On February 25, 2021 Nektar Therapeutics (Nasdaq: NKTR) reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Nektar Therapeutics, FEB 25, 2021, View Source [SID1234575668]).

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Cash and investments in marketable securities at December 31, 2020 were approximately $1.2 billion as compared to $1.6 billion at December 31, 2019.

"This past year, Nektar made significant progress advancing our clinical pipeline of novel cytokine therapeutics," said Howard W. Robin, President and CEO of Nektar. "Our broad registrational program evaluating bempegaldesleukin (BEMPEG) plus nivolumab is progressing well with five registrational studies underway in melanoma, renal cell carcinoma and bladder cancer. We also recently added a sixth study to the registrational program to evaluate BEMPEG in combination with pembrolizumab in head and neck cancer and are pleased to be collaborating with Merck on the study. For our PROPEL study, we look forward to reporting the first data for BEMPEG plus pembrolizumab in patients with metastatic non-small cell lung cancer in the second half of 2021."

"For our second cytokine program, NKTR-255, we were very encouraged by the early signs of clinical activity that we recently reported at the SITC (Free SITC Whitepaper) 2020 meeting, and are now advancing two Phase 1 clinical studies in combination with ADCC antibodies, one in hematological malignancies and one in solid tumors," continued Mr. Robin. "Finally, our partner Eli Lilly is conducting a broad Phase 2 program for NKTR-358, our T regulatory cell IL-2 agent, with Phase 2 studies in both lupus and ulcerative colitis and plans to initiate additional Phase 2 studies in immune-mediated diseases over the next 12-18 months."

Summary of Financial Results

Revenue in the fourth quarter of 2020 was $23.5 million as compared to $33.9 million in the fourth quarter of 2019. Revenue for the year ended December 31, 2020 was $152.9 million as compared to $114.6 million in 2019 and was higher primarily due to the recognition of $50.0 million in total milestones from Bristol-Myers Squibb related to the start of two new registrational trials of bempegaldesleukin plus Opdivo (nivolumab) in adjuvant melanoma and muscle-invasive bladder cancer.

Total operating costs and expenses in the fourth quarter of 2020 were $134.2 million as compared to $143.5 million in the fourth quarter of 2019. Total operating costs and expenses for 2020 were $578.0 million as compared to $554.7 million in 2019. Total operating costs and expenses for full year 2020 increased as compared to 2019 primarily as a result of $45.2 million in impairment charges in the first quarter of 2020 resulting from the discontinuation of the NKTR-181 program, partially offset by a decrease in R&D expense.

R&D expense in the fourth quarter of 2020 was $102.7 million as compared to $110.4 million for the fourth quarter of 2019. R&D expense for the year ended December 31, 2020 was $408.7 million as compared to $434.6 million in 2019. Excluding pre-commercial manufacturing costs for NKTR-181 incurred during 2019, research and development expense increased for the full year 2020 primarily due to the clinical development of bempegaldesleukin in five registrational trials.

G&A expense was $27.1 million in the fourth quarter of 2020 and $27.1 million in the fourth quarter of 2019. G&A expense for 2020 was $104.7 million as compared to $98.7 million in 2019.

Net loss for the fourth quarter of 2020 was $117.2 million or $0.65 basic and diluted loss per share as compared to a net loss of $112.2 million or $0.64 basic and diluted loss per share in the fourth quarter of 2019. Net loss for the year ended December 31, 2020 was $444.4 million or $2.49 basic and diluted loss per share as compared to net loss of $440.7 million or $2.52 basic and diluted loss per share in 2019.

2020 and Year-to-Date 2021 Business Highlights:

