On December 1, 2022 Theratechnologies Inc. ("Theratechnologies" or the "Company") (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, reported that it has decided to pause the enrollment of patients in its Phase 1 clinical trial of TH1902, the Company’s lead investigational peptide drug conjugate (PDC) for the treatment of sortilin-expressing cancers (Press release, Theratechnologies, DEC 1, 2022, View Source [SID1234624704]). The Company plans to submit an amendment to its protocol to the U.S. Food and Drug Administration (FDA) for approval.

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Theratechnologies voluntarily made the decision to pause enrollment and revisit the study design after consulting with its investigators. Efficacy results observed thus far were not convincing enough to pursue enrolling patients and did not outweigh the adverse events seen in some patients. As previously reported, these adverse events consist mainly of neuropathy and eye toxicity.

The current intent for the protocol amendment is to modify the dosage regimen to optimize the delivery of TH1902, with lower doses at more frequent intervals. The Company continues to investigate the results obtained thus far in the Phase 1 clinical trial.

"While we are disappointed with these developments, we remain committed to advancing our SORT1+ Technology platform and will continue investigating its potential in the treatment of advanced cancers," said Christian Marsolais, Ph.D., Senior Vice President and Chief Medical Officer, Theratechnologies.

About SORT1+ Technology and TH1902

Theratechnologies is currently developing a platform of proprietary peptides called SORT1+ TechnologyTM for cancer drug development targeting SORT1 receptors. The SORT1 receptor plays a significant role in protein internalization, sorting and trafficking. It is highly expressed in cancer cells compared to healthy tissue, which makes SORT1 an attractive target for cancer drug development. Expression of SORT1 is associated with aggressive disease, poor prognosis and decreased survival. It is estimated that the SORT1 receptor is expressed in 40% to 90% of cases of endometrial, ovarian, colorectal, triple-negative breast and pancreatic cancers.

TH1902 is currently Theratechnologies’ lead investigational PDC candidate for the treatment of cancer derived from its SORT1+ Technology. It is the Company’s proprietary peptide linked to docetaxel – a commonly used cytotoxic agent used to treat many cancers. The FDA granted fast track designation to TH1902 as a single agent for the treatment of all sortilin-positive recurrent advanced solid tumors that are refractory to standard therapy.

On December 1, 2022 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported results from a preclinical in vivo study for its highly selective CDK9 inhibitor, GFH009, that demonstrate robust inhibition of tumor growth in a mouse xenograft model of small cell lung cancer (SCLC) (Press release, Sellas Life Sciences, DEC 1, 2022, View Source [SID1234624703]).

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GFH009 was tested against NCI-H209 SCL xenografts in athymic nude mice in four treatment groups of eight mice each (n=32) consisting of GFH009 alone, olaparib (a PARP inhibitor) alone, a combined regimen of GFH009 and olaparib, and a vehicle control. Treatments were initiated after tumor xenograft volumes exceeded 120 mm3 in each animal group and mice were subsequently sacrificed after mean tumor volume exceeded 1,500 mm3 in the control group.

GFH009 treated mice exhibited a 40.4% decrease in mean tumor growth compared to the control group in this very aggressive cancer model which had a tenfold increase in average tumor volume over 20 days. Strongest effects were observed with GFH009 in combination with olaparib, with mean tumor growth decreased by 72.3%. Treatment with olaparib alone resulted in a 30.2% mean decrease in tumor growth. No significant toxicity or safety concerns were observed in any of the treatment groups.

"We are looking forward to expanding use of GFH009 into solid cancers and these results indicate CDK9 can be an actionable target not only in the apoptotic pathway but also the DNA damage response pathway," said Dragan Cicic, MD, Senior Vice President, Clinical Development, of SELLAS. "This opens up additional avenues for the use of GFH009 in an expanding array of difficult to treat cancers based on their genomic and proteomic traits, moving another step towards truly personalized medicine."

Olaparib is a commercially available targeted oncology drug that acts as a PARP inhibitor and induces synthetic lethality in BRCA mutated cancer cells via DNA damage and response (DDR) pathways. Recently published clinical trials have demonstrated early signals of efficacy for olaparib both as a single agent and in combination with agents targeting DDR, specifically ATR pathways in refractory SCLC patients. CDK9 is known to bind with cyclin K, forming a complex that plays a key role in the DDR pathway which could enhance synthetic lethality of olaparib.

