On May 9, 2022 Vaxart, Inc. (NASDAQ: VXRT) reported its business update for the first quarter of 2022, reporting continued progress on its oral vaccine candidates (Press release, Aviragen Therapeutics, MAY 9, 2022, View Source [SID1234614017]).

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"We continue to make progress on our pipeline of potentially game changing oral tablet vaccine programs, with norovirus studies that will report results in the second quarter as well as our Phase II study for our COVID-19 program that will report results from the first part of the planned two-part study in the third quarter," said Andrei Floroiu, Vaxart’s Chief Executive Officer. "Vaxart’s oral vaccine technology may address many public health challenges, including rapidly emerging COVID-19 variants because the cross-reactive nature of mucosal IgA response increases the likelihood of variant coverage."

Recent Business Highlights

Preclinical and Clinical

COVID-19 Vaccine Developments

In February 2022, Vaxart’s COVID-19 non-human primate study was published by bioRxiv. The study demonstrates that Vaxart’s S-only COVID-19 vaccine candidate, now being studied by Vaxart in Phase II trials, generated antibodies to the original COVID-19 virus strain and to the Beta, Delta, Alpha and Gamma variants of SARS-CoV-2 in the serum and nasal mucosa of non-human primates (NHPs).
Vaxart’s S-only candidate is believed to be the first vaccine candidate to demonstrate neutralizing antibody responses in mucosal sites, which is where primary infection occurs.
The candidate also induced a 1000-fold increase in nasal IgA responses to the variants, which may reduce community transmission.
In April 2022, an article was published in Vaccines that highlights the potential of Vaxart’s oral tablet vaccine platform to transform vaccine strategies for respiratory viral pathogens.
The article discusses one of the chief advantages of oral vaccines, which is to induce an immune response in the mucosa, the first line of defense against invading respiratory pathogens.
The article also outlines how Vaxart’s oral tablet vaccine candidates are very well suited to transform global vaccination strategies by potentially removing cold-chain requirements and the necessity of administration by healthcare professionals, enabling rapid deployment of new vaccines to address novel pathogens.
Vaxart now expects data from the dose selection portion of its two part dose-ranging and preliminary efficacy Phase II clinical trials of its oral tablet COVID-19 vaccine candidate to be available in the third quarter of 2022. This is an open-label dose and age escalation lead-in segment in naïve and previously vaccinated subjects.
Norovirus Vaccine Developments

Vaxart has dosed all subjects in its Phase IB placebo-controlled, dose-ranging, repeat dose trial of its oral norovirus vaccine candidate in elderly subjects aged 55 to 80 years. This study is designed to evaluate the safety and immunogenicity of Vaxart’s GI.1 vaccine candidate and results will be available in the second quarter of 2022.
Vaxart launched a Phase II GI.1 norovirus challenge study in January 2022 to evaluate the safety and clinical efficacy of its oral vaccine candidate. This double blind, placebo-controlled study uses a safe, well-characterized challenge with norovirus GI.1 of volunteers vaccinated with our monovalent norovirus vaccine candidate. The study will yield data on efficacy, safety, and immune correlates of protection, with data expected to be reported in the first quarter of 2023.
2022 Planned Clinical Milestones

Data from the first part of Vaxart’s two-part Phase II trial of its COVID-19 vaccine candidate is expected to be available in the third quarter of 2022.
Vaxart’s international Phase IB and Phase II COVID-19 trials, including a placebo-controlled efficacy trial in India, are anticipated to begin this year.
Results from Vaxart’s Phase IB trial of its norovirus vaccine candidate in elderly subjects are expected in the second quarter of 2022.
Financial Results for the Three Months Ended March 31, 2022

