Histogen Reports First Quarter 2022 Financial Results and Provides Business Update

On May 12, 2022 Histogen Inc. (NASDAQ: HSTO), a clinical-stage therapeutics company focused on developing both restorative therapeutics and pan-caspase and caspase selective inhibitors focused on treatments for infectious and inflammatory diseases, reported financial results for the first quarter ended March 31, 2022 and provided an update on its clinical pipeline and other corporate developments (Press release, Conatus Pharmaceuticals, MAY 12, 2022, View Source [SID1234614478]).

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"We continue to focus on clinical execution of HST-003 in knee cartilage repair, IND enabling activities for HST-004 in spinal disk regeneration, exploration of testing emricasan in animal studies for methicillin resistant staphylococcus aureus infections ("MRSA"), and evaluating our caspase-1 inhibitors that impact the inflammasome pathway," said Steven J. Mento, Ph.D., Interim President and Chief Executive Officer.

Highlights from the First Quarter 2022 and Business Updates

HST-003 – Our Phase 1/2 clinical study of HST-003 to evaluate the safety and efficacy of human extracellular matrix (hECM) implanted within microfracture interstices and the cartilage defect in the knee to regenerate hyaline cartilage in combination with a microfracture procedure is on-going. In the first quarter of 2022, we added three additional clinical sites to help supplement enrollment of patients due to the recruitment challenges we have experienced related to both the specific nature of the study inclusion criteria and the impact of COVID-19 on the elective surgery environment. We continue to anticipate top line results in the first half of 2023, assuming we complete enrollment in the fourth quarter of 2022.

HST-004 – Our initial preclinical research has shown that HST-004 stimulates stem cells from the spinal disc to proliferate and secrete aggrecan and collagen II, regenerate normal matrix and cell tissue structure and restores disc height. HST-004 was also shown to reduce inflammation and protease activity and upregulate aggrecan production in an ex vivo spinal disc model. We have initiated IND enabling activities for HST-004 and anticipate filing IND for HST-004 in the second half of 2023.

Emricasan COVID-19 Amerimmune Collaboration Agreement – On March 3, 2022, we filed our demand for arbitration as we believe that Amerimmune has failed to undertake commercially reasonable efforts toward conducting and completing the Phase 2 study as required by the Collaborative Development and Commercialization Agreement that we previously entered into with Amerimmune in October of 2020.

Emricasan MRSA – We continue to make progress on exploring the feasibility of testing emricasan in animal studies for MRSA. We expect to complete our feasibility assessment in the second half of 2022 and, subject to the outcome of the arbitration with Amerimmune, explore further development of emricasan for MRSA.

$3.75 Million Allergan Payment – In March 2022, we received a one-time $3.75 million payment from Allergan pursuant to execution of a letter agreement entered into on March 18, 2022. The payment represents a full and final satisfaction of all monies due to the Company pursuant to the Allergan License Agreement.

First Quarter Ended March 31, 2022 Financial Highlights

Product, License, and Grant Revenues

For the three months ended March 31, 2022 and 2021, we recognized product revenues of $0 and $0.3 million, respectively. The revenue for the first quarter of 2021 was related to the additional supply of CCM to Allergan. As of March 31, 2021, all obligations of the Company related to the additional supply of CCM to Allergan under the Allergan Agreements have been completed.

For the three months ended March 31, 2022 and 2021, we recognized license revenue of $3.8 million and $12 thousand, respectively. The increase in the current period is due to a one-time payment of $3.8 million received in March 2022 as consideration for execution of the Allergan letter agreement.

For the three months ended March 31, 2022 and 2021, we recognized grant revenue of $0 and $0.1 million, respectively. The related revenue is associated with a research and development grant awarded to the Company from the NSF. As of March 31, 2021, all work required by the Company under the grant has been completed.

Cost of revenues for the three months ended March 31, 2022 and 2021, we recognized $0 and $0.2 million, respectively, for cost of product sold to Allergan under the Allergan Agreements.

Research and development expenses for the three months ended March 31, 2022 and 2021 were $1.9 million and $2.1 million, respectively. The decrease of $0.2 million was primarily due to decreases in development costs of our clinical and pre-clinical product candidates and personnel related expenses, partially offset by facility rent increases.

General and administrative expenses for the three months ended March 31, 2022 and 2021 were $2.5 million and $2.3 million, respectively. The increase of $0.2 million was primarily due to increases in royalty expenses and legal fees, offset by reductions in personnel related expenses.

Cash and cash equivalents as of March 31, 2022 were $17.8 million. Histogen believes that its existing cash and cash equivalents and cash inflow from operations will be sufficient to meet Histogen’s anticipated cash needs into the third quarter of 2023.

