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 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported financial results for the first quarter of 2022 (Press release, CASI Pharmaceuticals, MAY 12, 2022, View Source [SID1234614360]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "We are pleased to report $9.0 million in EVOMELA sales revenue for the first quarter of 2022. This is an increase of 58% compared to the same period last year. Our first commercial product, EVOMELA has been instrumental in treating patients with multiple myeloma prior to stem cell transplantation in China. We have achieved our goal for full-year 2021 EVOMELA revenue to reach 100% growth. Our achievements in 2021 provide a great foundation that we plan to build upon throughout 2022 and beyond."

Dr. He continued, "Our strategic focus in 2022 will continue to advance the development and commercialization of the portfolio. Through our partner Juventas, the CNCT-19 NDA submission to the National Medical Products Administration (NMPA) is on track, and we are in preparation for the anticipated launch in China; We expect the start of the BI-1206 phase I trial in China; We expect CB-5339 to receive Clinical Trial Application approval from the NMPA during 2022; Meanwhile, our CID-103’s Phase I study continues. We are excited by our momentum and will continue to execute on several key milestones across our broad portfolio in the quarters ahead."

First Quarter 2022 Financial Highlights

Revenues consist primarily of product sales of EVOMELA. Revenue was $9.0 million for the three months ended March 31, 2022, compared to $5.7 million for the three months ended March 31, 2021.
Costs of revenues were $3.8 million for the three months ended March 31, 2022, compared to $2.4 million for the three months ended March 31, 2021, which included royalty payment of $1.8 million and $1.1 million, respectively.
Research and development expenses for the three months ended March 31, 2022, were $4.0 million, compared with $5.3 million for the three months ended March 31, 2021.
General and administrative expenses for the three months ended March 31, 2022, were $5.3 million, compared with $5.5 million for the three months ended March 31, 2021.
Selling and marketing expenses for the three months ended March 31, 2022, were $3.3 million, compared with $2.7 million for the three months ended March 31, 2021.
As of March 31, 2022, CASI had cash and cash equivalent of $29.3 million.
Further information regarding the Company, including its Quarterly Report on Form 10-Q for the quarter ended March 31, 2022, can be found at www.casipharmaceuticals.com.

Conference Call

The conference call can be accessed by dialing 1-866-218-2402 (U.S.) or 1-412-902-6605 (international) and ask to be joined into the CASI Pharmaceuticals call to listen to the live conference call. Confirmation #: 10165613

This call will be recorded and available for replay by dialing 1-877-344-7529 (U.S.) or 1-412-317-0088 (international) and enter 2360248 to access the replay.

On May 12, 2022 vTv Therapeutics Inc. (Nasdaq:VTVT) reported financial results for the first quarter ended March 31, 2022, and provided an update on the progress of its clinical programs (Press release, vTv Therapeutics, MAY 12, 2022, View Source [SID1234614377]).

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Recent Achievements and Outlook

Corporate

Strategic Focus. We are prioritizing the development of our lead program TTP399, a novel, oral liver selective glucokinase activator, as a potential treatment for patients with type 1 diabetes ("T1D"), as well as continuing to support our currently partnered programs. Given the strategic focus on these programs, we have paused our development activities in the United States on HPP737 while we evaluate strategic options for it. As part of this planned strategic focus, the Company has reduced its workforce. We are actively seeking to raise capital through licensing TTP399 in regions outside of North America and Europe and are also actively seeking licensing deals for HPP737 and other assets. We are currently in active discussions with respect to financing, partnering, and licensing transactions for the further development of TTP399.
Type 1 Diabetes

Mechanistic Study of Ketoacidosis with TTP399. In October 2021, we announced positive results from the Mechanistic study indicating no increased risk of ketoacidosis with TTP399 during acute insulin withdrawal in patients with T1D. Patients with type 1 diabetes taking TTP399 experienced no increase in ketone levels relative to placebo during a period of acute insulin withdrawal, indicating that treatment with TTP399 presents no increased risk of ketoacidosis. In addition, patients taking TTP399 had improved fasting plasma glucose levels and experienced fewer hypoglycemic events relative to those taking placebo, consistent and supportive of the previously announced phase 2 Simplici-T1 Study results. Full study results will be published in the Diabetes Obesity and Metabolism journal in conjunction with the 82nd American Diabetes Association Scientific Sessions on June 6th, 2022.

