On May 12, 2022 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company focused on developing novel therapeutics to treat severe autoimmune and inflammatory disorders with high unmet medical need, reported financial results for the first quarter 2022 and provided an update on its corporate development and clinical programs (Press release, Equillium, MAY 12, 2022, View Source [SID1234614348]).

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"It’s been a transformative start to the year, leading to significant enhancements in how we are building value going forward," said Bruce Steel, chief executive officer at Equillium. "Since the beginning of the year we announced the acquisition of Bioniz Therapeutics, adding two first-in-class therapeutic candidates to our pipeline and an underlying novel drug discovery platform. This enables us to further expand our approach to business development and partnering efforts and significantly adds to our future operational milestones and pending data catalysts. In March we also announced the initiation of our Phase 3 EQUATOR study of itolizumab in first-line acute graft-versus-host disease and look forward to interim data from the Phase 1b EQUALISE study of itolizumab in patients with lupus nephritis expected mid-year."

Clinical Highlights Since the Beginning of Q1 2022:

Initiated Phase 3 EQUATOR study of itolizumab in first-line acute graft-versus-host disease (aGVHD), a randomized, double-blind global pivotal study assessing the efficacy and safety of itolizumab versus placebo as a first-line therapy for aGVHD in combination with corticosteroids that will enroll up to 200 patients. The primary endpoint assessment is complete response rate at Day 29, with key secondary endpoints of overall response rate at Day 29 and durability of complete response rate from Day 29 through Day 99.
Published data in the Journal of Clinical Investigation confirming the role of T cells activated by the CD6-ALCAM pathway in the development of lupus nephritis.
Corporate Highlights Since the Beginning of Q1 2022:

Acquired Bioniz Therapeutics, a privately held clinical-stage biotechnology company, significantly expanding the company’s pipeline of novel immunomodulatory drug candidates, including two first-in-class clinical-stage assets (EQ101 & EQ102) and a proprietary product discovery platform. Lead assets are multi-specific inhibitors of key disease-driving, clinically validated cytokine targets aimed at addressing unmet needs across a range of immuno-inflammatory indications.
Appointed Barbara Troupin, M.D., formerly of Myokardia, ERX Pharmaceuticals, Aquinox and Apricus Biosciences to Equillium’s board of directors.
Anticipated Upcoming Milestones & Catalysts:

Itolizumab – EQUALISE Phase 1b study: interim data from the Type B part of the study in patients with lupus nephritis expected mid-2022
EQ101 – Phase 2 study in alopecia areata initiation expected 2H 2022
EQ102 – Phase 1 study initiation expected in 2H 2022, anticipated to include normal healthy volunteers and celiac disease patients
First Quarter 2022 Financial Results

Research and development (R&D) expenses for the first quarter of 2022 were $10.8 million, compared with $5.9 million for the same period in 2021. The increase was primarily due to greater clinical development expenses, driven by start-up expenses related to the EQUATOR study, greater employee compensation and benefit expenses driven by increased headcount, greater non-clinical research costs, and transaction costs associated with the Bioniz acquisition.

Acquired in-process research and development (IPR&D) expenses for the first quarter of 2022 were $23.0 million resulting from the accounting for the Bioniz acquisition. Those IPR&D expenses were comprised of $22.5 million in non-cash expense associated with the fair value of the equity consideration and $0.5 million for the net liabilities acquired. There were no IPR&D expenses in the first quarter of 2021.

General and administrative (G&A) expenses for the first quarter of 2022 were $3.5 million, compared with $2.8 million for the same period in 2021. The increase was primarily due to greater employee compensation and benefits, greater legal fees and consulting expenses.

Net loss for the first quarter of 2022 was $37.4 million, or $(1.17) per basic and diluted share, compared with a net loss of $9.0 million, or $(0.33) per basic and diluted share for the same period in 2021. The increase in net loss was largely attributable to greater operating expenses, especially the acquired IPR&D expenses.

Cash used in operations for the first quarter of 2022 was $12.1 million compared to $10.2 million in the fourth quarter of 2021. Key drivers of the quarter-over-quarter increase in cash used in operations include the 2021 annual bonuses that were paid in the first quarter of 2022, increased payments related to non-clinical research, and payments related to the Bioniz acquisition, partially offset by our annual directors and officers insurance premiums which were paid in the fourth quarter of 2021.

