On May 5, 2021 Vericel Corporation (NASDAQ:VCEL), a leader in advanced therapies for the sports medicine and severe burn care markets, reported financial results and business highlights for the first quarter ended March 31, 2021 (Press release, Vericel, MAY 5, 2021, View Source [SID1234579257]).

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First Quarter 2021 Financial Highlights

Total net revenue increased 30% to $34.6 million, compared to $26.7 million in the first quarter of 2020
MACI net revenue of $23.8 million, Epicel net revenue of $9.8 million and NexoBrid revenue of $0.9 million related to the U.S. Biomedical Advanced Research and Development Authority (BARDA) procurement for emergency response preparedness
Gross margin of 66%, compared to 63% in the first quarter of 2020
Net loss of $3.3 million, or $0.07 per share, compared to $4.7 million, or $0.10 per share, in the first quarter of 2020
Non-GAAP adjusted EBITDA of $4.6 million, or 13% of net revenue, compared to adjusted EBITDA loss of $0.7 million in the first quarter of 2020
Operating cash flow of $10.1 million
As of March 31, 2021, the Company had $110 million in cash and investments, compared to $100 million as of December 31, 2020, and no debt
Business Highlights and Updates

MACI implant and biopsy growth of more than 20% compared to the first quarter of 2020
Epicel net revenue growth of 54% compared to the first quarter of 2020, with record monthly volume in February and the second highest quarterly Epicel revenue in history
Expansion of UnitedHealthcare’s MACI medical policy to include patella and multiple cartilage defects in the knee
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"We entered 2021 with a great deal of momentum and delivered another quarter of strong results across both our sports medicine and burn care franchises," said Nick Colangelo, President and CEO of Vericel. "Our first quarter results demonstrate the strength of the Company’s financial profile as we continue to generate strong revenue growth and increase profitability and cash flow. Based on these results and our strong underlying business fundamentals, we remain on track for significant growth across both of our franchises and have raised our full-year 2021 revenue and adjusted EBITDA guidance."

Full-Year 2021 Financial Guidance

Total net revenue now expected to be in the range of $165-$168 million, compared to previous guidance of approximately $161-$164 million
Adjusted EBITDA margin now expected to be in the range of 21.5% to 23.5%, compared to previous guidance of 21 to 23%
Gross margin guidance of 70% to 71% and estimated operating expenses of approximately $115 million maintained
First Quarter 2021 Results
Total net revenue for the quarter ended March 31, 2021 increased 30% to $34.6 million, compared to $26.7 million in the first quarter of 2020. Total net product revenue for the quarter included $23.8 million of MACI (autologous cultured chondrocytes on porcine collagen membrane) net revenue and $9.8 million of Epicel (cultured epidermal autografts) net revenue, compared to $20.3 million of MACI net revenue and $6.4 million of Epicel net revenue, respectively, in the first quarter of 2020. Total net revenue for the quarter also included $0.9 million of revenue related to the procurement of NexoBrid (concentrate of proteolytic enzymes enriched in bromelain) by BARDA for emergency response preparedness.

Gross profit for the quarter ended March 31, 2021 was $23.0 million, or 66% of net revenue, compared to $16.8 million, or 63% of net revenue, for the first quarter of 2020.

Total operating expenses for the quarter ended March 31, 2021 were $26.3 million, compared to $21.8 million for the same period in 2020. The increase in operating expenses was primarily due to an increase in stock-based compensation expense driven by share price appreciation over the past year.

Net loss for the quarter ended March 31, 2021 was $3.3 million, or $0.07 per share, compared to $4.7 million, or $0.10 per share, for the first quarter of 2020.

Non-GAAP adjusted EBITDA for the quarter ended March 31, 2021 was $4.6 million, or 13% of net revenue, compared to an adjusted EBITDA loss of $0.7 million in the first quarter of 2020. A table reconciling non-GAAP measures is included in this press release for reference.

As of March 31, 2021, the Company had $110 million in cash and investments, compared to $100 million as of December 31, 2020, and no debt.