Vaxart ended the first quarter with cash, cash equivalents and available-for-sale debt securities of $157.0 million, compared to $182.7 million as of December 31, 2021. The decrease was primarily due to $25.1 million of cash used in operations.
The Company reported a net loss of $25.1 million for the first quarter of 2022, compared to $16.0 million for the first quarter of 2021. Net loss per share for the first quarter of 2022 was $0.20, compared to a net loss of $0.14 per share in the first quarter of 2021. The increase in net loss was primarily due to a significant increase in research and development expenses.
Revenue for the first quarter of 2022 was $85,000, compared to $506,000 in the first quarter of 2021. The decrease was due to lower royalty revenue from sales of Inavir in Japan.
Research and development expenses were $18.2 million for the first quarter of 2022, compared to $10.1 million for the first quarter of 2021. The increase was mainly due to increases in headcount and related costs and in manufacturing and clinical trial expenses related to our COVID-19 and norovirus vaccine candidates.
General and administrative expenses were $6.7 million for the first quarter of 2022, compared to $5.9 million the first quarter of 2021. The increase was mainly due to an increase in headcount and related costs.

On May 9, 2022 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing IgM antibodies, reported its financial results for the first quarter ended March 31, 2022 and provided an update on recent developments (Press release, IGM Biosciences, MAY 9, 2022, View Source [SID1234614001]).

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"IGM took a major step in expanding the scope and support of our research pipeline of IgM antibodies with the recent closing of an exclusive worldwide collaboration agreement with Sanofi for the creation, research and development of agonist IgM antibodies against three oncology targets and three autoimmune/inflammation targets," said Fred Schwarzer, Chief Executive Officer of IGM Biosciences. "We have also made significant progress in the clinical development of our wholly-owned pipeline of IgM antibodies with the start of our two Phase 2 studies for our T cell bispecific antibody, IGM-2323, and the continuation of our Phase 1 trial of IGM-8444. In addition to continued progress in these ongoing clinical trials, we also expect to file INDs for our targeted IL-15 IgM antibody, IGM-7354, and our CD38 x CD3 bispecific IgM antibody, IGM-2644, this year."

Corporate Updates

Closing of collaboration agreement with Sanofi. IGM announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended (the HSR Act), has expired in connection with the Company’s previously announced exclusive worldwide collaboration agreement with Sanofi. In connection with the closing of the collaboration agreement, Sanofi will pay IGM a $150 million upfront payment. The agreement is for the creation, development, manufacture, and commercialization of IgM antibody agonists against three oncology targets and three autoimmune/inflammation targets. In addition to the $150 million upfront payment, IGM is eligible for over $6 billion in aggregate development, regulatory and commercial milestones, a 50:50 profit share in certain major market countries and tiered royalties in the rest of world for oncology targets, and tiered royalties for autoimmune/inflammation targets.

Completed underwritten public offering of common stock. In April 2022, IGM closed a public offering of its common stock with gross proceeds of $230 million, before deducting the underwriting discounts and commissions and estimated offering expenses payable by IGM.
Pipeline Updates

IGM-2323 (CD20 x CD3)

Phase 2 studies. IGM previously announced the initiation of two Phase 2 studies to assess the safety and efficacy of two doses of IGM-2323, 100 mg and 300 mg, in patients with diffuse large B cell lymphoma (DLBCL) and follicular lymphoma (FL).
IGM-8444 (DR5)

Clinical development of IGM-8444. IGM continues to advance the clinical development of IGM-8444, the Company’s IgM DR5 agonist, in an open-label, multicenter, Phase I study of IGM-8444 in multiple combinations in subjects with relapsed and/or refractory solid and hematologic cancers.
IGM-7354 (IL-15 x PD-L1)

IND application expected to be filed this year. IGM expects to file an Investigational New Drug Application (IND) for IGM-7354, the Company’s targeted IL-15 IgM antibody, in solid tumors this year.
IGM-2644 (CD38 x CD3)

IND application expected to be filed this year. IGM expects to file an IND for IGM-2644, the Company’s CD38 x CD3 bispecific IgM antibody, in multiple myeloma this year.
First Quarter 2022 Financial Results

