On March 25, 2021 Immunome, Inc. (Nasdaq: IMNM), a biopharmaceutical company that utilizes its human memory B cell discovery engine platform to discover and develop first-in-class antibody therapeutics, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided a corporate update (Press release, Immunome, MAR 25, 2021, View Source [SID1234577176]).

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"The fourth quarter of 2020 was a transformational period for Immunome thanks to our successful initial public offering on the NASDAQ exchange, which raised $44.9 million in gross proceeds," said Purnanand Sarma, Ph.D., President and CEO of Immunome. "That positive momentum continued into 2021, with our recent announcements covering the isolation of antibodies capable of neutralizing SARS-CoV-2 variants in pseudovirus testing as part of our IMM-BCP-01 program, and the advancement of IMM-ONC-01, our proprietary antibody against IL-38 for the potential treatment of solid tumors including malignancies of high unmet need such as cancers of the head and neck and the lung, into IND-enabling studies."

Dr. Sarma continued: "Looking ahead, we expect to provide development program updates in the second quarter of 2021 and to file INDs for both IMM-BCP-01 and IMM-ONC-01 in 2021. We remain excited about the prospects for both of our current development programs and reiterate the confidence we have in our discovery engine to move one to two new candidates into IND-enabling studies per year. I very much look forward to updating you on our progress in the months ahead."

Financial Highlights

Cash and cash equivalents: As of December 31, 2020, cash and cash equivalents totaled $39.8 million.
Research and development (R&D) expenses: R&D expenses for the three months ended December 31, 2020 were $1.8 million. R&D expenses for the year ended December 31, 2020 were $7.5 million.
General and administrative (G&A) expenses: G&A expenses for the three months ended December 31, 2020 were $2.2 million. G&A expenses were $4.8 million for the year ended December 31, 2020.
Net loss: Net loss attributable to common stockholders was $4.1 million, or $0.40 per share, for the three months ended December 31, 2020. Net loss attributable to common stockholders was $17.8 million, or $5.26 per share, for the year ended December 31, 2020.
As of December 31, 2020, Immunome had 10,634,245 shares of common stock outstanding.

On March 25, 2021 PharmaEssentia reported that had repeatedly attempted to terminate the agreement with AOP Orphan concerning BESREMi (Ropeginterferon alfa-2b) (Press release, AOP Orphan Pharmaceuticals, MAR 25, 2021, View Source [SID1234577175]). After two and a half years of arbitral proceedings, in October 2020, the ICC Arbitral Tribunal issued its award in the matter. The arbitral award states that PharmaEssentia’s multiple attempts to terminate the agreement were unjustified, and that AOP Orphan is entitled to payment of damages of approximately EUR 142 million for project delays caused by PharmaEssentia.

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In December 2020, PharmaEssentia filed an application to set aside this arbitral award with the Frankfurt Higher Regional Court, arguing that the award violated public order and PharmaEssentia’s right to be heard. The Frankfurt Higher Regional Court, a German court with major experience in handling this kind of proceedings, called a hearing within less than two months of PharmaEssentia’s filing its setting aside application.

Award declared enforceable

Today, the Frankfurt Higher Regional Court announced its decision. As anticipated, the Court dismissed PharmaEssentia’s set aside application, confirming that both parties had sufficient opportunity to be heard, that all evidence brought before the Arbitral Tribunal was duly taken into consideration, and that the public order was fully respected.

Thus, the award as issued by the ICC Arbitral Tribunal in October 2020 has now been declared enforceable by Frankfurt Higher Regional Court, and AOP Orphan can pursue its enforcement in Germany and elsewhere. An appeal to the German Supreme Court is possible but would not hinder the enforcement of the award.

Rudolf Widmann, Chief Therapeutics Development Officer and Member of the Board of AOP Orphan, explains: "AOP Orphan has always complied with its contractual obligations and is entitled to compensation by PharmaEssentia, following their repeated attempts to boycott the joint project and success of BESREMi. We encourage PEC to accept the arbitral award and work towards cooperative efforts to exploit the full value of BESREMi in the interest of patients and stakeholders of both companies. AOP Orphan has tried to find solutions several times – I would have preferred to invest time and money into making this highly promising drug available to patients with blood cancer, instead of investing it into expensive lawsuits."