Conference Call Information
Today’s conference call will be available live at 8:30am Eastern Time and can be accessed through the Investor Relations section of the Vericel website at View Source." target="_blank" title="View Source." rel="nofollow">View Source A slide presentation with highlights from today’s conference call will be available on the webcast and in the Investor Relations section of the Vericel website. Please access the site at least 15 minutes prior to the scheduled start time in order to download the required audio software, if necessary. To participate in the live call by telephone, please call (877) 312-5881 and reference Vericel Corporation’s first quarter 2021 investor conference call. If calling from outside the U.S., please use the international phone number (253) 237-1173.

If you are unable to participate in the live call, the webcast will be available at View Source until May 5, 2022. A replay of the call will also be available until 11:30am (EDT) on May 12, 2021 by calling (855) 859-2056, or from outside the U.S. by calling (404) 537-3406. The conference ID is 9036676.

On May 5, 2021 Inivata, a leader in liquid biopsy, reported that NeoGenomics, Inc (NASDAQ: NEO.), a leading provider of cancer-focused genetic testing services and global oncology contract research services, has agreed to acquire Inivata (Press release, Inivata, MAY 5, 2021, View Source [SID1234579315]). The acquisition follows a $25 million minority equity investment by NeoGenomics in Inivata in May 2020, at which time NeoGenomics was granted a fixed price option to purchase the remainder of Inivata for $390 million.

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Inivata, with its leading liquid biopsy technology platform, will remain a separate business division alongside NeoGenomics’ growing clinical, pharma and informatics divisions. Current Inivata CEO Clive Morris will become the President of Inivata and will report to Mark Mallon, CEO of NeoGenomics.

Inivata will be focused on the continued development of leading liquid biopsy tests including RaDaR, the highly sensitive personalized assay for the detection of residual disease and recurrence (MRD). Proof-of-principle data presented at this year’s AACR (Free AACR Whitepaper) Annual Meeting showed RaDaR demonstrated excellent specificity (100%) and sensitivity (100%) in detecting MRD in patient cohorts with head and neck cancer and early-stage breast cancer. These results provided growing evidence in support of RaDaR’s capabilities in different cancer types, building on data previously reported in non-small cell lung cancer (NSCLC), where RaDaR detected ctDNA 6-12 months ahead of clinical progression in the majority of cases and predicted lower progression free survival.

Clive Morris, CEO of Inivata, commented: "Joining the NeoGenomics Group provides Inivata with an excellent foundation to support our growth ambitions. Our two organizations have highly complementary capabilities and we are excited to combine with NeoGenomics following a successful year of working together. By leveraging our combined resources, we expect to accelerate the development of our promising RaDaR minimal residual disease assay and bolster commercialization efforts with biopharma before driving a successful launch into the clinical setting."

Mark Mallon, CEO of NeoGenomics, commented: "NeoGenomics has spent the better part of the last year working in partnership with the exceptional team of professionals at Inivata while conducting confirmatory due diligence on Inivata and its world-leading liquid biopsy platform technology. We are exercising our option to purchase Inivata eight months ahead of plan and are delighted to welcome Inivata’s world class team of liquid biopsy experts and talented employees to NeoGenomics."

Douglas M. VanOort, Executive Chairman of NeoGenomics commented: "Testing for minimal residual disease has the potential to revolutionize oncology care to benefit millions of patients as they manage through their cancer journey and we are excited to innovate our offerings with Inivata’s best-in-class platform. Combining Inivata’s compelling technology with our unrivaled reach into the clinical community channel and existing relationships with biopharma is a winning strategy."

Perella Weinberg Partners LP acted as financial advisor to Inivata. Inivata was represented by K&L Gates LLP through a cross-border, multi-disciplinary transactional legal team spread across offices in Raleigh, Chicago, New York, San Francisco, Washington D.C. and London.

On May 5, 2021 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that preclinical data on its agonist anti-CD40 antibody mitazalimab has been published in the scientific journal Cancer Immunology, Immunotherapy (Press release, Alligator Bioscience, MAY 5, 2021, View Source [SID1234579145]).

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The published data show that mitazalimab activates dendritic cells and tumor-reactive T cells resulting in enhanced anti-tumor efficacy in combination with a model cancer vaccine.

Title: The human anti-CD40 agonist antibody mitazalimab (ADC-1013; JNJ-64457107) activates antigen-presenting cells, improves expansion of antigen-specific T cells and enhances anti-tumor efficacy of a model cancer vaccine in vivo

Journal: Cancer Immunology, Immunotherapy

View Source

Authors: Adnan Deronic et al.