Cash and Investments: Cash and investments as of March 31, 2022 were $187.5 million, compared to $229.5 million as of December 31, 2021.
The March 31, 2022 cash and investments balance does not include the $230.0 million in gross proceeds, before deducting the underwriting discounts and commissions and other offering expenses payable by IGM, received in connection with IGM’s 2022 public offering, which closed in April.
The March 31, 2022 cash and investments balance also does not include the $150.0 million upfront payment that IGM is expected to receive from Sanofi during the second quarter of 2022 under the terms of the collaboration agreement announced in March 2022.
Research and Development (R&D) Expenses: For the first quarter of 2022, R&D expenses were $38.9 million, compared to $23.6 million for the same period in 2021.
General and Administrative (G&A) Expenses: For the first quarter of 2022, G&A expenses were $13.1 million, compared to $8.1 million for the same period in 2021.
Net Loss: For the first quarter of 2022, net loss was $51.9 million, or a loss of $1.53 per share, compared to a net loss of $31.6 million, or a loss of $0.95 per share, for the same period in 2021. The net loss included non-cash stock-based compensation expense of $11.5 million and $5.5 million for the first quarter of 2022 and 2021, respectively.
2022 Financial Guidance

IGM reiterates its previously issued financial guidance expecting full year GAAP operating expenses of $250 million to $260 million including estimated non-cash stock-based compensation expense of approximately $50 million. IGM expects to end 2022 with a balance of over $390 million in cash and investments.

On May 9, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported financial results for the first quarter of 2022 and provided business updates (Press release, Caribou Biosciences, MAY 9, 2022, View Source [SID1234614000]).

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"Building on the momentum and significant achievements of 2021, we focused on execution and the advancement of our pipeline of genome-edited allogeneic CAR-T and CAR-NK cell therapies in the first quarter of 2022," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "We continue to enroll patients in our ANTLER Phase 1 clinical trial of CB-010 and we are slated to share initial data from patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) at EHA (Free EHA Whitepaper) next month. This year, we plan to submit an IND application for CB-011 to rapidly advance our second CAR-T program into the clinic, and we expect to share target selection for CB-020, the first solid tumor-targeted program from our CAR-NK platform."

Recent Business Highlights

Pipeline

CB-010: In April 2022, an abstract with initial clinical data from the ANTLER Phase 1 trial of CB-010 in adults with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) was accepted at EHA (Free EHA Whitepaper), being held in Vienna, Austria, June 9-17, 2022.
Caribou’s lead product candidate, CB-010, is an allogeneic anti-CD19 CAR-T cell therapy engineered using Cas9 CRISPR hybrid RNA-DNA (chRDNA) technology to insert a CD19-specific CAR into the TRAC gene and knock out PD-1 to boost the persistence of antitumor activity. More information can be found at www.clinicaltrials.gov (NCT04637763).
Caribou continues to enroll patients in ANTLER.
CB-011: Caribou is conducting IND-enabling studies to support an IND application submission in H2 2022 in patients with relapsed or refractory multiple myeloma (r/r MM).
In April 2022, promising preclinical data supporting the development of CB-011 were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
CB-011 is an allogeneic anti-BCMA CAR-T cell therapy engineered using Cas12a chRDNA technology to insert a BCMA-specific CAR into the TRAC gene and armor the cells with an immune cloaking strategy that includes a knockout of the endogenous B2M gene and site-specific insertion of a B2M–HLA-E fusion transgene into the B2M gene. This immune cloaking strategy is designed to blunt both T- and NK-mediated immune cell rejection, enabling more durable antitumor activity.
Corporate

In January 2022, Caribou appointed Syed Rizvi, M.D., as chief medical officer. Dr. Rizvi has more than two decades of experience in all stages of drug development, from clinical strategy and execution through regulatory submissions to support approval and commercialization of several cancer treatments, including three approved autologous CAR-T cell therapies ABECMA, BREYANZI, and CARVYKTI. Prior to joining Caribou, Dr. Rizvi served as chief medical officer of Chimeric Therapeutics and worked for Legend Biotech, Celgene Corporation (now Bristol Myers Squibb), Novartis, Merck, and Genta, Inc.
In January 2022, Zili An, M.D., joined Caribou as vice president of pharmacology. He brings over 20 years of drug development experience in multiple cancer therapeutic modalities, including CAR-T cell therapies.
Anticipated Milestones for 2022 and Beyond