Fortress Biotech Reports First Quarter 2022 Financial Results and Recent Corporate Highlights

On May 12, 2022 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on efficiently acquiring, developing and commercializing or monetizing promising therapeutic products and product candidates, reported financial results and recent corporate highlights for the first quarter ended March 31, 2022 (Press release, Fortress Biotech, MAY 12, 2022, View Source [SID1234614477]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "Together with our subsidiaries and partner companies, Fortress had an exciting start to the year with the acquisition and commercial launch of two dermatology products, Amzeeq and Zilxi, bringing our total number of marketed prescription products to nine. Fortress also has a growing portfolio of 30 product candidates across our partner companies, including 20 separate clinical programs in 30 ongoing clinical trials. Four product candidates are in seven1 ongoing pivotal clinical trials. We were pleased to announce positive topline results from the registration-enabling study of cosibelimab in metastatic cutaneous squamous cell carcinoma ("cSCC") in January 2022. Throughout the remainder of 2022 we anticipate multiple key regulatory and clinical inflection points, such as the submission of a Biologics License Application ("BLA") to the U.S. Food and Drug Administration ("FDA") for cosibelimab and the completion of Cyprium’s CUTX-101 rolling submission of its New Drug Application ("NDA"). CUTX-101 is eligible for a priority review voucher upon FDA approval. Moreover, we expect the availability of clinical data from many product candidates in ongoing clinical trials including MB-106, MB-107, cosibelimab and Dotinurad."

Dr. Rosenwald continued, "We ended the first quarter with $289.7 million in consolidated cash, cash equivalents and restricted cash. Additionally, we attained a new company quarterly record for net revenue, $23.9 million, which is an increase of 106% period-over-period. We believe that we are well-positioned for success with multiple product candidates and remain focused on creating long-term shareholder value through asset monetizations, equity holdings/appreciation in our subsidiaries and partner companies, annual equity dividends and royalty revenues."

Recent Corporate Highlights2:

Marketed Dermatology Products and Product Candidates

Journey Medical Corporation ("Journey Medical"), a Fortress partner company, currently has nine prescription dermatology products.
Our products generated net revenues of $20.8 million in the first quarter of 2022, compared to first quarter 2021 net revenues of $10.7 million, representing growth of 94%.
In March 2022, Journey Medical dosed the first patient in the Phase 3 clinical program of DFD-29 for the treatment of papulopustular rosacea. Topline data are anticipated in the first quarter of 2023 with an NDA filing expected in the second half of 2023.
In January 2022, Journey Medical received notice from its exclusive licensing partner in Japan, Maruho Co., Ltd., that Japan’s Ministry of Health, Labor and Welfare approved Rapifort Wipes 2.5% (glycopyrronium tosylate hydrate) for the treatment of primary axillary hyperhidrosis. This approval triggered a milestone payment of $10.0 million to Journey Medical, of which $7.5 million was paid to Dermira, Inc. ("Dermira"), a wholly owned subsidiary of Eli Lilly and Company, pursuant to the terms of the Asset Purchase Agreement between Journey Medical and Dermira, with net proceeds of $2.5 million to Journey Medical.
Also in January 2022, Journey Medical entered into a definitive agreement with VYNE Therapeutics, Inc. to acquire two FDA-approved topical minocycline products, Amzeeq and Zilxi, and a Molecule Stabilizing Technology platform for an upfront payment of $20.0 million and an additional $5.0 million on the one (1)-year anniversary of the closing of the transaction in January 2023.
Additionally, in January 2022, Journey Medical expanded the borrowing capacity of the East West Bank credit agreement to $30.0 million, which includes an increase to the working capital line of credit to $10.0 million and the addition of a $20.0 million term loan.
We intend to launch an additional prescription product in the second half of 2022.
CUTX-101 (Copper Histidinate for Menkes disease)

In December 2021, we initiated the rolling submission of an NDA to the FDA for CUTX-101. We intend to complete the rolling submission of the NDA for CUTX-101 in mid-2022.
In March 2022, our subsidiary company, Cyprium Therapeutics, Inc ("Cyprium") announced positive data on CUTX-101 were presented as a "Top-Rated Abstract" and Poster at the 2022 American College of Medical Genetics and Genomics Clinical Genetics Meeting. The abstract can be viewed here.
CUTX-101 is currently in development at Cyprium.
CAEL-101 (Light Chain Fibril-reactive Monoclonal Antibody for AL Amyloidosis)

On October 5, 2021, AstraZeneca acquired Caelum for an upfront payment of approximately $150 million paid to Caelum shareholders, of which approximately $56.9 million was paid to Fortress, net of Fortress’ $6.4 million portion of the $15 million, 24-month escrow holdback amount and other miscellaneous transaction expenses. The agreement also provides for additional potential payments to Caelum shareholders totaling up to $350 million, payable upon the achievement of regulatory and commercial milestones. Fortress is eligible to receive 42.4% of all potential milestone payments, totaling up to approximately $212 million.
There are two ongoing Phase 3 studies of CAEL-101 for AL amyloidosis. (ClinicalTrials.gov Identifiers: NCT04512235 and NCT04504825).
CAEL-101 was sourced by Fortress and was developed by Caelum until the acquisition by AstraZeneca in October 2021.
Cosibelimab (formerly CK-301, an anti-PD-L1 antibody)