"A publication in a prominent peer-reviewed journal such as Cancer Immunology, Immunotherapy is very encouraging and validates our findings. These data further highlight the strong potential for mitazalimab to be combined with other therapies, including chemotherapies, immune checkpoint inhibitors and cancer vaccines," said Malin Carlsson, interim CEO at Alligator.

A summary of the findings was presented at the World Immunotherapy Congress 2021 on March 31, 2021, by Alligator’s CSO, Peter Ellmark.

Mitazalimab is Alligator’s most advanced drug candidate for immunotherapy and is designed for the treatment of metastatic cancers, including pancreatic cancer. A phase II study, OPTIMIZE-1, in patients with pancreatic cancer is under preparation. The Phase II study is an open-label, multicenter study to assess the clinical efficacy of mitazalimab as a first-line therapy combined with chemotherapy (mFOLFIRINOX). It will be conducted at several European medical centers and inclusion of the first patient is planned for the first half of 2021.

On May 5, 2021 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the first quarter of 2021 and recent business highlights (Press release, Ionis Pharmaceuticals, MAY 5, 2021, View Source [SID1234579178]).

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"In the first quarter, we took important steps to maximize the value of our wholly owned pipeline. We recently initiated pivotal studies with our wholly owned FUS-ALS and Alexander disease programs. We delivered positive results from our IONIS-PKK-LRx program, demonstrating its potential to change the standard of care for patients with hereditary angioedema. We also further strengthened the business and continued executing on our strategic priorities," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "This summer, we expect data from our IONIS-MAPTRx program in Alzheimer’s disease patients. And later this year, we look forward to data from the Phase 3 VALOR study of tofersen in patients with SOD1-ALS. If results from the VALOR study are positive, we expect tofersen to be our next commercial medicine. These key upcoming catalysts, together with our recent achievements, position us well to have 12 or more products on the market in 2026."

First Quarter 2021 and Recent Summary Financial Results

On track to achieve 2021 financial guidance reflecting investments in Ionis’ wholly owned pipeline, based on the following first quarter results
$112 million in total revenues
$159 million of operating expenses on a non-GAAP basis(1) and $204 million on a GAAP basis
Net loss of $45 million on a non-GAAP basis(1) and $90 million on a GAAP basis
Further strengthened the Company’s balance sheet with pro forma cash of $2.1 billion, after reflecting the convertible notes transaction
Enables expansion of manufacturing and R&D capacity
$632.5 million principal due in April 2026 with 0% interest and an effective conversion price of $76.39 after the purchase of a call spread
Will realize interest expense savings while keeping potential future dilution nearly flat
Repurchased approximately 80% of previously outstanding 1% convertible notes due in November 2021
"So far this year, we have taken important steps in support of developing and commercializing our wholly owned medicines. In addition to completing the restructuring of our European operations, we expanded our Sobi distribution agreement to include North America. These transactions unlocked significant resources that we are now redirecting towards our highest priority programs, including IONIS-TTR-LRx and IONIS-APOCIII-LRx," said Elizabeth L. Hougen, chief financial officer of Ionis. "We are on track to meet our 2021 financial guidance. In the second half of this year, we expect R&D revenue to increase as many of our partnered programs continue to advance. Importantly, we are well-capitalized with the resources we need to expand our manufacturing and R&D capacity to support the future needs of our wholly owned pipeline. This large capital project, which is now underway, is necessary to successfully execute on our goal to drive growth."

(1)

All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards and expenses related to the Akcea acquisition and restructured European operations and the related tax effects. Please refer to the section below titled "Financial Impacts of Akcea Acquisition and Restructured Operations" for a summary of the costs specific to these transactions. Additionally, please refer to the detailed reconciliation of non-GAAP and GAAP measures, which is provided later in this release.

