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 Horizon Therapeutics plc (Nasdaq: HZNP) reported first-quarter 2021 financial results (Press release, Horizon Pharma, MAY 5, 2021, View Source [SID1234579256]). The Company updated its full-year 2021 net sales guidance and its adjusted EBITDA guidance to incorporate Viela Bio, Inc., which was acquired on March 15, 2021.

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"We continued to advance our position as one of the fastest-growing biotechs, completing the milestone acquisition of Viela with its strong biologics pipeline, R&D capabilities and rare disease medicine, UPLIZNA," said Tim Walbert, chairman, president and chief executive officer, Horizon. "We were very pleased with our performance in a quarter challenged by the supply disruption of TEPEZZA due to U.S. government-mandated COVID-19 vaccine orders. With the resumption of TEPEZZA supply, our expanded pipeline and our strong ability to execute, we are well positioned to continue to drive significant value for our patients, shareholders and all of our stakeholders."

First-Quarter and Recent Company Highlights

Completed Acquisition of Viela: On March 15, 2021, the Company completed the acquisition of Viela, representing a significant step forward in advancing the Company’s strategy to expand its pipeline to accelerate long-term sustainable growth. The acquisition advances the Company’s strategy in four ways: it adds a deep, mid-stage biologics pipeline with four candidates in nine development programs; it expands the capabilities of the Company’s R&D organization with Viela’s early-stage research and translational capabilities and deep scientific knowledge in autoimmune and severe inflammatory diseases; it supports the Company’s global expansion strategy; and it diversifies the Company’s on-market medicine portfolio with the addition of UPLIZNA, an infused biologic medicine indicated for the rare disease NMOSD.
Resumed TEPEZZA Supply: In March 2021, the U.S. Food and Drug Administration approved a prior approval supplement giving the Company authorization to manufacture an increased scale of TEPEZZA drug product, resulting in a greater number of vials per manufacturing slot at its third-party manufacturer, Catalent. The Company resumed supplying the market in April following a supply disruption that began in December 2020 due to U.S. government-mandated COVID-19 vaccine orders. The Company continues to make progress on adding a second drug product manufacturer by the end of 2021.
Advancing the Company’s Global Expansion with European UPLIZNA Launch Preparations: Today, the Company announced additional plans for its global expansion, preparing to build out its European infrastructure to support the potential approval by the first quarter of 2022 of UPLIZNA for NMOSD, which has been granted orphan designation by the European Commission. The Company is adding key infrastructure to support the potential launch of UPLIZNA in Europe in the near term as well as to support the potential launch of additional medicines over the long term. The Company anticipates building a global presence over time in several other markets outside of Europe, including Japan, one of the markets the Company is pursuing for TEPEZZA, where the Company is engaging with the Pharmaceutical and Medical Devices Agency and the Japanese medical community.
UPLIZNA Approved in Japan for NMOSD: In March 2021, the Company’s strategic partner Mitsubishi Tanabe Pharma Corporation received manufacturing and marketing approval of UPLIZNA for the prevention of relapses of NMOSD from the Japanese Ministry of Health, Labour and Welfare.
Presented New UPLIZNA Data at Medical Meetings: New UPLIZNA data were presented at the American Academy of Neurology’s 73rd Annual Meeting in April, including end-of-study data from the open-label extension period (OLP) of the pivotal N-MOmentum trial in patients with NMOSD. The data showed UPLIZNA was generally well-tolerated for at least four years, and that long-term UPLIZNA treatment provided a sustained reduction in NMOSD attack risk from baseline, regardless of when treatment was initiated. Data from the OLP were also recently presented at the American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) 2021 Forum, including a poster featuring data demonstrating the safety and efficacy of UPLIZNA in those with previous rituximab exposure. Additionally, a new analysis of the N-MOmentum trial demonstrating that the medicine consistently reduced the risk of worsening disability in people living with NMOSD was published in the May issue of Neurology Neuroimmunology & Neuroinflammation.
Initiated Enrollment in KRYSTEXXA Monthly Dosing Trial: In March 2021, the first patient was enrolled in an open-label trial to evaluate a monthly dosing regimen of KRYSTEXXA with methotrexate to treat people with uncontrolled gout. The goal of the trial is to explore whether a monthly dosing regimen can provide similar outcomes as the current dosing schedule, which is every other week.
Completed Enrollment in KRYSTEXXA PROTECT Trial: In January 2021, the Company completed enrollment in its PROTECT open-label trial. The trial, which is evaluating KRYSTEXXA to improve the management of uncontrolled gout for adults with a kidney transplant, enrolled a total of 20 patients. Results are expected in the fourth quarter of 2021.
Progressed the TEPEZZA Subcutaneous Administration Clinical Program: The Company completed dosing in late March for its first trial exploring a subcutaneous (SC) formulation of TEPEZZA. The trial is a small, single-dose Phase 1 pharmacokinetic trial which includes evaluating the use of the Halozyme ENHANZE drug-delivery technology for a SC formulation, which could potentially shorten drug administration time, reducing healthcare practitioner time and offering additional flexibility and convenience for patients.
Received Multiple Best Workplace Awards: The Company continued to be recognized as a best workplace. In March 2021, the Company ranked No. 1 on the "Fortune Best Workplaces in Biopharma 2021" list and was named to the "Best Workplaces in Ireland 2021" list. In April 2020, the Company was named one of Fortune’s "100 Best Companies to Work For" in the United States and placed on Crain’s Chicago Business’ 2021 "Best Places to Work in Chicago" list for the sixth consecutive year.
Key Clinical Development Programs

