On May 12, 2022 Karyopharm Therapeutics Inc. (NASDAQ: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that initial data from a Phase 1 portion of the Ph 1/2 study evaluating selinexor in combination with ruxolitinib in patients with treatment-naïve myelofibrosis has been selected for a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress taking place June 9-12, 2022, in Vienna, Austria (Press release, Karyopharm, MAY 12, 2022, View Source [SID1234614327]). The data are also being presented in a poster session at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on Saturday, June 4th.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are pleased to be sharing initial selinexor data in treatment-naïve patients with myelofibrosis at both EHA (Free EHA Whitepaper) and ASCO (Free ASCO Whitepaper)," said Reshma Rangwala, MD, PhD, Chief Medical Officer of Karyopharm. "JAK inhibitors remain the only class of approved therapies for myelofibrosis and there is a critical need for novel class of treatments that improve the hallmarks of this devastating blood disease including reductions in spleen size, as well as improvements in constitutional symptoms, quality of life, and anemia response. We look forward to sharing our findings and further exploring selinexor in this patient population."

Additional updated data will be presented during poster sessions at both EHA (Free EHA Whitepaper) and ASCO (Free ASCO Whitepaper).

Details for the EHA (Free EHA Whitepaper) 2022 Hybrid Congress presentation are as follows:
Poster Presentation

Title: A phase 1, open-label, dose-escalation study of selinexor plus ruxolitinib in patients with treatment-naïve myelofibrosis
Session type: Poster presentation
Presenter: Haris Ali, City of Hope
Abstract #: P1005
Date and time: Friday, June 10, 2022; 16:30 – 17:45 CEST

About XPOVIO (selinexor)
XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with Velcade (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in a growing number of ex-U.S. territories and countries, including Europe, the United Kingdom, China, South Korea, Singapore and Israel, and is marketed in those areas by Karyopharm’s global partners. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer and myelofibrosis. For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at:

Tel: +1 (888) 209-9326
Email: [email protected]

XPOVIO (selinexor) is a prescription medicine approved:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti–CD38 monoclonal antibody (Xd).
For the treatment of adult patients with relapsed or refractory diffuse large B–cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
SELECT IMPORTANT SAFETY INFORMATION
Warnings and Precautions
Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony–stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo–Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3–4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3–4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions were infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.

On May 12, 2022 Ad hoc announcement pursuant to Art. 53 LR: Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported its interim management statement for the quarter ending March 31, 2022 (Press release, Molecular Partners, MAY 12, 2022, View Source [SID1234614344]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Following a significant capital inflow in January, resulting from Novartis exercising its right to in-license global rights to ensovibep, we are leveraging our strong cash position to follow our strategy; to prioritize candidates where DARPins have an innate advantage over other approaches. Programs like ensovibep, the breakthrough design of MP0533 and our new research in radioligand therapies, showcase the success of our evolved strategy, which emphasizes highly differentiated molecules that can rapidly demonstrate clear activity and benefit for patients," said Patrick Amstutz, Molecular Partners’ CEO. "We continue to support Novartis on next steps for ensovibep’s global strategy as we advance our internal portfolio, building on the strong clinical performance our DARPins candidates have continually demonstrated."

Research & development highlights:

Ensovibep COVID-19 antiviral program: Positive Phase 2 results, Option exercised by Novartis
Part A of the EMPATHY global clinical trial met its primary endpoint with a statistically significant reduction in viral load over eight days in the ensovibep arms compared to placebo
Secondary EMPATHY endpoint of hospitalization and/or emergency room (ER) visits related to COVID-19, or death showed an overall 78% reduction in relative risk of events across all ensovibep arms compared to placebo
In April, the results of the EMPATHY Part A study were presented as a late breaker at the 2022 European Congress of Clinical Microbiology and Infectious Diseases (ECCMID)
Novartis exercised the option to license all rights to ensovibep, triggering a CHF 150 million payment to Molecular Partners
Novartis requested Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA) for ensovibep. As previously reported by Novartis, the EUA remains under review, and the FDA has indicated that Phase 3 data will be required prior to authorization. Novartis is currently engaged in developing a Phase 3 protocol for alignment with the FDA’s recommendations
MP0310 (FAP x 4-1BB)
Amgen returned global rights for MP0310 following a strategic pipeline review. The ongoing Phase 1 study is expected to continue as planned, with data expected later in 2022 that will inform further business development activity
MP0317 (FAP x CD40):
The Phase 1 open-label dose escalation study, initiated in Q4 2021, continues in patients with solid tumors known to express FAP. Initial data from this study is expected in the second half of 2022
In March 2022, preclinical data were published in the research journal Cancer Immunology Research1 supporting MP0317’s potential to deliver tumor-localized immune activation while avoiding systemic toxicity seen with other CD40-targeting agents
MP0533 (CD33 x CD70 x CD123 x CD3)
Following continued promising preclinical data supporting the unique design and mechanism of this candidate, a lead molecule was selected and named MP0533. It is expected to enter clinical development in 2022
New in vivo data from the MP0533 program will be presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA2022) which will be held in Vienna, Austria from June 9 to 12
Q1 2022 operational and financial highlights:

Strong financial position with CHF 296.2 million in cash (including short term deposits) as of March 31, 2022
Revenue of CHF 172.8 million primarily due to payment received from Novartis upon exercise of option to in-license global rights to ensovibep
Net cash from operating activities of CHF 163.6 million in Q1 2022
Operating profit of CHF 152.6 million and net profit of CHF 153.1 million in Q1 2022
Company expected to be funded into 2026, excluding any potential payments from R&D partnerships
The Q1 2022 Financial Statements are available on the company’s website
Ensovibep for COVID-19: In partnership with Novartis

Ensovibep is a first-in-class, multi-specific pan-variant DARPin therapeutic candidate, designed to bind three different epitopes on the receptor-binding domain (RBD) of the SARS-CoV-2 spike protein simultaneously.

Part A of the EMPATHY clinical trial – a randomized, placebo-controlled study which enrolled 407 symptomatic patients infected with SARS-CoV-2 – met its primary endpoint with a statistically significant reduction in viral load over eight days in the ensovibep arms compared to placebo. The secondary endpoint of hospitalization and/or emergency room (ER) visits related to COVID-19, or death showed an overall 78% reduction in relative risk of events across all ensovibep arms compared to placebo.

Pursuant to the Option and Equity Rights Agreement executed in October 2020 with Novartis and following positive Phase 2 (Part A) results, Novartis exercised its option for ensovibep in January 2022, triggering a milestone payment of CHF 150 million to Molecular Partners. Novartis is now responsible for further development, manufacturing, distribution and commercialization activities.

Novartis requested Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA) for ensovibep. As previously reported by Novartis, the application of the EUA remains under review and additional clinical data will be required to be authorized. While the current Omicron wave of SARS-Cov-2, and the lower incidence of hospitalization associated with it, has made clinical trials challenging to execute in this evolving environment, Novartis is continuing to supply additional documentation and data to the FDA in regards to the EUA review. The FDA has indicated that additional Phase 3 clinical data will be required to support EUA; Novartis is currently engaged in developing a Phase 3 study protocol in alignment with the FDA’s recommendations.

If approved or authorized by relevant regulatory authorities, ensovibep would be the first approved multi-specific antiviral candidate for the treatment of COVID-19 and the first DARPin therapy approved or authorized by a regulatory agency. Based on the strong clinical performance of ensovibep, the Company is assessing further viral disease areas where DARPins can offer advantages over existing antivirals or where no effective treatments exist.

Oncology: Phase 1 trial of MP0317; Phase 1 trial of MP0310; Progress of AML candidate MP0533; Development of a DARPin-based radioligand program
The ongoing Phase 1 trial of MP0317 is expected to enroll up to 30 patients across six dosing cohorts and up to 15 patients are then expected to be enrolled in a dose expansion cohort. In addition to evaluating monotherapy dynamics, the study plans to gather a wide variety of biomarker data to support the establishment of combination therapies with MP0317 in specific indications. MP0317 targets both the fibroblast activation protein (FAP) and the immunostimulatory protein CD40. MP0317 is designed to enable tumor-localized immune activation and fewer side effects compared to other CD40-targeting agents.