Cash, cash equivalents and short-term investments totaled $68.8 million as of March 31, 2022, compared to $80.7 million as of December 31, 2021. Of that $11.9 million reduction in cash and investments in the first quarter of 2022, approximately $1.0 million was estimated to be one-time costs related to the Bioniz acquisition transaction. Equillium believes that its cash and investments will be sufficient to fund operations for at least the next 12 months.

About Itolizumab

Itolizumab is a clinical-stage, first-in-class anti-CD6 monoclonal antibody that selectively targets the CD6-ALCAM pathway. This pathway plays a central role in modulating the activity and trafficking of T cells that drive a number of immuno-inflammatory diseases. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited.

About Multi-Cytokine Platform: EQ101 & EQ102

Our proprietary multi-cytokine platform (MCP) generates rationally designed composite peptides that selectively block key cytokines at the shared receptor level targeting pathogenic cytokine redundancies and synergies while preserving non-pathogenic signaling. This approach provides multi-cytokine inhibition at the receptor level and is expected to avoid the broad immuno-suppression and off-target safety liabilities that may be associated with other therapeutic classes, such as JAK inhibitors. Many immune-mediated diseases are driven by the same combination of dysregulated cytokines, and we believe identifying the key cytokines for these diseases will allow us to target and develop customized treatment strategies for multiple autoimmune and inflammatory diseases.
Current MCP assets include EQ101, a first-in-class, tri-specific inhibitor of IL-2, IL-9 and IL-15, and EQ102, a first-in-class, selective inhibitor of IL-15 and IL-21.

On May 12, 2022 Kinnate Biopharma Inc. (Nasdaq: KNTE) ("Kinnate"), a biopharmaceutical company focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers, reported financial results for the quarter ended March 31, 2022 (Press release, Kinnate Biopharma, MAY 12, 2022, View Source [SID1234614347]).

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"The company continues to make meaningful progress with our pipeline candidates, inclusive of our pan-RAF inhibitor, KIN-2787, and our FGFR inhibitor, KIN-3248, both in the clinic, and with initial data from the Phase 1 KN-8701 clinical trial forthcoming later this year for KIN-2787," said Nima Farzan, Chief Executive Officer of Kinnate. "We remain focused on developing a new generation of small molecule kinase inhibitors that target a wide range of cancers, including those that resist current treatment, and we are incredibly proud that in just four years we have advanced two compounds into clinical trials, with several more compounds in the pipeline. Our consistent, disciplined use of capital and updated cash runway guidance is expected to enable Kinnate to fund scientific innovation and operations into early 2024."

Recent Business Highlights and Corporate Update:

KIN-2787, pan-RAF inhibitor

Initial monotherapy data from the ongoing Phase 1 KN-8701 clinical trial expected in the fourth quarter of 2022.
Upon meeting a combination of pre-specified milestones in the ongoing Phase 1 KN-8701 clinical trial, initiated combination portion of KN-8701 to study KIN-2787 with binimetinib in NRAS-mutant melanoma; initial data expected in the first half of 2023.
Abstract highlighting the company’s KIN-2787 program was accepted for publication at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting taking place June 3-7, 2022, in Chicago, Illinois. The abstract is titled: Antitumor activity of KIN-2787, a next-generation pan-RAF inhibitor, in combination with MEK inhibition in preclinical models of human NRAS mutant melanoma.
Presented three separate poster presentations highlighting (1) preclinical activity of KIN-2787 in BRAF alteration positive and NRAS-mutant melanoma models, (2) an overview of the KN-8701 clinical trial design and (3) a clinico-genomics study and outcome analysis that documented the common occurrence of BRAF Class II and Class III alterations across solid tumors, at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. (View release)
KIN-3248, FGFR Inhibitor

Abstract highlighting KIN-3248 was accepted for poster presentation during the ASCO (Free ASCO Whitepaper) 2022 Annual Meeting. The abstract is titled: Design and rationale of a first-in-human (FIH) phase 1/1b study evaluating KIN-3248, a next-generation, irreversible (irrev), pan-FGFR inhibitor (FGFRi), in adult patients with solid tumors harboring FGFR2 and/or FGFR3 gene alterations (NCT05242822).
Announced that the first patient has commenced treatment in KN-4802 (NCT05242822), a Phase 1 clinical trial evaluating KIN-3248. KIN-3248 is a next-generation pan-FGFR inhibitor being developed for the treatment of intrahepatic cholangiocarcinoma (ICC) and urothelial carcinoma (UC), as well as other solid tumors. KN-4802 will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and anti-cancer activity of KIN-3248 in FGFR inhibitor naïve and pretreated cancer patients with FGFR2 and/or FGFR3 gene alterations. (View release)
Corporate Update