TEPEZZA: an insulin-like growth factor type 1 receptor (IGF-1R) antagonist monoclonal antibody.
Chronic Thyroid Eye Disease (TED) Trial: Phase 4 randomized, placebo-controlled trial evaluating TEPEZZA in chronic TED expected to initiate in mid-2021.
Subcutaneous Administration: Phase 1 pharmacokinetic trial exploring SC administration of TEPEZZA.
Diffuse Cutaneous Systemic Sclerosis (dcSSc) Exploratory Trial: Phase 1 exploratory trial in dcSSc expected to initiate in mid-2021.
KRYSTEXXA: a recombinant uricase enzyme that converts urate into a water-soluble liquid, allantoin, that can be easily excreted from the body.
MIRROR Randomized Controlled Trial: Phase 4 randomized, placebo-controlled trial evaluating KRYSTEXXA with methotrexate to increase the complete response rate in patients with uncontrolled gout.
PROTECT Trial: Phase 4 open-label trial evaluating KRYSTEXXA to improve management of uncontrolled gout in kidney transplant patients.
Shorter Infusion Duration Trial: Phase 4 open-label trial evaluating the impact of administering KRYSTEXXA with methotrexate over a shorter infusion duration in patients with uncontrolled gout.
Monthly Dosing Trial: Phase 4 open-label trial evaluating monthly dosing of KRYSTEXXA with methotrexate in patients with uncontrolled gout.
Retreatment Trial: Phase 4 open-label trial evaluating KRYSTEXXA with methotrexate in patients who have previously failed KRYSTEXXA monotherapy; expected to initiate in the second quarter of 2021.
UPLIZNA: an anti-CD19 humanized monoclonal antibody that depletes B cells and the pathogenic cells that produce autoantibodies.
Myasthenia Gravis Trial: Phase 3 randomized, placebo-controlled trial evaluating UPLIZNA in patients with myasthenia gravis, a chronic, rare, autoimmune neuromuscular disease that affects voluntary muscles, especially those that control the eyes, mouth, throat and limbs.
IgG4-Related Disease Trial: Phase 3 randomized, placebo-controlled trial evaluating UPLIZNA in patients with IgG4-related disease, which is a group of disorders marked by tumor-like swelling and fibrosis of affected organs, such as the pancreas, salivary glands and kidneys.
Kidney Transplant Desensitization Trial: Phase 2 open-label trial evaluating UPLIZNA, HZN-4920, or both in highly-sensitized patients waiting for a kidney transplant, which is paused due to COVID-19.
HZN-825: an oral lysophosphatidic acid receptor 1 (LPAR1) antagonist that prevents gene activation.
dcSSc Trial: Pivotal Phase 2b trial in dcSSc expected to initiate in the second quarter of 2021.
Interstitial Lung Disease Trial: Pivotal Phase 2b trial in idiopathic pulmonary fibrosis, the most common form of interstitial lung disease, expected to initiate in mid-2021.
HZN-4920: a CD40 ligand antagonist that blocks T cell interaction with the CD40-expressing B cells, disrupting the overactivation of the CD40 ligand co-stimulatory pathway. Several autoimmune diseases are associated with the overactivation this pathway.
Sjögren’s Syndrome Trial: Phase 2b randomized, placebo-controlled trial evaluating HZN-4920 in patients with Sjögren’s syndrome, a chronic, systemic autoimmune condition that impacts exocrine glands, including the salivary and tear glands.
Rheumatoid Arthritis Trial: Phase 2 randomized, placebo-controlled trial evaluating HZN-4920 in patients with rheumatoid arthritis.
Kidney Transplant Rejection Trial: Phase 2 open-label trial evaluating HZN-4920 in kidney transplant rejection patients.
HZN-7734: an anti-ILT7 human monoclonal antibody that depletes certain dendritic cells. Depleting these cells may interrupt the cycle of inflammation that causes tissue damage in diseases such as lupus, dermatomyositis and a variety of other autoimmune conditions.
Systemic Lupus Erythematosus (SLE) Trial: Phase 2 trial in SLE, a disease in which the body’s immune system attacks its own tissues and organs, expected to initiate in mid-2021.
COVID-19-Related Acute Lung Injury Trial: Phase 1 trial in COVID-19-related acute lung injury.
HZN-1116 Autoimmune Disease Trial: Phase 1 trial in autoimmune diseases expected to initiate in mid-2021.
First-Quarter Financial Results