On December 1, 2022 Seagen Inc. (Nasdaq: SGEN) reported that new data for ADCETRIS (brentuximab vedotin) will be featured at the upcoming 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 10-13, 2022 (Press release, Seagen, DEC 1, 2022, View Source [SID1234624702]). The abstracts, including five oral presentations, highlight updated and interim efficacy and safety clinical trial results for ADCETRIS in both early- and advanced stage settings of classical Hodgkin lymphoma (cHL), and in patients with other CD30-expressing lymphomas and other rare cancers.

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"The investigation of ADCETRIS as a single agent or in novel combinations with other agents demonstrates its continued potential to help people impacted by early- and advanced stage Hodgkin lymphoma," said Marjorie Green, M.D., Senior Vice President and Head of Late-Stage Development at Seagen. "Additional data from real-world settings further support the efficacy, safety and impact of ADCETRIS treatment in its approved indications for multiple types of lymphoma."

ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of cHL that is expressed on the surface of several types of lymphomas. It is approved in seven indications in the U.S.

Updated results will also be presented from a phase 1 study of the investigational agent SEA-BCMA in patients with relapsed/refractory multiple myeloma.

Presentations of Company-Sponsored ADCETRIS Clinical Development Trials

Abstract Title

Abstract #

Presentation Time

Lead Author

Brentuximab Vedotin, Nivolumab, Doxorubicin, and Dacarbazine (AN+AD) for Advanced Stage Classic Hodgkin Lymphoma: Updated Efficacy and Safety Results from the Single-Arm Phase 2 Study (SGN35-027 Part B)

314

Oral Presentation

Saturday, Dec. 10

4–5:30 p.m. CT

H. Lee

Population Pharmacokinetics (PPK) and Exposure-Response to Support Body Surface Area (BSA)-Based Dosing of Brentuximab Vedotin in Pediatric Patients with Advanced-Stage Newly Diagnosed Hodgkin Lymphoma (HL)

1616

Poster Session I
Saturday, Dec. 10

5:30–7:30 p.m. CT

X. Zhou

Brentuximab Vedotin, Nivolumab, Doxorubicin, and Dacarbazine (AN+AD) for Early-Stage Classic Hodgkin Lymphoma: Interim Efficacy and Safety Results from the Single-Arm Phase 2 Study (SGN35-027 Part C)

4230

Poster Session III
Monday, Dec. 12

6–8:00 p.m. CT

H. Lee

Brentuximab Vedotin in Frontline Therapy of Hodgkin Lymphoma in Patients with Significant Comorbidities Ineligible for Standard Chemotherapy (SGN35-015 Part E)

1598

Online Abstract

C. Yasenchak

Efficacy and Safety of Retreatment with Brentuximab Vedotin in Patients with Relapsed or Refractory Classical Hodgkin Lymphoma or CD30-Expressing Peripheral T-Cell Lymphoma

5469

Online Abstract

D. Sano

A Phase 4 Multicenter Study of Brentuximab Vedotin Treatment in Chinese Patients with CD30-Positive Cutaneous T-Cell Lymphoma

5491

Online Abstract

Q. Wang

A Spanish Medical Record Review of Adults with Relapsed or Refractory CD30+ Malignancies When Re-Treated with Brentuximab Vedotin (BV): Preliminary Analysis of the BELIEVE Study (NCT04998331)

5528

Online Abstract

A. Sureda

Presentations of Company-Sponsored Health Economic Outcomes Research

Abstract Title

Abstract #

Presentation Type

Lead Author

Real-World Treatment Patterns and Clinical Outcomes with Brentuximab Vedotin or Other Standard Therapies in Patients with Previously Treated Cutaneous T-Cell Lymphoma (CTCL): A Retrospective Chart Review Study in the United States

2265

Poster Session I
Saturday, Dec. 10

5:30–7:30 p.m. CT

S. Barta

Real-World Patient Characteristics, Treatment Patterns, and Outcomes in Patients with Stage III/IV Classic Hodgkin Lymphoma Treated with Frontline ABVD: A Retrospective Analysis Using a Real-World Database

3596

Poster Session II
Sunday, Dec. 11

6–8 p.m. CT

A. Winter

10-Year Impact on Productivity Costs Associated with Mortality in Stage III or IV Classical Hodgkin Lymphoma Based on the Overall Survival Update of the ECHELON-1 Trial: Application of an Oncology Simulation Model in the United States

