On May 13, 2022 POINT Biopharma Global Inc. (NASDAQ: PNT) (the "Company" or "POINT"), a company accelerating the discovery, development, and global access to life-changing radiopharmaceuticals, reported financial results for the first quarter ended March 31, 2022 and provided a business update (Press release, Point Biopharma, MAY 13, 2022, View Source [SID1234614535]).

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"There were two very significant milestones for POINT in the first quarter of 2022," said Dr. Joe McCann, CEO of POINT Biopharma. "First, we began supplying doses for the SPLASH trial out of our company-owned manufacturing facility. Second, we reported very encouraging dosimetry data from our Phase 3 SPLASH trial lead-in phase which is evaluating PNT2002 in pre-chemotherapy metastatic castration-resistant prostate cancer (mCRPC). The data positions us well as we continue with the randomization phase of SPLASH. The reason these milestones are very significant is they place POINT in the extremely small number of companies which have successfully manufactured their own radioligands, for their own Phase 3 trial. Combined with the upcoming advancement of our pan-cancer FAP-targeting program PNT2004 into the clinic this summer, POINT’s radioligand platform has the potential to be transformative for cancer care in a variety of indications with high unmet need."

Recent Developments and Upcoming Milestones

Pipeline Updates

PNT2002: 177Lu-based PSMA targeted radiopharmaceutical

In February 2022, the Company announced publication of the dosimetry results from the lead-in cohort of the Phase III SPLASH trial evaluating PNT2002 for the treatment of mCRPC at the 2022 Society of Nuclear Medicine and Molecular Imaging (SNMMI) Mid-Winter & American College of Nuclear Medicine (ACNM) Annual Meeting. The findings presented by Dr. Jean-Mathieu Beauregard concluded that "PNT2002 has a favorable and safe dosimetry profile in the patient population and dose regimen being studied." A link to the Abstract can be found in the Investors section of the company’s website found here: View Source

In March 2022, the Company hosted a virtual education event titled "Introduction to Dosimetry for Radiopharmaceuticals" led by a key opinion leader in the field, Dr. Ana Kiess, M.D., Ph.D. A replay of the event is accessible at View Source

In April 2022, the Company dosed its first European Union patient in the SPLASH trial. The SPLASH trial is currently enrolling patients across 42 sites in North America and Europe, and site activations all in jurisdictions remain ongoing to expedite accrual. The Company continues to expect to report top line data from SPLASH mid-2023.

PNT2004: fibroblast activation protein-alpha (FAP-alpha) targeted radiopharmaceutical

The Company filed a clinical trial application (CTA) with Health Canada at the end of the first quarter of 2022 for PNT6555, the lead of the pan-cancer PNT2004 fibroblast activation protein-alpha (FAP-alpha) targeted program. The clinical trial for PNT2004 is expected to first initiate in Canada in summer 2022.

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Manufacturing & Supply Chain Updates:

The Company’s Indianapolis manufacturing facility began supplying n.c.a. 177Lu PNT2002 to its SPLASH clinical trial in January 2022. The approximately 81,000 sq ft facility is licensed for alpha and beta emitting isotopes, and contains dedicated space for commercial-scale manufacturing. A virtual tour of the facility is accessible at View Source

In January 2022, the Company announced that it will receive 225Ac in 2022 from the U.S. Department of Energy Isotope Program to support its early-stage pipeline. The Company remains on track to launch its in-house n.c.a. 177Lu manufacturing program in 2023.

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First Quarter 2022 Financial Results

Cash and Cash Equivalents: As of March 31, 2022, POINT had approximately $227.4 million in cash and cash equivalents, which is anticipated to fund operations into the first quarter of 2024.

Net Loss: Net loss was $16.4 million, or $0.18 net loss per share, for the quarter ended March 31, 2022, as compared to a net loss of $5.8 million, or $0.10 net loss per share, for the same period in 2021.

Research and Development Expenses: Research and development expenses were $12.5 million for the quarter ended March 31, 2022, as compared to $4.3 million for the same period in 2021.

General and Administrative Expenses: General and administrative expenses were $3.8 million for the quarter ended March 31, 2022, as compared to $1.5 million for the same period in 2021.

On May 12, 2022 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a late clinical stage biopharmaceutical company focused on helping patients fight cancer with oncolytic viral immunotherapies, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Candel Therapeutics, MAY 12, 2022, View Source [SID1234614282]).