In January 2022, we announced positive topline results from our registration-enabling clinical trial evaluating the safety and efficacy of our anti-PD-L1 antibody, cosibelimab, administered as a fixed dose of 800 mg every two weeks in patients with metastatic cSCC. The study met its primary endpoint, with cosibelimab demonstrating a confirmed objective response rate of 47.4% (95% CI: 36.0, 59.1) based on independent central review of 78 patients enrolled in the metastatic cSCC cohort using Response Evaluation Criteria in Solid Tumors version 1.1 criteria. Our partner company, Checkpoint Therapeutics, Inc. ("Checkpoint") intends to submit a BLA for cosibelimab in 2022, followed by a Marketing Authorization Application submission in Europe and other territories worldwide. With a potentially favorable safety profile versus anti-PD-1 therapy and a plan to commercialize at a substantially lower price, we believe cosibelimab has the potential to be a market disruptive product in the $30 billion and growing PD-(L)1 class.
In April 2022, we announced that the results of our pivotal trial of cosibelimab in cSCC were selected for poster presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held at McCormick Place, in Chicago, June 3-7, 2022.
Cosibelimab was sourced by Fortress and is currently in development at Checkpoint.
MB-106 (CD20-targeted CAR T Cell Therapy)

In April 2022, we 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 non-Hodgkin lymphomas ("NHL") and chronic lymphocytic leukemia ("CLL"), were presented 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. Data demonstrated high efficacy and a very 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 ("WM"). An overall response rate ("ORR") of 96% and complete response ("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. We expect to dose the first patient in a Mustang-sponsored multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL in the second quarter of this year. A copy of the abstract can be viewed on the meeting website here.
Also in April 2022, MB-106 data focused on CLL were presented at the 4th International Workshop on CAR-T and Immunotherapies.
In May 2022, we announced that MB-106 CD20-targeted CAR T therapy data were selected for an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 ("EHA2022") Hybrid Congress scheduled to take place in June. 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.
MB-106 was sourced by Fortress and is currently in development at our partner company, Mustang Bio, Inc. ("Mustang Bio").
MB-107 and MB-207 (Lentiviral Gene Therapies for XSCID)

In May 2022, we announced that interim Phase 1/2 data on treatment with the same lentiviral vector used in MB-107, Mustang Bio’s lentiviral gene therapy for X-linked severe combined immunodeficiency ("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 American Society of Gene & Cell Therapy 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. Information on such website is not part of this release.
In the second half of 2022, we expect to enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang Bio’s Investigational New Product Drug Application ("IND") to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with XSCID.
Mustang Bio 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 a hematopoietic stem cell transplantation 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.
MB-107 and MB-207 were sourced by Fortress and are currently in development at Mustang Bio.
Dotinurad (Urate Transporter (URAT1) Inhibitor)

In May 2021, we announced an exclusive license agreement with Fuji Yakuhin Co. Ltd. to develop Dotinurad in North America and Europe. Dotinurad is a potential best-in-class urate transporter (URAT1) inhibitor for gout and possibly other hyperuricemic indications including chronic kidney disease (CKD) and heart failure. Dotinurad (URECE tablet) was approved in Japan in 2020 as a once-daily oral therapy for gout and hyperuricemia. Dotinurad was efficacious and well-tolerated in more than 500 Japanese patients treated for up to 58 weeks in Phase 3 clinical trials. Over 1,000 Japanese patients have been treated safely with this drug.
In December 2021, we filed an IND with the FDA. We expect to initiate a Phase 1 clinical trial to evaluate Dotinurad for the treatment of gout in the first half of 2022. We anticipate topline data from the Phase 1 trial in the second half of 2022.
Dotinurad was sourced by Fortress and is currently in development at our subsidiary company, UR-1 Therapeutics.
MB-105 (PSCA-targeted CAR T Cell Therapy)

In February 2022, Phase 1 data on MB-105, a prostate stem cell antigen ("PSCA")-targeted CAR T cell therapy administered systemically to patients with PSCA-positive metastatic castration-resistant prostate cancer ("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 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.
MB-105 was sourced by Fortress and is currently in development at Mustang Bio.
MB-109 (MB-101 (IL13Rα2-targeted CAR T Cell Therapy) + MB-108 Oncolytic Virus)

In April 2022, we announced our 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 recurrent glioblastoma ("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 American Association for Cancer Research (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.
MB-101 and MB-108 were sourced by Fortress and they are currently in development at Mustang Bio.
General Corporate