4846

Poster Session III
Monday, Dec. 12

6–8 p.m. CT

T. Phillips

Economic Burden of Hematopoietic Cell Transplantation (HCT) Among Commercially Insured Patients with Hematological Malignancies in the United States

4850

Poster Session III
Monday, Dec. 12

6–8 p.m. CT

M. Narkhede

Presentation of Company-Sponsored Trials of Pipeline Agents

Abstract Title

Abstract #

Presentation Type

Lead Author

SEA-BCMA Mono- and Combination Therapy in Patients with Relapsed/Refractory Multiple Myeloma: Updated Results of a Phase 1 Study (SGN-BCMA001)

4562

Poster Session III
Monday, Dec. 12

6–8 p.m. CT

J. Hoffman

Presentations of Investigator- and Cooperative-Group Sponsored ADCETRIS Trials

Abstract Title

Abstract #

Presentation Type

Lead Author

Treatment Related Morbidity in Patients with Classical Hodgkin Lymphoma: Results of the Ongoing, Randomized Phase III HD21 Trial by the German Hodgkin Study Group

317

Oral Presentation

Saturday, Dec. 10

4–5:30 p.m. CT

P. Borchmann

Frontline PET-Directed Therapy with Brentuximab Vedotin Plus AVD Followed by Nivolumab Consolidation in Patients with Limited Stage Hodgkin Lymphoma

728

Oral Presentation

Monday, Dec. 12

10:30 a.m.–12 p.m. CT

S. Park

Brentuximab Vedotin Combined with Chemotherapy in Newly Diagnosed, Early-Stage, Unfavorable-Risk Hodgkin Lymphoma: Extended Follow-Up with Evaluation of Baseline Metabolic Tumor Volume and PET2

730

Oral Presentation
Monday, Dec. 12
10:30 a.m.–12 p.m. CT

R. Stuver

Addition of Brentuximab Vedotin to Gemcitabine in Relapsed or Refractory T-Cell Lymphoma: Final Analysis of a LYSA Multicenter, Phase II Study "The TOTAL Trial"

956

Oral Presentation
Monday, Dec. 12
4:30–6 p.m. CT

O. Tournilhac

A Phase I Trial Assessing the Feasibility of Romidepsin Combined with Brentuximab Vedotin for Patients Requiring Systemic Therapy for Cutaneous T-Cell Lymphoma

2911

Poster Session II
Sunday, Dec. 11

6–8 p.m. CT

S. Barta

Peripheral Neuropathy in Children with High-Risk Hodgkin Lymphoma (HL): The Role of Protocol-Stipulated Dose Modification in the Children’s Oncology Group (COG) AHOD1331 Study

2914

Poster Session II
Sunday, Dec. 11

6–8 p.m. CT

S. Parsons,

F. Keller

Evaluating CHIPS in Pediatric High Risk Hodgkin Lymphoma Treated on AHOD1331

2921

Poster Session II

Sunday, Dec. 11

6–8 p.m. CT

C. Schwartz

Phase I Trial of Brentuximab Vedotin Plus Cyclosporine in Relapsed/Refractory Hodgkin Lymphoma

4239

Poster Session III
Monday, Dec. 12

6–8 p.m. CT

S. Kambhampati, M. Mei

About ADCETRIS

ADCETRIS is an antibody-drug conjugate (ADC) comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seagen’s proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing cells.

ADCETRIS is indicated for the treatment of:

Adult patients with previously untreated Stage III/IV cHL in combination with doxorubicin, vinblastine, and dacarbazine.
Pediatric patients 2 years and older with previously untreated high risk cHL in combination with doxorubicin, vincristine, etoposide, prednisone and cyclophosphamide.
Adult patients with cHL at high risk of relapse or progression as post-autologous hematopoietic stem cell transplantation (auto-HSCT) consolidation.
Adult patients with cHL after failure of auto-HSCT or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not auto-HSCT candidates.
Adult patients with previously untreated systemic anaplastic large cell lymphoma (sALCL) or other CD30-expressing peripheral T-cell lymphomas (PTCL), including angioimmunoblastic T-cell lymphoma and PTCL not otherwise specified, in combination with cyclophosphamide, doxorubicin, and prednisone.
Adult patients with sALCL after failure of at least one prior multi-agent chemotherapy regimen.
Adult patients with primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF) who have received prior systemic therapy.
Seagen and Takeda jointly develop ADCETRIS. Under the terms of the collaboration agreement, Seagen has U.S. and Canadian commercialization rights and Takeda has rights to commercialize ADCETRIS in the rest of the world. Seagen recognizes royalty revenues from Takeda based on a percentage of Takeda’s net sales of ADCETRIS in its licensed territories based on annual net sales tiers. Seagen and Takeda jointly fund development costs for ADCETRIS on a 50:50 basis, except in Japan where Takeda is solely responsible for development costs.

