Revenues for the first half of 2022 and update on AB Science’s activities

On September 30, 2022 AB Science SA (Euronext – FR0010557264 – AB) reportes its revenues for the first half of 2022 and provides an update on its activities (Press release, AB Science, SEP 30, 2022, View Source [SID1234621579]).

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CLINICAL DEVELOPMENT KEY EVENTS FOR THE FIRST HALF OF 2022 AND SINCE JUNE 30, 2022

Authorization by Health Canada to file a New Drug Submission for masitinib in the treatment of amyotrophic lateral sclerosis (ALS) under the Notice of Compliance with Conditions (NOC/c) policy

AB Science announced that Health Canada has granted authorization to file a New Drug Submission for masitinib in the treatment of amyotrophic lateral sclerosis (ALS) under the Notice of Compliance with Conditions (NOC/c) policy.

If granted, an NOC/c is authorization to market a drug with conditions. Such conditions will be discussed with Health Canada during the procedure.

An assessment named Advance Consideration, performed by a Health Canada Adjudicating Committee, is necessary before being granted authorization to file under NOC/c policy.

This assessment was made based on a pre-submission package sent by AB Science including, efficacy data of study AB10015, long-term survival data (75 months average follow-up from diagnosis) of study AB10015, and safety data.

An estimated 3,000 Canadians are currently living with ALS. Each year approximately 1,000 Canadians die from ALS. A similar number of Canadians are diagnosed with ALS each year.

Under the NOC/c policy, Health Canada has a target of 200 calendar days maximum to review the application.

Filing for conditional Marketing Authorization to the European Medicines Agency (EMA) for masitinib in the treatment of amyotrophic lateral sclerosis (ALS)

AB Science filed an application for conditional Marketing Authorization to the European Medicines Agency (EMA) for Alsitek (masitinib) in the treatment of amyotrophic lateral sclerosis (ALS). The application is based on results from the phase 2/3 AB10015 study and its long-term survival follow-up. Study AB10015 was a randomized, double-blind, placebo-controlled trial over a 48-week treatment period, conducted with 394 ALS patients and evaluating Alsitek in combination with riluzole versus riluzole alone.

This decision to file followed a pre-submission meeting held with the Committee for Medicinal Products for Human Use (CHMP) Rapporteur during which new data generated with Alsitek in ALS were presented, in particular, a clinical benefit in terms of a 25-month increase in median overall survival for patients with moderate ALS, which is a cohort that closely resembles newly diagnosed patients. During the pre-submission meeting, AB Science also presented how issues listed in the previous CHMP assessment for Alsitek on ALS (EMA/406203/2018) were resolved, in particular:

The mode of action of Alsitek in ALS, which has been well-demonstrated and published in peer-reviewed publications.
A remonitoring of all efficacy and safety data and a complete reassessment of the Alsitek safety database.
Additional analyses for the primary efficacy endpoint, imputing all missing data for early discontinuation, and a conservative analysis imputing missing data with a penalty for patients who discontinued Alsitek for lack of efficacy or toxicity. These analyses were positive, and all showed a treatment effect in favor of Alsitek and convergent with the primary analysis.
Long-term follow-up survival data showing a significant benefit in favor of Alsitek in moderate ALS patients (between group difference in median OS of +25 months, hazard ratio 0.56 (95%CI [0.32;0.96])).
This application has now been validated by EMA and review by the CHMP has begun. The CHMP has a target of 210 active evaluation days to review the application.

Launch of a confirmatory Phase 3 study with masitinib in progressive forms of multiple sclerosis

AB Science announced that it has been authorized by the French Medicine Agency, ANSM, to initiate a Phase III study (AB20009) evaluating masitinib in patients with Primary Progressive Multiple Sclerosis (PPMS) or non-active Secondary Progressive Multiple Sclerosis (nSPMS). The study will enroll 800 patients from numerous study centers with Expanded Disability Status Scale (EDSS) score between 3.0 to 6.0 and absence of T1 Gadolinium-enhancing brain lesions as measured by magnetic resonance imaging (MRI).

The primary objective of the study will be to evaluate the effect of masitinib on time to confirmed disability progression, with progression defined as 1-point worsening when EDSS baseline score ≤5.5, or 0.5 if baseline score >5.5 from randomization to week 96.

This confirmatory study follows successful completion of a first Phase 2B/3 study (AB07002) in primary progressive (PPMS) and non-active secondary progressive (nSPMS) multiple sclerosis. This study met its primary analysis endpoint, demonstrating a statistically significant reduction in cumulative change on EDSS with masitinib 4.5 mg/kg/day (p=0.0256).