Note: For additional detail and reconciliation of non-GAAP financial measures to the most directly comparable GAAP financial measures, please refer to the tables at the end of this release.

Net Sales: First-quarter 2021 net sales were $342.4 million. First-quarter 2020 net sales were $355.9 million.
Gross Profit: Under U.S. GAAP, the first-quarter 2021 gross profit ratio was 70.7 percent compared to 72.6 percent in the first quarter of 2020. The non-GAAP gross profit ratio in the first quarter of 2021 was 90.9 percent compared to 90.0 percent in the first quarter of 2020.
Operating Expenses: Research and development (R&D) expenses were 16.8 percent of net sales and selling, general and administrative (SG&A) expenses were 97.0 percent of net sales. Non-GAAP R&D expenses were 14.3 percent of net sales, and non-GAAP SG&A expenses were 63.8 percent of net sales.
Income Tax Expense: On a GAAP basis in the first quarter of 2021, income tax benefit was $47.8 million. First-quarter non-GAAP income tax expense was $25.8 million.
Net (Loss) Income: On a GAAP basis, first-quarter 2021 net loss was $123.4 million. First-quarter 2021 non-GAAP net income was $7.4 million.
Adjusted EBITDA: First-quarter 2021 adjusted EBITDA was $45.8 million.
(Loss) Earnings per Share: On a GAAP basis, diluted loss per share in the first quarter of 2021 and 2020 were $0.55 and $0.07, respectively. Non-GAAP diluted earnings per share in the first quarter of 2021 and 2020 were $0.03 and $0.40, respectively. Weighted average shares outstanding used for calculating GAAP and non-GAAP diluted earnings per share in the first quarter of 2021 were 223.9 million and 234.1 million, respectively.
First-Quarter Segment Results

Management uses net sales and segment operating income to evaluate the performance of the Company’s two segments, the orphan segment and the inflammation segment. While segment operating income contains certain adjustments to the directly comparable GAAP figures in the Company’s consolidated financial results, it is considered to be prepared in accordance with GAAP for purposes of presenting the Company’s segment operating results.

Orphan Segment

First-quarter 2021 net sales of the orphan segment, the Company’s strategic growth segment, were $257.5 million, an increase of 5 percent over the prior year’s quarter, driven by strong performance of KRYSTEXXA, RAVICTI, PROCYSBI and ACTIMMUNE. First-quarter 2021 net sales include a partial quarter of UPLIZNA net sales. First-quarter 2021 TEPEZZA net sales were negatively impacted by a short-term supply disruption due to U.S. government-mandated COVID-19 vaccine orders.
First-quarter 2021 net sales of the inflammation segment were $84.9 million, and segment operating income was $42.7 million.
Cash Flow Statement and Balance Sheet Highlights