In February 2021, Nektar announced a clinical trial collaboration and supply agreement with Merck for a Phase 2/3 study of bempegaldesleukin, Nektar’s investigational IL-2 pathway agent, in combination with Merck’s KEYTRUDA (pembrolizumab) for first-line treatment of patients with metastatic or unresectable recurrent squamous cell carcinoma of the head and neck (SCCHN) whose tumors express PD-L1. The study is planned to start in the second half of 2021.
In February 2021, Nektar announced a financing and co-development collaboration with SFJ Pharmaceuticals for the development of bempegaldesleukin plus pembrolizumab in SCCHN. SFJ has agreed to fund up to $150 million to support the planned Phase 2/3 study and manage clinical trial operations for the study. In return, Nektar agrees to pay SFJ success-based annual milestone payments over a period of seven to eight years which are contingent upon receipt of certain U.S. regulatory approvals for specified indications for bempegaldesleukin, and will begin following completion of the SCCHN study, which is projected to be completed in 2024.
In December 2020, Nektar sold its royalties on future sales of ADYNOVATE and MOVANTIK to Healthcare Royalty Management, LLC in exchange for $150 million.
In December 2020, Nektar announced dosing of the first patient in its Phase 1/2 study of its IL-15 agonist, NKTR-255, in combination with cetuximab in patients with relapsed or refractory head and neck squamous cell carcinoma or colorectal cancer. The study may enroll up to 80 patients at approximately 15 investigator sites in the United States and European Union.
In December 2020, Nektar presented preclinical data for NKTR-255 at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2020 Annual Meeting, underscoring the potential for NKTR-255 as an innovative immunotherapeutic agent in the treatment of multiple myeloma.
In November 2020, Nektar presented new data from its immuno-oncology pipeline at the virtual 2020 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. Updated clinical data from the PIVOT-02 study metastatic melanoma cohort showed that bempegaldesleukin with nivolumab resulted in a durable clinical benefit with median progression-free survival of 30.9 months. NKTR-255 showed biological activity in the first patients treated in the monotherapy dose-escalation phase of the ongoing Phase 1 study in multiple myeloma and non-Hodgkin’s lymphoma. In addition, new data showed that the combination of TLR agonist candidate, NKTR-262, plus bempegaldesleukin alters the tumor micro-environment through activation of both the innate and adaptive arms of the immune system.
In November 2020, Nektar presented new data from its NKTR-358 program at the American College of Rheumatology (ACR) virtual meeting. Data from the Phase 1b study in patients with mild to moderate systemic lupus erythematosus (SLE) showed that NKTR-358 produced a dose-dependent increase in expression of regulatory T cell (Treg) activation markers, providing a rationale for continued development in SLE and other inflammatory indications.
In October 2020, Nektar initiated a Phase 1b clinical study of bempegaldesleukin in adult patients with mild COVID-19 infection. The randomized, double-blind, placebo-controlled trial is designed to assess the safety, tolerability, and pharmacokinetic and pharmacodynamic profile of bempegaldesleukin in adult patients with mild COVD-19.
In August 2020, Vaccibody AS and Nektar announced that the first patient had been dosed in the Phase 1/2a study evaluating bempegaldesleukin with VB10.NEO, Vaccibody’s personalized neoantigen cancer vaccine, in patients with advanced squamous cell carcinoma of the head and neck.
In June 2020, Nektar announced the presentation of results from the Phase 1b study evaluating multiple ascending doses of NKTR-358 at the Annual European Congress of Rheumatology (EULAR 2020) virtual meeting. The data showed that treatment with NKTR-358 was safe and well tolerated in patients with mild-to-moderate SLE and led to a marked and selective, dose-dependent expansion of regulatory T cells (Tregs) that was maintained over multiple administrations.
In May 2020, Nektar announced the publication of clinical data from its PIVOT-02 study evaluating bempegaldesleukin in combination with nivolumab in immunotherapy-naïve patients with advanced solid tumors, including melanoma, renal cell carcinoma (RCC) and non-small cell lung cancer. The data, published in Cancer Discovery, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), showed that bempegaldesleukin plus nivolumab resulted in encouraging overall response rates across multiple tumor types, independent of baseline PD-L1 expression, with responses continuing to deepen over time.
In April 2020, Nektar repaid the principal and accrued interest of its senior notes totaling $254.8 million.
In February 2020, Nektar announced the publication of preclinical bempegaldesleukin data in two manuscripts in Nature Communications showing how bempegaldesleukin works synergistically with multiple immune-based therapies to enhance T-cell-mediated tumor control.
In January 2020, Nektar and Bristol-Myers Squibb announced a new joint development plan that expanded the ongoing registrational program for bempegaldesleukin plus Opdivo from three ongoing registrational trials in first-line metastatic melanoma, first-line cisplatin-ineligible metastatic urothelial cancer and first-line metastatic RCC to include two additional registrational trials in adjuvant melanoma and muscle-invasive bladder cancer. In addition, the expanded development plan includes a Phase 1/2 study to evaluate bempegaldesleukin plus nivolumab in combination with a tyrosine-kinase inhibitor in first-line RCC in order to support a future registrational trial.
In January 2020, Nektar made the strategic business decision to withdraw its New Drug Application (NDA) for NKTR-181, an investigational opioid medicine in development for chronic pain and make no further investment into the program.
Conference Call to Discuss Fourth Quarter and Year-End 2020 Financial Results
Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time, Thursday, February 25, 2021.