Expanded the organization to 68 full-time employees as of March 31, 2022, of which 52 were engaged in research and development activities, and subsequently appointed the following senior leaders:
Ben Powell, Vice President, Discovery Biology
Priyanka Shah, Vice President, IR & Communications
First Quarter 2022 Financial Results

Cash and Cash Equivalents and Investments Position: As of March 31, 2022, the total of cash and cash equivalents and investments was $302.4 million, exclusive of Kinnjiu’s cash. Existing cash and cash equivalents and investments as of March 31, 2022 with budget reallocation is expected to fund current operations, including initiation of multiple registrational studies, into early 2024.
Research and Development Expenses: First quarter research and development expenses for 2022 were $19.6 million, compared to $12.7 million for the same period in 2021.
General and Administrative Expenses: First quarter general and administrative expenses for 2022 were $7.4 million, compared to $4.8 million for the same period in 2021.
Net Loss: First quarter net loss for 2022 was $26.9 million, compared to $17.5 million for the same period in 2021.

On May 12, 2022 Applied DNA Sciences, Inc. (NASDAQ: APDN) (the "Company"), a leader in cell-free, enzymatic DNA production, reported consolidated financial results for the second quarter of fiscal 2022, ended March 31, 2022 (Press release, Applied DNA Sciences, MAY 12, 2022, View Source [SID1234614346]).

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"We delivered strong momentum in revenue growth with a second consecutive quarter of record revenues that reduced cash burn while advancing our strategic priority to develop and further position our LinearDNA platform as a novel approach for the production of the increasing number of nucleic acid-based therapeutic applications under development by the biotherapeutics industry," stated Dr. James A. Hayward, president and CEO of Applied DNA. "Our clinical lab subsidiary, ADCL (Applied DNA Clinical Labs), continues to power our topline performance in the first half of fiscal 2022 with revenues that exceed total revenues for the entirety of fiscal 2021. During the quarter, COVID-19 testing demand remained durable due to our largely academic and corporate client base that continues to test to protect their stakeholders and mitigate disruptions to their operations.

"Similarly, we are starting to realize the value from investments made last year to optimize and advance our LinearDNA platform to commercialization. We are generating compelling data, most recently concluding in vitro studies that demonstrated that LinearDNA encapsulated by LNP expresses well and giving us greater confidence that a LinearDNA-LNP platform is the best delivery means for LinearDNA as a direct therapeutic agent. We concurrently progressed the use of the platform as a rapid IVT templating and production system for RNA-based therapeutics that is, we feel, the most proximal path to incremental biotherapeutic revenues. The second half of the fiscal year should also feature data on the applicability of our LinearDNA platform to the manufacture of advanced therapies, including mRNA, adoptive cell therapies, and DNA-based vaccines. In many instances, a key gating factor to rapid and broader adoption of novel and potentially clinically invaluable therapies is the bottleneck of plasmid DNA."

Continued Dr. Hayward, "Looking ahead to the second half of the fiscal year, our ability to continue to mitigate cash burn and further commercialize the LinearDNA platform will be informed by the durability of ADCL-generated revenues, as well as the conversion of supply chain security opportunities into CertainT platform orders. We expect COVID-19 testing demand to attenuate over the summer months, given our concentration of academic clients but foresee a path to demand resumption in the fall with the start of the new academic year. We have continued to diversify our client base, most notable of which was the addition of an investment management organization after the close of the reported quarter. In addition, we will begin validation of a new testing platform in ADCL that empowers several forms of high-value genetic testing, including pharmacogenetics, for which we believe consumer demand is growing.

"Furthermore, after the close of the reported quarter, our cotton merchant partner received a request to ship the first quantities for traceable tagged cotton that is directly attributable to the recent passage of the Uyghur Forced Labor Prevention Act (the "Act"), a new Federal law. Our team has presented to many members of Congress, Federal agencies, and Committees regarding the utility of our platform in enforcing the Act. Though not expected to be material to revenue in the current fiscal year, the shipment anticipates a global brand’s multi-year commitment to our CertainT platform through a scaled deployment across its many supply chains. We believe that the passage of the Act is a trigger point for the wider adoption of our CertainT platform that holds the potential for molecular taggant sales for textile fiber applications to become a second material revenue stream along with ADCL revenue. With less than 45 days before the Act goes into force, we believe interest in CertainT by brands and their supply chains has never been higher."