On a GAAP basis in the first quarter of 2021, cash used in operating activities was $3.7 million. Non-GAAP operating cash flow was $63.5 million.
As of March 31, 2021, the Company had cash and cash equivalents of $811.6 million.
As of March 31, 2021, the total principal amount of debt outstanding was $2.618 billion. As of March 31, 2021, the gross-debt-to-last-12-months adjusted EBITDA leverage ratio was 2.8 times.
2021 Guidance

The Company now expects full‐year 2021 net sales to range between $2.75 billion and $2.85 billion, updated from the previous range of $2.70 billion and $2.80 billion. Full-year 2021 adjusted EBITDA is now expected to range between $1.02 billion and $1.06 billion, updated from the previous guidance of $1.14 billion and $1.18 billion. The updated guidance ranges incorporate Viela, which was acquired on March 15, 2021. The Company continues to expect TEPEZZA full-year 2021 net sales of greater than $1.275 billion and KRYSTEXXA full-year 2021 net sales of greater than $500 million.

Webcast

At 8 a.m. EST / 1 p.m. IST today, the Company will host a live webcast to review its financial and operating results and provide a general business update. The live webcast and a replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay of the webcast will be available approximately two hours after the live webcast.

On May 5, 2021 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the quarter ended March 31, 2021 and provided a general business update (Press release, Ophthotech, MAY 5, 2021, View Source [SID1234579255]).

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"Iveric Bio is entering an important period as we remain focused on the execution of our ongoing Zimura GATHER2 clinical trial, which is our second Phase 3 clinical trial for Zimura for the treatment of geographic atrophy secondary to age-related macular degeneration. We are committed to completing recruitment for the GATHER2 trial in the third quarter of this year," stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. "To date, both the recruitment and retention of patients in GATHER2 have exceeded our expectations. We are on track for initial, topline data from GATHER2 to be available approximately one year after the recruitment of the last patient in the GATHER2 clinical trial, plus the time needed for database closure and analysis of the initial, topline data."

"A key goal of ours is to expand and advance our footprint in multiple stages and types of AMD," stated Pravin U. Dugel, M.D., President of Iveric Bio. "We are excited by the opportunity to potentially expand the reach of Zimura beyond GA and to continue the development of IC-500, our HtrA1 inhibitor, which we expect could be complementary to Zimura in treating AMD patients. We remain committed to developing safe and effective therapeutic and gene therapy treatment options for retinal diseases with significant unmet medical needs."

Therapeutics Programs Targeting Geographic Atrophy Secondary to Age-Related Macular Degeneration

Zimura (avacincaptad pegol): Complement C5 Inhibitor

Enrollment and retention for GATHER2, the Company’s pivotal clinical trial of Zimura in development for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD), are progressing well with enrollment on target. In March 2021, the Company announced it accelerated the timeline for when it expects to complete enrollment in GATHER2 to the third quarter of 2021.
The Phase 2b screening clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease, referred to as the STAR trial, is ongoing with the goal of enrolling approximately 120 patients.
IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor

During the first quarter of 2021, the Company revised its development plans for IC-500 to include plans to investigate multiple dosing schedules for this product candidate. In April 2021, the Company commenced its first preclinical tolerability study for IC-500 and is currently planning additional preclinical studies, including pharmacokinetic and target engagement studies. Formulation optimization and other manufacturing activities are also ongoing. The Company expects to submit an IND to the FDA for IC-500 in GA secondary to AMD in the second half of 2022.
Iveric Bio to Host Dry AMD Virtual Symposium for Investors/Analysts

The Company will host a dry AMD Virtual Symposium for investors and analysts on Friday, June 18, 2021 from 10:00am – 12:00pm Eastern Time. The event will include presentations and discussions with retinal specialists and key opinion leaders on the dry AMD landscape, Zimura pivotal program in GA and highlights from the Company’s IC-500 program in AMD. The event will be accessible via webcast on the Iveric Bio website at www.ivericbio.com. For more information, please contact Kathy Galante at [email protected].

Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

IC-200: BEST1-Related IRDs
The Company is completing a preclinical efficacy and toxicology study for IC-200, in the naturally occurring canine model of Best disease. Published data have demonstrated long-term rescue in this model following a single sub-retinal injection. The Company is on track to release the recently manufactured cGMP batch of IC-200 in preparation for the planned IND filing and plans to move IC-200 into the clinic, in a Phase 1/2 trial in the second half of 2021.
IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP).
The Company continues to evaluate the results of preclinical toxicology studies for IC-100. In the Company’s preclinical efficacy and toxicology study in a naturally occurring canine model of RHO-adRP, efficacy was demonstrated at all three doses tested. The Company also tested the same three doses in a GLP toxicology study in non-human primates. Ocular inflammation on clinical exam was observed in the high dose group in canines and to varying degrees at different dosing levels tested in non-human primates. Due to the different findings in the two different species, and the Company’s high commitment to the safety of its patients, the Company is planning to discuss with regulators the design of its planned first-in-human clinical trial for IC-100 prior to submitting an IND. The Company now believes that IC-100 will likely be delayed from entering into a Phase 1/2 clinical trial this year.
Minigene Programs
The Company, in its minigene collaboration with the University of Massachusetts Medical School, has identified a lead construct for its Leber Congenital Amaurosis Type 10 (LCA10) program and is currently considering development plans for this program. The Company expects to obtain additional results from its Stargardt Disease (ABCA4) program in the second quarter of 2021, and expects to obtain preliminary results from its USH2A-related inherited retinal diseases program in the second half of 2021.
The Company announced today the formation of its Gene Therapy Inherited Retina Disease Scientific Advisory Committee that will work closely with senior management as the Company advances its gene therapy inherited retinal disease programs. The members of the advisory committee include:

Elias Traboulsi, MD, MEd
Head of the Department of Pediatric Ophthalmology
Director of the Center for Genetic Eye Diseases
Cole Eye Institute
Professor of Ophthalmology, Cleveland Clinic Lerner College of Medicine,
Cleveland Clinic
Andreas K. Lauer, MD
Chair, Department of Ophthalmology, Casey Eye Institute
Professor of Ophthalmology, School of Medicine
Bart P. Leroy, MD, PhD
Head, Department of Ophthalmology, Ghent University Hospital
Senior Staff Member, Center for Medical Genetics Ghent, Ghent University Hospital
Professor of Ophthalmology & Ophthalmic Genetics, Ghent University
Director of the Retinal Degenerations Clinic Children’s Hospital of Philadelphia
Mark Pennesi, MD, PhD
Division Chief, Ophthalmic Genetics
Associate Professor in Ophthalmology, Oregon Health & Science University
Eleonora Lad, MD, PhD
Director of Grading, Duke Reading Center
Associate Professor of Ophthalmology, Duke University Medical Center
Board of Directors and Management

Today the Company announced the promotions of Pravin U. Dugel, MD, to President, and Kathy Galante to Senior Vice President, Investor Relations, both effective as of May 1.
In April 2021, the Company announced that David R. Guyer, MD, was stepping down from the Iveric Bio Board of Directors after 14 years, effective following Iveric Bio’s 2021 Annual Stockholder Meeting scheduled to be held on May 19, 2021.
First Quarter Financial Results and 2021 Cash Guidance

As of March 31, 2021, the Company had $180.2 million in cash, cash equivalents and available for sale securities.
The Company estimates its year-end 2021 cash, cash equivalents and available for sale securities to range between $125 and $135 million. The Company also estimates that its cash, cash equivalents and available for sale securities will be sufficient to fund its planned capital expenditure requirements and operating expenses, excluding any potential approval or sales milestones payable to Archemix Corp. or any commercialization expenses for Zimura, into 2024. These estimates are based on the Company’s current business plan, including the continuation of its ongoing clinical development programs for Zimura, the progression of its IC-100 and IC-200 programs into the clinic, and the advancement of its IC-500 development program. These estimates also assume that the Company will enroll approximately 400 patients in the GATHER2 trial. These estimates do not reflect any additional expenditures related to potentially studying Zimura in other indications or resulting from the potential in-licensing or acquisition of additional product candidates or technologies or commencement of new sponsored research programs, and any associated development the Company may pursue.
2021 Q1 Financial Highlights