ADCETRIS (brentuximab vedotin) for injection U.S. Important Safety Information

BOXED WARNING

PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY (PML): JC virus infection resulting in PML and death can occur in ADCETRIS-treated patients.

CONTRAINDICATION

Contraindicated with concomitant bleomycin due to pulmonary toxicity (e.g., interstitial infiltration and/or inflammation).

WARNINGS AND PRECAUTIONS

Peripheral neuropathy (PN): ADCETRIS causes PN that is predominantly sensory. Cases of motor PN have also been reported. ADCETRIS-induced PN is cumulative. Monitor for symptoms such as hypoesthesia, hyperesthesia, paresthesia, discomfort, a burning sensation, neuropathic pain, or weakness. Patients experiencing new or worsening PN may require a delay, change in dose, or discontinuation of ADCETRIS.

Anaphylaxis and infusion reactions: Infusion-related reactions (IRR), including anaphylaxis, have occurred with ADCETRIS. Monitor patients during infusion. If an IRR occurs, interrupt the infusion and institute appropriate medical management. If anaphylaxis occurs, immediately and permanently discontinue the infusion and administer appropriate medical therapy. Premedicate patients with a prior IRR before subsequent infusions. Premedication may include acetaminophen, an antihistamine, and a corticosteroid.

Hematologic toxicities: Fatal and serious cases of febrile neutropenia have been reported with ADCETRIS. Prolonged (≥1 week) severe neutropenia and Grade 3 or 4 thrombocytopenia or anemia can occur with ADCETRIS.

Administer G-CSF primary prophylaxis beginning with Cycle 1 for adult patients who receive ADCETRIS in combination with chemotherapy for previously untreated Stage III/IV cHL or previously untreated PTCL, and pediatric patients who receive ADCETRIS in combination with chemotherapy for previously untreated high risk cHL.

Monitor complete blood counts prior to each ADCETRIS dose. Monitor more frequently for patients with Grade 3 or 4 neutropenia. Monitor patients for fever. If Grade 3 or 4 neutropenia develops, consider dose delays, reductions, discontinuation, or G-CSF prophylaxis with subsequent doses.

Serious infections and opportunistic infections: Infections such as pneumonia, bacteremia, and sepsis or septic shock (including fatal outcomes) have been reported in ADCETRIS-treated patients. Closely monitor patients during treatment for infections.

Tumor lysis syndrome: Patients with rapidly proliferating tumor and high tumor burden may be at increased risk. Monitor closely and take appropriate measures.

Increased toxicity in the presence of severe renal impairment: The frequency of ≥Grade 3 adverse reactions and deaths was greater in patients with severe renal impairment. Avoid use in patients with severe renal impairment.

Increased toxicity in the presence of moderate or severe hepatic impairment: The frequency of ≥Grade 3 adverse reactions and deaths was greater in patients with moderate or severe hepatic impairment. Avoid use in patients with moderate or severe hepatic impairment.

Hepatotoxicity: Fatal and serious cases have occurred in ADCETRIS-treated patients. Cases were consistent with hepatocellular injury, including elevations of transaminases and/or bilirubin, and occurred after the first ADCETRIS dose or rechallenge. Preexisting liver disease, elevated baseline liver enzymes, and concomitant medications may increase the risk. Monitor liver enzymes and bilirubin. Patients with new, worsening, or recurrent hepatotoxicity may require a delay, change in dose, or discontinuation of ADCETRIS.

PML: Fatal cases of JC virus infection resulting in PML have been reported in ADCETRIS-treated patients. First onset of symptoms occurred at various times from initiation of ADCETRIS, with some cases occurring within 3 months of initial exposure. In addition to ADCETRIS therapy, other possible contributory factors include prior therapies and underlying disease that may cause immunosuppression. Consider PML diagnosis in patients with new-onset signs and symptoms of central nervous system abnormalities. Hold ADCETRIS if PML is suspected and discontinue ADCETRIS if PML is confirmed.