CB-010: Caribou is scheduled to share initial data from its ongoing ANTLER Phase 1 trial for CB-010, an anti-CD19 CAR-T cell therapy for r/r B- NHL, at EHA (Free EHA Whitepaper) in June 2022.
CB-011: Caribou expects to submit an IND application for CB-011, an anti-BCMA CAR-T cell therapy for r/r MM, in H2 2022.
CB-020: Caribou expects to announce target selection for CB-020, an iPSC-derived CAR-NK cell therapy for solid tumors, in Q4 2022. Additionally, Caribou expects to disclose multiple armoring strategies under development for its CAR-NK platform in Q4 2022.
CB-012: Caribou expects to submit an IND application for CB-012, an anti-CD371 CAR-T cell therapy for r/r AML, in 2023.
Upcoming Meetings

May 16-19, 2022: 25th Annual Meeting of the American Society for Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper). A poster with data highlighting the mechanism underlying the superior specificity of its CRISPR hybrid RNA-DNA (chRDNA) guides for genome editing of primary human T cells will be presented on Monday, May 16, 2022, 5:30 – 6:30 pm ET
June 9-17: European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress. Initial ANTLER Phase 1 clinical data for CB-010 is scheduled to be presented
June 23-24: SingHealth Duke-NUS Cell Therapy Centre (SDCT) and Regenerative Medicine Institute of Singapore (REMEDIS) Annual Conference. An encore presentation of initial ANTLER data will be presented
July 18-22: Pan Pacific Lymphoma Conference 2022. An encore presentation of initial ANTLER data will be presented
First Quarter 2022 Financial Results

Cash, cash equivalents, and marketable securities: Caribou had $390.8 million in cash, cash equivalents, and marketable securities as of March 31, 2022 compared to $413.5 million as of December 31, 2021.

Licensing and collaboration revenue: Revenue from Caribou’s licensing and collaboration agreements was $2.7 million for the three months ended March 31, 2022 compared to $1.6 million for the same period in 2021. The increase was primarily due to an increase in revenue recognized pursuant to the AbbVie collaboration and license agreement.

R&D expenses: Research and development expenses were $13.9 million for the three months ended March 31, 2022 compared to $10.2 million for the same period in 2021. The increase was primarily due to personnel-related expenses attributable to increased headcount, costs associated with clinical trial and preclinical study activities, and facilities expenses, partially offset by a decrease in expenses related to licensing, sublicensing revenue, and milestones.

G&A expenses: General and administrative expenses were $9.6 million for the three months ended March 31, 2022 compared to $4.6 million for the same period in 2021. The increase was primarily due to personnel-related expenses attributable to increased headcount, legal and accounting services, insurance, other expenses associated with operating as a public company, and facilities and other expenses.

Other income (expense): The Company recorded other income of $1.8 million for the three months ended March 31, 2022 compared to less than $0.1 million for the same period in 2021. This increase was primarily due to the non-cash change in fair value of the success payments liability under the Memorial Sloan Kettering Cancer Center (MSKCC) exclusive license agreement.

Net loss: For the three months ended March 31, 2022, net loss was $19.1 million compared to $13.2 million for the same period in 2021.

About Caribou’s Novel Next-Generation CRISPR Platform
CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of Class 2 CRISPR systems: the nuclease protein that cuts DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to an edit at the targeted genomic site. CRISPR systems are capable of editing unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function and phenotype. In response to this challenge, Caribou has developed CRISPR hybrid RNA-DNA guides (chRDNAs; pronounced "chardonnays") that direct substantially more precise genome editing compared to all-RNA guides. Caribou is deploying the power of its Cas12a chRDNA technology to carry out high efficiency multiple edits, including multiplex gene insertions, to develop CRISPR-edited therapies.

