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 Brickell Biotech, Inc. ("Brickell" or the "Company") (Nasdaq: BBI), a clinical-stage pharmaceutical company striving to transform patient lives by developing innovative and differentiated prescription therapeutics for the treatment of autoimmune, inflammatory, and other debilitating diseases, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Vical, MAY 12, 2022, View Source [SID1234614343]).

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"Last week, we announced the sale of our rights to sofpironium bromide to Botanix Pharmaceuticals, which we believe will provide an optimal pathway for sofpironium bromide gel, 15% to become a potential best-in-class treatment for millions of patients suffering from primary axillary hyperhidrosis," commented Robert Brown, Chief Executive Officer of Brickell. "Through this transaction, we have immediately started to unlock the value of sofpironium bromide while eliminating the significant investment required by Brickell through the NDA review process and commercialization. Importantly, this sale aligns with our new business strategy to develop innovative therapeutics in the immunology and inflammatory fields. The proceeds and potential future economics from this transaction allow us to directly invest additional resources to advance our pipeline of novel, potential first-in-class therapies."

Mr. Brown continued, "As we look ahead, there are several exciting company milestones planned for 2022 and beyond, which we believe will help us address the needs of several high-impact patient populations afflicted by autoimmune and inflammatory diseases, while creating shareholder value. Our plans include conducting a first-in-human Phase 1 clinical study starting in the second quarter of 2022 for our lead DYRK1A inhibitor, BBI-02, and progressing the development of our lead STING inhibitor, BBI-10, and other next-generation kinase inhibitors through early preclinical stage studies this year."

Research and Development Highlights

BBI-02: a potential first-in-class DYRK1A inhibitor for the treatment of autoimmune and inflammatory diseases

On track to initiate the Phase 1 clinical trial of BBI-02 (BBI-02-101) in Canada in the second quarter of 2022, which is a randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of BBI-02 capsules in both healthy subjects and patients with atopic dermatitis (AD).

Successfully submitted the Clinical Trial Application for BBI-02 to Health Canada and subsequently received a No Objection Letter in the first quarter of 2022, allowing the BBI-02-101 study to proceed as planned.
Part 1A of the study will be a single ascending dose (SAD) assessment of BBI-02 capsules or placebo in healthy subjects. Part 1B of the study will be a multiple ascending dose (MAD) assessment of BBI-02 capsules or placebo administered once daily for 14 days in healthy subjects. Part 2 of the study will compare BBI-02 to placebo in patients with moderate-to-severe AD over 28 days of dosing, and will include a preliminary assessment of efficacy.
Topline results from the SAD and MAD parts of the Phase 1 trial are expected to be announced by early 2023.
BBI-10: a covalent STING inhibitor for the potential treatment of autoimmune, inflammatory, and rare genetic diseases

In February 2022, Brickell acquired exclusive global rights to develop and commercialize a portfolio of novel, potent, and orally available Stimulator of Interferon Genes (STING) inhibitors from Carna Biosciences, Inc., an established Japanese drug discovery company.
Preclinical development activities for BBI-10 are underway, and the Company expects to conduct experimental characterization of the STING inhibitor library throughout 2022.
Next-Generation Kinase Inhibitors: a cutting-edge platform with the potential to produce treatments for autoimmune, inflammatory, and other debilitating diseases

Currently engaged in research to identify both brain penetrant and non-brain penetrant kinase inhibitors from the Company’s library of novel compounds, including next-generation DYRK1A inhibitors and other new chemical entities that specifically inhibit LRRK2, TTK, and CLK kinases, as potential treatments for autoimmune, inflammatory, and other debilitating diseases.
Recent Events

On May 3, 2022, Brickell signed and closed a definitive asset purchase agreement with Botanix SB Inc., a subsidiary of Botanix Pharmaceuticals Limited ("Botanix"). Under the terms of the agreement, Botanix acquired all of Brickell’s rights and assets primarily related to sofpironium bromide. In exchange, Brickell received $3 million at closing and is eligible to receive up to $6 million in potential near-term regulatory milestone payments over the next 18 months from Botanix. Brickell also is eligible to receive additional success-based regulatory and sales milestone payments of up to $168 million and tiered earnout payments ranging from high-single digits to mid-teen digits on net sales of sofpironium bromide gel. Certain of these amounts are subject to payments by Brickell to its former licensor. Brickell additionally will receive certain payments from sales by its former sublicensee, Kaken Pharmaceutical Co. ("Kaken"). Botanix will be responsible for all further research, development, and commercialization of sofpironium bromide globally. In connection with the sale of sofpironium bromide, Brickell and Botanix entered into a transition services agreement whereby Brickell will provide consulting services to Botanix as an independent contractor through submission and potential approval of the U.S. NDA for sofpironium bromide gel, 15%.