Concluded Dr. Hayward, "We believe the business model of Applied DNA is unique in the biotechnology sector. Our expertise in polymerase chain reaction (PCR) empowers the Company to commercialize DNA technologies across targeted industries to give us multiple sources of revenue growth and cash flow to help support the development of the LinearDNA platform to produce biotherapeutic DNA."

Recent Operational Highlights:

Further to a recent Letter of Intent entered into with Spindle Biotech Inc. ("Spindle Biotech"), Applied DNA and Spindle Biotech have formalized a research collaboration and initiated a Proof-of-Concept study (the "PoC") to generate mRNA at high yields. The companies believe the combination of their respective platforms provides for a simplified, high yield, and 100% cell-free workflow that is differentiated from current mRNA production that uses pDNA. In addition to increased speed and purity, the use of LinearDNA as an IVT template for mRNA production removes several complex manufacturing steps necessitated by plasmid DNA. The companies intend to present results from the PoC study upon its conclusion.
The Company entered into a research agreement to advance LinearDNA-based vaccine research and discovery for animal diseases with agricultural biosecurity implications with a leading college of veterinary medicine at a leading university on the East Coast, USA. The research agreement seeks to combine LinearDNA as a platform for rapid drug development with the college’s expertise in viral vector design to advance a differentiated approach to animal vaccine development.
Corporate Updates:

The Company initiated a branding refresh aligned with its positioning of the LinearDNA platform as a novel, cell-free manufacturing foundation for nucleic acid-based therapies. As part of the brand refresh, a LinearDNA-specific website will be launched in the coming months dedicated to showcasing LinearDNA’s attributes to therapy developers and manufacturers.
Dr. Hayward voluntarily waived 50% of his cash compensation effective March 7, 2022, as part of a cost management program implemented by the Company in the reported quarter.
Second Quarter Fiscal 2022 Financial Highlights:

Revenues increased 130% for the second quarter of fiscal 2022 to $6.1 million, compared with $2.7 million reported in the same period of the prior fiscal year and increased 48% from $4.2 million for the first quarter of fiscal 2022. The increase in revenues year-over-year was due primarily to an increase in clinical laboratory service revenues from the safeCircle COVID-19 testing platform of $3.9 million. This increase was offset by a decrease in product revenues of approximately $557 thousand due mainly to a decrease of approximately $605 thousand in sales of the Linea 1.0 COVID-19 Assay Kit.
Gross profit for the three months ended March 31, 2022, was $2.5 million, or 40%, compared with $1.7 million and 65% for the same period in the prior fiscal year. The decline in gross margin was primarily the result of a higher portion of clinical laboratory service revenues coming from the managed services testing contracts where ADCL also provides and staffs test collection centers, as these contracts have higher costs associated with them compared with ADCL’s surveillance testing contracts. The Company saw an improvement in gross profit percentages for the second quarter of fiscal 2022 to 40% as compared to 28% for the first quarter of fiscal 2022. The improvement was the result of the decrease in COVID-19 positivity rates as sample pooling returned during the second fiscal quarter, and sample numbers remained at higher levels.
Total operating expenses increased to $4.5 million for the second quarter of fiscal 2022, compared with $4.0 million in the prior-year quarter and decreased from $5.7 million for the first quarter of fiscal 2022. The year-over-year increase is primarily attributable to an increase in payroll of approximately $740 thousand. The increase in total payroll is due to the three months ended March 31, 2021, having a reversal of an accrual of approximately $817 thousand for an accrued bonus that was forgiven by the CEO. The increase was also due to an increase in insurance expense of approximately $129 thousand, which was primarily the result of increased Directors and Officers insurance premiums. These increases were offset by a decrease of approximately $376 thousand and $169 thousand in stock-based compensation and professional fees, respectively. To a lesser extent, the increase was attributable to an increase in Research and Development expenses of $114 thousand.
Net loss applicable to common stockholders for the second quarter of fiscal 2022, was $1.8 million, or $0.23 per share, compared with a net loss of $1.5 million, or $0.21 per share, for the prior-year quarter.
Excluding non-cash expenses, Adjusted EBITDA was negative $1.6 million and negative $1.5 million for the second quarters of fiscal 2022 and 2021, respectively. See below for information regarding non-GAAP measures.
Cash and cash equivalents stood at $6.5 million on March 31, 2022, compared with $6.6 million as of September 30, 2021. Cash and cash equivalents include net proceeds of $3.7 million from a registered direct offering closed on February 24, 2022.
Second Quarter Fiscal 2022 Conference Call Information