R&D Expenses: Research and development expenses were $18.5 million for the quarter ended March 31, 2021, compared to $13.8 million for the same period in 2020. Research and development expenses increased primarily due to the initiation of our GATHER2 trial and commencement of patient enrollment and increased manufacturing activities for Zimura, increased manufacturing and preclinical development activities associated with the Company’s IC-100 and IC-200 gene therapy programs and the progression of its IC-500 development program.
G&A Expenses: General and administrative expenses were $8.3 million for the quarter ended March 31, 2021, compared to $5.0 million for the same period in 2020. General and administration expenses increased primarily due to legal costs associated with ongoing litigation.
Income Tax Benefit: For the quarter ended March 31, 2021, the Company recorded no income tax benefit. An income tax benefit of $3.3 million was recognized in the quarter ended March 31, 2020 to reflect a favorable settlement of a state corporate income tax audit.
Net Loss: The Company reported a net loss for the quarter ended March 31, 2021 of $26.8 million, or ($0.29) per diluted share, compared to a net loss of $15.1 million, or $(0.28) per diluted share, for the same period in 2020.
Conference Call/Web Cast Information

Iveric Bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for May 5, 2021 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-317-6003 (USA) or 412-317-6061 (International), passcode 5841649. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the Iveric Bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 877-344-7529 (USA) or 412-317-0088, passcode 10153477.

On May 5, 2021 Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) reported that the Company, together with Bristol-Myers Squibb Company (NYSE: BMY), has launched beLAB1407, a new $ 20 m academic BRIDGE to identify and advance novel and breakthrough drug discovery opportunities across therapeutic areas from the UK’s top-tier academic institutions (Press release, Evotec, MAY 5, 2021, View Source;announcements/press-releases/p/evotec-launches-belab1407-to-accelerate-translational-research-from-the-uks-academic-life-science-ecosystem-in-collaboration-with-bristol-myers-squibb-6064 [SID1234579254]). Through a unique combination of Evotec’s drug discovery and development platforms and early-stage therapeutic concepts from the Universities of Birmingham, Edinburgh, Nottingham, and Dundee, beLAB1407 offers a unique route to the advancement of first-in-class therapeutics and the creation of spin-out companies.

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Evotec’s BRIDGE (Biomedical Research, Innovation & Development Generation Efficiency) collaborations provide an integrated fund and award framework to validate exciting academic projects in collaborations with Pharma and biotech with the goal to form new companies. Since implementing the first academic BRIDGE ‘LAB282’ in Oxford in November 2016, Evotec has continued to evolve similar collaborations with a variety of academic, Pharma and venture capital partners across Europe and North America.

With beLAB1407 representing the continued refinement of the approach, Evotec links one of Europe’s foremost academic clusters of excellence in the life sciences with Bristol Myers Squibb as a major biopharmaceutical partner dedicated to advancing innovation in therapeutics from academia to patient benefit. The beLAB1407 collaboration builds on the longstanding and successful relationship between Evotec and Bristol Myers Squibb in drug discovery across several therapeutic areas.

Dr Werner Lanthaler, Chief Executive Officer of Evotec, said: "We are thrilled to launch beLAB1407 together with our partners at Bristol Myers Squibb with whom we’ve worked on a variety of projects over a period of many years. beLAB1407 provides researchers from the member institutions with a unique way to fast-track their projects, to validate them on our industrial-grade platform and have partnering options including company formations readily available to them."

Dr Thomas Hanke, Head of Academic Partnerships at Evotec, added: "We are delighted to see the BRIDGE concept gaining further traction in the UK and we very much look forward to working closely with our academic partners in the Midlands and Scotland as well as our colleagues at BMS to identify and accelerate the next generation of first-in-class therapeutics across all modalities and therapeutic areas."

Dr Rupert Vessey, Executive Vice President and President, Research and Early Development at Bristol Myers Squibb, commented: "This collaboration builds on our important connection to leading European universities. With beLAB1407, we are supporting U.K.-based universities that are exploring many interesting lines of scientific research and discovery. That research combined with Evotec’s proprietary data platforms has the potential to identify new and novel therapies for areas of unmet medical need."

The name beLAB1407 refers to the distance between Land’s End in the far southwest of Great Britain to its north-easternmost point near the village of John o’ Groats in Scotland, which – if travelled by bike – adds up to 1,407 kilometres. To learn more about beLAB1407, please visit www.belab1407.org.

On May 5, 2021 vTv Therapeutics Inc. (Nasdaq:VTVT) reported financial results for the first quarter ended March 31, 2021, and provided an update on the progress of its clinical programs (Press release, vTv Therapeutics, MAY 5, 2021, View Source [SID1234579253]).