On May 3, 2022, Brickell adjourned its 2022 annual meeting of stockholders to May 17, 2022 at 10 a.m. MDT. At the time the annual meeting was adjourned, proxies had been submitted by stockholders constituting a quorum, but there were not sufficient votes to approve two of the proposals, one related to an increase in the number of authorized shares of common stock and the other to approve a reverse stock split, each of which requires approval by the holders of a majority of the outstanding shares of common stock of Brickell. Brickell continues to solicit votes from its stockholders with respect to all of the proposals for the annual meeting.

First Quarter 2022 Financial Results

The Company reported cash and cash equivalents of $17.3 million as of March 31, 2022, compared to $26.9 million as of December 31, 2021. The Company expects its cash and cash equivalents as of March 31, 2022, combined with $3.0 million in upfront fees it received from Botanix on May 3, 2022, and other expected near-term milestone payments under the agreement with Botanix, will support its operations for at least the next 12 months.

Revenue was $92.0 thousand for the first quarter of 2022, compared to $17.0 thousand for the first quarter of 2021. Revenue in both periods resulted from royalty revenue related to sales of ECCLOCK (sofpironium bromide gel, 5%) in Japan by Kaken.

Research and development expenses were $6.0 million for the first quarter of 2022, compared to $6.1 million for the first quarter of 2021. During the first quarter of 2022, Brickell incurred $3.3 million in lower clinical costs related to its U.S. Phase 3 pivotal clinical program for sofpironium bromide gel, 15%, which was completed in the fourth quarter of 2021. This decrease was almost fully offset by increases of $2.0 million in upfront costs related to Brickell’s acquisition of the STING inhibitor platform, $0.7 million in development costs related to its DYRK1A inhibitor program, and $0.4 million related to personnel and other expenses.

General and administrative expenses were $3.5 million for the first quarter of 2022, compared to $3.0 million for the first quarter of 2021. The increase was primarily due to higher compensation-related expenses, professional fees, insurance, and other miscellaneous expenses.

Brickell’s net loss was $9.4 million for the first quarter of 2022 compared to $9.0 million for the first quarter of 2021.

Conference Call and Webcast Information

Brickell’s management will host a conference call today at 4:30 p.m. EDT to discuss the financial results and recent corporate developments. The dial-in number for the conference call is 1-877-705-6003 for domestic participants and 1-201-493-6725 for international participants, with Conference ID #: 13728736. A live webcast of the conference call can be accessed at (click here) or through the Investors section of the Brickell website at View Source A replay will be available on this website shortly after conclusion of the event for approximately 90 days.

On May 12, 2022 CTI BioPharma Corp. (Nasdaq: CTIC) reported its financial results for the first quarter ended March 31, 2022 (Press release, CTI BioPharma, MAY 12, 2022, View Source [SID1234614342]).

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"With the accelerated FDA approval and U.S. commercial launch of VONJO, the first quarter was transformational for CTI and the myelofibrosis community. We are thrilled to now be delivering VONJO for patients with cytopenic myelofibrosis who have platelet counts below 50 x 109/L. Our U.S. commercial team has been in the field since early March and has delivered net product revenue of $2.3 million in less than a month, exceeding our internal expectations and establishing a great foundation for future performance," said Adam Craig, President and Chief Executive Officer of CTI BioPharma. "We are also pleased that NCCN quickly recommended VONJO for the treatment of myelofibrosis, making VONJO the only approved JAK inhibitor recommended by NCCN for these patients regardless of platelet counts."