MP0310 is also designed to deliver tumor-localized immune activation and activates the immunostimulatory 4-1BB protein. A Phase 1 study of this candidate as a treatment for solid tumors is ongoing, with a full dataset expected later in 2022. Following Amgen’s strategic pipeline review and return of global rights to MP0310, the Phase 1 dataset will inform further business development activity.

MP0533, Molecular Partners’ novel acute myeloid leukemia (AML) candidate, is a DARPin designed to engage CD3 on T-cells while binding up to three tumor-associated antigens (CD33, CD70 and CD123) on AML cells. Preclinical studies have shown the binding strength of MP0533 increasing significantly with the number of tumor-associated antigens present. This ‘avidity-dependent’ mechanism, enabled by the DARPin platform, leads to preferential targeting of AML cells which, unlike healthy cells, generally express two or more of these antigens. Once bound, the AML cells are marked for termination by nearby T-cells activated by MP0533. Clinical development is expected to begin in 2022.

Molecular Partners is also collaborating with Novartis to develop, manufacture and commercialize DARPin-conjugated radioligand therapies (DARPin-RLTs). The collaboration combines DARPins’ unique properties, including small size and very high affinity and specificity, with the RLT capabilities and expertise of Novartis. DARPin-RLTs have the potential to deliver molecule-targeted radiation deeply into the tumor thereby harnessing the power of radioactive atoms for tumor-killing. Under the terms of the agreement, Molecular Partners will collaborate with Novartis to discover DARPin-RLTs that target specific tumor-associated antigens. Both parties will collaborate on the discovery and optimization of the therapeutic candidates for further development.

Balance sheet: Strong cash and equity positions as of March 2022

Ongoing strong financial position with CHF 296.2 million in cash and short-term deposits as of March 31, 2022
Net cash inflow from operating activities of CHF 163.6 million in the first three months of 2022
Financial outlook 2022

For the full year 2022, at constant exchange rates, the Company expects total expenses of CHF 75 – 85 million, of which approximately CHF 9 million will be non-cash effective costs for share-based payments, IFRS pension accounting and depreciation. This cash flow guidance does not include any potential receipts from R&D partnerships.

With CHF 296.2 million in cash and short-term time deposits and no debt as of March 31, 2022, the Company expects to be funded into 2026, excluding any potential receipts from R&D partners.

Financial Calendar

25 August 2022 – Publication of Half-year Results 2022 (unaudited)

27 October 2022 – Interim Management Statement Q3 2022

About DARPin therapeutics

DARPin therapeutics are a new class of custom-built protein therapeutics based on natural binding proteins that open a new dimension of multi-functionality and multi-target specificity in drug design. A single DARPin candidate can engage more than five targets, and its flexible architecture and small size offer benefits over conventional monoclonal antibodies or other currently available protein therapeutics. DARPin therapeutics have been clinically validated through to registration via the development of abicipar, Molecular Partners’ most advanced DARPin drug candidate. The DARPin platform is a fast and cost-effective drug discovery engine, producing drug candidates with optimized properties for development and very high production yields.