Pulmonary toxicity: Fatal and serious events of noninfectious pulmonary toxicity, including pneumonitis, interstitial lung disease, and acute respiratory distress syndrome, have been reported. Monitor patients for signs and symptoms, including cough and dyspnea. In the event of new or worsening pulmonary symptoms, hold ADCETRIS dosing during evaluation and until symptomatic improvement.

Serious dermatologic reactions: Fatal and serious cases of Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) have been reported with ADCETRIS. If SJS or TEN occurs, discontinue ADCETRIS and administer appropriate medical therapy.

Gastrointestinal (GI) complications: Fatal and serious cases of acute pancreatitis have been reported. Other fatal and serious GI complications include perforation, hemorrhage, erosion, ulcer, intestinal obstruction, enterocolitis, neutropenic colitis, and ileus. Lymphoma with pre-existing GI involvement may increase the risk of perforation. In the event of new or worsening GI symptoms, including severe abdominal pain, perform a prompt diagnostic evaluation and treat appropriately.

Hyperglycemia: Serious cases, such as new-onset hyperglycemia, exacerbation of pre-existing diabetes mellitus, and ketoacidosis (including fatal outcomes) have been reported with ADCETRIS. Hyperglycemia occurred more frequently in patients with high body mass index or diabetes. Monitor serum glucose and if hyperglycemia develops, administer anti-hyperglycemic medications as clinically indicated.

Embryo-fetal toxicity: Based on the mechanism of action and animal studies, ADCETRIS can cause fetal harm. Advise females of reproductive potential of this potential risk, and to avoid pregnancy during ADCETRIS treatment and for 6 months after the last dose of ADCETRIS.

ADVERSE REACTIONS

The most common adverse reactions (≥20% in any study) are peripheral neuropathy, fatigue, nausea, diarrhea, neutropenia, upper respiratory tract infection, pyrexia, constipation, vomiting, alopecia, decreased weight, abdominal pain, anemia, stomatitis, lymphopenia, mucositis, thrombocytopenia, and febrile neutropenia.

DRUG INTERACTIONS

Concomitant use of strong CYP3A4 inhibitors has the potential to affect the exposure to monomethyl auristatin E (MMAE). Closely monitor adverse reactions.

USE IN SPECIAL POPULATIONS

Lactation: Breastfeeding is not recommended during ADCETRIS treatment.

Females and Males of Reproductive Potential: Advise females to report pregnancy immediately and advise males with female sexual partners of reproductive potential to use effective contraception during ADCETRIS treatment and for 6 months after the last dose of ADCETRIS.

Please see full Prescribing information, including BOXED WARNING, for ADCETRIS here.

On December 1, 2022 Scandion Oncology (Scandion), a biotech company developing first-in-class medicines aimed at treating cancer which is resistant to current treatment options, reported that the company’s Board of Directors has appointed Francois Martelet as new Chief Executive Officer (CEO) (Press release, Scandion Oncology, DEC 1, 2022, View Source;m-d–as-new-chief-executive-officer,c3675834 [SID1234624701]). Francois will take up his new position on January 2, 2023.

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Trained as a Medical Doctor with a Master’s degree in Business, Francois Martelet is an experienced executive and CEO with a career spanning more than 30 years in the global pharmaceutical and biotech industry and more than 20 years of experience as an executive. He has significant experience within early and late-stage clinical development in oncology (cancer) as well as business and partnership development.

"We are delighted to appoint Francois Martelet as the new CEO of Scandion. Francois brings a wealth of highly relevant experiences to Scandion as an experienced and passionate business leader with the qualifications to successfully develop Scandion and our lead asset, SCO-101", says Martin Møller, Chairman of the Board of Directors of Scandion.

Francois Martelet has worked and held leadership positions in large pharmaceutical companies including Roche, Eli Lilly, Novartis and MSD. He has also been the CEO of a number of biotech companies, most recently Vivesto AB, where he completed a full turnaround and led the build-up of an oncology pipeline through organic and external growth.