Recent Accomplishments and Updates

FDA accelerated approval for VONJO for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L.
U.S. commercial launch in early March by fully funded sales and marketing team.
$60 million payment from DRI Healthcare Trust for the acquisition of a tiered royalty for VONJO.
VONJO included as recommended treatment in the latest National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for Myeloproliferative Neoplasms, as a first-line treatment for high-risk patients with myelofibrosis with platelet counts <50 x 109/L who are not candidates for transplant, and as a second-line treatment for lower-risk and higher-risk patients with myelofibrosis with platelet counts ≥50 x 109/L who are not candidates for transplant.
Accepted abstract at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held June 3–7, 2022, in Chicago and virtually:
Abstract Title: Risk-adjusted safety analysis of pacritinib (PAC) in patients (pts) with myelofibrosis (MF)
Abstract Number: 7058
Session Name: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant
Session Date: Saturday, June 4, 2022
Presentation Time: 8:00 – 11:00 a.m. CDT (9:00 a.m. – 12:00 p.m. ET)
Presenter: Dr. Naveen Pemmaraju
Accepted abstracts at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Congress being held June 9–12, 2022, in Vienna, Austria:
Abstract Title: Risk-adjusted safety analysis of pacritinib in patients with myelofibrosis
Abstract Number: P1068
Session Name: Poster session
Session Date: Friday, June 10, 2022
Presentation Time: 16:30 – 17:45 CEST (10:30 – 11:45 a.m. ET)
Presenter: Dr Naveen Pemmaraju
Abstract Title: Retrospective comparison of patient outcomes on pacritinib versus ruxolitinib in patients with myelofibrosis and thrombocytopenia
Abstract Number: P1069
Session Name: Poster session
Session Date: Friday, June 10, 2022
Presentation Time: 16:30 – 17:45 CEST (10:30 – 11:45 a.m. ET)
Presenter: Prof. Claire Harrison
First Quarter Financial Results

Net product sales of $2.3 million for the first quarter ended March 31, 2022 were attributable to VONJO product sales in the United States. There were no product sales for the comparable period in 2021. Our realization of future product sales will be dependent, in part, upon our commercialization efforts and the market acceptance of VONJO among physicians, patients, healthcare payers and the medical community.

Operating loss was $35.1 million and $17.1 million for the three months ended March 31, 2022 and 2021, respectively. The increase in operating loss between periods resulted primarily from increases in selling, general and administrative activities related to the commercial-launch of VONJO and the growth in our commercial infrastructure, as well as a $10.3 million milestone expense related to FDA approval of VONJO, which was included in other operating expenses for the three months ended March 31, 2022.

Net loss for the three months ended March 31, 2022 was $37.2 million, or $0.37 for basic and diluted loss per share, compared to net loss of $17.3 million, or $0.23 for basic and diluted loss per share, for the same period in 2021.

As of March 31, 2022, our cash and cash equivalents totaled $96.9 million. We expect our present financial resources, including expected cash receipts from receivables arising from historical net product sales of VONJO (but excluding any proceeds of future net product sales of VONJO), will enable us to fund our operations into the first quarter of 2023. In accordance with applicable accounting standards, our evaluation of our expected cash runway considers only relevant conditions and events that are known or reasonably knowable at the date that the financial statements are issued. As a result, our cash runway evaluation did not include VONJO sales that we may recognize in the future. We expect to include future net product sales of VONJO in our cash runway projections once we have an established history of such sales.

Conference Call and Webcast

CTI will host a conference call and webcast to review its first quarter 2022 financial results and provide an update on business activities today, May 12 at 4:30 p.m. ET. To access the live call by phone please dial (877) 735-2860 (domestic) or (602) 563-8791 (international); the conference ID is 7291915. A live audio webcast of the event may also be accessed through the "Investors" section of CTI’s website at www.ctibiopharma.com. A replay of the webcast will be available for 30 days following the event.

About VONJO (pacritinib)
Pacritinib is an oral kinase inhibitor with activity against wild type Janus Associated Kinase 2 (JAK2), mutant JAK2V617F form and FMS-like tyrosine kinase 3 (FLT3), which contribute to signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. Myelofibrosis is often associated with dysregulated JAK2 signaling. Pacritinib has higher inhibitory activity for JAK2 over other family members, JAK3 and TYK2. At clinically relevant concentrations, pacritinib does not inhibit JAK1. Pacritinib exhibits inhibitory activity against additional cellular kinases (such as CSF1R and IRAK1), the clinical relevance of which is unknown.

VONJO is indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Important VONJO Safety Information
Hemorrhage:
Serious (11%) and fatal (2%) hemorrhages have occurred in VONJO-treated patients with platelet counts <100 × 109/L. Serious (13%) and fatal (2%) hemorrhages have occurred in VONJO-treated patients with platelet counts <50 × 109/L. Grade ≥3 bleeding events (defined as requiring transfusion or invasive intervention) occurred in 15% of patients treated with VONJO compared to 7% of patients treated on the control arm. Due to hemorrhage, VONJO dose-reductions, dose interruptions, or permanent discontinuations occurred in 3%, 3%, and 5% of patients, respectively.