In April 2022, Fortress participated in a two-day summit hosted by the B. Riley Securities’ Healthcare Equity Research team that featured multiple programs from Fortress’ diversified pipeline. Webcast replays are available on Fortress’ website here.
Financial Results:

To assist our stockholders in understanding our company, we have prepared non-GAAP financial results for the three months ended March 31, 2022 and 2021. These results exclude the operations of our four public partner companies: Avenue Therapeutics, Inc. ("Avenue"), Checkpoint, Journey Medical and Mustang Bio, as well as any one-time, non-recurring, non-cash transactions. The goal in providing these non-GAAP financial metrics is to highlight the financial results of Fortress’ core operations, which are comprised of our privately held development-stage entities, as well as our business development and finance functions. See "Use of Non-GAAP Measures" below.

As of March 31, 2022, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $289.7 million, compared to $308.0 million as of December 31, 2021, a decrease of $18.3 million during the quarter.
On a GAAP basis, Fortress’ net revenue totaled $23.9 million for the first quarter of 2022, which included $20.8 million in net revenue generated from our marketed dermatology products. This compares to net revenue totaling $11.6 million for the first quarter of 2021, which included $10.7 million in net revenue generated from our marketed dermatology products.
On a GAAP basis, consolidated research and development expenses including license acquisitions were $36.7 million for the first quarter of 2022, compared to $20.2 million for the first quarter of 2021. On a non-GAAP basis, Fortress research and development expenses were $2.8 million for the first quarter of 2022, compared to $4.1 million for first quarter of 2021.
On a GAAP basis, consolidated selling, general and administrative expenses were $26.3 million for the first quarter of 2022, compared to $17.5 million for the first quarter of 2021. On a non-GAAP basis, Fortress selling, general and administrative expenses were $6.2 million, for the first quarter of 2022, compared to $6.7 million for the first quarter of 2021.
On a GAAP basis, consolidated net loss attributable to common stockholders was $15.8 million, or $0.18 per share, for the first quarter of 2022, compared to consolidated net loss attributable to common stockholders of $8.8 million, or $0.11 per share for the first quarter of 2021.
Fortress’ non-GAAP loss attributable to common stockholders was $5.7 million, or $0.07 per share, for the first quarter of 2022, compared to Fortress’ non-GAAP loss attributable to common stockholders of $8.5 million, or $0.11 per share, for the first quarter of 2021.
Use of Non-GAAP Measures:

In addition to the GAAP financial measures as presented in this press release and that will be presented in our Form 10-Q to be filed with the Securities and Exchange Commission ("SEC") on May 12, 2022, the Company, in this press release, has included certain non-GAAP measurements. The non-GAAP net loss attributable to common stockholders is defined by the Company as GAAP net loss attributable to common stockholders, less net losses attributable to common stockholders from our public partner companies Avenue, Checkpoint, Journey Medical and Mustang Bio ("public partner companies"), as well as our former subsidiary, Caelum. In addition, the Company has also provided a Fortress non-GAAP loss attributable to common stockholders which is a modified EBITDA calculation that starts with the non-GAAP loss attributable to common stockholders and removes stock-based compensation expense, non-cash interest expense, amortization of licenses and debt discount, changes in fair values of investment, changes in fair value of derivative liability, and depreciation expense. The Company also provides non-GAAP research and development expenses including license acquisitions, defined as GAAP research and development costs, less research and development costs of our public partner companies and non-GAAP consolidated selling, general and administrative expenses, defined as GAAP selling, general and administrative expenses, less selling, general and administrative costs of our public partner companies.

Management believes each of these non-GAAP measures provide meaningful supplemental information regarding the Company’s performance because (i) it allows for greater transparency with respect to key measures used by management in its financial and operational decision-making; (ii) it excludes the impact of non-cash or, when specified, non-recurring items that are not directly attributable to the Company’s core operating performance and that may obscure trends in the Company’s core operating performance; and (iii) it is used by institutional investors and the analyst community to help analyze the Company’s standalone results separate from the results of its public partner companies. However, non-GAAP loss attributable to common stockholders and any other non-GAAP financial measures should be considered as a supplement to, and not as a substitute for, or superior to, the corresponding measures calculated in accordance with GAAP. Further, non-GAAP financial measures used by the Company and the manner in which they are calculated may differ from the non-GAAP financial measures or the calculations of the same non-GAAP financial measures used by other companies, including the Company’s competitors.