This press release and a live audio-only Webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through March 25, 2021.

In the event that any non-GAAP financial measure is discussed on the conference call that is not described in the press release, or explained on the conference call, related information will be made available on the Investors page at the Nektar website as soon as practical after the conclusion of the conference call.

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 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported financial results for the fourth quarter and full year ending December 31, 2020 and provided a corporate update (Press release, PTC Therapeutics, FEB 25, 2021, View Source [SID1234575666]).

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"Despite the challenges of the pandemic, PTC has been able to make significant progress in moving our pipeline forward and has been able to continue to bring therapies to our patients throughout 2020," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "I am very proud of our team and their abilities to execute on our 2020 goals even in such turbulent times. I am confident that we will continue to progress on our 2021 goals that will create value for all of our stakeholders."

Key 2020 Corporate Highlights:

Strong continued revenue growth for the DMD franchise, with total net product revenue of $331 million for Translarna (ataluren) and Emflaza (deflazacort) in 2020.
Translarna growth was driven by broader uptake due to new patients in existing geographies, geographic expansion and label updates.
Emflaza experienced strong 38% year-over-year revenue growth in 2020 due to increased new prescriptions and high compliance.
Evrysdi (risdiplam) was approved by the FDA in August 2020 for treatment of spinal muscular atrophy (SMA) patients aged 2 months and older. A strong global launch is under way, leading to increased patient uptake across all disease subtypes. Evrysdi is a product of a collaboration between PTC, Roche, and the SMA Foundation.
PTC initiated five clinical trials in 2020, three of which are registration-directed clinical studies:
The MIT-E Phase 2/3 trial with vatiquinone for mitochondrial epilepsy with data anticipated in the third quarter of 2022.
The MOVE-FA Phase 3 trial with vatiquinone for Friedreich ataxia with data anticipated in 2023.
The FITE19 Phase 2/3 clinical trial for PTC299 in patients with COVID-19.
A healthy volunteer study for PTC857, the second Bio-e compound.
A healthy volunteer study for PTC518 for the Huntington disease program from the splicing platform.
Submitted a Marketing Authorization Application (MAA) to European Medicines Agency (EMA) for PTC-AADC, a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency.
PTC strengthened its balance sheet with a $650 million upfront payment for a portion of the Evrysdi royalties.
2021 Potential Value-Creating Milestones:

Results from Phase 1 healthy volunteer study of PTC518 for Huntington disease program from the splicing platform are expected in the first half.
Data from the healthy volunteer study for PTC857 are expected in the first half.
PTC expects an opinion on the PTC-AADC MAA from the EMA’s Committee for Medicinal Products for Human Use (CHMP) in the second quarter.
PTC is on track for a submission of a Biologics License Application (BLA) to the FDA in the second quarter for PTC-AADC.
PTC anticipates an opinion from the CHMP for Evrysdi in the first quarter.
A registration-directed study, APHENITY, for PTC923 in patients with phenylketonuria (PKU) is expected to initiate mid-year.
The second stage of the FITE19 Phase 2/3 clinical trial to assess PTC299 in patients with COVID-19 has been initiated. Data from this study is expected in the second half of the year.
PTC expects to dose the first patient with PTC-FA gene therapy for Friedreich ataxia before year end.
Results from the ongoing clinical trials evaluating PTC596 in Leiomyosarcoma (LMS) and Diffuse Intrinsic Pontine Glioma (DIPG) are expected by year end.
Fourth Quarter and Full Year 2020 Financial Highlights:

Total revenues were $118.9 million for the fourth quarter of 2020, compared to $96.5 million for the fourth quarter of 2019. Total revenues were $380.8 million for the full year 2020, compared to $307.0 million for the full year 2019.
Total revenue includes net product revenue across the commercial portfolio of $107.3 million for the fourth quarter of 2020 and $333.4 million for full year 2020, and collaboration and royalty revenue of $11.6 million for the fourth quarter of 2020 and $47.4 million for full year 2020.
Translarna net product revenues were $69.4 million for the fourth quarter of 2020, compared to $48.4 million for the fourth quarter of 2019. Translarna net product revenues were $191.9 million for the full year 2020, compared to $190.0 million for the full year 2019. In October 2020, PTC secured a purchase agreement for Translarna with the Ministry of Health in Brazil specifying two shipments, both of which were fulfilled in 2020.
Emflaza net product revenues were $36.8 million for the fourth quarter of 2020, compared to $32.7 million for the fourth quarter of 2019. Emflaza net product revenues were $139.0 million for the full year 2020, compared to $101.0 million for the full year 2019.
Roche reported Evrysdi 2020 year-to-date sales of approximately CHF 55 million. During the third quarter of 2020, the acceptance of the MAA filed by Roche for Evrysdi for the treatment of SMA triggered a $15 million milestone payment to PTC and the first commercial sale of Evrysdi in the U.S. triggered a $20 million milestone payment to PTC. Additionally, in October 2020, Chugai Pharmaceutical, Co. Ltd, a member of the Roche group, announced that a new drug application for Evrysdi for the treatment of SMA was filed in Japan. The filing in Japan triggered a $7.5 million milestone payment to PTC in the fourth quarter of 2020. As a result, PTC recognized $35.0 million in the third quarter of 2020 and $7.5 million in the fourth quarter of 2020 of collaboration revenue associated with Roche milestone events.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $118 million for the fourth quarter of 2020, compared to $81.8 million for the fourth quarter of 2019. GAAP R&D expenses were $477.6 million for the full year 2020, compared to $257.4 million for the full year 2019. The increase in R&D expenses for the fourth quarter and full year 2020 reflects costs associated with advancing the gene therapy and Bio-e platforms, increased investment in research programs, and advancement of the clinical pipeline. Additionally, the increase in R&D expenses full year 2020 includes one-time charges in 2020 of $53.6 million related to the acquisition of Censa Pharmaceuticals, and $41.4 million related to the MassBiologics of the University of Massachusetts Medical School agreement for commercial manufacturing of our lead gene therapy program in AADC deficiency.
Non-GAAP R&D expenses were $105.3 million for the fourth quarter of 2020, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $76.2 million for the fourth quarter of 2019, excluding $5.6 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $438.9 million for the full year 2020, excluding $38.7 million in non-cash, stock-based compensation expense, compared to $236.6 million for the full year 2019, excluding $20.8 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $75.5 million for the fourth quarter of 2020, compared to $63.5 million for the fourth quarter of 2019. GAAP SG&A expenses were $245.2 million for the full year 2020, compared to $202.5 million for the full year 2019. The increase reflects continued investment to support PTC’s commercial activities including the expanding commercial portfolio, and rent and related expenses associated with entering into a long-term lease for the Hopewell facility that commenced on July 1, 2020.
Non-GAAP SG&A expenses were $66.8 million for the fourth quarter of 2020, excluding $8.7 million in non-cash, stock-based compensation expense, compared to $57.7 million for the fourth quarter of 2019, excluding $5.8 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $213.6 million for the full year 2020, excluding $31.6 million in non-cash, stock-based compensation expense, compared to $181.2 million for the full year 2019, excluding $21.3 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $6.3 million for the fourth quarter of 2020, compared to $12.4 million for the fourth quarter of 2019. Change in the fair value of deferred and contingent consideration was $23.3 million for the full year 2020, compared to $48.4 million for the full year 2019. The change is related to the fair valuation of the potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis), as a result of our merger with Agilis which closed in August 2018. Changes in the fair value were due to the re-calculation of discounted cash flows for the passage of time and changes to certain other estimated assumptions.
Settlement of deferred and contingent consideration was $10.6 million for the full year 2020. The settlement of deferred and contingent consideration is related to a loss upon the settlement of the deferred and contingent consideration liabilities as a result of the rights exchange agreement with certain former shareholders of Agilis, whereby such former shareholders exchanged their pro rata share of specific future cash milestone payments in the aggregate amount of $225 million for a mixture of cash and equity of PTC. Under this agreement, which the former shareholders and PTC entered into on April 29, 2020, PTC has paid $36.9 million in cash and issued 2,821,176 shares of common stock in exchange for the cancellation and forfeiture of the participating shareholders’ rights to receive (i) $174.0 million, in the aggregate, of potential milestone payments based on the achievement of certain regulatory milestones and (ii) $37.6 million, in the aggregate, of $40.0 million in development milestone payments that would have been due upon the passing of the second anniversary of the closing of PTC’s merger with Agilis, regardless of whether the milestones are achieved.
Net loss was $74.4 million for the fourth quarter of 2020, compared to net loss of $77.7 million for the fourth quarter of 2019. Net loss was $438.2 million for the full year 2020, compared to net loss of $251.6 million for the full year 2019.
Cash, cash equivalents, and marketable securities was $1.1 billion at December 31, 2020, compared to $686.6 million at December 31, 2019.
Shares issued and outstanding as of December 31, 2020 were 69,718,096.
PTC Reaffirms Full Year 2021 Guidance as Follows:

PTC anticipates net product revenues for the DMD franchise for the full year 2021 to be between $355 and $375 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $825 and $855 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2021 to be between $725 and $755 million, excluding estimated non-cash, stock-based compensation expense of $100 million.
Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 2174406. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. The accompanying slide presentation will be posted on the investor relations section of the PTC website. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

On February 25, 2021 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, reported fourth quarter and full year financial results for the periods ended December 31, 2020 (Press release, Allogene, FEB 25, 2021, View Source [SID1234575665]).

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"By all measures, 2020 was a year of exceptional growth and success as we progressed development of our AlloCAR T candidates and continued to establish Allogene as a leader in the cell therapy field. We’ve now treated over 75 patients with our AlloCAR T therapies, more than any other company in the field. Continued progress on our first three Phase 1 trials, ALPHA, ALPHA2, and UNIVERSAL, two new IND submissions, including our first in solid tumors, and the establishment of our Allogene Overland Biopharm joint venture highlight our executional capabilities," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "Looking ahead to key milestones this year, we are looking forward to presenting an update on our CD19 program and the possibility of launching our first pivotal trial as well as operationalizing our state-of-the-art AlloCAR T production facility in Newark, California."

Pipeline Highlights

Anti-CD19 AlloCAR T Program

Additional data from the Phase 1 ALPHA study of ALLO-501 in relapsed/refractory non-Hodgkin lymphoma (NHL) and initial data from the Phase 1 ALPHA2 study of ALLO-501A are planned for Q2 2021. The Company intends to initiate a potentially pivotal Phase 2 trial of ALLO-501A by the end of 2021.
ALLO-501A was recently granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA) for the treatment of relapsed/refractory diffuse large B cell lymphoma (DLBCL), a type of NHL. FTD is intended to facilitate the development, and expedites the review of, medicines to treat serious conditions and fill unmet medical need. FTD allows for potentially greater access to the FDA for the purpose of expediting the drug product candidate’s development, review and potential approval.
Anti-BCMA AlloCAR T Program
The Company continues to expand its portfolio of anti-BCMA therapies to realize the potential benefits of an allogeneic approach to patients with multiple myeloma.

ALLO-715 UNIVERSAL Trial
In December 2020, at an oral session of the 62nd Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), the Company reported initial data on ALLO-715, its first AlloCAR T candidate for relapsed/refractory multiple myeloma (MM). The Phase I UNIVERSAL trial utilizes a proprietary lymphodepletion regimen consisting of ALLO-647 (anti-CD52 mAb) and chemotherapy.
As per the ASH (Free ASH Whitepaper) presentation, 31 ALLO-715 treated patients were evaluable for safety and 26 patients were evaluable for efficacy.
Higher CAR T cell doses were associated with an increased response rate and greater AlloCAR T cell expansion.
In the DL3 cohort (320M CAR T+ cells), the overall response rate (ORR) was 60% with 40% of patients achieving a very good partial response (VGPR) or better (VGPR+).
Minimal Residual Disease (MRD) was assessed in five patients with VGPR+ and all five were MRD negative.
Approximately 90% of patients were treated within five days of study enrollment. No bridging therapy was required.
No graft-vs-host disease or Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) was observed. Cytokine Release Syndrome (Grade 1 or 2) was reported in 14 patients (45%) and was manageable with standard therapies. The rate of Grade 3+ infection events was similar to what has been reported in other advanced MM studies. Any Grade 3+ adverse events reported as serious adverse events occurred in 19% of patients. A single Grade 5 event related to progressive myeloma and a cyclophosphamide and ALLO-647 conditioning regimen was reported.
ALLO-715 + nirogacestat
The FDA cleared the Investigational New Drug Application (IND) to evaluate ALLO-715 in combination with the investigational gamma secretase inhibitor nirogacestat, in patients with relapsed/refractory MM. Enrollment has been initiated. Nirogacestat is being developed by SpringWorks Therapeutics.
ALLO-605 TurboCAR
An IND submission is planned for 1H 2021 for ALLO-605, the first anti-BCMA TurboCAR T cell therapy, for use in relapsed/refractory MM. Upon clearance, the IGNITE trial is expected to begin this year. The Company presented preclinical findings supporting ALLO-605 at ASH (Free ASH Whitepaper) in December 2020. TurboCAR technology allows cytokine activation signaling to be engineered selectively into CAR T cells. TurboCAR has the potential to improve efficacy, overcome cell exhaustion, and reduce dosing requirements of AlloCAR T therapy.
Solid Tumor AlloCAR T Program