Avoid use of VONJO in patients with active bleeding and hold VONJO 7 days prior to any planned surgical or invasive procedures. Assess platelet counts periodically, as clinically indicated. Manage hemorrhage using treatment interruption and medical intervention.

Diarrhea:
VONJO causes diarrhea in approximately 48% of patients compared to 15% of patients treated on the control arm. The median time to resolution in VONJO-treated patients was 2 weeks. The incidence of reported diarrhea decreased over time with 41% of patients reporting diarrhea in the first 8 weeks of treatment, 15% in Weeks 8 through 16, and 8% in Weeks 16 through 24. Diarrhea resulted in treatment interruption in 3% of VONJO-treated patients. None of the VONJO-treated patients reported diarrhea that resulted in treatment discontinuation. Serious diarrhea adverse reactions occurred in 2% of patients treated with VONJO compared to no such adverse reactions in patients in the control arm.

Control pre-existing diarrhea before starting VONJO treatment. Manage diarrhea with antidiarrheal medications, fluid replacement, and dose-modification. Treat diarrhea with anti–diarrheal medications promptly at the first onset of symptoms. Interrupt or reduce VONJO dose in patients with significant diarrhea despite optimal supportive care.

Thrombocytopenia:
VONJO can cause worsening thrombocytopenia. VONJO dosing was reduced due to worsening thrombocytopenia in 2% of patients with pre–existing moderate to severe thrombocytopenia (platelet count <100 × 109/L). VONJO dosing was reduced due to worsening thrombocytopenia in 2% of patients with pre–existing severe thrombocytopenia (platelet count <50 × 109/L).

Monitor platelet count prior to VONJO treatment and as clinically indicated during treatment. Interrupt VONJO in patients with clinically significant worsening of thrombocytopenia that lasts for more than 7 days. Restart VONJO at 50% of the last given dose once the toxicity has resolved. If toxicity recurs hold VONJO. Restart VONJO at 50% of the last given dose once the toxicity has resolved.

Prolonged QT interval:
VONJO can cause prolongation of the QTc interval. QTc prolongation of >500 msec was higher in VONJO-treated patients than in patients in the control arm (1.4% vs 1%). QTc increase from baseline by 60 msec or higher was greater in VONJO-treated patients than in control arm patients (1.9% vs 1%). Adverse reactions of QTc prolongation were reported for 3.8% of VONJO-treated patients and 2% of control arm patients. No cases of torsades de pointes were reported.

Avoid use of VONJO in patients with a baseline QTc of >480 msec. Avoid use of drugs with significant potential for QTc prolongation in combination with VONJO. Correct hypokalemia prior to and during VONJO treatment. Manage QTc prolongation using VONJO interruption and electrolyte management.

Major Adverse Cardiac Events (MACE):
Another Janus associated kinase (JAK)-inhibitor has increased the risk of MACE, including cardiovascular death, myocardial infarction, and stroke (compared to those treated with TNF blockers) in patients with rheumatoid arthritis, a condition for which VONJO is not indicated.

Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with VONJO particularly in patients who are current or past smokers and patients with other cardiovascular risk factors. Patients should be informed about the symptoms of serious cardiovascular events and the steps to take if they occur.

Thrombosis:
Another JAK-inhibitor has increased the risk of thrombosis, including deep venous thrombosis, pulmonary embolism, and arterial thrombosis (compared to those treated with TNF blockers) in patients with rheumatoid arthritis, a condition for which VONJO is not indicated.

Patients with symptoms of thrombosis should be promptly evaluated and treated appropriately.

Secondary Malignancies:
Another JAK-inhibitor has increased the risk of lymphoma and other malignancies excluding non-melanoma skin cancer (NMSC) (compared to those treated with TNF blockers) in patients with rheumatoid arthritis, a condition for which VONJO is not indicated. Patients who are current or past smokers are at additional increased risk.

Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with

VONJO, particularly in patients with a known malignancy (other than a successfully treated NMSC), patients who develop a malignancy, and patients who are current or past smokers.

