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.

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.

On January 11, 2021 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, reported its key 2021 milestones that will drive its recently announced strategic pivot to focus on developing and commercializing innovative treatments for genetically defined diseases, as well as its five-year vision for the company (Press release, Agios Pharmaceuticals, JAN 11, 2021, View Source [SID1234573824]). Agios will present at the virtual 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11 at 10:50 a.m. ET, and a live webcast will be available at investor.agios.com.

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"Agios is at an exciting inflection point as we prepare to move forward with a singular focus on genetically defined diseases," said Jackie Fouse, Ph.D., chief executive officer of Agios. "2021 will be a year of significant momentum and further evidence of our potential to meaningfully impact the lives of patients with unmet needs in pyruvate kinase deficiency, thalassemia, sickle cell disease and other genetically defined diseases. This year, we expect to file for the approval of mitapivat in pyruvate kinase deficiency, which currently has no disease-modifying treatment options, and to initiate pivotal development programs in thalassemia and sickle cell disease. We also expect to further unlock the potential of PK activation across a range of genetically defined diseases by advancing our rich and sustainable research pipeline. As we reimagine the future of Agios, we look forward to building on our core values and pioneering leadership in cellular metabolism to expand and accelerate our work on behalf of patients."

Anticipated 2021 Key Milestones

Agios expects to achieve the following key milestones in 2021:

Corporate

Complete sale of oncology portfolio to Servier, in a transaction worth up to $2 billion plus royalties, in the second quarter of 2021 subject to shareholder approval and satisfaction of regulatory conditions, and commence return of at least $1.2 billion to shareholders post-closing
Genetically Defined Disease Program Milestones

File for regulatory approval for mitapivat in adults with PK deficiency: submit new drug application (NDA) in the U.S. in the second quarter of 2021 and marketing authorization application (MAA) in the EU in mid-2021
Initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassemia in the second half of 2021
Announce pivotal development plan for mitapivat in adults with sickle cell disease in the first half of 2021 and initiate pivotal development program by year-end
Prioritize new indications for pyruvate kinase R (PKR) and pyruvate kinase M2 (PKM2) activator clinical development by year-end
Genetically Defined Disease Data Presentations

Report topline data from the Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions in the first quarter of 2021
Submit data from the following clinical studies for presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress, which will be held June 9-17, 2021:
Phase 3 ACTIVATE study of mitapivat in adults with PK deficiency who do not receive regular transfusions
Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions
Phase 2 study of mitapivat in adults with α- and β-thalassemia who do not receive regular transfusions
Submit data from ongoing clinical studies of mitapivat in sickle cell disease for presentation at medical meetings throughout 2021
Present data from the single ascending dose (SAD) and multiple ascending dose (MAD) cohorts of the Phase 1 study of AG-946, the company’s next-generation PKR activator, in healthy volunteers by year-end
Oncology Milestones & Data Presentations

Present mature overall survival data from the Phase 3 ClarIDHy study of TIBSOVO (ivosidenib tablets) in patients with previously treated IDH1-mutant cholangiocarcinoma at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO-GI), which will be held virtually January 15-17, 2021
Submit supplemental new drug application (sNDA) in the U.S. for TIBSOVO in patients with previously treated IDH1-mutant cholangiocarcinoma in the first quarter of 2021
Enrollment in the Phase 3 AGILE trial of TIBSOVO in combination with azacitidine in adult patients with previously untreated IDH1-mutant acute myeloid leukemia is expected to be complete by year-end
Enrollment in the relapsed or refractory myelodysplastic syndrome arm of the TIBSOVO Phase 1 study of IDH1-mutant advanced hematologic malignancies is expected to be complete by year-end
Full-year net product revenue for TIBSOVO is expected to be $160-170 million
Agios 2025 Strategic Vision
The Agios 2025 strategic vision reflects the company’s expected evolution over the next five years in light of its singular focus on genetically defined diseases. As part of this vision, Agios expects to achieve the following milestones by the end of 2025:

Receive regulatory approval for mitapivat in three initial indications: PK deficiency, thalassemia and sickle cell disease
Advance a broad clinical pipeline of at least 5 molecules exploring at least 10 indications
Foster a robust research pipeline poised to deliver an investigational new drug (IND) every 12-24 months
Achieve cash-flow positivity
2020 Year-End Cash and Guidance
Agios ended 2020 with approximately $670.5 million of cash, cash equivalents and marketable securities. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2020, together with anticipated product and royalty revenue, interest income and expense reimbursements under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its planned operating expenses and capital expenditure requirements to the end of 2022. Following the completion of the transaction with Servier and the subsequent shareholder returns, Agios expects its cash runway to extend to cash-flow positivity in 2025.

Presentation at 39th Annual J.P. Morgan Healthcare Conference
Agios will webcast its corporate presentation from the virtual 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2021 at 10:50 a.m. ET (7:50 a.m. PT). A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On January 11, 2021 Bristol Myers Squibb (NYSE: BMY) reported that its Board of Directors has authorized incremental share repurchases of up to an additional $2 billion of the company’s outstanding shares of common stock (Press release, Bristol-Myers Squibb, JAN 11, 2021, View Source [SID1234573823]). With this increase, the company’s total outstanding share repurchase is approximately $6.4 billion.