Results for the three months ended March 31, 2021 have been adjusted to present Journey Medical separately as a public entity.
Avenue net loss for the three months ended March 31, 2022 and 2021 of $2.9 million and $1.0 million, respectively, net of non-controlling interest of $2.4 million and $0.8 million, respectively.
Checkpoint net loss of $16.8 million net of non-controlling interest of $13.6 million, Fortress MSA fee of $0.1 million, and Fortress financing fee of $0.2 million for the three months ended March 31, 2022; and net loss of $6.5 million net of non-controlling interest of $4.6 million, Fortress MSA fee of $0.1 million, and Fortress financing fee of $0.6 million for the three months ended March 31, 2021.
Journey Medical net loss for the three months ended March 31, 2022 of $1.4 million net of non-controlling interest of $0.5 million and tax expense recognized on a stand-alone basis of $0.1 million; and net income for the three months ended March 31, 2021 of $0.3 million, net non-controlling interest of approximately $35,000.
Mustang Bio net loss of $19.8 million net of non-controlling interest of $16.2 million, Fortress MSA fee of $0.3 million and Fortress financing fee of $0.8 million for the three months ended March 31, 2022; and net loss of $15.0 million net of non-controlling interest of $10.9 million, Fortress MSA fee of $0.1 million and Fortress financing fee of $1.2 million for the three months ended March 31, 2021.
Reconciliation to non-GAAP research and development and general and administrative costs:

Includes Research and development expense and Research and development – licenses acquired expense for the three months ended March 31, 2021.

Excludes $0.1 million of Fortress MSA expense for each of the three months ended March 31, 2022 and 2021.

Excludes $0.1 million of Fortress MSA expense and $0.2 million Fortress financing fee for the three months ended March 31, 2022; and $0.1 million of Fortress MSA expense and $0.6 million Fortress financing fee for the three months ended March 31, 2021.

Excludes $0.1 million of Fortress MSA expense and $0.8 million Fortress financing fee for the three months ended March 31, 2022; and $0.1 million of Fortress MSA expense and $1.2 million Fortress financing fee for the three months ended March 31, 2021.

Salarius Pharmaceuticals Reports Business Highlights with First Quarter 2022 Financial Results

On May 12, 2022 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company developing cancer therapies for patients in need of new treatment options, reported important corporate events and its financial results for the first quarter ended March 31, 2022 (Press release, Salarius Pharmaceuticals, MAY 12, 2022, View Source [SID1234614476]).

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"We have high expectations for 2022 as we build on the progress achieved during the first quarter and recent weeks," stated David Arthur, CEO of Salarius Pharmaceuticals. "Recent highlights include our acquisition of an intellectual property portfolio, including the drug candidate SP-3164, which is now the foundation of our new drug development program focused on targeted protein degradation (TPD), and the subsequent completion of SP-3164’s pre-IND meeting process with the FDA. TPD is a fast-growing field of cancer drug research with significant potential to improve patient’s lives and a market potential estimated in the billions of dollars. Coupled with our existing clinical programs for seclidemstat, our most advanced cancer drug candidate, Salarius is now pursuing multiple drug development programs built around two exciting approaches to cancer drug development – protein inhibition and protein degradation."

Financial Highlights:
•Cash and cash equivalents totaled $24.2 million on March 31, 2022; $2.3 million direct offering with institutional investors closed on April 26, 2022; Estimated cash runway extends into 2023.
•For the three-month period ended March 31, 2022, net loss per common share, basic and diluted, of $0.13, compared to $0.06 for the same period in 2021
Recent Business and Corporate Highlights:
•2022 Annual Meeting of Stockholders announced for June 15, 2022; Company is seeking stockholder approval or authorization of four proposals and is encouraging stockholders to participate and vote their shares of Salarius Pharmaceuticals stock.

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•Advancing new TPD cancer drug development program built around an intellectual property portfolio acquired from DeuteRx LLC in January 2022. The lead development candidate is SP-3164 for which the company has:
◦Completed the FDA Pre-IND meeting process, which has informed planned IND-enabling studies and activities; and
◦Initiated preclinical studies to support the potential submission of an Investigational New Drug (IND) application with initial preclinical data anticipated in the second half of 2022 and potential for first clinical trial in 2023.
•Salarius continues its enrollment activities to advance clinical exploration of seclidemstat in the treatment of sarcomas, while MD Anderson Cancer Center is enrolling patients in the investigator-initiated trial exploring seclidemstat as a treatment for hematologic cancers.
◦Data updates expected in 2022 from both ongoing Phase 1/2 clinical trials.

"As both CEO and a stockholder of Salarius, I am excited about the upcoming value-building opportunities we expect to achieve" continued Mr. Arthur. "On June 15, the Company will hold its Annual Meeting of Stockholders, where Salarius is asking stockholders to vote on several proposals that we believe will augment these opportunities. All stockholders are encouraged to vote their shares through their brokerage website or through the proxy materials."

"We believe the combination of multiple drug development programs built around two exciting approaches to cancer drug development – protein inhibition and protein degradation – coupled with upcoming value-building opportunities will benefit both current and future stockholders" concluded Mr. Arthur.