Positive recommendation of the Data and Safety Monitoring Board to continue both Phase 2 studies in Covid-19

AB Science announced the continuation of the Phase 2 study evaluating masitinib in combination with isoquercetin in COVID-19, following the recommendation of the Data and Safety Monitoring Board (DSMB). This randomized (1:1), open-label, phase 2 study (AB20001) is designed to evaluate the safety and efficacy of masitinib plus isoquercetin in hospitalised patients with moderate COVID-19 (WHO 7-point ordinal scale level 4) or severe COVID-19 (level 5). The study is planned to recruit 200 patients (over 18 years of age with no upper age limit). The primary objective is to improve the clinical status of patients after 15 days of treatment, as measured by the WHO 7-point ordinal scale.

The interim analysis was conducted with one third of the patients evaluated, as planned. The purpose of the interim analysis was to assess the safety and efficacy of the treatment. The DSMB recommends continuing the study without restrictions in moderate patients (level 4, i.e. hospitalized patients with oxygen supply <6 L/min with SpO2 maintained ≥92%). In line with this recommendation, AB Science has made the decision to continue the study only in moderate patients. The study is therefore now planned to include 200 patients at level 4 of the ordinal scale.

AB Science also announced the continuation of the second Phase 2 study evaluating the antiviral activity of masitinib in patients who have a confirmed diagnosis of COVID-19, following the recommendation of the Data and Safety Monitoring Board (DSMB). This randomized (1:1), double-blind, phase 2 study (AB21002) in 78 patients, is designed to evaluate the anti-viral efficacy of masitinib in non-hospitalized patients who are at risk of developing severe COVID-19 and in hospitalized patients with need of oxygen (via face mask or nasal cannula).

The analysis was to assess the safety of the treatment and was based on the first 50% of the patient targeted recruitment. The DSMB indicated that there was no safety concern and recommended continuation of the study without restrictions.

CONSOLIDATED FINANCIAL INFORMATION FOR THE FIRST HALF OF 2022

The operating loss as of June 30, 2022 was 9,562 K€, compared to a loss of 6,040 K€ as of June 30, 2021, i.e. an increase in the operating loss of 3,522 K€ (58.3%).

Operating income, exclusively made up of sales related to the operation of a veterinary medicine, amounted to 629 K€ as of June 30, 2022 compared to 818 K€ one year earlier
Operating expenses amounted to 10,192 K€ as of June 30, 2022, compared to 6,858 K€ as of June 30, 2021, an increase of 48.6%.
Marketing expenses increased by 7.2% from 236 K€ as of June 30, 2021 to 253 K€ as of June 30, 2022.
Administrative expenses increased by 26.9%, from 1,326 K€ as of June 30, 2021 to 1,682 K€ as of June 30, 2022.
Research and development expenses increased by 2,800 K€, i.e 52.8 %, from 5,299 K€ as of June 30, 2021 to 8,099 K€ as of June 30, 2022. This variation is mainly explained by:
the decrease in the research tax credit (600 K€), due to the end of the doubling of subcontracting expenses to public research laboratories in the calculation of the research tax credit base from 2022,
the recognition at 30 June 2021 of income related to the cancellation of old accounts payable (860 K€), and
the recognition on 30 June 2022 of the valuation of warrants (414 K€)
The financial income as of June 30, 2022 is a gain of 2,424 K€ compared to a loss of 1,386 K€ one year earlier. The gain of 2,424 K€ as of 30 June 2022 is mainly related to the recognition of the change in fair value between 31 December 2021 and 30 June 2022 of the preference shares resulting from the conversion of the bonds in December 2016 (class C) and of the preference shares issued in September 2020 (class D), i.e. a financial gain of 2,244 K€ with no impact on cash over the period.

The net loss as of June 30, 2022 amounts to 7,141 K€ compared to a loss of 4,655 K€ as of June 30, 2021.

The following table summarizes the consolidated financial statements for the first half of 2022 prepared in accordance with IFRS, and comparative information with the first half of 2021:

OTHER CORPORATE INFORMATION FOR THE FIRST HALF OF 2022 AND SINCE JUNE 30, 2022

Financing of USD 8.5 million through the issuance of bonds with attached warrants

AB Science reached an agreement with a historical investor on a financing of USD 8.5 million through the issuance of bonds convertible into new ordinary shares with attached warrants (OCABSA).

50,000 OCABSA will be issued, representing a nominal value of USD 8.5 million. It will reinforce the cash position of AB Science for the development of its clinical research program.

50,000 convertible bonds will be issued at their par value of USD 170,0 each (the "PV"), representing a total par value of USD 8.5 million.

