases/inhibrx-reports-third-quarter-2021-financial-results-and-recent-corporate-highlights-301420241.html [SID1234594914]).

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Recent Corporate Highlights

On October 12, 2021, Inhibrx announced interim results from a Phase 1 clinical trial evaluating the safety and pharmacokinetics of INBRX-101, an optimized recombinant human AAT-Fc fusion protein, in patients with alpha-1 antitrypsin deficiency, or AATD. The data from the single ascending dose cohorts revealed the potential to achieve normal AAT levels with monthly dosing and also showed a favorable safety and tolerability profile with no drug-related severe or serious adverse events at doses up to and including 120 mg/kg single dose and 80 mg/kg multi-dose administered intravenously.
On October 28, 2021, Inhibrx announced the appointment of three executives: David Matly, M.B.A., as Chief Commercial Officer; David Kao, PharmD, M.B.A., RPh, as Vice President of Regulatory Affairs; and Jack Tsai, M.D., M.B.A., as Vice President of Business Development. Additionally, the Company announced the appointment of Kristiina Vuori, M.D., Ph.D. to its Board of Directors.
On November 1, 2021, Inhibrx announced it will be presenting at the Connective Tissue Oncology Society (CTOS) 2021 Virtual Annual Meeting taking place November 10-13, 2021. This presentation will provide an update on the Phase 1 expansion cohort for INBRX-109 in patients with conventional chondrosarcoma. Inhibrx will also present a poster on preclinical data that will guide the planned Phase 1 expansion cohort for INBRX-109 in Ewing Sarcoma.
On November 3, 2021, Inhibrx announced that three abstracts had been accepted for presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting, taking place November 10-14, 2021, in Washington D.C. and virtually. The three abstracts will highlight preclinical data from two of its clinical programs, INBRX-105 and INBRX-106, as well as introduce the first emerging pipeline candidate from its targeted cytokine platform, INBRX-121.
Financial Results

Cash and Cash Equivalents. As of September 30, 2021, Inhibrx had cash and cash equivalents of $112.7 million, compared to $128.7 million as of December 31, 2020. As of November 9, 2021, Inhibrx had cash and cash equivalents of $141.1 million.
R&D Expense. Research and development expenses were $18.5 million during the third quarter of 2021, compared to $19.8 million during the third quarter of 2020. This overall decrease was primarily due to the timing of work performed during 2020 by Inhibrx’s contract development and manufacturing organization partners for the formulation and manufacturing of certain of its therapeutic candidates, offset in part by an increase in clinical trial expenses based on the initiation of a Phase 2 trial in conventional chondrosarcoma and the progression of ongoing Phase 1 trials during 2021. Additionally, personnel-related costs increased during the third quarter of 2021 as compared to the same period in the prior year as a result of the continued expansion of its organization.
G&A Expense. General and administrative expenses were $2.8 million during the third quarter of 2021, compared to $1.6 million during the third quarter of 2020. This was primarily driven by increases in personnel-related costs, as well as increases in professional service fees related to Inhibrx’s expanding intellectual property portfolio and other expenses associated with operating as a public company following its initial public offering in August 2020.
Net Loss. Net loss was $20.6 million during the third quarter of 2021, or $0.54 per share, compared to $20.5 million during the third quarter of 2020, or $0.77 per share.
About the Inhibrx sdAb Platform
Inhibrx utilizes diverse methods of protein engineering in the construction of therapeutic candidates that can address the specific requirements of complex target and disease biology. A key tool for this effort is the Inhibrx proprietary sdAb platform, which enables the development of therapeutic candidates with attributes superior to other monoclonal antibody and fusion protein approaches. This platform allows the combination of multiple binding units in a single molecule, enabling the creation of therapeutic candidates with defined valency or multiple specificities that can achieve enhanced cell signaling or conditional activation. An additional benefit of this platform is that these optimized, multi-functional entities can be manufactured using the established processes that are commonly used to produce therapeutic proteins.

On November 9, 2021 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the third quarter and nine months ended September 30, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, NOV 9, 2021, View Source [SID1234594913]).