Pivotal Study Planning. The Company is planning two pivotal, placebo-controlled clinical trials of TTP399 in subjects withT1D and has engaged with the Food and Drug Administration ("FDA") on the optimal clinical trial designs for these studies. The studies will recruit a total of approximately 1000 patients and at least one of the studies will be one year of treatment. The FDA and the company have agreed on the primary endpoint for the studies as the difference between placebo and TTP399-treated group in number of hypoglycemia events. These pivotal studies are expected to start in 3Q 2022.
First Quarter 2022 Financial Results

Cash Position: The Company’s cash position as of March 31, 2022, was $12.1 million compared to $13.4 million as of December 31, 2021.

Revenue: Revenue in first quarter of 2022 was $2.0 million and relates to an increase in the transaction price for a license performance obligation, that was fully recognized due to the satisfaction of a development milestone under the amended license agreement with Huadong. The revenue for the fourth quarter of 2021 was immaterial.

R&D Expenses: Research and development expenses were $3.1 million and $5.4 million in each of the three months ended March 31, 2022 and December 31, 2021, respectively. The changes are attributable to (i) decreases of $2.0 million for a license payment to Novo Nordisk for the completion of TTP399 phase 2 studies in Q4 2021, (ii) decreased severance costs of $0.7 million and payroll costs of $0.1 million in connection with the Company’s restructuring plan that occurred in Q4 2021, (iii) decreased spending of $0.5 million related to the multiple ascending dose study for HPP737 offset by (iv) increases of $1.3 million due to manufacturing and analytical work related to chemistry manufacturing and control "CMC" for pivotal TTP399 studies, and the progression of TTP399 toxicology studies in Q1 2022.

G&A Expenses: General and administrative expenses were consistent between periods at $5.3 million and $5.7 million for each of the three months ended March 31, 2022, and December 31, 2021. However, individual changes in the quarters are attributable to (i) lower payroll costs of $0.4 million and lower severance costs of $0.7 million due to the Company’s restructuring plan that occurred in December 2021 and separation agreement with the Company’s former CEO in Q1 2022, (ii) lower shared-based expense of $0.6 million due to the modification of awards related to the retirement and separation agreements with several key employees that occurred in Q4 2021, offset by (iii) higher other G&A operating costs of $0.2 million and (iv) increases of $1.1 million in legal expense.

Other Income/(Expense): Other expense for the three months ended March 31, 2022, was $2.7 million and was driven by an unrealized loss related to the Company’s investment in Reneo Pharmaceuticals, Inc. ("Reneo"), as well as gains related to a reduction in the fair value of the outstanding warrants to purchase shares of our own stock issued to a related party ("Related Party Warrants"). Other income for the three months ended December 31, 2021, was $1.6 million and was driven by changes in the fair value of our investment in Reneo, as well as the gains related to a reduction in fair value of the Related Party Warrants.
Net Loss Before Non-Controlling Interest: Net loss before non-controlling interest was $9.4 million for the first quarter of 2022 compared to net loss before non-controlling interest of $9.5 million for the fourth quarter of 2021. The decrease in net loss before Non-Controlling Interest was attributable to (i) increases in other expense of $4.3 million driven by changes in the fair value of our investment in Reneo, as well as the gains related to a reduction in the fair value of the outstanding warrants to purchase shares of our own stock issued to a related party, offset by (ii) lower R&D expenses of $2.3 million, and (iii) higher revenue of $2.0 million due to an increase in the transaction price for a license performance obligation, that was fully recognized due to the satisfaction of a development milestone under the amended license agreement with Huadong.
Net Loss Per Share: Diluted net loss per share was ($0.10) for the three months ended March 31, 2022 compared to diluted net loss per share of ($0.11) for the three months ended December 31, 2021, based on weighted-average diluted shares of 66.9 million and 66.8 million for the three-month periods ended March 31, 2022 and December 31, 2021, respectively.

On May 12, 2022 Purple Biotech Ltd. ("Purple Biotech", or the "Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class, effective and durable therapies by overcoming tumor immune evasion and drug resistance, reported financial results for the first quarter ended March 31, 2022 (Press release, Purple Biotech, MAY 12, 2022, View Source [SID1234614394]).

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"We were focused in the clinical advancement of our promising first in class oncology drug candidates during this quarter," said Gil Efron, President and Chief Financial Officer of Purple Biotech. "For NT219, we completed enrollment in the fourth dose level of the monotherapy arm of the ongoing Phase 1/2 clinical trial and will soon start the last dose level planned in the dose escalation part of the monotherapy arm. For CM24, we will soon initiate the expansion arms of the study. We had $42.2 million in cash, cash equivalent, short and long-term deposits at the end of the first quarter this year and we are well positioned to execute our plans."