Decision of the Enforcement Committee of the French market regulator (AMF) following the investigation relating to the financial information and the market for AB Science shares, opened in September 2017

On March 24, 2022, the AMF Enforcement Committee ruled that there was no privileged information, neither at the time of the two capital increases carried out by AB Science on March 24 and 27, 2017, nor at the time Alain Moussy sold a part of his shares on March 31, 2017. The AMF Enforcement Committee therefore completely exonerated Alain Moussy, prosecuted for insider trading, and found that AB Science had not failed to comply with its disclosure obligations at the time of these capital increases in March 2017.

The AMF Enforcement Committee nevertheless considered that AB Science should have communicated as early as April 7, 2017 the high probability of a negative opinion from the European Medicine Agency (EMA) on the marketing authorization application for masitinib for the treatment of mastocytosis and ordered AB Science to pay the sum of one million euros.

In application of its internal procedures, AB Science had nevertheless put in place a deferral of privileged information from this date of April 7, 2017, considering that the delay in communication was in the interest of the Company and in line with industry practices of not communicating before the final vote of the CHMP, or else withdrawing the registration dossier, which AB Science had no intention to do.

Given this difference in assessment concerning a technical point relating to one of the criteria for the deferred communication of privileged information, as well as the amount of penalty, AB Science has decided to appeal to the Paris Court of Appeal. The President of AMF also appealed against the Enforcement Committee’s decision.

Other events

Considerations arising from the Russia-Ukraine war

Russia launched invasion of Ukraine in February 2022, which, alongside humanitarian concerns, may also have an impact on the health research ecosystem in the form of delays in the conduct of clinical trials. At the date of publication of the December 31, 2021 annual report, there were no significant delays or impacts on the studies monitored in Russia and Ukraine.

Other securities transactions

During the first half of 2022, 56,990 share subscription warrants and 5,000 stock-options were granted.

Other information

AB Science confirms its eligibility for PEA-PME (a share savings plan aimed at providing finance to SMEs) in accordance with decree no. 2014-283 of 4 March 2014 taken for the application of article 70 of law no. 2013-1278 of 29 December 2013 of finance for 2014 fixing the eligibility of companies for PEA-PME, i.e. less than 5,000 employees on the one hand, an annual turnover of less than 1,500 million euros or a total balance sheet of less than 2,000 million euros, on the other hand.

Panbela Announces Pricing of Approximately $6 Million Public Offering

On September 30, 2022 Panbela Therapeutics, Inc. (Nasdaq: PBLA), ("Panbela" or the "Company"), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported the pricing of a public offering of (i) 20,100,000 shares of its common stock (or pre-funded warrants in lieu thereof) and (ii) warrants to purchase up to 30,150,000 shares of its common stock (the "Public Warrants") at a purchase price of $0.30 per share and associated Public Warrant (Press release, Panbela Therapeutics, SEP 30, 2022, View Source [SID1234621578]). The Public Warrants will have an exercise price of $0.30 per share, are exercisable upon issuance, and will expire five years following the date of issuance. The offering is expected to close on or about October 4, 2022, subject to customary closing conditions.

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Roth Capital Partners, LLC is acting as lead placement agent, Craig-Hallum Capital Group LLC and Maxim Group LLC are each acting as co-placement agent of the offering.

Gross proceeds, before deducting placement agent fees and commissions and offering expenses are expected to be approximately $6 million. The Company intends to use the net proceeds from the proposed offering for the continued clinical development of its product candidates ivospemin (SBP-101) and eflornithine (CPP-1X), working capital, business development and other general corporate purposes, which may include repayment of debt.

The securities described above are being offered pursuant to a registration statement on Form S-1 (File No. 333-267000), as amended, that was declared effective by the U.S. Securities and Exchange Commission ("SEC"), on September 29, 2022. Copies of the accompanying prospectus relating to and describing the terms of the offering may be obtained, when available, at the SEC’s website at www.sec.gov or by contacting Roth Capital Partners, LLC, 888 San Clemente Drive, Suite 400, Newport Beach, CA 92660 or by email at [email protected].

This press release does not and shall not constitute an offer to sell or the solicitation of an offer to buy any 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 registration or qualification under the securities laws of any such state or other jurisdiction. Any offer, if at all, will be made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement.

About our Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of catalysts with programs ranging from pre-clinical to registration studies.

SBP-101 Ivospemin

Ivospemin is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial. For more information, please visit View Source .

Flynpovi

Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increase polyamine export and catabolism. In a Phase 3 clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase 3 trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), Flynpovi showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X

CPP-1X (eflornithine) is being developed as a single agent tablet or high dose power sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase 1 or Phase 2 investigator-initiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity.