About BESREMi

BESREMi is a long-acting, mono-pegylated proline interferon (ATC L03AB15). Its unique pharmacokinetic properties offer a new level of tolerability. BESREMi is designed to be conveniently self-administered subcutaneously with a pen once every two weeks, or monthly after stabilization of hematological parameters. This treatment schedule is expected to lead to overall better safety, tolerability and adherence compared to conventional pegylated interferons. Ropeginterferon alfa-2b was discovered by PharmaEssentia, a long-term partner of AOP Orphan. In 2009, AOP Orphan in-licensed the exclusive rights for clinical development and commercialization of Ropeginterferon alfa-2b in PV and other MPNs for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.

On March 25, 2021 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported the publication of a cross-sectional study of dermatologists that found its respondents are increasingly incorporating DecisionDx-Melanoma into the management of their patients with melanoma (Press release, Castle Biosciences, MAR 25, 2021, View Source [SID1234577174]). DecisionDx-Melanoma is Castle’s gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as sentinel lymph node positivity, independent of traditional staging factors.

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The article, titled "Assessment of the 31-Gene Expression Profile Test by Dermatologists: A Cross-Sectional Survey from National Dermatology Conferences," was published in SKIN: The Journal of Cutaneous Medicine. The cross-sectional study was offered to attendees of two national, virtual dermatology conferences during the end of 2020 and beginning of 2021 to assess the professional understanding, opinions and clinical usage of DecisionDx-Melanoma by dermatologists. Participants were asked questions regarding practice demographics, factors considered prior to ordering DecisionDx-Melanoma, their integration of the test’s results into clinical management and their opinions on the usefulness of the test.

Data from 589 U.S. dermatological clinicians showed:

45% of participants ordered the DecisionDx-Melanoma test in the prior twelve months.
Going forward, 82% of participants were "somewhat to very likely" to order the test, with 66% stating they would recommend the test to a friend or family member as part of their melanoma care.
In melanomas less than or equal to 1.0mm (T1), which make up the majority of melanomas, previous studies have demonstrated that a DecisionDx-Melanoma Class 1A test result (lowest risk) has a 5-year recurrence free survival rate of 96.8%, compared to 64.6% for a Class 2B test result (highest risk). 61% of participants stated they would change their treatment plan in this T1 population with a Class 2B test result.
Participants who use DecisionDx-Melanoma indicated that they use the results to impact follow-up schedules, referrals, surveillance imaging, sentinel lymph node biopsy procedure recommendations and other treatment decisions. These uses largely follow published appropriate-use criteria for the test.
Participants responded that patients gain various benefits from DecisionDx-Melanoma test results, including increased knowledge and understanding (70%), personalized treatment options (58%) and eased uncertainty about the future (59%). Even regarding test results indicating the lowest risk of recurrence (i.e. Class 1A), 66% of participants reported potential benefits for ameliorating patients’ anxiety and 46% reported increasing confidence in their management.
"The sample surveyed demonstrated that dermatology specialists are using DecisionDx-Melanoma in increased numbers, and concluded that melanoma patients whose healthcare providers incorporate DecisionDx-Melanoma into their practice may benefit from decreased anxiety and uncertainty from the improved prognosis, reduced need for unwarranted procedures and optimized healthcare resources for patients who need it most," said study author, Darrell Rigel, M.D., M.S., Clinical Professor at New York University School of Medicine.

About DecisionDx-Melanoma

DecisionDx-Melanoma is a gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as sentinel lymph node positivity, independent of traditional staging factors, and has been studied in more than 5,700 patient samples. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in four archival risk of recurrence studies of 901 patients and six prospective risk of recurrence studies including more than 1,600 patients. To predict likelihood of sentinel lymph node positivity, the Company utilizes its proprietary algorithm, i31-GEP, to produce an integrated test result. i31-GEP is an artificial intelligence-based neural network algorithm (independently validated in a cohort of 1,674 prospective, consecutively tested patients with T1-T4 cutaneous melanoma) that integrates the DecisionDx-Melanoma test result with the patient’s traditional clinicopathologic features. Impact on patient management plans for one of every two patients tested has been demonstrated in four multicenter and single-center studies including more than 560 patients. The consistent performance and accuracy demonstrated in these studies provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results. Through December 31, 2020, DecisionDx-Melanoma has been ordered more than 68,920 times for use in patients with cutaneous melanoma.