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The timing and amount of any share repurchases under the authorization will be determined by management at its discretion and based on market conditions and other considerations. Share repurchases under the authorizations may be made through a variety of methods, which may include open market purchases, pursuant to pre-set trading plans meeting the requirements of Rule 10b-1 under the Securities Exchange Act of 1934, in privately negotiated transactions, block trades, accelerated share repurchase transactions, or any combination of such methods. The program does not obligate Bristol Myers Squibb to acquire any particular amount of its common stock, and the repurchase program may be suspended or discontinued at any time at the Company’s discretion.

On January 11, 2021 Aileron Therapeutics, Inc. (Nasdaq: ALRN) reported the completion of its previously announced registered direct offering of 32,630,983 of its shares of common stock at a purchase price of $1.10 per share, for gross proceeds of $35.9 million, before deducting placement agent fees and other offering expenses payable by Aileron (Press release, Aileron Therapeutics, JAN 11, 2021, View Source [SID1234573822]). Aileron is developing ALRN-6924 as a novel medicine to selectively protect healthy cells in patients with cancers that harbor p53 mutations to reduce or eliminate chemotherapy-induced side effects while preserving chemotherapy’s effects against cancer cells, a concept known as chemoprotection.

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New fundamental investors, including Acorn Bioventures, BVF Partners, L.P., Maven Investment Partners and Grand Oaks Capital, participated in the offering, in addition to several existing Aileron investors, including Satter Medical Technology Partners and Lincoln Park Capital Fund, LLC.

JonesTrading Institutional Services LLC ("JonesTrading") acted as the placement agent for the offering.

In addition to the $35.9 million registered direct offering, between November 12, 2020 and January 5, 2021, Aileron sold an aggregate of 9,894,519 shares of its common stock in "at the market" offerings under the Capital on DemandTM Sales Agreement with JonesTrading resulting in aggregate gross proceeds of approximately $12.7 million. Gross proceeds combined from both offerings were $48.6 million before deducting commissions and fees.

With the proceeds from these transactions, Aileron believes that its cash, cash equivalents and investments will enable it to fund its current strategic plan into the second half of 2023, including the planned clinical trial of ALRN-6924 in patients with advanced non-small cell lung cancer (NSCLC).

"We are thrilled to welcome Acorn Bioventures, BVF Partners and Maven Investment Partners as fundamental healthcare investors in Aileron. We believe that the participation of these funds, in addition to the continued support of key existing Aileron investors, is further validation of the potential of ALRN-6924 as an important medicine in the emerging chemoprotection field," said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer of Aileron. "With the completed offering, we are well positioned to continue advancing toward our vision to bring chemoprotection to all patients with p53-mutant cancer regardless of cancer type or chemotherapy."

Dr. Aivado continued, "For decades, the medical community has largely been resigned to the sad reality that chemotherapy destroys healthy cells while destroying cancer cells. 2021 holds the promise to begin a shift in this long-held mindset with the imminent PDUFA date and potential approval of the industry’s first chemoprotective agent1 that, similar to ALRN-6924, aims to protect healthy cells from chemotherapy’s side effects. Given the increasing interest in chemoprotection and the significant unmet medical need, we believe ALRN-6924 has the potential to have an important and broad role in proactively preventing chemotherapy’s harmful effects on cancer patients."

Aileron plans to begin enrollment in a Phase 1b randomized, double-blind, placebo-controlled clinical trial of ALRN-6924 in patients with advanced p53-mutated NSCLC undergoing treatment with first-line carboplatin doublet chemotherapy (with or without immune checkpoint inhibitors), in the second quarter of 2021. The planned Phase 1b NSCLC trial follows Aileron’s presentation in October 2020 of clinical data from its ongoing Phase 1b clinical trial of ALRN-6924 in small cell lung cancer (SCLC) demonstrating clinical proof-of-concept that treatment with ALRN-6924 resulted in a protective effect against severe anemia, thrombocytopenia and neutropenia in patients with p53-mutated SCLC treated with topotecan. Aileron anticipates reporting initial results from the trial late in the fourth quarter of 2021 and full results in mid-2022.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

How ALRN-6924 Is Designed to Protect Healthy Cells from Chemotherapy

ALRN-6924 is being developed by Aileron as a novel chemoprotective medicine to selectively protect healthy cells in patients with cancers that harbor p53 mutations to reduce or eliminate chemotherapy-induced side effects.

Chemotherapy preferentially acts on cells that are cycling or undergoing the process of cell division. In cancer cells, the cell cycle is unchecked, which leads to uncontrolled cell proliferation, a hallmark of cancer. Certain types of healthy cells also naturally need to cycle, such as bone marrow cells, hair follicle cells, skin cells, and cells lining the oral cavity and the gastrointestinal tract. As a result, chemotherapy preferentially targets and kills both cycling healthy cells and cycling cancer cells. This, in turn, can lead to a spectrum of chemotherapy-induced side effects, from unpleasant to life-threatening and fatal.

ALRN-6924, an investigational first-in-class MDM2/MDMX dual inhibitor, is administered prior to chemotherapy to patients with p53-mutant cancers. ALRN-6924 is designed to activate normal p53 protein in patients’ healthy cells, temporarily and reversibly pausing cell cycling to selectively shield the patients’ healthy cells from chemotherapy. The protection is limited to healthy cells, as ALRN-6924 cannot work in p53-mutated cancer cells given that mutated p53 has lost its function in those cells. Therefore, cancer cells continue to cycle uninterrupted and remain fully susceptible to destruction by chemotherapy.

1 The U.S. Food & Drug Administration has assigned a Prescription Drug User Fee Act (PDUFA) date of February 15, 2021 for G1 Therapeutics, Inc.’s investigational therapy trilaciclib.