INTERIM RESULTS for the six months ended 30 June 2022

On September 30, 2022 ImmuPharma PLC (LSE:IMM), ("ImmuPharma" or the "Company"), the specialist drug discovery and development company, reported its interim results for the six months ended 30 June 2022 (the "Period") (Press release, ImmuPharma, SEP 30, 2022, View Source [SID1234621577]).

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Key Highlights (including post Period review)

Financials

Financial performance in line with expectations over the Period
Loss for the Period of £1.7m (30 June 2021: £3.7m)
Research and development expenses of £1.0m (30 June 2021: £1.1m)
Administrative expenses of £0.6m (30 June 2021: £0.9m)
Share based expense of £0.1m (30 June 2021: £0.3m)
Cash balance of £0.2m as at 30 June 2022 (31 December 2021: £1.6m)
Derivative financial asset of £0.6m as at 30 June 2022 (31 December 2021: £0.9m)
Incanthera financial asset of £0.6m (£1.2m at 31 December 2021) and warrants financial asset of £Nil (£0.2m at 31 December 2021)
Basic and diluted loss per share of 0.58p (30 June 2021: 1.46p)
Reorganisation of share capital structure in June 2022
Successful fundraising in August 2022 raising £2.04m (gross), including Lanstead subscription, placement and broker option
Total proceeds of £0.3m from exercise of share options by L1 Capital in August 2022 and in September 2022
‘Autoimmunity’: Lupuzor ("P140")

P140 Pharmokinetic ("PK") study successfully completed with key endpoints met. P140 was safe and well tolerated across all doses and in all subjects
FDA Type C written response received on 14 September 2022: The FDA response was detailed and included significant guidance on next steps for the clinical programme. This included advice on the dosing regime and on study protocol that can be amended to improve the regulatory outcome
The Company is currently reviewing the written response with Avion and will make a further notification in due course
An adaptive Phase 2/3 clinical study protocol of P140 in CIDP is being finalised for IND submission. Commercial partnering discussions ongoing
‘Anti-Infection’

BioAMB – further pre-clinical studies are in preparation
BioCin – further pre-clinical studies are in preparation
New website launched: www.immupharma.co.uk

Commenting on the statement and outlook, Tim McCarthy, CEO and Chairman, said:

"The last six months have been pivotal for the Company, positively concluding the PK study of Lupuzor ("P140"). Post the recent written response from the FDA, in respect to the Lupuzor Phase 3 protocol, we continue to have positive discussions with our partner, Avion, on the way forward.

The new fundraising, supported by existing and new shareholders, allows us to further invest in the other parts of our product portfolio, whilst being focused on preserving cash where required.

Moving forward, we remain focused on creating a robust and successful Company that, with a diversity of assets, will build future value for our shareholders.

The Board would like to take this opportunity to thank its shareholders for their continued patience and support, as well as its staff, corporate and scientific advisers and our partners including, CNRS and Avion".

Market Abuse Regulation (MAR) Disclosure

THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION AS STIPULATED UNDER THE UK VERSION OF THE MARKET ABUSE REGULATION NO 596/2014 WHICH IS PART OF UK LAW BY VIRTUE OF THE EUROPEAN UNION (WITHDRAWAL) ACT 2018, AS AMENDED. ON PUBLICATION OF THIS ANNOUNCEMENT VIA A REGULATORY INFORMATION SERVICE, THIS INFORMATION IS CONSIDERED TO BE IN THE PUBLIC DOMAIN.

Scandion Oncology announce topline results from part 2 of the CORIST phase II trial

On September 30, 2022 Scandion Oncology (Scandion), a biotech company developing first-in-class medicines aimed at treating cancer which is resistant to current treatment options, reported the topline results from the second part of the ongoing CORIST phase II trial studying Scandion’s lead compound SCO-101 as a combination treatment with FOLFIRI chemotherapy in patients with metastatic colorectal cancer (mCRC) (Press release, Scandion Oncology, SEP 30, 2022, View Source,c3639117 [SID1234621576]).

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The results confirm the safety and tolerability of SCO-101 in this indication and combination. Further, tumor reduction has been observed in some patients, however below the +30% threshold defined as the trial’s primary endpoint. Also, evidence of prolonged progression free survival and stable disease (secondary endpoints) were observed in this hard-to-treat refractory patient population.

Importantly, the data is obtained from the 25 patients enrolled in CORIST part 2, more of whom have only been evaluated after eight weeks of treatment. Patients are still participating in the study, which means that the topline results are not final and could change with longer treatment.