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"2022 is off to a great start as we continue to advance our late-stage pipeline of novel oncolytic viral immunotherapies for cancer," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "Today, we are well positioned for major catalysts over the next 12 months, namely multiple clinical data readouts across our product candidates and advancement of our pipeline of promising therapeutics with potential to treat various solid tumors including lung, brain, pancreatic and prostate cancer. We look forward to delivering on our mission to develop oncolytic viral immunotherapies aimed at tipping the balance in favor of the patient’s immune system to fight cancer."

First Quarter 2022 & Recent Highlights

•Enhanced executive leadership with the promotion of Francesca Barone, MD, PhD to Chief Scientific Officer and appointment of Seshu Tyagarajan, PhD, RAC as Chief Technical and Development Officer.
•Strengthened cash position with a $20.0 million non-dilutive debt financing with Silicon Valley Bank (SVB) in February 2022.

Key Upcoming Milestones

•To present initial data from an ongoing Phase 2 clinical trial of CAN-2409 and valacyclovir combined with PD-1 or PD-L1 targeting agents in patients with stage III/IV non-small cell lung cancer at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting to be held in-person June 3-7, 2022 at the McCormick Place Convention Center in Chicago, Illinois.
•In the fourth quarter of 2022, the Company expects to present data from two high grade glioma clinical trials – a Phase 1b clinical trial of CAN-2409 in combination with

nivolumab (Opdivo) combined with standard of care first line treatment and a Phase 1 clinical trial of CAN-3110 in recurrent high-grade glioma.

Financial Results for the Quarter Ended March 31, 2022

Cash Position: Cash and cash equivalents as of March 31, 2022 were $94.3 million compared to $82.6 million as of December 31, 2021. The net increase was due to receipt of $20.0 million from a term loan with SVB and the use of $8.0 million in cash to fund operating activities. Based on current plans and assumptions, the Company expects that its existing cash and cash equivalents will be sufficient to fund its operations into the fourth quarter of 2023.

Research and Development Expenses: Research and development expenses were $5.4 million for the three-month period ended March 31, 2022 compared to $2.8 million for the comparable period in 2021. The increase was primarily due to increased personnel-related costs, including stock-based compensation, for additional headcount to support the ongoing clinical trials for Candel’s product candidates and $1.0 million of severance in the first quarter of 2022, as well as increased clinical development costs related to our clinical trial sites and the cost of treatment and follow-up on patients. Excluding stock-based compensation expense of $142,000 for the three-month period ended March 31, 2022, research and development expenses for the three-month period ended March 31, 2022 were $5.3 million.

General and Administrative Expenses: General and administrative expenses were $3.6 million for the three-month period ended March 31, 2022 compared to $1.9 million for the comparable period in 2021. The increase was primarily due to increased insurance costs, personnel-related costs including stock-based compensation for additional headcount required to support the growth of the Company and operate as a public company, and professional and consulting fees associated with public relations consultants, legal firms, and accounting firms associated with operating as a public company. Excluding stock-based compensation expense of $350,000 for the three-month period ended March 31, 2022, general and administrative expenses for the three-month period ended March 31, 2022 were $3.3 million.

Total Operating Expenses: Total operating expenses were $9.0 million for the three-month period ended March 31, 2022 compared to $4.7 million for the comparable period in 2021.The increase was primarily due to increased personnel-related costs including stock-based compensation for additional headcount required to support the growth of the Company and to operate as a public company, severance costs, insurance costs, and professional and consulting fees associated with operating as a public company. Excluding stock-based compensation expense of $492,000 for the three-month period ended March 31, 2022, total operating expenses for the three-month period ended March 31, 2022, were $8.5 million.

Net Loss: Net loss was $874,000 for the three-month period ended March 31, 2022 compared to a net loss of $4.5 million for the comparable period in 2021. The net loss for the three-month period ended March 31, 2022 includes a non-cash credit of $8.3 million for the change in the fair value of the Company’s warrant liability and stock-based compensation expense of $492,000. Excluding the non-cash credit for the change in the warrant liability and the charge for stock-based compensation, the net loss for the three-month period ended March 31, 2022 was $8.7 million.

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.

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"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]).

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"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]).

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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.