Three-Month Financial Results:
For the three-month period ended March 31, 2022, Salarius’ reported net loss was $6.1 million, or $0.13 per basic and diluted share, compared to a net loss of $1.9 million, or $0.06 per basic and diluted share for the same period in 2021. The loss for the three-month period ended March 31, 2022, increased by $4.2 million compared to the same time span last year, primarily because the Company had higher research and development costs and no grant revenue in the current period.
Net cash used for operating activities during the three-month period ended March 31, 2022, totaled $3.5 million, compared to $2.7 million during the same span last year.
As of March 31, 2022, total cash, cash equivalents, and restricted cash were $24.2 million, compared to $36.6 million as of March 31, 2021. The decrease in cash was primarily driven by the Company’s continued spend on operating activities, especially research and development activities and less financing activities involved since Q1 2021. Current cash and cash equivalents are expected to fund our current planned operations into 2023.

Conference Call Information:
Salarius Pharmaceuticals will host a conference call and live audio webcast on Thursday, May 12, 2022, at 5:00 p.m. ET, to discuss its corporate and financial results for the first quarter 2021. Interested participants and investors may access the conference call by dialing either:

An audio webcast will be accessible via the Investors Events and Presentations section of the Company’s website View Source An archive of the webcast will remain available for 90 days beginning at approximately 6:00 p.m. ET on May 12, 2022.

Achieve Reports Financial Results for First Quarter 2022 and Provides Corporate Update

On May 12, 2022 Achieve Life Sciences, Inc. (Nasdaq: ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine addiction, reported first quarter 2022 financial results and provided an update on the cytisinicline clinical development program (Press release, OncoGenex Pharmaceuticals, MAY 12, 2022, View Source [SID1234614475]).

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Recent Events & Highlights

Reported clinically robust and statistically significant Phase 3 topline results from the ORCA-2 clinical trial of cytisinicline in adult smokers

Following positive ORCA-2 results, Achieve updated its agreements with Silicon Valley Bank, providing access to an additional $10.0 million of capital

Cytisinicline development program overview presented at Society for Research on Nicotine and Tobacco (SRNT) Annual Meeting

Appointed Dr. Vaughn Himes to Achieve’s Board of Directors

"Cytisinicline exceeded our expectations in the ORCA-2 trial by delivering impressive efficacy and tolerability in a difficult-to-treat population of smokers, who had lengthy and intense smoking histories, but who courageously attempted to quit smoking in the height of the COVID-19 pandemic," said John Bencich, Chief Executive Officer of Achieve. "We are delighted for those who were successful in ORCA-2 and inspired to continue our efforts to bring this important cytisinicline treatment to the FDA for review and potential approval as a new cessation option for the millions of people battling nicotine addiction."

Positive Phase 3 ORCA-2 Topline Results

The Phase 3 ORCA-2 trial of cytisinicline achieved statistically significant results in primary and secondary endpoints for both 6- and 12-week cytisinicline treatment compared to placebo, and was very well tolerated with single-digit rates of adverse events observed.

Subjects who received 12 weeks of cytisinicline treatment had 6.3 times higher odds, or likelihood, to have quit smoking during the last 4 weeks of treatment compared to subjects who received placebo (p<0.0001). The abstinence rate during weeks 9-12 was 32.6% for cytisinicline compared to 7.0% for placebo.

Subjects who received 6 weeks of cytisinicline treatment had 8 times higher odds, or likelihood, to have quit smoking during the last 4 weeks of treatment compared to subjects who received placebo (p<0.0001). The abstinence rate during weeks 3-6 was 25.3% for cytisinicline compared to 4.4% for placebo.

Updated Debt Facility Agreement with SVB

In connection with the positive ORCA-2 trial results, Achieve received approval from Silicon Valley Bank to access the remaining capital available under its $25.0 million debt facility put in place in December 2021. Per the terms of the agreement, the remaining $10.0 million of commitments are being made available to the Company through April 30, 2023. Amounts drawn under the remaining commitments will incur interest at the greater of 3.50% and the WSJ prime rate, and will be subject to interest only payments through April 30, 2024, and then amortize fully over the following 24 months. No amounts have been drawn to date and the Company will have the discretion to make draws under the facility through April 30, 2023.

Cytisinicline Development Program Presented at SRNT Annual Meeting

An overview of the cytisinicline development program was included in the "Promising New Smoking Cessation Pharmacotherapies" Symposium during the Society for Research on Nicotine and Tobacco (SRNT) Annual Meeting on March 16, 2022.

