J.P. Morgan Healthcare Conference Presentation – January 11, 2021

On January 11, 2021 Mirati Presented the Corporate Presentation (Presentation, Mirati, JAN 11, 2021, View Source [SID1234573829]).

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Investor Presentation dated January 2021

On January 11, 2021, Moleculin Biotech, Inc. (the "Company") presented the corporate presentations (Press release, Moleculin, JAN 11, 2021, View Source [SID1234573828]).

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Vericel Announces Preliminary Fourth-Quarter and Full-Year 2020 Financial Results and Provides Business Updates

On January 11, 2021 Vericel Corporation (NASDAQ:VCEL), a leader in advanced therapies for the sports medicine and severe burn care markets, reported preliminary unaudited financial results for the fourth quarter and year ended December 31, 2020, and provided business updates (Press release, Vericel, JAN 11, 2021, View Source [SID1234573827]).

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Preliminary Unaudited Fourth-Quarter and Full-Year 2020 Financial Results
Preliminary total net revenues for the fourth-quarter 2020 are expected to be in the range of $44.9-$45.4 million, with MACI (autologous cultured chondrocytes on porcine collagen membrane) net revenue in the range of $34.4-$34.9 million, Epicel (cultured epidermal autografts) net revenue of approximately $9.6 million, and NexoBrid (concentrate of proteolytic enzymes enriched in bromelain) revenue of approximately $1 million related to the U.S. Biomedical Advanced Research and Development Authority’s (BARDA) procurement of NexoBrid for emergency response preparedness.

Preliminary total net product revenues for the full-year 2020 are expected to be in the range of $123.9-$124.4 million, with MACI net revenue in the range of $94.1-$94.6 million, Epicel net revenue of approximately $27.5 million, and NexoBrid revenue of approximately $2.2 million related to the BARDA procurement.

Cash and investments increased by approximately $14.5 million in the fourth quarter. As of December 31, 2020, the company had approximately $100 million in cash and investments and no debt, compared to $79.0 million as of December 31, 2019.

Business Highlights and Updates

Record fourth-quarter and full-year total net revenues;
Record quarterly and full-year MACI implants and net revenue;
Record fourth-quarter and full-year Epicel grafts and net revenue, and the second highest quarterly Epicel grafts and revenue in history;
Received MACI biopsies from approximately 1,500 surgeons in 2020, up from approximately 1,400 surgeons in 2019;
Record quarterly high in the number of surgeons taking MACI biopsies in the fourth quarter; and
Double-digit growth in MACI biopsies in the fourth quarter, achieving a record quarterly high and a record monthly high in December.
"We delivered strong financial and operational results in the fourth quarter and for the full year, including record product volumes and revenue for both MACI and Epicel for the year," said Nick Colangelo, President and CEO of Vericel. "Epicel performance was very strong in the fourth quarter, with revenue growing over 60% for the quarter. MACI performance was in line with our expectations through mid-December. However, due to the recent surge in COVID-19 cases in the United States, the scheduling of MACI pipeline cases for the last two weeks of December slowed compared to historical trends and there was an increase in case cancellations during that period. We expect, based on our experience earlier this year, that the majority of those MACI cases, which represent approximately $2 million in revenue, will move forward in 2021. Moreover, the underlying growth drivers for MACI were in line with our expectations, which we believe will drive strong revenue and profitability growth in 2021."

The company will host a webcast and conference call to discuss its fourth quarter 2020 financial results and business highlights on February 24, 2020, at 8:30am Eastern Time. Webcast information can be found on the events and presentation section of the Investor Relations website at View Source

Horizon Therapeutics plc Provides Preliminary 2020 Financial Results, Exceeding Full-Year 2020 Net Sales and Adjusted EBITDA Guidance; Provides Update on TEPEZZA® (teprotumumab-trbw) Supply and New KRYSTEXXA® (pegloticase injection) Trials

On January 11, 2021 Horizon Therapeutics plc (Nasdaq: HZNP) reported updates on its 2020 financial results, TEPEZZA supply and new KRYSTEXXA trials (Press release, Horizon Pharma, JAN 11, 2021, View Source [SID1234573826]).