On May 9, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported financial results for the first quarter of 2022 and provided business updates (Press release, Caribou Biosciences, MAY 9, 2022, View Source [SID1234614000]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Building on the momentum and significant achievements of 2021, we focused on execution and the advancement of our pipeline of genome-edited allogeneic CAR-T and CAR-NK cell therapies in the first quarter of 2022," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "We continue to enroll patients in our ANTLER Phase 1 clinical trial of CB-010 and we are slated to share initial data from patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) at EHA (Free EHA Whitepaper) next month. This year, we plan to submit an IND application for CB-011 to rapidly advance our second CAR-T program into the clinic, and we expect to share target selection for CB-020, the first solid tumor-targeted program from our CAR-NK platform."

Recent Business Highlights

Pipeline

CB-010: In April 2022, an abstract with initial clinical data from the ANTLER Phase 1 trial of CB-010 in adults with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) was accepted at EHA (Free EHA Whitepaper), being held in Vienna, Austria, June 9-17, 2022.
Caribou’s lead product candidate, CB-010, is an allogeneic anti-CD19 CAR-T cell therapy engineered using Cas9 CRISPR hybrid RNA-DNA (chRDNA) technology to insert a CD19-specific CAR into the TRAC gene and knock out PD-1 to boost the persistence of antitumor activity. More information can be found at www.clinicaltrials.gov (NCT04637763).
Caribou continues to enroll patients in ANTLER.
CB-011: Caribou is conducting IND-enabling studies to support an IND application submission in H2 2022 in patients with relapsed or refractory multiple myeloma (r/r MM).
In April 2022, promising preclinical data supporting the development of CB-011 were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
CB-011 is an allogeneic anti-BCMA CAR-T cell therapy engineered using Cas12a chRDNA technology to insert a BCMA-specific CAR into the TRAC gene and armor the cells with an immune cloaking strategy that includes a knockout of the endogenous B2M gene and site-specific insertion of a B2M–HLA-E fusion transgene into the B2M gene. This immune cloaking strategy is designed to blunt both T- and NK-mediated immune cell rejection, enabling more durable antitumor activity.
Corporate

In January 2022, Caribou appointed Syed Rizvi, M.D., as chief medical officer. Dr. Rizvi has more than two decades of experience in all stages of drug development, from clinical strategy and execution through regulatory submissions to support approval and commercialization of several cancer treatments, including three approved autologous CAR-T cell therapies ABECMA, BREYANZI, and CARVYKTI. Prior to joining Caribou, Dr. Rizvi served as chief medical officer of Chimeric Therapeutics and worked for Legend Biotech, Celgene Corporation (now Bristol Myers Squibb), Novartis, Merck, and Genta, Inc.
In January 2022, Zili An, M.D., joined Caribou as vice president of pharmacology. He brings over 20 years of drug development experience in multiple cancer therapeutic modalities, including CAR-T cell therapies.
Anticipated Milestones for 2022 and Beyond

CB-010: Caribou is scheduled to share initial data from its ongoing ANTLER Phase 1 trial for CB-010, an anti-CD19 CAR-T cell therapy for r/r B- NHL, at EHA (Free EHA Whitepaper) in June 2022.
CB-011: Caribou expects to submit an IND application for CB-011, an anti-BCMA CAR-T cell therapy for r/r MM, in H2 2022.
CB-020: Caribou expects to announce target selection for CB-020, an iPSC-derived CAR-NK cell therapy for solid tumors, in Q4 2022. Additionally, Caribou expects to disclose multiple armoring strategies under development for its CAR-NK platform in Q4 2022.
CB-012: Caribou expects to submit an IND application for CB-012, an anti-CD371 CAR-T cell therapy for r/r AML, in 2023.
Upcoming Meetings