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"This past quarter we initiated our Phase 1/2 MARINATM trial for AOC 1001, which transitions us into a clinical stage company. This trial is important for both AOC 1001 and our platform as it is expected to generate the first proof of concept data on the AOC platform’s potential to deliver RNA therapy outside of the liver – a first for the field of RNA conjugates and for Avidity," said Sarah Boyce, president and chief executive officer. "This is also an important milestone for the myotonic dystrophy community who currently have no therapeutic options. We look forward to sharing preliminary data on AOC 1001 in the second half of next year as we work to achieve our ambitious agenda of having AOCs for DM1, DMD and FSHD in the clinic by the end of 2022."

"Following completion of our successful first follow-on financing in August, we are well funded into 2024 with a cash balance of $413 million at quarter end. Given these significant financial resources, we are well positioned to complete the ongoing MARINA trial for AOC 1001, as well as initiate proof of concept clinical trials in 2022 for AOC 1044, the first of our DMD programs, and for our AOC FSHD program while we continue our investment in expanding our AOC platform," said Mike MacLean, chief financial officer.

Recent Highlights:

Initiated first clinical trial for an AOC – transitioning Avidity into a clinical stage company
Initiated the Phase 1/2 MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1)
Received clearance from the U.S. Food and Drug Administration (FDA) to proceed with the Phase 1/2 MARINA trial under Avidity’s initial new drug application (IND) in July 2021
Enrolled the first patient in the MARINA trial in October 2021, marking the first time a person has been dosed with an AOC
AOC 1001 was granted Orphan Designation by the FDA and the European Medicines Agency (EMA)
AOC 1001 was granted Fast Track Designation by the FDA in October 2021
Entered a collaboration with a key physician network for a natural history study in FSHD
Avidity is supporting the natural history study called the Motor Outcomes to Validate Evaluations Plus (MOVE+) Study run by the facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial Research Network (FSHD CTRN) to enhance the understanding of how to utilize whole-body MRI and other tools to identify specific biomarkers for FSHD that can accelerate and support future clinical trial design.
Completed a follow-on financing, resulting in net proceeds of $155.1 million
Upcoming Events

Volume 3 of Avidity’s Virtual Investor and Analyst Event Series will be focused on FSHD and will be held on Thursday, December 9, 2021 at 8am PT/11am ET. To register for the live video webcast, please visit the "Events and Presentations" page in the "Investors" section of Avidity’s website. A replay of the webcast will be archived on Avidity’s website following the event.

Art Levin, Ph.D., Avidity’s chief scientific officer, and W. Michael Flanagan, Ph.D., Avidity’s chief technical officer, will discuss the scientific rationale and potential benefits of leveraging AOCs for FSHD.

The virtual event will also feature Jeffrey Statland, M.D., professor, Department of Neurology, University of Kansas Medical Center, to discuss the clinical impact of FSHD and the ongoing natural history studies. Dr. Statland is one of the principal investigators in the ongoing MOVE and MOVE+ natural history studies for people living with FSHD.

Third Quarter 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $413.0 million as of September 30, 2021, compared to $328.1 million as of December 31, 2020.
Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $2.2 million for the third quarter of 2021 compared with $1.7 million for the third quarter of 2020, and $7.5 million for the first nine months of 2021 compared with $4.6 million for the first nine months of 2020.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $24.8 million for the third quarter of 2021 compared with $9.5 million for the third quarter of 2020, and $68.2 million for the first nine months of 2021 compared with $24.0 million for the first nine months of 2020. The increases were primarily driven by the advancement of AOC 1001, AOC 1044 and the AOC FSHD program, as well as costs related to the expansion of the company’s overall research capabilities.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $6.6 million for the third quarter of 2021 compared with $3.8 million for the third quarter of 2020, and $18.8 million for the first nine months of 2021 compared with $8.6 million for the first nine months of 2020. The increases were primarily due to higher personnel costs. The year-to-date increase was also due to higher professional fees and insurance costs.

On November 9, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the third quarter of 2021 and updated financial guidance for 2021 (Press release, Jazz Pharmaceuticals, NOV 9, 2021, View Source [SID1234594912]).

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"Last year, we set the ambitious corporate objective of completing five key commercial launches through 2020 and 2021. With the launch of Xywav for idiopathic hypersomnia earlier this month, we have now accomplished this goal, demonstrating our significant execution capabilities and commitment to bring important new medicines forward for patients," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "The successful integration of GW underscores our ability to deliver on transformative M&A to grow our business. While there’s more work to be done, I’m confident we have the right strategy, teams and capabilities in place to realize the blockbuster potential of Epidiolex and to discover, develop and launch additional novel, innovative medicines leveraging cannabinoid science. Our commercial execution, productive R&D engine and culture of commitment to patients and their families provide a strong foundation for significant and sustained growth."