ALLO-316 (anti-CD70) – TRAVERSE Trial
The FDA cleared an IND to evaluate ALLO-316, Allogene’s first CAR T candidate for solid tumors. The Company expects to initiate the Phase 1 TRAVERSE trial in Q1 2021 to examine safety, tolerability, anti-tumor efficacy, pharmacokinetics, and pharmacodynamics of ALLO-316 in patients with advanced or metastatic clear cell renal cell carcinoma (ccRCC).
ALLO-316 also has potential application in hematologic malignancies. The Company presented preclinical findings of ALLO-316 targeting CD70 in models of acute myeloid leukemia (AML) at ASH (Free ASH Whitepaper) in December and plans to explore AML as a potential second indication for ALLO-316.
Corporate Highlights

Establishment of Allogene Overland Biopharm
The Company and Overland Pharmaceuticals, which is backed by Hillhouse Capital, announced the formation of Allogene Overland Biopharm. The joint venture will have an exclusive license to develop, manufacture and commercialize specific Allogene candidates targeting BCMA, CD70, FLT3, and DLL3 in the licensed territories.
Cell Forge 1 Manufacturing Facility
Construction of the Company’s new state-of-the-art cGMP cell manufacturing facility, Cell Forge 1, in Newark, California has been completed. cGMP manufacturing from this facility is expected to begin in 2021.
Fourth Quarter Financial Results

Research and development expenses were $52.2 million for the fourth quarter of 2020, which includes $7.9 million of non-cash stock-based compensation expense. For the full year of 2020, research and development expenses were $193.0 million. Research and development expense for the year includes $31.3 million of non-cash stock-based compensation expense.
General and administrative expenses were $17.1 million for the fourth quarter of 2020, which includes $8.6 million of non-cash stock-based compensation expense. For the full year of 2020, general and administrative expenses were $65.3 million, which includes $34.0 million of non-cash stock-based compensation expense.
Net loss for the fourth quarter of 2020 was $68.6 million, or $0.53 per share, including non-cash stock-based compensation expense of $16.5 million. For the full year of 2020, net loss was $250.2 million, or $2.08 per share, including non-cash stock-based compensation expense of $65.3 million.
The Company had $1.0 billion in cash, cash equivalents, and investments as of December 31, 2020.
2021 Financial Guidance

Allogene expects full year GAAP Operating Expenses to be between $300 million and $330 million including estimated non-cash stock-based compensation expense of $80 million to $90 million and excluding any impact from potential new business development activities.

Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at 2:00 p.m. Pacific Time /5:00 p.m. Eastern Time to discuss financial results and provide a business update. To access the live conference call by telephone, please dial 1 (866) 940-5062 (U.S.) or 1 (409) 216-0618 (International). The conference ID number for the live call is 4973969. The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

On February 25, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, BioMarin, FEB 25, 2021, View Source [SID1234575664]).

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Net Product Revenues for the fourth quarter of 2020 were essentially flat as compared to the fourth quarter of 2019. The change in Net Product Revenues was primarily attributed to the following:

Kuvan Net Product Revenues decreased by $33.6 million, primarily due to the U.S. loss of market exclusivity in October 2020 resulting from generic competition; and
Aldurazyme Net Product Revenues decreased by $22.7 million due to timing of product fulfillment to Genzyme. Aldurazyme is marketed by Genzyme and BioMarin Aldurazyme revenues are driven by the timing of when the product is released and control is transferred to Genzyme. Revenues for the fourth quarter of 2020 were comparatively lower than 2019 due to such timing. Based on data provided to us by Genzyme, patients receiving commercial Aldurazyme increased by 10% during 2020; partially offset by
Naglazyme and Vimizim Net Product Revenues increased by an aggregate of $35.1 million primarily due to timing of sales in the Middle East and Latin America;
Palynziq Net Product Revenues increased by $17.9 million driven by a combination of revenue from U.S. patients achieving maintenance dosing and new patients initiating therapy; and
Brineura Net Product Revenues increased by $9.8 million driven by growth in the number of patients in all regions.
The increase in GAAP Net Income for the fourth quarter of 2020, compared to the same period in 2019 was primarily due to the following:

decreased research and development (R&D) expense of $16.1 million primarily due to lower clinical activity spend for valoctocogene roxaparvovec gene therapy programs and decreased tralesinidase alfa costs as the program was licensed to a third-party in 2019; and
an increase in the benefit from income taxes of $37.5 million primarily due to the change in the jurisdictional mix of earnings and the related tax impact from the completion of an intra-entity transfer of certain intellectual property rights to an Irish subsidiary where the Company’s ex-US regional headquarters are located during the third quarter of 2020; partially offset by
an increase in Cost of Sales of $30.2 million primarily due to inventory reserves and higher sales volumes of products with lower margins.
Non-GAAP Income for the fourth quarter of 2020 decreased to $39.5 million, compared to Non-GAAP Income of $46.4 million for the same period in 2019. The decrease in Non-GAAP Income for the quarter, compared to the same period in 2019, was primarily attributed to lower gross profits and higher SG&A expenses, partially offset by lower R&D expenses.

As of December 31, 2020, BioMarin had cash, cash equivalents and investments totaling $1.35 billion, which includes net proceeds of $535.8 million from the Company’s May 2020 convertible debt offering, as compared to $1.17 billion as of December 31, 2019. On October 15, 2020, the Company’s 1.50% senior subordinate convertible notes matured and were settled in cash for approximately $375.0 million.

Commenting on full-year 2020 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "Despite the impact in 2020 from the COVID-19 pandemic and a delay in the potential approval of valoctocogene roxaparvovec for severe hemophilia A, demand for our current product portfolio continued to drive steady revenue growth and expansion of our pipeline. Excluding contributions from Kuvan, for which a generic became available during 2020, total revenues grew 13% in 2020, and generated $85 million of positive operating cash flows for the full year, underscoring the essential nature of our medicines."

Mr. Bienaimé continued, "The most recent Phase 3 data updates from our latest-stage development programs in achondroplasia and severe hemophilia A demonstrated significant efficacy. In the largest gene therapy trial ever conducted for the treatment of severe hemophilia A, we were pleased that valoctocogene roxaparvovec was the first in hemophilia A to demonstrate statistically significant evidence of annualized bleed rate superiority over standard of care recombinant FVIII. Based on these results, we are very encouraged that one infusion of valoctocogene roxaparvovec gene therapy may potentially address the high treatment burden for people with severe hemophilia A. We are targeting submission of the one-year Phase 3 results to the European Medicines Agency in the second quarter of 2021 and planning to dialog with the FDA to align on steps to obtain approval in the United States."

"Also in 2021, we look forward to the potential approval of vosoritide, which would be the first pharmacological treatment to address the underlying cause of achondroplasia, the most common form of dwarfism. We announced in the fourth quarter of 2020 that vosoritide demonstrated sustained growth effects for over two years in children with achondroplasia participating in our Phase 3 extension study. In addition to the large, Phase 3 program currently in the extension phase, we have built a multi-pronged dossier of additional studies to support our understanding of the unmet medical need for children with achondroplasia and the effects of vosoritide in this condition. In addition to the highly statistically significant placebo-controlled Phase 3 results, the program includes the long-term clinical results in 5 to 18 year-olds from our Phase 2 study, natural history data, and the ongoing study of newborns through 5 years. Many families are keen to seek early treatment for their children so we are hopeful that, if approved, vosoritide will become available later in 2021 upon potential approvals."

Key Program Highlights

Valoctocogene roxaparvovec gene therapy for severe hemophilia A: On January 10, 2021, the Company announced positive top-line, one-year data results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. Data from the study in the pre-specified primary analysis for Annualized Bleeding Rate (ABR) showed that a single dose of valoctocogene roxaparvovec significantly reduced ABR by 84% compared with prior treatment with prophylactic FVIII infusions. These results were from a pre-specified group of participants in a non-interventional prospective baseline observational study (rollover population; N=112) with a median follow-up of 60.1 weeks after dosing with valoctocogene roxaparvovec. 80% of the rollover participants were bleed-free starting at week five after treatment.
Additionally, at the end of the first year post-infusion with valoctocogene roxaparvovec, participants in the modified intent-to-treat (mITT) population (N=132) had a mean endogenous Factor VIII expression level of 42.9 (SD 45.5, median 23.9) IU/dL, as measured by the chromogenic substrate (CS) assay, supporting the marked clinical benefits observed with abrogation of bleeding episodes and Factor VIII infusion treatment rate. Factor VIII expression declined at a slower rate compared to the Phase 1/2 study, and remained in a range to provide hemostatic efficacy. In a subset of the mITT population that had been dosed at least two years prior to the data cut date (N=17), Factor VIII expression declined from a mean of 42.2 (SD 50.9, median 23.9) IU/dL at the end of year one to a mean of 24.4 (SD 29.2, median 14.7) IU/dL at the end of year two with continued hemostatic efficacy demonstrated by a mean ABR of 0.9 (median 0.0) bleeding episodes per year.

Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII usage in the rollover population by 99% from 135.9 (median 128.6) to 2.0 (median 0.0) infusions per year (p-value <0.0001).

In the U.S., the FDA recommended that the Company complete the Phase 3 study and submit two-year follow-up safety and efficacy data on all study participants. The Company plans to meet with FDA to review the two-year data request and share the Phase 3 GENEr8-1 results announced on January 10, 2021. BioMarin is targeting submission of the Marketing Authorization Application (MAA) with these results to the EMA in the second quarter of 2021 pending confirmation in presubmission meetings.

Vosoritide for children with achondroplasia: On December 21, 2020 the Company announced that children in the open-label long-term extension of the Phase 3 study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP), maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment. An analysis, comparing all children randomized and treated with vosoritide for two years (n=52) to all children from the run-in study who were randomized to receive placebo with an untreated observation period of two years (n=38), showed improvement in one-year height change in the treated group relative to the untreated group that was similar in the second year of treatment, 1.79 cm, as in the first year of treatment, 1.73 cm. The cumulative height gain over the 2-year treatment period was 3.52 cm more than the untreated children.
In 2020, marketing applications for vosoritide were validated and accepted by EMA and FDA, respectively. The CHMP opinion is expected in Europe in June of 2021. The U.S. New Drug Application (NDA) for vosoritide is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of August 20, 2021.

In January 2021, the Company received notice from FDA that the NDA for vosoritide had been granted Priority Review Designation. Under this designation, the vosoritide NDA may qualify for a Priority Review Voucher (PRV) upon approval. A PRV confers priority review to a subsequent drug application that would not otherwise qualify for that designation. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

Palynziq for PKU: On October 7, 2020 the Company announced that the FDA approved the supplemental Biologics License Application (sBLA) to increase the maximum allowable dose of Palynziq (pegvaliase-pqpz) Injection for treatment of adults with PKU to 60 mg daily. Previously, the maximum dose was 40 mg daily. In the Phase 3 PRISM studies, 19% of study participants required a 60 mg dose to achieve adequate response to Palynziq.
Palynziq is indicated to reduce blood Phe concentrations in adults with PKU, who have uncontrolled blood Phe concentrations greater than 600 μmol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first and only approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe.

BMN 307 gene therapy product candidate for PKU: The Company announced that it plans to dose escalate in PHEarless, the Phase 1/2 study of BMN 307 based on encouraging Phe lowering and safety signals observed in study participants who were treated with the lowest dose. Both the FDA and EMA granted BMN 307 Orphan Drug Status. Additionally, the FDA has granted Fast Track status to BMN 307. Product for use in the Phase 1/2 study was made at commercial scale from BioMarin’s award-winning gene therapy manufacturing facility.
BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): IND-enabling studies are ongoing with BMN 331, BioMarin’s third gene therapy candidate, for the treatment of HAE. BioMarin plans to leverage its broad expertise in developing gene therapies for severe hemophilia A and PKU to improve efficiencies in the development process of BMN 331.
DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR received an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales.
BMN 255 for a subset of chronic renal disease: On January 11, 2021 the Company announced that it filed an IND in 2020 for BMN 255, a small molecule for a subset of chronic renal disease. BMN 255 was driven by genetic discoveries for both mechanism and for identifying individuals for treatment.
BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies are underway with BMN 351, an oligonucleotide therapy that has demonstrated a high-level of protein expression in experimental animals possessing skippable dystrophic mutations and at doses that are promising in regard to safety. The Company intends to determine timing of a potential IND filing at the end of the year based on results of ongoing IND-enabling studies.
BioMarin will host a conference call and webcast to discuss fourth quarter and full-year 2020 financial results today, Thursday, February 25, 2021 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.