On March 25, 2021 MAIA Biotechnology, Inc., a biotechnology company focused on the development of targeted, first-in-class oncology drugs, reported that company management is presenting at the following virtual investor conferences (Press release, MAIA Biotechnology, MAR 25, 2021, View Source [SID1234577173]):

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Keiretsu Midwest Angel Forum: March 23-25, 2021
Keiretsu Forum is a global investment community of accredited private equity angel investors, venture capitalists and corporate/institutional investors.
MedInvest Spring 2021 Oncology Investor Conference: March 29 – April 2, 2021
MedInvest Spring is sponsored by the National Foundation for Cancer Research and is a well-known event for cancer related companies seeking investment.
During these conferences, MAIA will present an overview of the company including several recent and upcoming milestones that will advance the company and its pipeline. In February 2021, MAIA entered into a clinical supply agreement with Regeneron Pharmaceuticals, Inc. to evaluate its lead program, THIO (aka 6-thio-dG), followed by the PD-1 inhibitor Libtayo (cemiplimab), in a Phase 1/2 clinical trial in second-line or later advanced non-small cell lung cancer (NSCLC) patients who have progressed following treatment with standard-of-care regimen that includes a checkpoint inhibitor. Libtayo was approved by the US FDA to treat first-line advanced NSCLC in February 2021.

This clinical trial will evaluate the safety and efficacy of four dose levels of THIO, the only telomere-by-telomerase targeting agent in development for the treatment of cancer, followed by Libtayo. The lead-in portion of the study will assess the safety and immunogenic effects of each of the THIO doses and overall response rate (ORR) as the basis for potentially expanding individual patient cohorts and evaluation in other cancer types. The Phase 1/2 clinical trial is expected to begin enrolling patients in 2021.

The presentations made by MAIA at Keiretsu and MedInvest will be available to registered conference attendees.

About THIO

THIO (aka 6-thio-dG, 6-thio-2’-deoxyguanosine) is a first-in-class small molecule that is the only telomere-by-telomerase targeting agent currently in development. THIO selectively kills cancer cells by modifying telomeric DNA structure and function utilizing telomerase. Telomerase is present in >85% of human cancers and contributes significantly to the proliferation and reproductive immortality of cancer cells. THIO’s activity was shown to be cancer-specific in tumor types with active telomerase. THIO is recognized by telomerase and incorporated into telomeres selectively in cancer cells. Once incorporated, it compromises the telomere structure and function, leading to ‘uncapping’ of the chromosome ends and thus resulting in rapid tumor cell death. Low doses of THIO, followed by anti-PD-L1 or anti-PD1 therapy, completely eliminated advanced tumors in preclinical models and produced cancer cell specific immune memory, where the immune system continued to be active against the cancer cells after extended periods of time, with no additional treatment. These results demonstrate how the THIO-produced telomere stress increases innate sensing and adaptive anti-tumor immunity, which provides a strong rationale for sequentially combining telomere-targeted therapy with immunotherapy. THIO is investigational and has not been approved for any use by regulatory authorities.

On March 25, 2021 Gilead Sciences, Inc. (Nasdaq: GILD) reported that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for sacituzumab govitecan-hziy (SG) for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have received at least two prior therapies, including at least one prior therapy for locally advanced or metastatic disease (Press release, Gilead Sciences, MAR 25, 2021, View Source [SID1234577172]). The MAA is now under accelerated review by the EMA, in recognition of the product being considered of major interest for public health and therapeutic innovation.