Dr. Vaughn Himes Appointed to the Board of Directors

In March, Achieve announced the appointment of Vaughn B. Himes, PhD, to Achieve’s Board of Directors. Dr. Himes currently serves as the Chief Technology Officer at Seagen, where he leads the manufacturing, supply chain, process sciences, and quality functions. He has more than 30 years of experience in product development and commercialization in the pharmaceutical and biotech industry, including previous roles as Senior Vice President of Technical Operations at ZymoGenetics, and Vice President of Worldwide Manufacturing Operations at Corixa Corporation.

Financial Results

As of March 31, 2022, the company’s cash, cash equivalents, and restricted cash was $36.4 million. Total operating expenses for the three months ended March 31, 2022 was $7.2 million. Total net loss for the three months ended March 31, 2022 was $7.6 million. As of May 12, 2022, Achieve had 9,681,855 shares outstanding.

Conference Call Details

Achieve will host a conference call at 4:30 PM EDT, Thursday, May 12, 2022. To access the webcast, please use the following link 1Q22 Earnings Webcast. Alternatively, you may access the live conference call by dialing (877) 472-9809 (U.S. & Canada) or (629) 228-0791 (International) and referencing conference ID 3887417. A webcast replay will be available approximately two hours after the call and will be archived on the website for 90 days.

Chinook Therapeutics Provides Business Update and Reports First Quarter 2022 Financial Results

On May 12, 2022 Chinook Therapeutics, Inc. (Nasdaq: KDNY), a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, reported financial results for the first quarter ended March 31, 2022 (Press release, Aduro Biotech, MAY 12, 2022, View Source [SID1234614474]).

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"During the first quarter of 2022, we made strong progress advancing our pipeline of clinical, research and discovery programs for rare, severe chronic kidney diseases. We continue to enroll patients in the phase 3 ALIGN and phase 2 AFFINITY trials for atrasentan as well as the phase 1/2 trial of BION-1301, and we are pleased to have recently initiated the phase 1 healthy volunteer trial of CHK-336, our first internally-discovered program for the treatment of primary and idiopathic hyperoxaluria," said Eric Dobmeier, president and chief executive officer of Chinook Therapeutics. "We look forward to the upcoming 59th ERA Congress being held May 19th – 22nd, where we will present clinical data from both our lead programs, atrasentan and BION-1301, in patients with IgA nephropathy (IgAN)."

Recent Accomplishments and Updates

Atrasentan

Atrasentan is a potent and selective endothelin A (ETA) receptor antagonist that has the potential to provide benefit in multiple chronic kidney diseases by reducing proteinuria and having direct anti-inflammatory and anti-fibrotic effects to preserve kidney function. The phase 3 ALIGN trial of atrasentan is currently enrolling patients with IgAN, and the phase 2 AFFINITY basket trial of atrasentan is currently enrolling patients with proteinuric glomerular diseases.

Enrollment of the phase 3 ALIGN trial of atrasentan continues to advance with the activation of new trial sites and expansion into additional countries. Chinook expects to report topline data from the six-month interim proteinuria endpoint analysis in 2023 to support an application for accelerated approval under Subpart H in the United States.

Chinook plans to present data from the IgAN patient cohort of the phase 2 AFFINITY trial in an oral presentation at the 59th ERA Congress on May 20, 2022, and provide a program update on atrasentan during an investor conference call and webcast at 4:15 pm EDT that day. Chinook has completed enrollment of the IgAN patient cohort of this trial, and continues to enroll the other three cohorts, including patients with focal segmental glomerulosclerosis (FSGS), Alport syndrome and diabetic kidney disease in combination with SGLT2 inhibitors.

Chinook will deliver a mini-oral presentation at the 59th ERA Congress on May 19, 2022 on preclinical mechanistic work describing atrasentan’s effect to block mesangial cell injury and the pathogenic transcriptional networks driving IgAN progression in a model system.

In March 2022, Chinook presented an overview of the phase 2 AFFINITY clinical trial at the 4th Annual Chronic Kidney Disease (CKD) Drug Development Summit.

In February 2022, Chinook delivered an encore trials-in-progress presentations on the phase 3 ALIGN and phase 2 AFFINITY clinical trials at the ISN World Congress of Nephrology 2022.

BION-1301

BION-1301 is a novel anti-APRIL monoclonal antibody currently in phase 1/2 development for patients with IgAN. BION-1301’s potentially disease-modifying approach to treating IgAN by reducing circulating levels of galactose-deficient IgA1 (Gd-IgA1) to prevent the formation of pathogenic immune complexes has been demonstrated preclinically as well as clinically in both healthy volunteers and patients with IgAN.

Chinook will present additional data from Cohort 1 of Part 3 in a mini-oral presentation at the 59th ERA Congress on May 19, 2022. After at least 24 weeks of treatment, all eight patients in Cohort 1 transitioned from IV dosing at 450 mg every two weeks to SC dosing at 600 mg every two weeks.