Risk of Infection:
Another JAK-inhibitor has increased the risk of serious infections (compared to best available therapy) in patients with myeloproliferative neoplasms. Serious bacterial, mycobacterial, fungal and viral infections may occur in patients treated with VONJO. Delay starting therapy with VONJO until active serious infections have resolved. Observe patients receiving VONJO for signs and symptoms of infection and manage promptly. Use active surveillance and prophylactic antibiotics according to clinical guidelines.

Interactions with CYP3A4 Inhibitors or Inducers:
Co-administration of VONJO with strong CYP3A4 inhibitors or inducers is contraindicated. Avoid concomitant use of VONJO with moderate CYP3A4 inhibitors or inducers.

Drug interruptions due to an adverse reaction occurred in 27% patients who received VONJO 200 mg twice daily compared to 10% of patients treated with BAT. Dosage reductions due to an adverse reaction occurred in 12% of patients who received VONJO 200 mg twice daily compared to 7% of patients treated with BAT. Permanent discontinuation due to an adverse reaction occurred in 15% of patients receiving VONJO 200 mg twice daily compared to 12% of patients treated with BAT.

Please visit View Source for full Prescribing Information and the Medication Guide.

About Myelofibrosis

Myelofibrosis is bone marrow cancer that results in formation of fibrous scar tissue and can lead to thrombocytopenia and anemia, weakness, fatigue and an enlarged spleen and liver. Within the United States, there are approximately 21,000 patients with myelofibrosis, 7,000 of which have severe thrombocytopenia (defined as blood platelet counts of less than 50 x109/L). Severe thrombocytopenia is associated with poor survival and high symptom burden and can occur as a result of disease progression or from drug toxicity with other JAK2 inhibitors, such as JAKAFI and INREBIC.

On May 12, 2022 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Vincerx Pharma, MAY 12, 2022, View Source [SID1234614341]).

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"In April, we presented positive preclinical and preliminary clinical data on PTEFb/CDK9 inhibitor VIP152 at AACR (Free AACR Whitepaper). These data, coupled with our findings presented at ASH (Free ASH Whitepaper) last year, suggest that VIP152 has the potential to provide new treatment options for patients across various MYC and MCL-1-driven tumor types," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx.

"We continue dosing patients in our VIP152 clinical program and are seeing improvements in trial enrollment following a challenging pandemic year," added Dr. Hamdy. "This June, we are excited for our poster presentation at the annual European Hematology Association (EHA) (Free EHA Whitepaper) meeting, which will show data from patients with lymphoma treated with VIP152. We remain on track to initiate Phase 2 studies of VIP152 in the second half of this year."

"Looking to the second half of this year and into 2023, we look forward to continuing to advance our preclinical bioconjugation platform – a diverse, modular platform of linkers and payloads that can be conjugated with antibodies and small molecules to create novel targeted therapeutics for a broad range of solid tumors and hematologic malignancies. We remain on track to file an IND in the second half of this year for VIP236, a small molecule drug conjugate (SMDC) engineered to bind an αvß3 integrin adhesion molecule that is abundantly expressed in advanced metastatic solid tumors. αvß3 is a hallmark of aggressive cancers and poor prognosis. We also remain on-target to file INDs in the second half of 2023 for our two initial antibody drug conjugates (ADCs), VIP943, an anti-IL3RA-KSPi, and VIP924, an anti-CXCR5-KSPi, both with a CellTrapper moiety. From a financial perspective, our current cash resources position us to continue to pursue our upcoming regulatory and clinical milestones," concluded Dr. Hamdy.

RECENT CORPORATE HIGHLIGHTS
VIP152

Abstract accepted for poster presentation at the upcoming European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting, titled "VIP152 is a novel CDK9 inhibitor with improved selectivity, target modulation, and cardiac safety in patients with lymphoma."
Presenting author: Melanie Frigault, PhD
Abstract number: P1269
Session date and time: Friday, June 10, 2022; 16:30-17:45 CEST
Presented poster, "VIP152, a selective CDK9 inhibitor, demonstrates sensitivity in gynecologic cell lines that are cisplatin sensitive or resistant and delivers in vivo antitumor efficacy," at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022.
Bioconjugation Platform

Invited talk on the Company’s modular assets that can build innovative and high-potency ADCs titled, "ADCs with KSP-Inhibitor Payloads and a Tailored Design of Linker and Metabolite Profile," at the Festival of Biologics meeting March 2022.