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"Amid the most challenging environment we have ever faced, we had a record year of performance, exceeding our full-year 2020 net sales and adjusted EBITDA guidance, driven by the significant outperformance of TEPEZZA and the strong second-half performance of KRYSTEXXA," said Tim Walbert, chairman, president and chief executive officer, Horizon. "We delivered exceptional shareholder value while expanding our pipeline, with six programs expected to begin this year, including two new KRYSTEXXA programs. Additionally, we successfully completed the first increased scale TEPEZZA lot and we are on track to submit the resultant data to the FDA by the end of this month."

Full-Year 2020 Preliminary Financial Results (unaudited)

Full-year 2020 net sales exceeded the high end of the Company’s guidance range of $2.12 billion to $2.14 billion; exceeding the high end of this guidance range represents year-over-year growth of more than 65 percent.
Full-year 2020 adjusted EBITDA exceeded the high end of the Company’s guidance range of $920 million to $940 million; exceeding the high end of this guidance range represents year-over-year growth of more than 95 percent and an adjusted EBITDA margin expansion of more than 700 basis points compared to 2019.
The outperformance was driven by strong net sales from its two key growth drivers, TEPEZZA, its biologic for the treatment of Thyroid Eye Disease (TED), and KRYSTEXXA, its biologic for the treatment of uncontrolled gout (chronic gout refractory to conventional therapy), as well as continued growth of its Rare Disease Business Unit.
TEPEZZA full-year 2020 net sales exceeded $800 million, with double-digit sequential growth in the fourth quarter of 2020.
KRYSTEXXA full-year 2020 net sales exceeded $400 million, with double-digit sequential growth in the fourth quarter of 2020.
Cash and cash equivalents at Dec. 31, 2020 were $2.08 billion and gross leverage was less than 1.1 times.
These preliminary financial results are unaudited and subject to adjustment. Horizon will report its final fourth quarter and full-year 2020 financial results in February.

TEPEZZA Supply Update

As previously announced on Dec. 17, 2020 the Company expects a short-term disruption in TEPEZZA supply as a result of government-mandated COVID-19 vaccine production orders related to Operation Warp Speed that dramatically restricted capacity available for TEPEZZA at its drug product contract manufacturer, Catalent. The Company anticipates that this drug supply shortage could last through the first quarter. The length of the TEPEZZA supply disruption will depend on whether future manufacturing slots are successfully completed as well as decisions by the U.S. Food and Drug Administration (FDA) regarding the increased scale manufacturing process of TEPEZZA.

The Company has made significant progress with TEPEZZA drug product supply over the last several weeks, successfully completing its first manufacturing lot at increased scale and beginning its second manufacturing lot at increased scale. The Company continues to expect to submit data this month from the first increased scale drug product manufacturing lot to the FDA for its review and approval.

Pipeline Update

The Company also announced two new KRYSTEXXA development programs it expects to begin in 2021, bringing the total number of programs in its pipeline to 14.

KRYSTEXXA monthly dosing trial: This open-label trial will evaluate a monthly dosing regimen of KRYSTEXXA, with methotrexate, to treat people with uncontrolled gout. The current dosing schedule for KRYSTEXXA is every other week. The goal of the trial is to explore whether a monthly dosing regimen can provide similar outcomes as the current dosing schedule.
KRYSTEXXA retreatment trial: This open-label trial will evaluate KRYSTEXXA, with methotrexate, in patients who have previously failed KRYSTEXXA. The goal of the trial is to evaluate whether patients can benefit from KRYSTEXXA, with methotrexate, after developing an immune response to KRYSTEXXA when taken alone. Patients who have previously failed KRYSTEXXA have limited options available to address their uncontrolled gout.
Presentation Information

Mr. Walbert will present at the J.P. Morgan Healthcare Conference at 11:40 a.m. ET on Jan. 12, 2021. The conference presentation will be webcast live and may be accessed by visiting Horizon’s website at View Source A replay of the webcast will be available following the event.