Renée Galá, executive vice president and chief financial officer, added, "This is an exciting time of transformation for Jazz, underpinned by operational execution, financial discipline and strategic capital allocation across our business. We continue to deliver on our business and financial targets which has enabled us to rapidly reduce our net leverage ratio to 4.41 times in just five months following the close of the GW transaction. We have also delivered on revenue growth and diversification. Recently launched or acquired products now make up over 50% of net product sales, and we remain on track to meet our goal of at least 65% in 2022. In addition, our prior investments in corporate development are translating into near-term catalysts as we advance JZP385, JZP150 and Zepzelca into important new clinical trials. We will continue to prioritize disciplined capital allocation to assets and activities that drive growth and value, while remaining focused on achieving our net leverage ratio target of less than 3.51 times by the end of next year."

Key Highlights

Total revenues increased 39% to $838.1 million compared to 3Q20
52% of net product sales from recently launched or acquired products
Exceptional Xywav adoption in narcolepsy with approximately 6,000 active patients exiting 3Q21
Xywav for idiopathic hypersomnia (IH) launched November 1, 2021
Continued Epidiolex revenue growth of 21% compared to 3Q20 despite COVID-19 pressure
Top-tier launch has established Zepzelca as second-line SCLC treatment of choice
Rylaze launch progressing well; positive feedback from key stakeholders
Pipeline advancing with key trial initiations underway for JZP385, JZP150 and Zepzelca
Raising full year 2021 earnings guidance
Net leverage ratio reduced by 0.5x to 4.4×1 in the five months following GW transaction close
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1.

On a pro forma, non-GAAP adjusted basis

Business Updates

Neuroscience

Oxybate (Xyrem and Xywav):

Net product sales for the combined oxybate business increased 3% to $460.4 million in 3Q21 compared to the same period in 2020.
Average active oxybate patients on therapy was approximately 16,000 in 3Q21, an increase of approximately 6% compared to the same period in 2020.
Xywav for Narcolepsy (calcium, magnesium, potassium, and sodium oxybates) oral solution:

The Company continues to drive market-leading adoption of Xywav in narcolepsy.
Xywav net product sales were $153.1 million in 3Q21.
There were approximately 6,000 active patients on Xywav exiting 3Q21.
In June 2021, FDA recognized seven years of Orphan Drug Exclusivity, through July 2027, for Xywav and published its summary of clinical superiority findings stating that "Xywav is clinically superior to Xyrem by means of greater safety because Xywav provides a greatly reduced chronic sodium burden compared to Xyrem." Further, FDA stated that "the differences in the sodium content of the two products at the recommended doses will be clinically meaningful in reducing cardiovascular morbidity in a substantial proportion of patients for whom the drug is indicated."
Xywav for Idiopathic Hypersomnia

On August 12, 2021, FDA approved Xywav for the treatment of IH in adults.
The Company launched Xywav for IH on November 1, 2021.
Xywav is the first-and-only medicine approved by FDA for the treatment of IH in adults, underscoring the Company’s patient-focused R&D strategy and concept-to-commercial capabilities.
Xywav for IH is a significant value driver, with initial launch efforts focused on the approximately 37,000 currently diagnosed patients in the U.S. who are actively seeking healthcare.
Xywav demonstrated robust clinical data with statistically significant improvements across all primary and secondary endpoints in the Phase 3 clinical trial.
Xywav has broad patent protection to 2033 and is eligible for Orphan Drug Exclusivity for IH.
Xyrem (sodium oxybate) oral solution:

Xyrem net product sales decreased 31% to $307.3 million in 3Q21 compared to the same period in 2020, reflecting the continued strong adoption of Xywav.
Epidiolex/Epidyolex (cannabidiol):