Continue to advance our next generation modular bioconjugation platform, comprised of a first-in-class SMDC for solid tumors (VIP236) and two best-in-class preclinical-stage assets for hematologic malignancies (VIP943 and VIP924).
VIP236 consists of an avß3 integrin binder and a linker that is cleavable by neutrophil elastase. The payload is an optimized camptothecin derivative designed for high cellular permeability and low efflux. IND filing in solid tumors expected in 2H 2022.
VIP943 (anti-IL3RA) and VIP924 (anti-CXCR5) are two antibodies linked to a KSP inhibitor – a novel payload class in ADCs. These ADCs also have a stable linker specifically cleaved by legumain, a tumor associated protease. The payload (i.e., KSP inhibitor) is modified to be trapped in the cell by the CellTrapper moiety. Manufacturing is underway and IND filings expected in 2H 2023.
FIRST QUARTER FINANCIAL RESULTS

Vincerx Pharma had $96.5 million in cash as of March 31, 2022, as compared to $111.5 million as of December 31, 2021. Based on its current business plans and assumptions, Vincerx believes its available cash will be sufficient to meet its operating requirements through 2023.
Research and development (R&D) expenses for the quarter ended March 31, 2022 were $16.0 million, as compared to $4.8 million for the same period in 2021. The increase was primarily related to increases in manufacturing services of approximately $4.0 million, including the initiation of manufacturing associated with our ADC program, new employee salaries of approximately $2.5 million, third party research and preclinical work of approximately $2.0 million, clinical services of approximately $1.4 million and stock-based compensation of approximately $0.8 million.
General and administrative (G&A) expenses for the quarter ended March 31, 2022 were $5.6 million, as compared to $4.8 million for the same period in 2021. The increase was primarily related to new employee salaries and increases in legal (patent protection and filings), accounting and other professional services in support of our operations as a public company.
For the quarter ended March 31, 2022, Vincerx reported a net loss of $16.4 million, or $0.79 per share. For the quarter ended March 31, 2021, Vincerx reported a net loss of $6.3 million, or $0.46 per share.

On May 12, 2022 2seventy bio, Inc. (Nasdaq: TSVT), a leading immuno-oncology cell therapy company, reported financial results and recent highlights for the first quarter ended March 31, 2022 (Press release, 2seventy bio, MAY 12, 2022, View Source [SID1234614340]).

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"2seventy bio has started 2022 in a strong position, and we’ve already begun to execute on some key clinical milestones," said Nick Leschly, chief kairos officer. "This quarter, we enrolled the first patients in clinical studies of SC-DARIC33 and bbT369. We look forward to continuing to enroll patients in these studies and providing an update at the appropriate time. In addition, we continue to focus on other foundational elements of 2seventy bio: growth of ABECMA and a sound financial position. Despite a challenging external environment, I’m pleased with the steady progress on both fronts with continued demand for ABECMA as well securing additional capital in March that we anticipate will provide cash runway into 2025. We’re proud of the momentum we’ve established as a team at 2seventy bio because we know that every day matters as we focus on delivering more time to patients with cancer and their families."

COMMERCIAL PROGRESS
Bristol Myers Squibb reported total U.S. ABECMA (idecabtagene vicleucel; ide-cel) first quarter revenues of $56 million, consistent with our 2022 plan. 2seventy bio and Bristol Myers Squibb share equally in all profits and losses related to developing, manufacturing and commercializing ABECMA in the U.S. In 2022, 2seventy anticipates total U.S. ABECMA revenues of $250-$300 million and we are continuing to track to the high end of the range bolstered by continued high demand for a proven treatment and increasing manufacturing capacity.

Demand for ABECMA is expected to continue to fully utilize the expanding manufacturing capacity throughout 2022 and 2023.