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SG is a first-in-class therapy targeting Trop-2, a protein frequently expressed in multiple types of epithelial tumors, such as TNBC, where high expression is associated with poor survival and relapse. Currently, in the European Union (EU), there is no authorized standard treatment regimen with proven benefit in overall survival (OS) for patients with previously treated metastatic TNBC.

"Metastatic triple-negative breast cancer is an aggressive and life-threatening cancer. Unfortunately, for many people with this cancer, there are not enough effective treatment options and their prognosis is extremely poor," said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. "The validation of our EU marketing application is an important step toward addressing the significant unmet medical need for people with metastatic triple-negative breast cancer."

SG (under the tradename Trodelvy) received accelerated approval by the U.S. Food and Drug Administration (FDA) to treat adult patients with metastatic TNBC who have received at least two prior therapies for metastatic disease. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

In addition to the European Union, regulatory reviews of SG in metastatic TNBC are currently underway in the U.K., Canada, Switzerland and Australia, as well as in Singapore through our partner Everest Medicines. SG is also under review by the FDA for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a platinum containing chemotherapy in the neoadjuvant/adjuvant, locally advanced, or metastatic setting, and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor.

About Triple-Negative Breast Cancer (TNBC)

TNBC is an aggressive type of breast cancer, accounting for approximately 15% of all breast cancers. The disease is diagnosed more frequently in younger and premenopausal women and is highly prevalent in African American and Hispanic women. TNBC cells do not have estrogen and progesterone receptors and have limited human epidermal growth factor receptor 2 (HER2). Medicines targeting these receptors therefore are not typically effective in treating TNBC.

About the ASCENT Study

The Phase 3 ASCENT study, an international, multi-center, randomized confirmatory trial, enrolled more than 500 patients with relapsed/refractory metastatic TNBC who had received at least two prior therapies for metastatic disease. Patients were randomized to receive either sacituzumab govitecan-hziy or a chemotherapy chosen by the patients’ treating physicians. The primary endpoint of the study was progression-free survival (PFS) in patients without brain metastasis at baseline. Secondary endpoints include PFS for the full study population, OS, objective response rate (ORR), duration of response (DoR), and time to response. More information about ASCENT is available at View Source

About Sacituzumab Govitecan-Hziy

SG is an antibody and topoisomerase inhibitor conjugate directed to the Trop-2 cell surface antigen, a protein frequently expressed in multiple types of epithelial tumors, including TNBC, where high expression is associated with poor survival and relapse.

In addition to multiple ongoing studies of SG in triple-negative breast cancer, it is being developed as an investigational treatment for metastatic urothelial cancer, hormone receptor-positive/HER 2-negative metastatic breast cancer, and metastatic non-small cell lung cancer, either as a monotherapy or in combination with other agents.

Important Safety Information for SG as included in the U.S. Prescribing information for Trodelvy.

Recommendations for the use of SG outside of the U.S. (including final safety information for prescribers) will be assessed as part of ongoing and future Marketing Authorization Applications.

WARNING: NEUTROPENIA AND DIARRHEA

SG can cause severe or life-threatening neutropenia. Withhold SG for absolute neutrophil count (ANC) below 1500/mm3 on Day 1 of any cycle or ANC below 1000/mm3 on Day 8 of any cycle. Withhold SG for neutropenic fever.

Monitor blood cell counts periodically during treatment. Consider Granulocyte Colony-Stimulating Factor (G-CSF) for secondary prophylaxis. Initiate anti-infective treatment in patients with febrile neutropenia without delay.

Dose modifications may be required due to neutropenia. Febrile neutropenia occurred in 6% (24/408) of patients treated with SG, including 8% (9/108) of patients with mTNBC after at least 2 prior therapies. Less than 1% (1/408) of patients had febrile neutropenia leading to permanent discontinuation. The incidence of Grade 1-4 neutropenia was 64% in patients with mTNBC (n=108). In all patients treated with SG (n=408), the incidence of Grade 1-4 neutropenia was 54%; Grade 4 neutropenia occurred in 13%. Less than 1% (2/408) of patients permanently discontinued treatment due to neutropenia.
Severe diarrhea may occur. Monitor patients with diarrhea and give fluid and electrolytes as needed. Administer atropine, if not contraindicated, for early diarrhea of any severity. At the onset of late diarrhea, evaluate for infectious causes and, if negative, promptly initiate loperamide. If severe diarrhea occurs, withhold SG until resolved to ≤ Grade 1 and reduce subsequent doses.