"I am very excited by the opportunity to join and lead Scandion in its pursuit to revert cancer drug resistance, which would make a tremendous difference to patients. Scandion has a huge medical and therefore commercial potential which I am looking forward to help fulfil", says Francois Martelet.

Acting CEO & CFO Johnny Stilou will continue in this role until January 2, 2023, and afterwards resume his role as CFO of Scandion.

On December 1, 2022 Salarius Pharmaceuticals, Inc. (NASDAQ: SLRX), a clinical-stage biopharmaceutical company using targeted protein inhibition and targeted protein degradation to develop therapies for patients with cancer in need of new treatment options, reported interim clinical trial results from the company’s Phase 1/2 trial of its novel oral, reversible, targeted LSD1 inhibitor, seclidemstat, as a treatment for Ewing sarcoma and FET-rearranged sarcomas (Press release, Salarius Pharmaceuticals, DEC 1, 2022, View Source [SID1234624700]).

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These interim results appear to indicate that first- and second-relapse Ewing sarcoma patients treated with seclidemstat in combination with topotecan and cyclophosphamide who achieve disease control may have an increased time to tumor progression (TTP) compared with treatment of topotecan and cyclophosphamide alone, per the Phase 3 rEECur study described below. The Ewing sarcoma and FET-rearranged sarcoma trial is currently on a partial clinical hold as described below.

As of October 31, 2022, 13 first- and second-relapse Ewing sarcoma patients were evaluable for confirmed disease control rate assessment. Of these 13 patients, five patients (38%) achieved confirmed disease control with no tumor progression observed while treated with seclidemstat in combination with topotecan and cyclophosphamide. Treatment duration for the 13 patients ranged from 0.7 months to 13.8 months.

Five first-relapse Ewing sarcoma patients were treated and three (60%) achieved confirmed disease control, including one complete response, one partial response and one stable disease at 12.8 months and continuing. First-relapse patients had a median TTP of 7.4 months and treatment duration ranged from 1.4 months to 13.8 months.

At ASCO (Free ASCO Whitepaper) 2022, Euro Ewing Consortium presented rEECur2 Phase 3 study results in relapsed/refractory Ewing sarcoma patients that showed median event-free survival of 3.5 months in the topotecan/ cyclophosphamide arm (n=73) compared with 5.7 months in the high-dose ifosfamide arm (n=73)2. The rEECur data includes approximately 80% primary refractor or first-relapse. Ewing sarcoma patients after relapse have 5-year overall survival of about 13% and 10-year overall survival of about 9%3.

"Ewing sarcoma is a devastating bone and soft tissue cancer that affects children, adolescents and young adults, and treatment options primarily consist of chemotherapies, radiation and surgical resection," commented Damon Reed M.D., Program Leader, Adolescent Young Adult Program, Chair of Department of Individualized Cancer Management Moffitt Cancer Center and principal investigator in this sarcoma trial. "As an oncologist treating Ewing sarcoma patients, ultimately new treatments that extend a
1 Confirmed disease control rate includes complete remission, partial remission or stable disease confirmed by both cycle 2 and cycle 4 scans. Treatment cycles are 21 days in length, with cycle 2 scans occurring on or about day 42 and cycle 4 scans occurring on or about day 84.

2 rEECur – International Randomized Controlled Trial of Chemotherapy for the Treatment of Recurrent and Primary Refractory Ewing Sarcoma.
3 Risk of recurrence and survival after relapse in patients with Ewing sarcoma, Pediatric Blood & Cancer, Volume 57, Issue 4. First published October 2011.

David Arthur, President and CEO of Salarius, said, "These interim results are encouraging, and I believe they show the potential for seclidemstat to provide a more durable response among Ewing sarcoma patients when used in combination with topotecan and cyclophosphamide. This trial has more than 15 clinical trial sites with more than 20 locations throughout the United States, and when needed Salarius provides travel assistance to patients who want to participate in the sarcoma trial. It is our hope that the potential for seclidemstat to improve outcomes coupled with our support will make it easier for patients to participate when enrollment is restarted."

As previously reported, on October 18, 2022, enrollment of new patients in the Salarius-sponsored seclidemstat sarcoma clinical trial and the MD Anderson investigator-initiated hematologic clinical trial was voluntarily paused due to a suspected unexpected serious adverse reaction (SUSAR) observed in the sarcoma trial; patients currently enrolled in both studies are able to continue treatment after consulting with their physician. The U.S. Food and Drug Administration (FDA) subsequently agreed with Salarius’ approach and placed the sarcoma trial on partial clinical hold; Salarius is working with the FDA to further analyze the available data with the goal of understanding how best to proceed and restart enrollment.