About KRYSTEXXA

INDICATIONS AND USAGE

KRYSTEXXA (pegloticase injection) is a PEGylated uric acid specific enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy.

Gout refractory to conventional therapy occurs in patients who have failed to normalize serum uric acid and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated.

Important Limitations of Use: KRYSTEXXA is not recommended for the treatment of asymptomatic hyperuricemia.

IMPORTANT SAFETY INFORMATION
WARNING: ANAPHYLAXIS AND INFUSION REACTIONS

Anaphylaxis and infusion reactions have been reported to occur during and after administration of KRYSTEXXA. Anaphylaxis may occur with any infusion, including a first infusion and generally manifests within 2 hours of the infusion. However, delayed-type hypersensitivity reactions have also been reported. KRYSTEXXA should be administered in healthcare settings and by healthcare providers prepared to manage anaphylaxis and infusion reactions. Patients should be premedicated with antihistamines and corticosteroids. Patients should be closely monitored for an appropriate period of time for anaphylaxis after administration of KRYSTEXXA. Serum uric acid levels should be monitored prior to infusions, and healthcare providers should consider discontinuing treatment if levels increase to above 6 mg/dL, particularly when 2 consecutive levels above 6 mg/dL are observed.

The risk of anaphylaxis and infusion reactions is higher in patients who have lost therapeutic response.

Concomitant use of KRYSTEXXA and oral urate-lowering agents may blunt the rise of sUA levels. Patients should discontinue oral urate-lowering agents and not institute therapy with oral urate-lowering agents while taking KRYSTEXXA.

In the event of anaphylaxis or infusion reaction, the infusion should be slowed, or stopped and restarted at a slower rate.

Patients should be informed of the symptoms and signs of anaphylaxis and instructed to seek immediate medical care should anaphylaxis occur after discharge from the healthcare setting.

CONTRAINDICATIONS: G6PD DEFICIENCY ASSOCIATED HEMOLYSIS AND METHEMOGLOBINEMIA

Patients should be screened for G6PD deficiency prior to starting KRYSTEXXA. Hemolysis and methemoglobinemia have been reported with KRYSTEXXA in patients with G6PD deficiency. KRYSTEXXA should not be administered to these patients.

GOUT FLARES

An increase in gout flares is frequently observed upon initiation of anti-hyperuricemic therapy, including treatment with KRYSTEXXA. If a gout flare occurs during treatment, KRYSTEXXA need not be discontinued. Gout flare prophylaxis with a non-steroidal anti-inflammatory drug (NSAID) or colchicine is recommended starting at least 1 week before initiation of KRYSTEXXA therapy and lasting at least 6 months, unless medically contraindicated or not tolerated.

CONGESTIVE HEART FAILURE

KRYSTEXXA has not been studied in patients with congestive heart failure, but some patients in the clinical trials experienced exacerbation. Caution should be exercised when using KRYSTEXXA in patients who have congestive heart failure, and patients should be monitored closely following infusion.

ADVERSE REACTIONS

The most commonly reported adverse reactions in clinical trials with KRYSTEXXA were gout flares, infusion reactions, nausea, contusion or ecchymosis, nasopharyngitis, constipation, chest pain, anaphylaxis and vomiting.

Please see Full Prescribing Information and Medication Guide for more information.

About TEPEZZA

INDICATION

TEPEZZA is indicated for the treatment of Thyroid Eye Disease.