Diarrhea occurred in 63% (68/108) of patients with mTNBC and 62% (254/408) of all patients treated with SG. In each population, events of Grade 3-4 occurred in 9% (10/108) of mTNBC patients and 9% (36/408) of all patients treated with SG. Four out of 408 patients (<1%) discontinued treatment because of diarrhea. Neutropenic colitis was observed in 2% (2/108) of patients in the mTNBC cohort and 1% of all patients treated with SG.
Contraindications: Severe hypersensitivity reaction to SG.

Hypersensitivity

SG can cause severe and life-threatening hypersensitivity, including anaphylactic reactions. Hypersensitivity reactions occurred within 24 hours of dosing in 37% (151/408) and Grade 3-4 hypersensitivity occurred in 1% (6/408) of all patients treated with SG (n=408). The incidence of hypersensitivity reactions leading to permanent discontinuation of SG was 1% (3/408).
Pre-infusion medication for patients receiving SG is recommended. Observe patients closely for infusion-related reactions during each SG infusion and for at least 30 minutes after completion of each infusion. Medication to treat such reactions, as well as emergency equipment, should be available for immediate use.
Nausea and Vomiting

SG is emetogenic. Nausea occurred in 69% (74/108) of patients with mTNBC and 69% (281/408) of all patients treated with SG. Grade 3 nausea occurred in 6% (7/108) and 5% (22/408) of these populations, respectively. Vomiting occurred in 49% (53/108) of patients with mTNBC and 45% (183/408) of all patients treated with SG. Grade 3 vomiting occurred in 6% (7/108) and 4% (16/408) of these patients, respectively.
Premedicate with a 2- or 3-drug combination regimen (e.g., dexamethasone with either a 5-HT3 receptor antagonist or an NK-1 receptor antagonist as well as other drugs as indicated) for prevention of chemotherapy-induced nausea and vomiting (CINV).
Withhold SG doses for Grade 3 nausea or Grade 3-4 vomiting at the time of scheduled treatment administration and resume with additional supportive measures when resolved to Grade ≤ 1. Additional antiemetics and other supportive measures may also be employed as clinically indicated. All patients should be given take-home medications with clear instructions for prevention and treatment of nausea and vomiting.
Use in Patients with Reduced UGT1A1 Activity

Individuals who are homozygous for the uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1)*28 allele are at increased risk for neutropenia and may be at increased risk for other adverse events following initiation of SG treatment. Closely monitor patients with reduced UGT1A1 activity for severe neutropenia. The appropriate dose for patients who are homozygous for UGT1A1*28 is not known and should be considered based on individual patient tolerance to treatment.
In 84% (343/408) of patients who received SG (up to 10 mg/kg on Days 1 and 8 of a 21-day cycle) and had retrospective UGT1A1 genotype results available, the incidence of Grade 4 neutropenia was 26% (10/39) in patients homozygous for the UGT1A1*28 allele, 13% (20/155) in patients heterozygous for the UGT1A1*28 allele, and 11% (16/149) in patients homozygous for the wild-type allele.
Embryo-Fetal Toxicity

SG contains a genotoxic component and can cause teratogenicity and/or embryo-fetal lethality when administered to a pregnant woman. Advise pregnant women and females of reproductive potential of the potential risk to a fetus.
Advise females of reproductive potential to use effective contraception during treatment with SG and for 6 months following the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with SG and for 3 months after the last dose.
Lactation

Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 1 month after the last dose of SG.

Adverse Reactions

Most common adverse reactions (incidence >25%) in patients with mTNBC are nausea (69%), neutropenia (64%), diarrhea (63%), fatigue (57%), anemia (52%), vomiting (49%), alopecia (38%), constipation (34%), rash (31%), decreased appetite (30%), abdominal pain (26%), and respiratory infection (26%).