Epidiolex/Epidyolex net product sales were $160.4 million in 3Q21, an increase of 21% compared to the same period of 2020 on a pro-forma basis, despite short-term COVID-19 pressure.
Recent market research indicates approximately 40% of prescribers are moving Epidiolex up in their treatment algorithm.
The Company has made significant progress on its European rollout with launches in Spain, Italy and Switzerland in 3Q21. Epidyolex is now commercially available and fully reimbursed in four of the five key European markets: United Kingdom, Germany, Italy and Spain, with an anticipated launch in France in 2022.
The Company expects to initiate a Phase 3 pivotal trial of Epidiolex for Epilepsy with Myoclonic-Atonic Seizures (EMAS), the fourth target indication for Epidiolex, in 1H22.
The Company continues to strengthen the durability of Epidiolex, and expects a composition of matter-like patent, extending through 2039, to be issued later this year.
Sunosi (solriamfetol):

Sunosi net product sales increased by 111% to $19.3 million in 3Q21 compared to the same period of 2020.
In 3Q21, U.S. prescriptions increased by 8% compared to 2Q21.
Nabiximols:

The Company has initiated the third Phase 3 nabiximols clinical trial in multiple sclerosis (MS)-related spasticity. This is a randomized, double-blind, placebo-controlled trial with a primary endpoint of muscle tone, expected to enroll approximately 190 patients.
The Company expects data from its first Phase 3 trial in 1H22, followed by data from the two additional Phase 3 trials in late 2022 and early 2023.
The Company anticipates that if the results of the first trial are positive, there is potential for regulatory submission to FDA in the next 18-24 months.
JZP385:

The Company has initiated a Phase 2b trial and expects top-line data to read out in 1H24.
JZP385, a highly selective modulator of T-type calcium channels, is in clinical development for the potential treatment of essential tremor.
JZP150:

The Company is on track to initiate a Phase 2 trial this year.
JZP150, a fatty acid amide hydrolase (FAAH) inhibitor, is in clinical development for the potential treatment of post-traumatic stress disorder.
Oncology

Zepzelca (lurbinectedin):

Zepzelca net product sales increased 94% to $71.7 million in 3Q21 compared to the same period in 2020.
Zepzelca net product sales in 3Q21 were favorably impacted by approximately $10 million, relating to a reduction in the returns accrual rate, due to lower than estimated actual returns. Excluding this impact, net product sales in 3Q21 increased by 10% compared to 2Q21.
The Company has established Zepzelca as the treatment of choice in the second-line small cell lung cancer (SCLC) setting. Zepzelca has near-term growth opportunities, as the Company expects that it will continue to gain share among patients being re-challenged with platinum-based chemotherapies or receiving other chemotherapy regimens.
Zepzelca development program updates:
Jazz and collaborator F. Hoffmann-La Roche Ltd (Roche) have initiated a Phase 3 trial to evaluate first-line use of Zepzelca in combination with Tecentriq (atezolizumab), compared to Tecentriq alone, as maintenance therapy, in patients with extensive stage SCLC after induction chemotherapy. The trial is now listed on clinicaltrials.gov (NCT05091567); enrollment of the first patient is anticipated later this year.
The Company’s partner, PharmaMar, plans to initiate a confirmatory trial in second-line SCLC later this year. If positive, this trial would confirm the benefit of Zepzelca in the treatment of SCLC when patients progress following first-line treatment with a platinum-based regimen.
Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn):

Rylaze net product sales were $20.7 million in 3Q21, following commercial launch on July 15, 2021.
The Company has been granted Real-Time Oncology Review by FDA and plans to submit a supplemental Biologics License Application (sBLA) with additional data in support of a Monday/Wednesday/Friday (M/W/F) intramuscular dosing schedule in early 2022.
The Company is presenting data, for the first time, from the Phase 2/3 study of Rylaze in patients with ALL/LBL who developed hypersensitivity or silent inactivation to a long-acting E. coli–derived asparaginase, at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held December 11-14, 2021.
The Company anticipates that data from the current development program will support regulatory filings in Europe in mid-2022, with potential for approval in 2023. The Company is also working with a partner to advance the program for potential filing, approval and launch in Japan.
Rylaze is the only recombinant Erwinia asparaginase manufactured product that maintains a clinically meaningful level of asparaginase activity throughout the entire duration of treatment. It was developed by the Company to address the needs of patients and healthcare providers for an innovative, high-quality Erwinia asparaginase with reliable supply.
Vyxeos (daunorubicin and cytarabine) liposome for injection:

Vyxeos net product sales increased 13% to $34.7 million in 3Q21 compared to the same period in 2020.
Defitelio (defibrotide sodium) / defibrotide:

Defitelio/defibrotide net product sales increased 15% to $57.7 million in 3Q21 compared to the same period in 2020.