May 16-19, 2022: 25th Annual Meeting of the American Society for Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper). A poster with data highlighting the mechanism underlying the superior specificity of its CRISPR hybrid RNA-DNA (chRDNA) guides for genome editing of primary human T cells will be presented on Monday, May 16, 2022, 5:30 – 6:30 pm ET
June 9-17: European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress. Initial ANTLER Phase 1 clinical data for CB-010 is scheduled to be presented
June 23-24: SingHealth Duke-NUS Cell Therapy Centre (SDCT) and Regenerative Medicine Institute of Singapore (REMEDIS) Annual Conference. An encore presentation of initial ANTLER data will be presented
July 18-22: Pan Pacific Lymphoma Conference 2022. An encore presentation of initial ANTLER data will be presented
First Quarter 2022 Financial Results

Cash, cash equivalents, and marketable securities: Caribou had $390.8 million in cash, cash equivalents, and marketable securities as of March 31, 2022 compared to $413.5 million as of December 31, 2021.

Licensing and collaboration revenue: Revenue from Caribou’s licensing and collaboration agreements was $2.7 million for the three months ended March 31, 2022 compared to $1.6 million for the same period in 2021. The increase was primarily due to an increase in revenue recognized pursuant to the AbbVie collaboration and license agreement.

R&D expenses: Research and development expenses were $13.9 million for the three months ended March 31, 2022 compared to $10.2 million for the same period in 2021. The increase was primarily due to personnel-related expenses attributable to increased headcount, costs associated with clinical trial and preclinical study activities, and facilities expenses, partially offset by a decrease in expenses related to licensing, sublicensing revenue, and milestones.

G&A expenses: General and administrative expenses were $9.6 million for the three months ended March 31, 2022 compared to $4.6 million for the same period in 2021. The increase was primarily due to personnel-related expenses attributable to increased headcount, legal and accounting services, insurance, other expenses associated with operating as a public company, and facilities and other expenses.

Other income (expense): The Company recorded other income of $1.8 million for the three months ended March 31, 2022 compared to less than $0.1 million for the same period in 2021. This increase was primarily due to the non-cash change in fair value of the success payments liability under the Memorial Sloan Kettering Cancer Center (MSKCC) exclusive license agreement.

Net loss: For the three months ended March 31, 2022, net loss was $19.1 million compared to $13.2 million for the same period in 2021.

About Caribou’s Novel Next-Generation CRISPR Platform
CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of Class 2 CRISPR systems: the nuclease protein that cuts DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to an edit at the targeted genomic site. CRISPR systems are capable of editing unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function and phenotype. In response to this challenge, Caribou has developed CRISPR hybrid RNA-DNA guides (chRDNAs; pronounced "chardonnays") that direct substantially more precise genome editing compared to all-RNA guides. Caribou is deploying the power of its Cas12a chRDNA technology to carry out high efficiency multiple edits, including multiplex gene insertions, to develop CRISPR-edited therapies.

On May 9, 2022 Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, reported first quarter 2022 financial results (Press release, Pliant Therapeutics, MAY 9, 2022, View Source [SID1234613999]).

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"Our achievements so far in 2022 have built on the momentum we generated last year. We’ve advanced our portfolio, built upon the favorable safety profile of our lead asset and strengthened our financial position," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant Therapeutics. "Solid execution across all areas of our business has allowed us to advance with confidence toward multiple near-term clinical milestones. The first of these will be the expected mid-year data readout of our INTEGRIS-IPF Phase 2a trial."