The Company will hold a conference call and webcast to discuss its second quarter fiscal 2022 financial results today, Thursday, May 12, 2022, at 4:30 PM ET. To participate in the conference call, please follow the instructions below. While every attempt will be made to answer investors’ questions on the Q&A portion of the call, not all questions may be answered.

Presentation slides will also be posted to the ‘Company Events’ sub-page of the Company’s Investor Relations website and embedded into the live webcast.

Information about Non-GAAP Financial Measures

As used herein, "GAAP" refers to accounting principles generally accepted in the United States of America. To supplement our condensed consolidated financial statements prepared and presented in accordance with GAAP, this earnings release includes Adjusted EBITDA, which is a non-GAAP financial measure as defined in Rule 101 of Regulation G promulgated by the Securities and Exchange Commission. Generally, a non-GAAP financial measure is a numerical measure of a company’s historical or future performance, financial position, or cash flows that either excludes or includes amounts that are not normally excluded or included in the most directly comparable measure calculated and presented in accordance with GAAP. The presentation of this non-GAAP financial information is not intended to be considered in isolation or as a substitute for, or superior to, the financial information presented in accordance with GAAP. We use this non-GAAP financial measure for internal financial and operational decision-making purposes and as a means to evaluate period-to-period comparisons of the performance and results of operations of our core business. Our management believes that these non-GAAP financial measures provide meaningful supplemental information regarding the performance of our business by excluding non-cash expenses that may not be indicative of our recurring operating results. We believe this non-GAAP financial measure is useful to investors as they allow for greater transparency with respect to key metrics used by management in its financial and operational decision making.

"EBITDA"- is defined as earnings (loss) before interest expense, income tax expense and depreciation and amortization expense.

"Adjusted EBITDA"- is defined as EBITDA adjusted to exclude (i) stock-based compensation and (ii) other non-cash expenses.

On May 12, 2022 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the first quarter ended March 31, 2022 (Press release, CymaBay Therapeutics, MAY 12, 2022, View Source [SID1234614345]).

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Sujal Shah, President and CEO of CymaBay, stated, "Despite facing ongoing external challenges, we have made steady progress over the past few months enrolling patients in RESPONSE, our second, global phase 3 registration study of seladelpar for patients with primary biliary cholangitis (PBC). We now have over 150 clinical sites activated across 26 countries where we have continued to increase our direct site engagement initiatives. As we approach what we project to be the final months of screening, we have greater visibility into monthly metrics and forecast completion of enrollment in the third quarter. We were also excited to have the results of our 52-week, open-label, Phase 2 study of seladelpar in patients with PBC published in the Journal of Hepatology last month. The opportunity to have these data featured in one of the world’s preeminent medical journals for liver diseases elevates seladelpar’s visibility as a differentiated drug candidate for patients with PBC. Finally, we have continued to navigate the difficult market environment with a focus on diligent expense management leaving us with a strong balance sheet after completing two successful financings in 2021. In the first quarter, we received the third $25 million funding tranche from our non-dilutive, clinical funding agreement with Abingworth and ended the quarter with $193 million of cash, cash equivalents and investments."