On May 12, 2022 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the first quarter ended March 31, 2022 (Press release, Mustang Bio, MAY 12, 2022, View Source [SID1234614361]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "Mustang started strong in 2022, with multiple data announcements and key clinical milestones achieved across our robust pipeline of CAR T cell and gene therapies. In the first quarter, data were presented on three of our CAR T cell therapies and our oncolytic virus clinical candidate at prestigious medical conferences by our academic collaborators at City of Hope and Fred Hutchinson Cancer Center ("Fred Hutch"). City of Hope’s Dr. Tanya Dorff presented Phase 1 clinical trial data on MB-105, our prostate stem cell antigen ("PSCA") chimeric antigen receptor ("CAR") T-cell therapy for treatment of PSCA-positive metastatic castration-resistant prostate cancer ("mCRPC") at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Genitourinary ("GU") Cancers Symposium that demonstrated its potential as a PSCA-targeted CAR T-cell therapy for prostate cancer and other solid tumors that express PSCA. Fred Hutch’s Dr. Mazyar Shadman presented updated interim Phase 1/2 clinical trial data on MB-106, our CD20-targeted, autologous CAR T cell therapy for treatment of relapsed or refractory B-cell non-Hodgkin lymphomas ("NHL") and chronic lymphocytic leukemia ("CLL"), at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research ("2022 Tandem Meetings") and at the 4th International Workshop on CAR-T and Immunotherapies ("iwCAR-T"). The data demonstrated high efficacy and a favorable safety profile, with an overall response rate ("ORR") of 96% and complete response ("CR") rate of 72% observed in all patients across all dose levels. The data included two patients previously treated with CD19-directed CAR T therapy who relapsed and responded to MB-106, demonstrating its potential as an immunotherapy option for these patients. Additionally, City of Hope’s Dr. Christine Brown presented Phase 1 clinical trial data from a late-breaking poster regarding MB-108 (C134 oncolytic virus licensed from Nationwide Children’s Hospital) and MB‐101 (IL13Rα2‐targeted CAR T cell therapy licensed from City of Hope) for the treatment of recurrent glioblastoma ("rGBM"), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting 2022 that support the safety of administering these two therapies sequentially to optimize treatment in a regimen designated as MB-109."

"Looking ahead, we have additional upcoming data presentations for MB-106 at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 ("EHA2022") Hybrid Congress and for our MB-107 gene therapy for the treatment of X-linked severe combined immunodeficiency ("XSCID") in newly diagnosed infants under the age of two at the American Society of Gene & Cell Therapy ("ASGCT") 25th Annual Meeting. We plan to dose the first patient under Mustang’s IND in a multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL at the end of this quarter. In the second half of 2022, we plan to file an IND to initiate a Phase 1 clinical trial for MB-109 for the treatment of rGBM and enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang’s IND to evaluate MB-107. It’s an incredibly exciting time for our team and we look forward to continuing to advance our cell and gene therapies to help meet the needs of patients with hard-to-treat diseases."

Financial Results:

●As of March 31, 2022, Mustang’s cash and cash equivalents and restricted cash totaled $123.2 million, compared to $110.6 million as of December 31, 2021, an increase of $12.6 million year-to-date.
●Research and development expenses were $16.3 million for the first quarter of 2022, compared to $11.6 million for the first quarter of 2021. Non-cash, stock-based expenses included in research and development were $0.5 million for the first quarter of 2022, compared to $0.7 million for the first quarter of 2021.
●General and administrative expenses were $3.3 million for the first quarter of 2022, compared to $3.5 million for the first quarter of 2021. Non-cash, stock-based expenses included in general and administrative expenses were $1.0 million for the first quarter of 2022, compared to $1.5 million for the first quarter of 2021.
●Net loss attributable to common stockholders was $19.8 million, or $0.20 per share, for the first quarter of 2022, compared to a net loss attributable to common stockholders of $15.0 million, or $0.19 per share, for the first quarter of 2021.
Recent Corporate Highlights:

●In February 2022, Mustang was selected as the Bronze winner for the Central region in the Eighteenth Annual Team Massachusetts Economic Impact Awards presented by MassEcon. The award winners were honored at Gillette Stadium on April 7, 2022.
●Also in February 2022, Phase 1 data on MB-105, a PSCA-targeted CAR T cell therapy administered systemically to patients with PSCA-positive mCRPC, were presented by City of Hope at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium. The data indicated that PSCA-targeted CAR T-cell therapy is feasible in patients with mCRPC with dose-limiting toxicity ("DLT") of cystitis and is associated with preliminary anti-tumor effect at a dose of 100 million cells plus lymphodepletion. It was concluded that escalation up to the next dose level of 300 million cells plus modified lymphodepletion can proceed in the trial.
●In March 2022, Mustang completed a $75 million long-term debt facility with Runway Growth Capital LLC (with $30.0 million funded on the closing date and the remaining $45.0 million becoming available if Mustang achieves certain predetermined milestones).
●In April 2022, Mustang announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL and CLL, were presented at the 2022 Tandem Meetings. Data demonstrated high efficacy and a favorable safety profile in all patients (n=25). Five dose levels were used during the study, and complete responses were observed at all dose levels. Durable responses were observed in a wide range of hematologic malignancies including follicular lymphoma ("FL"), CLL, diffuse large B-cell lymphoma ("DLBCL"), and Waldenstrom macroglobulinemia. An ORR of 96% and CR rate of 72% was observed in all patients across all dose levels. Additionally, two patients had been previously treated with CD19-directed CAR T therapy and subsequently relapsed, and both responded to treatment, one patient with FL with a CR and the other with DLBCL with a partial response. A copy of the abstract can be viewed on the meeting website here. Mustang expects to dose the first patient in a multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL later this quarter.
●Also in April 2022, MB-106 data focused on CLL were presented at the 4th iwCAR-T.
●Additionally, in April 2022, Mustang announced its plan to file an IND in the second half of 2022 to initiate a Phase 1 clinical trial combining CAR T cells and an oncolytic virus for the treatment of rGBM, supported by interim data from two ongoing investigator-sponsored Phase 1 clinical trials evaluating two clinical candidates, MB‐101 (IL13Rα2‐targeted CAR T cell therapy licensed from City of Hope) and MB-108 (C134 oncolytic virus licensed from Nationwide Children’s Hospital). The data are from a late-breaking poster presented at the AACR (Free AACR Whitepaper) Annual Meeting 2022. Preclinical data also presented support the safety of administering these two therapies sequentially to optimize treatment in a regimen designated as MB-109.
●In May 2022, Mustang announced that MB-106 CD20-targeted CAR T therapy data were selected for an oral presentation at EHA (Free EHA Whitepaper)2022 taking place June 9 – 12 in Vienna. Dr. Mazyar Shadman of Fred Hutch will present updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL. A copy of the abstract can be viewed online through the EHA (Free EHA Whitepaper)2022 website here.
●In May 2022, Mustang announced that interim Phase 1/2 data on MB-107, Mustang’s lentiviral gene therapy for the treatment of XSCID, also known as bubble boy disease, in newly diagnosed infants under the age of two, were selected for an oral presentation during the Clinical Trials Spotlight Symposium at the ASGCT (Free ASGCT Whitepaper) 25th Annual Meeting taking place May 16-19, 2022, both virtually and in Washington, D.C. The presentation will include updated data from a multicenter Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Children’s Research Hospital, UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital. The abstract can be viewed on the meeting website here.
●In the second half of 2022, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang’s IND to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with XSCID.
●Mustang filed an IND application in December 2021 for its pivotal multicenter Phase 2 clinical trial of MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with hematopoietic stem cell transplantation ("HSCT") and for whom re-treatment is indicated. The trial is currently on hold pending CMC clearance from the FDA and, based on feedback from the Agency, Mustang Bio expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial in the first quarter of 2023.

On May 12, 2022 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported pipeline and business progress and first quarter 2022 financial results (Press release, Nuvalent, MAY 12, 2022, View Source [SID1234614378]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"At Nuvalent, we’re building a fully integrated biotech company with the goal to design, develop, and deliver a portfolio of best-in-class therapies for patients with cancer. We view 2022 as a critical year of execution and we have already made meaningful progress across our pipeline. Enrollment is progressing well in the ongoing Phase 1 portion of our ARROS-1 Phase 1/2 trial of NVL-520 for advanced ROS1-positive NSCLC and other solid tumors, and following the recently announced clearance of the IND for NVL-655, we are on track for planned initiation of our ALKOVE-1 Phase 1/2 study for advanced ALK-positive NSCLC and other solid tumors in the second quarter of 2022," said James Porter, Ph.D., Chief Executive Officer at Nuvalent. "By year-end, our team has the potential to have delivered four novel development candidates since company formation, with two clinical trials underway. With an exceptional team and strong foundation in place, I am confident in our ability to bring precisely targeted therapies that can enable deeper and more durable responses to patients with cancer."