IMPORTANT SAFETY INFORMATION
Warnings and Precautions

Infusion Reactions: TEPEZZA may cause infusion reactions. Infusion reactions have been reported in approximately 4% of patients treated with TEPEZZA. Reported infusion reactions have usually been mild or moderate in severity. Signs and symptoms may include transient increases in blood pressure, feeling hot, tachycardia, dyspnea, headache and muscular pain. Infusion reactions may occur during an infusion or within 1.5 hours after an infusion. In patients who experience an infusion reaction, consideration should be given to premedicating with an antihistamine, antipyretic, or corticosteroid and/or administering all subsequent infusions at a slower infusion rate.

Preexisting Inflammatory Bowel Disease: TEPEZZA may cause an exacerbation of preexisting inflammatory bowel disease (IBD). Monitor patients with IBD for flare of disease. If IBD exacerbation is suspected, consider discontinuation of TEPEZZA.

Hyperglycemia: Increased blood glucose or hyperglycemia may occur in patients treated with TEPEZZA. In clinical trials, 10% of patients (two-thirds of whom had preexisting diabetes or impaired glucose tolerance) experienced hyperglycemia. Hyperglycemic events should be managed with medications for glycemic control, if necessary. Monitor patients for elevated blood glucose and symptoms of hyperglycemia while on treatment with TEPEZZA. Patients with preexisting diabetes should be under appropriate glycemic control before receiving TEPEZZA.

Adverse Reactions

The most common adverse reactions (incidence ≥5% and greater than placebo) are muscle spasm, nausea, alopecia, diarrhea, fatigue, hyperglycemia, hearing impairment, dysgeusia, headache and dry skin.

Replimune Provides 2020 Year End Review and Overview of Expected 2021 Milestones

On January 11, 2021 Replimune Group Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported that corporate update, highlighting the progress of its key programs (Press release, Replimune, JAN 11, 2021, View Source [SID1234573825]).

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"Replimune made great progress in 2020 providing positive data read outs in multiple tumor types which support our registration-directed programs with RP1, as well as with RP2, demonstrating the potential of our platform to redefine the cancer treatment paradigm by providing what we believe to be the most practical and effective way to ignite a systemic immune response to a patient’s cancer. During the year we also completed the construction of and technology transfer to our state of the art commercial scale manufacturing facility" said Philip Astley-Sparke, Chief Executive Officer of Replimune. "We start this year with the recent news that dosing of patients has commenced with our third product candidate, RP3, which like RP2 is intended to treat tumor types which are not traditionally thought of as ‘immune-responsive’, and look forward to releasing further data on all of our programs during the course of 2021. We continue to enroll into our two registration-directed clinical trials in cutaneous squamous cell carcinoma (CSCC), the "CERPASS" study, and anti-PD1 failed melanoma, the "IGNYTE" study, and commercial planning activities are underway. We also expect to start dosing RP1 combined with Opdivo in anti-PD1 failed non-small cell lung cancer (NSCLC) patients and anti-PD1 failed CSCC patients this quarter. I am proud of the entire Replimune team for our accomplishments to date, as we continue to execute upon our mission to make oncolytic immuno-gene therapy a foundational cornerstone of cancer treatment."

Program Highlights and Upcoming Milestones

RP1 in combination with Libtayo in CSCC: The Company is actively enrolling patients into its global registration-directed Phase 2, randomized, controlled, clinical trial. The Company remains on track to provide the primary data read out in 2022.

RP1 in combination with Opdivo in anti-PD-1 failed melanoma: The Company initiated recruitment into a new registration-directed 125-patient cohort Phase 2 clinical trial of RP1 in combination with Opdivo in the first half of 2020 and continues to enroll patients. The Company remains on track to provide the primary data readout in 2022.

RP1 in combination with Opdivo in melanoma and non-melanoma skin cancers (NMSC): In October 2020, Replimune provided positive Phase 2 data updates in melanoma and NMSC which demonstrated deep and durable responses to RP1 combined with Opdivo, including in anti-PD1 failed melanoma that continues to support the Company’s ongoing registration-directed development in this setting and in CSCC. Enrollment of the initial melanoma cohort (including anti-PD1 naïve and anti-PD1 failed patients) was completed in the first half of 2020 with the NMSC cohort now being expanded from 30 to 45 patients to also include 15 patients with anti-PD1 failed disease.