"While we do not achieve a clinical proof of concept for efficacy through these topline results, we are encouraged by the signals observed in the trial, confirming the rationale for combining SCO-101 and FOLFIRI in this indication. We look forward to continuing the development by progressing the CORIST trial further as planned," says Johnny Stilou, acting CEO of Scandion.

Scandion will continue the CORIST trial enrolling up to 36 patients to be treated according to an optimized dosing schedule as part 3 of the trial. Based on an improved understanding of the pharmacokinetics of SCO-101 in combination with FOLFIRI, increased doses of SCO-101 and chemotherapy will be explored aiming to exploit the full potential of SCO-101 in this indication and combination. The first patient is expected to be enrolled in CORIST part 3 shortly.

Depending on the dose escalation results, CORIST part 3 may be completed by Q3 2023, with an update on the timeline expected in Q1 2023. Scandion then expects to conduct part 4 of the trial, which will conclude the combined CORIST trial.

Webcast and conference call on October 4 at 10:00 am CET

On October 4 at 10:00 am, Scandion Oncology’s executive management will host a webcast and conference call about the topline results from the second part of CORIST.

The event can be accessed via www.scandiononcology.com or through dial in on below numbers:

This information is information that Scandion Oncology A/S is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above on September 30, 2022, at 10.30 CET.

Mersana Therapeutics Announces Launch of Oncology FACETS, a Resource for Healthcare Providers to Advance Knowledge in Gynecologic Malignancies

On September 30, 2022 Mersana Therapeutics, Inc. (NASDAQ: MRSN), a clinical-stage biopharmaceutical company, reported the launch of Oncology FACETS: an educational platform for healthcare providers that will address clinically impactful topics in gynecologic oncology (Press release, Mersana Therapeutics, SEP 30, 2022, View Source [SID1234621575]). Oncology FACETS is driven by a Steering Committee of experts in gynecologic oncology and offers content developed in collaboration with Mersana.

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"Our goal is to create a destination where oncologists can find up-to-date, peer-reviewed educational content on emerging topics about ovarian, endometrial, cervical, and other gynecological cancers," said Dr. Thomas J. Herzog of the University of Cincinnati, who is serving as Steering Committee Chair. "We believe Oncology FACETS will provide a valuable resource for physicians seeking to keep pace with this field’s rapidly evolving science and data."

Each Oncology FACETS educational module is led by a Steering Committee member. The first module is "Current and Emerging Biomarkers in Gynecologic Cancer," led by Dr. Rebecca C. Arend, a leading gynecologic oncologist at the University of Alabama at Birmingham. The module is now available for download at OncologyFACETS.com.

Dr. Arend noted, "Precision medicine is now at the forefront of the oncology field. Biomarkers are central to this movement, and their use in gynecologic malignancies promises to shape the treatment paradigm in the near future. This module is intended to both educate and facilitate peer-to-peer dialogue about this important topic."

Oncology FACETS will release new modules on a regular basis. The next module, planned to be released in December 2022, will focus on antibody-drug conjugates (ADCs) in gynecologic malignancies.

"We are proud to collaborate with this esteemed group of gynecologic oncologists to advance knowledge and patient care," said Anna Protopapas, President and Chief Executive Officer of Mersana. "Oncology FACETS is a natural extension of our commitment to driving innovation aimed at improving outcomes for women with gynecologic cancers."

About Oncology FACETS
Oncology FACETS is the outcome of a collaboration between the Oncology FACETS Steering Committee and Mersana Therapeutics, Inc. Oncology FACETS disseminates educational resources to advance the learning of practicing healthcare professionals; it is not intended as medical advice and includes information on both approved medications and investigational agents not yet approved for use in any country. The committee of experts in gynecologic oncology was assembled to provide guidance and approval of the material contained within this website. Mersana provides financial, administrative, and writing support to the Steering Committee in developing these resources. Oncology FACETS is not an accredited provider of continuing medical education.

The Oncology FACETS Steering Committee members include Thomas J. Herzog, MD, University of Cincinnati (serving as Chair); Rebecca C. Arend, MD, MSPH, University of Alabama at Birmingham; John L. Hays, MD, PhD, Ohio State University; Bhavan Pothuri, MD, PhD, New York University; Leslie M. Randall, MD, MAS, Virginia Commonwealth University; and Ritu Salani, MD, MBA, University of California, Los Angeles. Committee members provide guidance on the development of all educational material. They represent diverse geographic regions across the United States.

Each educational module is led by a Steering Committee member. The first educational module is available for download at OncologyFACETS.com. A second module is in progress and will be released in December 2022. Associated content and updates can be followed @OncologyFACETS on Twitter.