Upcoming Pipeline Milestones
ALKOVE-1 Trial of NVL-655 for Patients with ALK-positive Non-Small Cell Lung Cancer (NSCLC) Expected to Begin in Second Quarter of 2022: Nuvalent has received confirmation from the U.S. Food and Drug Administration (FDA) that its Investigational New Drug (IND) application for NVL-655 has been cleared. The company is on track to initiate the ALKOVE-1 Phase 1/2 study of NVL-655 for patients with advanced ALK-positive NSCLC and other solid tumors, anticipated in the second quarter of 2022. NVL-655 is a novel ALK-selective inhibitor designed to address the clinical challenges of emergent treatment resistance, CNS adverse events, and brain metastases that may limit the use of currently available ALK kinase inhibitors.
Selection of Two Additional Development Candidates from Discovery Pipeline Targeted in 2022: Nuvalent continues to advance its pipeline expansion efforts with multiple discovery-stage research programs. The company expects to select development candidates for its programs directed toward ALK IXDN compound resistance mutations and HER2 Exon 20 insertions in 2022.
Recent Business Highlights
Appointed Anna Protopapas as Chair of Board of Directors: Nuvalent appointed Anna Protopapas, President and Chief Executive Officer of Mersana Therapeutics, as Chair of its Board of Directors in March 2022. Ms. Protopapas joined Mersana in March 2015 and has a substantial track record of executive leadership and business growth in the field of oncology. Her broad industry experience ranges from global development to commercial expertise with a focus on building companies from start-ups to leaders in their categories.
Presented Expanded Preclinical Profiles of NVL-520 and NVL-655 at AACR (Free AACR Whitepaper) 2022: Nuvalent presented new preclinical data supporting the potential for broad clinical utility of NVL-520 and NVL-655 across an expanded set of ROS1 and ALK fusion partners, resistance mutations, and tumor types beyond NSCLC.
Upcoming Investor Conference Presentations
2022 H.C. Wainwright Hybrid Global Investment Conference: Dr. Porter will conduct an in-person corporate presentation during the 2022 H.C. Wainwright Hybrid Global Investment Conference on Wednesday May 25, 2022 at 4:00 p.m. ET. The conference is being held May 23-26, 2022, with in-person presentations and meetings in Miami, FL. A live webcast will be available in the Investors section of the company’s website at www.nuvalent.com and will be archived for 30 days following the presentation.
First Quarter 2022 Financial Results
Cash Position: Cash, cash equivalents and marketable securities were $272.7 million as of March 31, 2022. Nuvalent continues to expect the existing cash, cash equivalents, and marketable securities to be sufficient to fund its planned operations into 2024.
R&D Expenses: Research and development (R&D) expenses were $12.7 million for the first quarter of 2022.
G&A Expenses: General and administrative (G&A) expenses were $5.0 million for the first quarter of 2022.
Net Loss: Net loss for the first quarter of 2022 was $17.5 million, or $0.36 per share.

On May 12, 2022 Veracyte, Inc. (Nasdaq: VCYT) reported that six abstracts demonstrating the ability of its genomic and immuno-oncology diagnostic tests and technology to improve outcomes for cancer patients will be shared at the upcoming 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Veracyte, MAY 12, 2022, View Source [SID1234614395]). The meeting will take place in person and virtually from June 3-7, in Chicago.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The findings being presented at the ASCO (Free ASCO Whitepaper) annual meeting expand the performance and utility data for our clinical tests, while demonstrating the role our novel immuno-oncology offerings may play in advancing new frontiers in personalized medicine," said Marc Stapley, Veracyte’s chief executive officer. "We are especially excited to present new data focused on determining which patients with metastatic non-small cell lung cancer may benefit from immune checkpoint inhibitors – an important clinical need that is not being met with current biomarker testing."