Financial Results for the Three Months Ended March 31, 2022

Research and Development Expenses were $6.0 million, an increase of $1.1 million, or 22%, compared to $4.9 million in the same period of 2021. The increase was mainly due to CMC expenses in support of our clinical studies.

Sales, General and Administrative Expenses were $1.4 million, compared to $1.7 million in the same period of 2021, a decrease of $0.3 million. The decrease was mainly due to a decrease in employee equity-based compensation (ESOP) costs.

Operating Loss from continuing operations was $7.3 million, an increase of $0.8 million, or 12%, compared to $6.5 million in the same period of 2021.

On a non-IFRS basis (as reconciled below), adjusted operating loss was $7.0 million, an increase of $1.2 million, compared to $5.8 million in the same period of 2021, mainly due to a decrease in Selling, General and Administrative expenses offset by an increase in R&D expenses.

Net Loss for the first three months ended March 31, 2022 was $7.3 million, or $0.41 per basic and diluted ADS, compared to a net loss of $6.6 million, or $0.38 per basic and diluted ADS, in the same period of 2021. The increase in net loss was mainly due to an increase of $0.8 million in operating expenses. Adjusted net loss for the first three months ended March 31, 2022 was $7.0 million, an increase from $5.8 million in the first three months ended March 31, 2021.

During the three months ended March 31, 2022, the Company sold, under the Open Market Sale Agreementsm with Jefferies LLC, approximately 59 thousand ADSs, at an average price of $3.932 per ADS. Net proceeds to the Company, were approximately $0.22 million, net of issuance expenses.

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 that results from the follicular lymphoma ("FL") cohort of the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T cell therapy, in patients with relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHLs") and chronic lymphocytic leukemia ("CLL") were selected for an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress ("EHA2022") taking place June 9-12, 2022, both virtually and in Vienna, Austria (Press release, Mustang Bio, MAY 12, 2022, View Source [SID1234614413]).

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The on-site presentation by Mazyar Shadman, M.D., M.P.H., Associate Professor and physician at Fred Hutchinson Cancer Center ("Fred Hutch") and University of Washington will provide updated data on patients with FL beyond what is available in the abstract published today on the EHA (Free EHA Whitepaper)2022 website and what was presented at the recent Tandem meetings. MB-106 is being developed in a collaboration between Mustang and Fred Hutch.

"Acceptance for oral presentation at major international meetings such as EHA (Free EHA Whitepaper)2022 is a prestigious accomplishment, and we’re pleased that the Scientific Program Committee has granted Dr. Shadman this highly visible opportunity to present Fred Hutch’s compelling data on patients with relapsed or refractory follicular lymphoma in the ongoing Phase 1/2 clinical trial of MB-106," said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang. "Furthermore, as we present our data to more investigators at peer-reviewed meetings, we are encouraged by the enthusiasm of these investigators for the durability of the responses and for the expansion of enrollment at Fred Hutch from CAR T naïve follicular lymphoma patients to patients previously treated with CAR Ts and to patients with other CD20-positive histologies such as diffuse large B cell lymphoma, Waldenstrom macroglobulinemia and CLL. Finally, as Mustang continues to advance our CD20-targeted CAR T cell therapy program, we look forward to the planned dosing of 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 under Mustang’s IND this quarter."

Details of the presentation are as follows:

Title: Efficacy and Safety of a Third Generation CD20 CART (MB-106) for Treatment of Relapsed/Refractory Follicular Lymphoma (FL)
Session: Indolent and mantle-cell lymphoma
Session Date and Time: Saturday, June 11, 11:30 am – 12:45 pm Central European Time
Session room: Hall C1
Abstract Code: S207

For more information about EHA (Free EHA Whitepaper)2022, please visit: View Source

Scientists at Fred Hutch played a role in developing these discoveries, and Fred Hutch and certain of its scientists may benefit financially from this work in the future.

About MB-106 (CD20-targeted autologous CAR T Cell Therapy)
CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustang’s research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients’ cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody, and MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch will be used to transduce patients’ cells to create the MB-106 drug product produced at Mustang Bio’s Worcester, MA, cell processing facility for administration in the planned multicenter Phase 1/2 clinical trial to be initiated shortly under Mustang Bio’s IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on the trial can be found at View Source using the identifier NCT03277729.