RP1 in anti-PD1 failed NSCLC: The Company has expanded the clinical study of RP1 combined with Opdivo to include a new cohort of 30 anti-PD1 failed NSCLC patients and plans to dose its first patient in early 2021.

RP1 as monotherapy in solid organ transplant recipients with CSCC: The Company is currently enrolling a 30 patient Phase 1b clinical trial assessing the safety and efficacy of RP1 in liver and kidney transplant recipients. The Company expects to dose its first patient in early 2021. Progress on this study has been particularly impacted by COVID-19 due to the immune suppression that solid organ transplant patients receive. Initial data from this clinical trial is intended to be presented in the second half of 2021.

RP1 in combination with Opdivo in MSI-H/dMMR tumors: The Company is accumulating data from the MSI-H/dMMR (anti-PD1 naïve) cohort. Based on the data, the Company expects to decide whether to pursue MSI-H/dMMR tumors into registration-directed development in 2021.

RP2 alone and in combination with Opdivo: RP2 is an enhanced potency oncolytic immunotherapy which expresses an anti-CTLA-4 molecule, intended to improve on the safety and efficacy profile of systemic antibody approaches to targeting CTLA-4, in addition to expressing the GALV-GP R- fusogenic protein and GM-CSF. RP2 is being evaluated in a Phase 1 clinical trial alone and combined with Opdivo in advanced solid tumor patients. In October 2020, Replimune presented positive data from the single agent RP2 portion of the clinical trial that showed deep and durable responses, including in patients with immune insensitive tumor types. Following the monotherapy phase, enrollment is currently underway in a 30-patient cohort in combination with Opdivo. Updated data from this clinical trial is expected to be presented mid-year.

RP3 alone and in combination with anti-PD-1 therapy: Replimune initiated dosing in its Phase 1 clinical trial of RP3 in December 2020. RP3 is the Company’s third product candidate, which in addition to GALV-GP R- and anti-CTLA-4 also expresses CD40L and 4-1BBL. The Phase 1 clinical trial is designed to evaluate RP3 alone and combined with anti-PD1 therapy in advanced solid tumor patients. Initial data is expected to be presented in the second half of 2021.

Targeted evaluation for new indications is currently underway: An analysis of the solid tumor space is currently underway to define the later stage and registrational clinical development pathway initially intended for RP2 and/or RP3. This is from the perspective that RP2 and RP3 are intended to target less immune responsive tumor types, and follows from initial promising data having been generated with single agent RP2, including in immune non-responsive tumor types. The details of this initial development plan are intended to be announced mid-year.
Corporate Updates

Manufacturing: The Company has completed buildout of its 63,000-square-foot state-of-the-art manufacturing facility in Framingham, MA to support late-stage development and full commercialization of all of its products. GMP production is underway.

Potential impact of COVID-19 on milestones: Enrollment into the Company’s clinical trials, in particular the clinical trial of RP1 in solid organ transplant patients with CSCC which represents a highly immune compromised patient population, has been slower than expected, which the Company attributes to the global pandemic. While mitigation plans have been implemented, as the clinical trial sites continue to evaluate their capacity to enroll patients into clinical trials, the Company could see additional impact on the pace of enrollment across its clinical trial programs.

Cash Position: Based on its current operating plan, Replimune expects that its preliminary cash, cash equivalents and short-term investments of $493 million as of December 31, 2020 is expected to fund its operating expenses and capital expenditure requirements into the second half of 2024.
J.P. Morgan Conference Presentation and Webcast.

As previously announced, the Company will be virtually presenting at the 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12th at 5:20 p.m. ET. A simultaneous webcast of the presentation will be available in the Investors section of Replimune’s website at www.replimune.com. A replay will be available for 30 days following the conference.