First Quarter and Recent Highlights

PLN-74809 Highlights

INTEGRIS-IPF Phase 2a trial data readout anticipated in mid-2022. All patients enrolled in the trial have completed treatment. This is a 12-week, randomized, double-blind, placebo-controlled trial evaluating PLN-74809 in patients with idiopathic pulmonary fibrosis (IPF) at once daily doses of 40, 80 and 160 mg. Data will include the primary and secondary endpoints of safety, tolerability, and pharmacokinetics. Exploratory efficacy endpoints will include quantitative lung fibrosis (QLF) imaging as well as pulmonary function tests and biomarkers.
Positive independent Data Safety Monitoring Board (DSMB) review provides additional support for tolerability profile for PLN-74809. The DSMB recommended the INTEGRIS-IPF trial continue without modification. This review occurred after completion of enrollment and included all patients enrolled in all dose cohorts of the trial. To date, no safety concerns have been identified by the DSMB. The INTEGRIS-IPF trial completed enrollment in December 2021.
Phase 2a trial evaluating PLN-74809 at 320 mg for 6 months currently enrolling patients with IPF. This randomized, double-blind, placebo-controlled trial will evaluate PLN-74809 at a once daily dose of 320 mg administered for at least six months and up to 48 weeks in approximately 28 patients with IPF. The trial will evaluate primary and secondary endpoints of safety, tolerability and pharmacokinetics. Exploratory efficacy endpoints will include QLF imaging as well as pulmonary function tests and biomarkers over 6 months of treatment.
FDA Fast Track designation received for PLN-74809 for the treatment of idiopathic pulmonary fibrosis (IPF). FDA’s Fast Track designation is intended to facilitate and expedite the development and review of new drugs to treat serious or life-threatening conditions. The benefits of Fast Track designation include opportunities for frequent meetings with the FDA to discuss trial design, development plans and data needed to support drug approval, as well as the ability to submit a New Drug Application (NDA) on a rolling basis, and eligibility for priority review, if relevant criteria are met.
Expanded PLN-74809 Phase 1b proof-of-mechanism trial demonstrated durable inhibition of TGF-β signaling in the lungs of healthy participants. Results from an expanded Phase 1b proof-of-mechanism trial of PLN-74809 demonstrated on-target biological activity in the lungs of 36 healthy participants with inhibition of TGF-β activation by up to 92% and 76% at 6- and 24-hours, respectively, following seven days of once-daily dosing. TGF-β activation strongly contributes to fibrotic disorders including IPF.
EMA Orphan Drug designation received for PLN-74809 for the treatment of primary sclerosing cholangitis (PSC). Benefits of the European Medicines Association (EMA) Orphan Drug designation include trial design assistance, a centralized EU approval process, and 10 years of market exclusivity. To qualify, an investigational medicine must target a seriously debilitating or life-threatening condition affecting fewer than five in 10,000 people in the EU and must show sufficient non-clinical or clinical data to suggest it may produce clinically relevant outcomes. PLN-74809 received Orphan Drug designation from the United States Food and Drug Administration (FDA) in 2018. Topline data from the ongoing Phase 2a INTEGRIS-PSC 12-week, randomized, double-blind, placebo-controlled trial is expected in the first half of 2023.
Early-Stage Development Programs

Oncology and muscular dystrophy programs progressing through Investigational New Drug (IND) enabling studies. IND application submissions for both programs expected by the end of 2022.
Corporate Highlights

Cash runway extended to mid-2024 with $100 million loan facility from Oxford Finance. Under the terms of the loan agreement, Pliant drew $10 million of an initial $25 million tranche at closing, with the remaining $15 million available through the end of the year. The Company has access to an additional $75 million over three tranches, $50 million of which is based on pre-determined milestones, and $25 million at Oxford’s discretion. The loan carries an interest-only period of 48 months (extendable to 60 months) and total term of 60 months (extendable to 72 months). Interest is based on a floating rate which is subject to both a floor and a cap. There are no warrants or financial covenants in the agreement.
First Quarter 2022 Financial Results

Research and development expenses were $20.9 million, as compared to $18.5 million for the prior-year quarter. The increase was due primarily to employee related expenses and higher costs related to the advancement of several programs and ongoing Phase 1/2 clinical trials.
General and administrative expenses were $8.6 million, as compared to $6.6 million for the prior-year quarter. The increase was due to higher personnel-related and professional services expenses.
Net loss of $28.1 million as compared to $22.9 million for the prior-year quarter due to an increase in operating expenses and a decrease in collaboration revenue.
As of March 31, 2022, the Company had cash, cash equivalents and short-term investments of $178.3 million. With the initial drawdown of $10.0 million from the Oxford Finance loan facility, the Company had pro-forma cash, cash equivalents and short-term investments of $188.3 million as of March 31, 2022. With the facility in place, Pliant expects to be able to fund operations to mid-2024.