RECENT HIGHLIGHTS

SC-DARIC33 FIRST PATIENT ENROLLED – Last quarter, the first patient with acute myeloid leukemia (AML) was enrolled in PLAT-08, a phase 1 study of SC-DARIC33 led by Seattle Children’s Therapeutics in relapsed or refractory pediatric and young adult AML. This is the first-in-human application of 2seventy bio’s proprietary DARIC T cell platform.
BBT369 FIRST PATIENT ENROLLED – Last quarter, the first patient with B cell non-Hodgkin lymphoma (B-NHL) was enrolled in CRC-403, a phase 1/2 study of bbT369 in patients with relapsed and/or refractory B-NHL. This study serves as a safety and proof-of-concept assessment of 2seventy bio’s proprietary megaTAL gene editing platform, dual-targeting strategies and split co-stimulation signaling technology.
bbT369 PRECLINICAL DATA AT AACR (Free AACR Whitepaper) – New preclinical data on bbT369 was presented in a poster session (poster #581) at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 in New Orleans, LA on Sunday, April 10. The data presented at AACR (Free AACR Whitepaper) show the anti-lymphoma activity of bbT369 observed to date and suggest that, as intended in the design, bbT369 has the potential to overcome failure modes of anti-CD19 CAR therapies.
ASCO ABSTRACTS ACCEPTED– In April 2022, the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) released the abstract titles for its 2022 Annual Meeting. 2seventy bio will present updates from its portfolio of oncology cell therapies at the meeting, including a correlative analysis, in partnership with Bristol Myers Squibb, defining patient profiles associated with manufacturing and clinical endpoints in patients treated with ide-cel, and a trial in progress poster on CRB-403, a phase 1/2 study on bbT369.
Poster Discussion [#8021]: Correlative analysis to define patient profiles associated with manufacturing and clinical endpoints in relapsed/refractory multiple myeloma (RRMM) patients treated with idecabtagene vicleucel (ide-cel; bb2121), an anti-BCMA CAR T cell therapy.
Presenting Author: Julie Rytlewski, PhD, Bristol Myers Squibb
Date/Time: Saturday, June 4, 2022, 5:30 PM – 7:00 PM ET
Poster [#TPS7580]: CRC-403: A phase 1/2 study of bbT369, a dual targeting CAR T-cell drug product with a gene edit, in relapsed and/or refractory B-cell non-Hodgkin lymphoma (NHL).
Presenting Author: Frederick L. Locke, MD, H. Lee Moffitt Cancer Center & Research Institute
Date/Time: Saturday, June 4, 2022, 9:00 AM – 12:00 PM ET
UPCOMING ANTICIPATED MILESTONES

ABECMA

Anticipated $250-300 million total U.S. commercial revenue in 2022; profits and losses shared with Bristol Myers Squibb
Increasing manufacturing capacity expected over 2022 and 2023
KarMMa-2 study in high-risk multiple myeloma proof-of-concept data in 2022
KarMMa-3 study in 3L+ registrational data in 2023 with potential FDA approval in 2023-2024
Pipeline

Initial assessment of feasibility of bbT369 drug product manufacturing and patient safety in 2H 2022
Initial assessment of feasibility of SC-DARIC33 drug product manufacturing and drug regulated anti-CD33 activity in 2H 2022
SELECT FIRST QUARTER 2022 FINANCIAL RESULTS

Bristol Myers Squibb reported total U.S. revenues of $56 million for ABECMA for the three months ended March 31, 2022. 2seventy bio and Bristol Myers Squibb share equally in all profits and losses related to development, manufacturing and commercializing ABECMA in the U.S. We reported share of collaboration loss of $5.4 million for the three months ended March 31, 2022, which includes our share of gross profit/loss less costs associated with the commercialization of ABECMA in the U.S. The collaboration reported a loss this quarter due to continued investment in manufacturing scale-up and commercialization.
Total revenues were $8.4 million for the three months ended March 31, 2022, compared to $11.9 million for the three months ended March 31, 2021. The decrease for the three-month period was primarily driven by a decrease in royalty and other revenue as a result of the termination of the Company’s license agreement with Novartis in March 2021.
Research and development expenses were $69.2 million for the three months ended March 31, 2022, compared to $77.6 million for the three months ended March 31, 2021. The decrease for the period was primarily driven by decreased collaboration research costs, which represent the Company’s share of research and development costs under the collaboration with Bristol Myers Squibb.
Selling, general and administrative expenses were $23.9 million for the three months ended March 31, 2022, compared to $24.6 million for the three months ended March 31, 2021. The slight decrease was primarily driven by a decrease in employee compensation expense. In 2021, the Company recorded higher stock-based compensation and bonus expense related to a retention plan that was enacted during the separation of 2seventy bio from bluebird bio. The retention plan was completed at the end of 2021.
Net loss was $85.7 million for the three months March 31, 2022, compared to $87.2 million for the three months ended March 31, 2021.
2seventy bio ended the first quarter of 2022 with cash, cash equivalents and marketable securities of $452.5 million, including net proceeds from our March 2021 private placement of $165.7 million, after deducting placement agent fees and other offering expenses payable by the Company.