GAAP net income (loss) for 3Q21 was ($52.8 million), or ($0.86) per diluted share, compared to $148.2 million, or $2.64 per diluted share, for 3Q20.

Non-GAAP adjusted net income for 3Q21 was $261.4 million, or $4.20 per diluted share, compared to $242.1 million, or $4.31 per diluted share, for 3Q20.

Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.

Total revenues increased 39% in 3Q21 compared to the same period in 2020.

Products launched or acquired since 2019 comprised 52% of total net product sales in 3Q21.
Neuroscience net product sales in 3Q21 increased 41% to $646.1 million compared to the same period in 2020. In 3Q21, oxybate net product sales increased to $460.4 million led by strong Xywav net product sales of $153.1 million partially offset by a decrease in Xyrem net product sales as a result of the strong adoption of Xywav by existing Xyrem patients. Epidiolex/Epidyolex net product sales in 3Q21 were $160.4 million, following the GW Acquisition in 2Q21.
Oncology net product sales in 3Q21 increased 34% to $184.8 million compared to the same period in 2020 primarily driven by an increase in Zepzelca net product sales of $34.8 million. Zepzelca launched in the U.S. in July 2020.
Operating expenses changed over the prior year period primarily due to the following:

Cost of product sales increased in 3Q21 compared to the same period in 2020, on a GAAP and on a non-GAAP adjusted basis, due to increased net product sales as a result of the GW Acquisition. In addition, an acquisition accounting inventory fair value step-up expense of $82.6 million impacted GAAP cost of product sales.
Selling, general and administrative (SG&A) expenses increased in 3Q21 compared to the same period in 2020, on a GAAP and on a non-GAAP adjusted basis, primarily due to an increase in compensation-related expenses driven by higher headcount as a result of the GW Acquisition and the addition of costs related to Epidiolex, as well as an increase in other expenses related to the expansion of our business including investments to support the Company’s recent product launches. SG&A expenses in 3Q21 on a GAAP basis also included transaction and integration related expenses of $53.4 million related to the GW Acquisition.
Research and development expenses increased in 3Q21 compared to the same period in 2020, on a GAAP and on a non-GAAP adjusted basis, primarily due to the addition of costs related to clinical programs for Epidiolex, nabiximols and cannabinoids and an increase in compensation-related expenses due to higher headcount primarily driven by the GW Acquisition.
Cash Flow and Balance Sheet

As of September 30, 2021, cash and cash equivalents were $671.8 million, and the outstanding principal balance of the Company’s long-term debt was $6.6 billion compared to $7.1 billion as of June 30, 2021. In addition, the Company had undrawn borrowing capacity under a revolving credit facility of $500.0 million.

For the nine months ended September 30, 2021, the Company generated $600.8 million of cash from operations.

During the third quarter, and aligned to its stated deleveraging target, the Company made significant debt repayments of $477.6 million which included the repayment on maturity of the remaining balance on its 1.875% exchangeable senior notes due 2021 and a voluntary payment on its term loan B.

2021 Financial Guidance1

Jazz Pharmaceuticals is updating its full year 2021 financial guidance. This guidance reflects the Company’s current and future expected operational performance, including COVID-19 related impacts, the strength of its underlying operations and the prioritization of new and ongoing value creating development projects.

The Company is raising its full-year earnings guidance, resulting in a reduced GAAP net loss and increased non-GAAP adjusted net income (ANI) on an absolute and per share basis. The updated non-GAAP ANI range exceeds the upper end of the prior range. The Company is reducing both SG&A and R&D expense guidance on a GAAP and non-GAAP adjusted basis, reflecting progress within its transformation initiatives, improved financial discipline and strategic capital allocation. The Company is narrowing its net sales guidance range for neuroscience and oncology, with a reduced mid-point for oncology net sales guidance which reflects the ongoing impacts of COVID-19 on our legacy products and the Rylaze competitive landscape at launch in 3Q21, resulting in a reduced mid-point for total revenues guidance.

Conference Call Details

Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. ET (9:30 p.m. GMT) to provide a business and financial update and discuss its 3Q21 results. The live webcast may be accessed from the Investors section of the Company’s website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 5888822.

A replay of the conference call will be available through November 16, 2021 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 5888822. An archived version of the webcast will be available for at least one week in the Investors section of the Company’s website at www.jazzpharmaceuticals.com.