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"The vTv team continued to build on the clinical success of our type 1 diabetes program during the quarter by obtaining Breakthrough Therapy designation from the FDA for TTP399 and by initiating a phase 1 study to understand TTP399’s impact on ketoacidosis. The FDA’s grant of Breakthrough is an acknowledgment of the significant unmet need for patients with type 1 diabetes, and the encouraging clinical results we have generated to date," said Steve Holcombe, president and CEO, vTv Therapeutics. "We look forward to working closely with the FDA to optimize the development pathway for TTP399 to bring this potential treatment to patients with type 1 diabetes as quickly as possible."

Recent Achievements and Outlook

Type 1 Diabetes

Breakthrough Therapy Designation. As announced in April, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for TTP399 as an adjunctive therapy to insulin for the treatment of type 1 diabetes. Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned for the treatment of a serious or life-threatening disease where initial clinical evidence indicates that the drug may demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints.

Mechanistic Study of Ketoacidosis with TTP399. The Company began dosing patients in a mechanistic study of TTP399 in people with type 1 diabetes to determine the impact of TTP399 on ketone body formation during a period of acute insulin withdrawal. vTv expects to report topline results from the mechanistic study in the third quarter of 2021.

Pivotal Study Planning. The Company is planning to initiate the first of two pivotal, placebo-controlled, six-month clinical trials of TTP399 in subjects with type 1 diabetes in the fourth quarter of 2021. The current study designs will be shared with the FDA for its input and advice as part of the collaborative process enabled by the Breakthrough Therapy designation.
Psoriasis

Multiple Ascending Dose Study with HPP737. The Company began dosing healthy subjects in a phase 1 multiple ascending dose study to assess the safety, tolerability, and pharmacokinetic profile of HPP737, an oral PDE4 inhibitor. This phase 1 study is expected to report topline results in the third quarter and will inform dose selection for a planned phase 2 study in psoriasis.
License Partner Updates

Reneo Pharmaceuticals Completes Initial Public Offering. We congratulate our licensee, Reneo Pharmaceuticals, Inc. (Nasdaq: RPHM), for successfully completing an initial public offering in April. The Company is party to a license agreement with Reneo for its lead asset REN001, which Reneo is currently developing in three rare genetic diseases: primary mitochondrial myopathies, long-chain fatty acid oxidation disorders, and glycogen storage disease type V (McArdle disease).
First Quarter 2021 Financial Results

Cash Position: The Company’s cash position as of March 31, 2021, was $8.4 million compared to $5.7 million as of December 31, 2020.

Revenue: Revenue for the first quarter of 2021 was $1.0 million and was $6.4 million for the fourth quarter of 2020. The revenue for the first quarter was non-cash and related to the recognition of revenue pertaining to the Huadong license agreement. The revenue for the fourth quarter was primarily attributable to the upfront consideration, consisting of cash and an equity interest, received in connection with the Company’s license agreement with Anteris Bio.

R&D Expenses: Research and development expenses were $3.1 million and $2.5 million for the three months ended March 31, 2021 and December 31, 2020, respectively. This increase of $0.6 million was driven primarily by the increase in spending for our study of HPP737 in psoriasis.

G&A Expenses: General and administrative expenses were consistent between periods at $2.2 million and $2.0 million for the three months ended March 31, 2021 and December 31, 2020, respectively.

Net (Loss)/Income Before Non-Controlling Interest: Net loss before non-controlling interest was $5.9 million for the first quarter of 2021 compared to net income of $1.6 million for the fourth quarter of 2020.

Net (Loss)/Income Per Share: Diluted net loss per share was ($0.08) for the three months ended March 31, 2021 compared to a diluted net income per share of $0.02 for the three months ended December 31, 2020, based on weighted-average diluted shares of 56.5 million and 74.4 million for the three-month periods ended March 31, 2021 and December 31, 2020, respectively. Non-GAAP net loss per fully exchanged share was ($0.09) for the three months ended March 31, 2021 compared to a net income per fully exchanged share of $0.02 at December 31, 2020, based on non-GAAP fully exchanged weighted-average shares of 79.6 million and 74.4 million for the three months ended March 31, 2021 and December 31, 2020, respectively.