Additional interim trial results include the following:
•Among the 13 Ewing sarcoma patients treated with seclidemstat, topotecan and cyclophosphamide
oThere was a 38% confirmed disease control rate and 1.6 months median TTP which ranged from 0.7 months to 13.8 months
oOne second relapse patient with confirmed stable disease withdrew from the study with no observed progression at 3.5 months due to a non-study related adverse event
oOne second relapse patient with a confirmed partial response withdrew from the study with no observed progression at 3.1 months
•Among the five first-relapse Ewing sarcoma patients treated, three (60%) achieved confirmed disease control
oOne patient achieved a complete response and withdrew from the study at 7.4 months
oOne patient achieved a partial response with 78% target lesion reduction; this patient subsequently elected radiation treatment as consolidation and withdrew from the study at 13.8 months
oOne patient achieved stable disease and continues on treatment after 12.8 months
•Single-agent activity has not been observed in the FET-rearranged sarcoma cohort of the trial
•More than 85 patients have been treated in either the dose-escalation or the dose-expansion portions of the trial with seclidemstat as a standalone therapy or in combination with standard-of-care chemotherapy

Conference call and webcast
Salarius plans to hold an investor call in mid-December to discuss these interim results and other clinical and preclinical data following presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting by MD Anderson Cancer Center and Salarius. Details about the conference call including how to participate will be provided in a separate news release.

About the Phase 1/2 Ewing’s and other FET-rearranged sarcomas trial
The Phase 1/2 trial currently is in its dose-expansion stage, which includes three patient arms. The first arm is planned to enroll up to 30 patients with Ewing’s sarcoma, a rare and deadly pediatric bone cancer, and will investigate seclidemstat in combination with topotecan and cyclophosphamide, a commonly used second- and third-line chemotherapy regimen. Salarius believes data released during ASCO (Free ASCO Whitepaper) 2021 demonstrated synergy in an Ewing’s sarcoma cell line when seclidemstat was used in combination with these agents. Salarius also believes this treatment combination and its use as a second- and third-line therapy has the potential to expand the addressable patient population for seclidemstat and improve outcomes by allowing physicians to introduce seclidemstat earlier in the Ewing’s sarcoma continuum of care.

The trial’s second patient arm is planned to investigate seclidemstat as a single agent in up to 15 patients with myxoid liposarcoma. The third patient arm is planned to investigate seclidemstat as a single agent in up to 15 patients with select sarcomas that share a similar biology to Ewing’s sarcoma, also referred to as FET-rearranged or Ewing’s-related sarcomas. In data released at ASCO (Free ASCO Whitepaper) 2021, a subset of patients with advanced FET-rearranged sarcomas treated with single-agent seclidemstat resulted in stable disease and prolonged time to progression, which Salarius believes suggests disease control, a clinically relevant endpoint for soft tissue sarcomas.

All three patient arms are designed to evaluate safety and efficacy in patients with advanced disease.

Salarius supports patient referrals to their sarcoma trial sites and provides travel assistance for clinical trial patients and families who are evaluating and participating in the sarcoma trial.

Clinical trial sites include Seattle Cancer Care Alliance (SCCA) – which is comprised of the Fred Hutchinson Cancer Research Center, Seattle Children’s Hospital and University of Washington Medical Center; Oregon Health & Sciences University Portland, OR; Johns Hopkins All Children’s Hospital in St. Petersburg, FL; Children’s Hospital of Los Angeles in Los Angeles, CA; Moffitt Cancer Center in Tampa, FL; Dana-Farber Cancer Institute in Boston, MA; MD Anderson Cancer Center in Houston, TX; Nationwide Children’s Hospital in Columbus, OH; Memorial Sloan Kettering Cancer Center in New York NY; the Sarcoma Oncology Center in Santa Monica, CA; Virginia Cancer Specialists, Fairfax, Virginia; Cleveland Clinic, in Cleveland, OH; Washington University, St. Louis, MO and Fox Chase Cancer Center in Philadelphia PA. Information on the Salarius Sarcoma trial SALA-002-EW16 (NCT03600649) can be found at clinicaltrial.gov.