First Quarter 2021 Marketed Products Highlights

SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages
$521 million in worldwide sales in the first quarter
More than 11,000 patients worldwide were on therapy at the end of the first quarter across post-marketing, expanded access and clinical trial settings
Higher-dose SPINRAZA demonstrated safety and tolerability consistent with the currently approved dose in the open-label safety cohort of the DEVOTE study, enabling enrollment in the blinded, pivotal cohort to get underway
TEGSEDI and WAYLIVRA: important medicines approved for the treatment of patients with severe rare diseases
Completed the transition of European operations to Swedish Orphan Biovitrum AB (Sobi) and expanded the distribution agreement to include North American TEGSEDI operations
First Quarter 2021 and Recent Pipeline Events

Phase 3 Pipeline: growing and positioned for 12 or more products on the market in 2026
Advanced ION363 into a Phase 3 study in patients with FUS-ALS
Advanced tofersen into the Phase 3 ATLAS study in presymptomatic SOD1-ALS patients
Roche reported tominersen data related to the dosing halt in the Phase 3 program
Mid-stage Pipeline: advancing multiple medicines with potential to change the standard of care for patients with severe diseases
Reported positive topline IONIS-PKK-LRx results in patients with hereditary angioedema
Advanced ION373 into the Phase 2 portion of a pivotal study in patients with Alexander disease
Advanced the IONIS-AGT-LRx development program:
Reported positive Phase 2 data in JACC: Basic to Translational Science
Advanced into a Phase 2b study in patients with hypertension uncontrolled with three or more antihypertensive medications
Advanced into a Phase 2 study in patients with chronic heart failure with reduced injection fraction
Advanced the ongoing Phase 2 study of ION541 in patients with ALS regardless of family history, resulting in a $10 million payment from Biogen
Upcoming 2021 Pipeline Catalysts(2)

(2) Timing of partnered program catalysts based on partners’ most recent publicly available disclosures

First Quarter 2021 Financial Results

TEGSEDI and WAYLIVRA revenue, net

Amortization from upfront payments

20The Company’s commercial revenue in the first quarter of 2021 was consistent with the same period last year. As the Company completes its transition of TEGSEDI operations in North America to Sobi, the Company’s commercial revenue from product sales will shift to distribution fees based on net sales generated by Sobi.

The Company’s R&D revenue decreased in the first quarter of 2021 compared to the same period last year primarily because the Company earned more milestone payments in the first quarter of 2020 than the same period this year. The Company expects its R&D revenue to increase in the second half of 2021 compared to the first half.

Financial Impacts of Akcea Acquisition and Restructured Operations

In conjunction with the Akcea acquisition and restructured European operations, in the first quarter of 2021, the Company incurred $7 million of costs, which it excluded from its non-GAAP amounts for the period. Refer to the detailed reconciliation of non-GAAP and GAAP measures that is provided later in this release. The Company expects to incur additional expenses in the range of $11 million to $14 million related to the restructuring of its North American TEGSEDI operations from the expanded distribution agreement with Sobi. The company will reflect the North American TEGSEDI restructuring costs primarily in the second quarter of 2021.

Operating Expenses

Ionis’ operating expenses for the first quarter of 2021 increased compared to the same period last year driven primarily by the Company’s investments in advancing its late-stage wholly owned pipeline.

Net Loss Attributable to Ionis Common Stockholders

Ionis’ net loss attributable to Ionis’ common stockholders for the first quarter of 2021 increased compared to the same period in the prior year for the reasons discussed above.

Balance Sheet

Ionis ended March 2021 with cash, cash equivalents and short-term investments of $1.8 billion, compared to $1.9 billion at December 31, 2020. In April 2021, Ionis issued $632.5 million of 0% senior convertible notes due in April 2026 and repurchased $247.9 million of its 1% senior convertible notes. After reflecting these transactions, Ionis’ pro forma cash, cash equivalents and short-term investments was $2.1 billion.

The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.

Webcast

Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast to discuss this earnings release and related activities. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address.

On May 5, 2021 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported business updates and financial results for the first quarter ended March 31, 2021 (Press release, Alector, MAY 5, 2021, View Source [SID1234579189]).

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"Positive momentum continues to build throughout our early and late-stage product pipeline," said Arnon Rosenthal, Ph.D., co-founder and chief executive officer of Alector. "Our pivotal AL001 INFRONT-3 Phase 3 trial and AL002 INVOKE-2 placebo-controlled Phase 2 trial continue to enroll patients, and we look forward to sharing additional data from the AL001 Phase 2 clinical development program in people with FTD-GRN this summer at AAIC. In parallel, we continue to prepare for the initiation of a Phase 2 study evaluating AL001 in people with amyotrophic lateral sclerosis and advance our early-stage novel immuno-oncology candidates toward first-in-human studies. This is an exciting time for Alector and we look forward to continued execution on our clinical and corporate objectives."