Enrollment of Cohort 2 of Part 3 of the ongoing phase 1/2 trial of BION-1301 is ongoing. Patients in Cohort 2 receive a SC dose of 600 mg of BION-1301 every two weeks. Data from Cohort 2 is expected in the second half of 2022.

Chinook will present a trials-in-progress mini-oral presentation at the 59th ERA Congress on May 19, 2022 on the ongoing phase 1/2 trial of BION-1301.

In March 2022, Chinook presented a detailed overview of the BION-1301 program at the 4th Annual CKD Drug Development Summit.

In February 2022, Chinook delivered encore presentations on data from Cohort 1 of Part 3 as well as a trials-in-progress of the ongoing phase 1/2 trial of BION-1301 at the ISN World Congress of Nephrology 2022.

CHK-336

CHK-336 is an oral small molecule lactate dehydrogenase A (LDHA) inhibitor with liver-targeted tissue distribution that Chinook is developing for the treatment of patients with primary hyperoxaluria (PH), secondary hyperoxaluria due to increased endogenous oxalate production and idiopathic stone formation.

In April 2022, Chinook initiated dosing in a phase 1 clinical trial evaluating CHK-336 in healthy volunteers. Data from this trial is expected in the first half of 2023.

Precision Medicine Research & Discovery

Chinook is focused on the discovery and development of novel precision medicines for rare, severe chronic kidney diseases (CKDs) with defined genetic or molecular drivers of disease initiation and progression, and efficient development paths. Chinook has multiple preclinical programs across the discovery, target validation, lead identification and lead optimization stages to generate future clinical pipeline candidates. Chinook is leveraging its ongoing strategic collaboration with Evotec to identify and validate novel targets and enable patient stratification strategies through access to the NURTuRE CKD Patient Biobank, which provides comprehensive PANOMICS characterization of thousands of CKD patients with prospective clinical follow-up and retained bio-samples of urine and blood for exploratory biomarker analysis.

Chinook will deliver an oral presentation at the 59th ERA Congress on May 20, 2022 on the approach used in collaboration with Evotec to leverage the NURTuRE CKD biobank to generate mechanistic disease understanding for patient-centric, integrated target and biomarker discovery that will enable the development of novel precision treatments for CKD patient subsets.

In March 2022, Chinook participated in a panel on the challenges and opportunities in drug development for rare kidney diseases at the 4th Annual CKD Drug Development Summit.

Corporate

In April 2022, Chinook announced an outreach initiative in collaboration with the IgA Nephropathy Foundation and Komodo Health, leveraging data and technology to drive awareness of IgAN and engage key medical providers at nephrology practices across the U.S., with the goal of ensuring patients have access to optimal support and treatment options earlier in their disease journey.

In March 2022, Chinook announced the appointment of Dr. Mahesh Krishnan, group vice president of research and development at DaVita Inc., to its Board of Directors.

In January 2022, Chinook announced the appointment of Dr. Charlotte Jones-Burton as senior vice president of product development and strategy.

First Quarter 2022 Financial Results

Cash Position – Cash, cash equivalents and marketable securities totaled $330.0 million at March 31, 2022, compared to $355.1 million at December 31, 2021.

Revenue – Revenue for the quarter ended March 31, 2022 was $2.7 million compared to $0.4 million for the same period in 2021. The increase was primarily due to revenue recognized under Chinook’s license agreement with SanReno.

Expenses –

Research and development expenses for the quarter ended March 31, 2022 were $26.3 million compared to $25.7 million for the same period in 2021. The increase was primarily due to higher employee-related costs from increased staff to build out our clinical and development capabilities; increased spending for consulting and outside services; and an increase in facilities and other costs. These increases were partially offset by a decrease in licensing and contract research and manufacturing costs. The decrease resulted from an upfront fee of $3.3 million to Evotec International GmbH included in the quarter ended March 31, 2021 and lower costs from clinical trials initiated in 2021.

General and administrative expenses for the quarter ended March 31, 2022 were $7.9 million compared to $9.5 million for the same period in 2021. The decrease was primarily due to lower consulting and other professional services costs; lower employee-related costs; and a decrease in facilities and other costs. These decreases were partially offset by an increase in stock-based compensation expense resulting from new grants.

The change in fair value of contingent consideration and contingent value rights liabilities for the quarter ended March 31, 2022 was a benefit of $1.0 million compared to expense of $1.8 million for the same period in 2021. The decrease in these non-cash expenses primarily resulted from a change in estimate of the potential future proceeds derived from the Merck collaboration.

Other –

A $10.0 million development milestone under the Merck collaboration was earned in the fourth quarter of 2021 and received in the first quarter of 2022. We expect to pay this milestone, net of taxes and expenses, to the CVR holders in the second quarter of 2022.

Net Loss – Net loss for the first quarter of 2022 was $31.7 million, or $0.54 per basic share, compared to a net loss of $37.2 million, or $0.88 per share for the same period in 2021.