Recent Corporate Highlights

Moving towards completion of enrollment in RESPONSE, a 52-week, placebo-controlled, randomized, global, Phase 3 registrational study evaluating the safety and efficacy of seladelpar in patients with PBC. This study is targeting enrollment of 180 patients who have an inadequate response to, or intolerance to, ursodeoxycholic acid, in a 2:1 randomization to oral, once daily seladelpar 10 mg or placebo. The primary outcome measure is the responder rate at 52 weeks. A responder is defined as a patient who achieves an alkaline phosphatase level < 1.67 times the upper limit of normal with at least a 15% decrease from baseline and has a normal level of total bilirubin. Additional key outcomes of efficacy will compare the rate of normalization of alkaline phosphatase at 52 weeks and the level of pruritus at 6-months for patients with moderate to severe pruritus at baseline assessed by a numerical rating scale recorded with an electronic diary. To date we have over 150 sites activated across 26 countries.
Continued strong enrollment in ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional long-term safety data to support registration. In April, we successfully rolled over patients completing RESPONSE into ASSURE. Together with patients that entered into ASSURE from prior studies with seladelpar, there are now approximately 140 patients in this study taking daily seladelpar.
Supported enrollment efforts in a Phase 2a proof-of-pharmacology study to evaluate the potential for MBX-2982, a GPR119 agonist, to prevent hypoglycemia in patients with type 1 diabetes. The study is being conducted by the AdventHealth Translational Research Institute in Orlando, Florida and fully funded by The Leona M. and Harry B. Helmsley Charitable Trust with CymaBay retaining full rights to MBX-2982.

Published results from Phase 2, 52-week study of seladelpar in patients with primary biliary cholangitis (PBC) in the Journal of Hepatology. This 52-week, phase 2, dose-ranging, open-label study examined the efficacy and safety of seladelpar in PBC patients who were receiving or intolerant to first-line therapy with ursodeoxycholic acid., The results included:

An interim primary efficacy analysis of ALP change from baseline at Week 8 found that seladelpar treatment provided 26%, 33%, and 41% reductions for the 2 mg, 5 mg and 10 mg doses, respectively (all p<0.005).
Responses were maintained or improved at Week 52, which included dose escalation in 91% and 80% of the 2 mg and 5 mg cohorts, respectively.
At Week 52, the composite biochemical response (ALP <1.67×ULN, ≥15% ALP decrease from baseline, and normal total bilirubin) rates were 64%, 53%, and 67%, and ALP normalization rates were 9%, 13%, and 33% in the 2 mg, 5 mg, and 10 mg cohorts, respectively.
The pruritus visual analog scale score decreased in the 5 mg and 10 mg cohorts.
There were no treatment-related serious adverse events (AEs), and 4 patients discontinued due to AEs.
Expanded the Board of Directors to include Dr. Éric Lefebvre, the Chief Medical Officer of Pliant Therapeutics. Prior to joining Pliant, Dr. Lefebvre served as the Vice President of Research and Development of Allergan plc and before that was Chief Medical Officer of Tobira Therapeutics, Inc. Dr. Lefebvre also led global clinical development and global medical affairs at Janssen Pharmaceuticals for 10 years prior to starting his pharmaceutical career at GlaxoSmithKline Canada.
Received $25 million of funding in January 2022 from Abingworth through a non-dilutive financing agreement for the development of seladelpar, which was executed in July 2021. $75 million has been received to date through the financing agreement. CymaBay also has an option to receive an additional $25 million after the completion of enrollment of the RESPONSE clinical trial.
Held $193.4 million in cash, cash equivalents and investments as of March 31, 2022. We believe that cash and investments on hand, together with committed capital available through the development financing agreement with Abingworth, is sufficient to fund CymaBay’s operating plan through 2023.
First Quarter Ended March 31, 2022 Financial Results

Research and development expenses for the three months ended March 31, 2022 and 2021 were $18.4 million and $12.4 million, respectively. Research and development expenses in the three months ended March 31, 2022 were higher than the corresponding period in 2021 primarily due to an increase in clinical trial activities associated with the ongoing late-stage development of seladelpar in PBC. In particular, cost increases were primarily driven by an expansion of our site activation, patient enrollment, and other clinical trial activities associated with RESPONSE and ASSURE, our two active global late-stage clinical trials in PBC.

General and administrative expenses for the three months ended March 31, 2022 and 2021 were $6.1 million and $5.2 million, respectively. General and administrative expenses in the three months ended March 31, 2022 were higher than the corresponding period in 2021 due to higher employee compensation associated with the hiring of additional personnel and an increase in consulting and other expenses to support our late-stage development of seladelpar in PBC.

Net loss for the three months ended March 31, 2022 and 2021 was $27.8 million and $17.6 million, or ($0.32) and ($0.25) per diluted share, respectively. Net loss was higher largely due to increases in clinical operating expenses, as clinical activity related to our late-stage development of seladelpar in PBC continued to expand and accretion of interest expense related to the Abingworth development financing arrangement. We expect our operating expenses to increase in the future as we continue to execute on our clinical development plans.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss fourth quarter and fiscal year end 2021 financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13728967. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

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.