Key Business Updates

Progranulin Portfolio:

Alector plans to present updated data from the ongoing AL001 Phase 2 open-label study at the upcoming Alzheimer’s Association International Conference (AAIC), taking place July 26-30, 2021.

Enrollment is ongoing in INFRONT-3, a randomized, placebo-controlled, pivotal Phase 3 trial evaluating AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). The ongoing pivotal trial is evaluating the efficacy and safety of AL001 in at-risk and symptomatic participants with FTD-GRN. Participants in the trial will be given the option to continue receiving treatment in an open-label extension study.

The Company is on track to initiate a Phase 2 study evaluating AL001 in people with amyotrophic lateral sclerosis (ALS) caused by C9orf72 repeats, which share TDP-43 pathology with FTD-GRN in the second half of 2021.
Alzheimer’s Disease Portfolio:

Alector continues patient enrollment in INVOKE-2, a Phase 2 trial evaluating AL002 in people with early Alzheimer’s disease. The randomized, double-blind, placebo-controlled, dose-ranging, multi-center Phase 2 study is expected to enroll approximately 265 participants with early Alzheimer’s disease (AD) at up to 90 sites globally. The AL002 clinical program is being developed in collaboration with AbbVie.

Alector plans to present two posters highlighting AL002 at AAIC, taking place July 26-30, 2021. The first poster will include the results of the AL002 Phase 1 study in healthy volunteers and the second poster will highlight the study design of the ongoing INVOKE-2 trial in people with early AD.

Data from the Phase 1b study evaluating AL003 in participants with AD is expected in 2021. Alector initiated the Phase 1b study in January 2020 and completed enrollment in 2020. The AL003 clinical development program is being developed in collaboration with AbbVie. Preliminary results from the study are expected to be presented at a scientific medical meeting in the second half of 2021.
Early-Stage Pipeline with Potential in Neurodegenerative Diseases and Oncology

The Company continues to progress AL044, a first-in-class antibody targeting the MS4A4A receptor. MS4A4A is a major risk gene for AD that encodes a transmembrane receptor protein that is expressed selectively in microglia in the brain and is associated with control of microglia functionality and potential viability.

Planning is underway to initiate first-in-human studies for AL008 and AL009, two oncology programs, in 2022. AL008 is a novel, investigational, antibody product candidate with a dual mechanism of action that combines inhibition of the CD47-SIRP-alpha (SIRPα) pathway with stimulation of activating Fc receptors and has the potential to yield a best-in-class product. AL009 is a first-in-class multi-Siglec inhibitor that works to enhance the innate and adaptive immune system response by blocking a critical glycan checkpoint pathway that drives immune inhibition.
First Quarter 2021 Financial Results

Revenue. Collaboration revenue for the quarter ended March 31, 2021, was $4.1 million, compared to $7.2 million for the same period in 2020. Revenue is recognized as the program costs are incurred by measuring actual costs incurred to date compared to the overall total expected costs to satisfy the performance obligation. Changes in estimates for revenue recognized over time are recognized on a cumulative basis.

R&D Expenses. Total research and development expenses for the quarter ended March 31, 2021, were $45.7 million, compared to $34.6 million for the same period in 2020. This increase was mainly driven by an increase in expenses to support the advancement of clinical and pre-clinical programs across several therapeutic initiatives as well as an increase in personnel-related expenses.

G&A Expenses. Total general and administrative expenses for the quarter ended March 31, 2021, were $11.1 million, compared to $14.6 million for the same period in 2020. This decrease was primarily due to a decrease in legal fees related to the conclusion of our arbitration proceedings for certain intellectual property matters.

Net Loss. For the quarter ended March 31, 2021, Alector reported a net loss of $52.2 million, compared to a net loss of $40.0 million for the same period in 2020.

Cash Position. Cash, cash equivalents, and marketable securities were $362.7 million as of March 31, 2021.