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.

On May 9, 2022 ALX Oncology Holdings Inc., ("ALX Oncology") (Nasdaq: ALXO) a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported financial results for the first quarter ended March 31, 2022 and provided clinical development and operational highlights (Press release, ALX Oncology, MAY 9, 2022, View Source [SID1234613989]).

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"Throughout the first quarter, we continued to realize significant progress advancing our lead program, evorpacept, through multiple clinical trials," said Jaume Pons, Ph.D., Founder, President and Chief Executive Officer of ALX Oncology. "Our highlights include dosing the first patient in ASPEN-06, our Phase 2/3 study testing evorpacept in combination with ramucirumab, trastuzumab, and paclitaxel for the treatment of patients with HER2-positive gastric cancer or gastroesophageal junction ("GEJ") cancer and the U.S. Food and Drug Administration ("FDA") granting Orphan Drug Designation ("ODD") to evorpacept for the treatment of patients with gastric/GEJ cancer."

"Looking ahead, we are excited for clinical milestones by year-end including the expected dose optimization readout of a Phase 1b clinical trial of evorpacept in combination with azacitidine in patients with myelodysplastic syndromes ("MDS") (ASPEN-02) and updates on our Phase 1/2 collaboration with Zymeworks in evaluating the combination of evorpacept and zanidatamab in patients with HER2-positive breast cancer and other solid tumors," Dr. Pons continued.

Recent Clinical Developments for Evorpacept

Initiation of a Phase 2/3 Study of Evorpacept for the Treatment of Patients with Advanced Gastric or Gastroesophageal Junction Cancer (ASPEN-06)
In March 2022, the first patient was dosed in the Phase 2/3 ASPEN-06 study evaluating the combination of evorpacept, a next generation CD47 blocker, and CYRAMZA (ramucirumab), Eli Lilly and Company’s anti-VEGFR2 antibody, added to trastuzumab and paclitaxel for the treatment of patients with HER2-positive gastric/GEJ cancer.
ASPEN-06 (NCT05002127) is a randomized Phase 2 (open-label) / Phase 3 (double-blind), international, multi-center study to evaluate the efficacy of evorpacept in combination with ramucirumab, trastuzumab, and paclitaxel for the treatment of patients whose tumors have progressed following treatment with HER2-targeted therapy and chemotherapy. Approximately 450 adult patients will be enrolled in the study across both phases.
FDA Granted ODD for Evorpacept for the Treatment of Patients with Gastric/GEJ Cancer
In January 2022, ALX Oncology announced that the FDA granted ODD to evorpacept, a next-generation CD47 blocker, for the treatment of patients with gastric/GEJ cancer.
Recent Corporate Updates

Appointed Itziar Canamasas, Ph.D., to its Board of Directors
In April 2022, ALX Oncology announced the appointment of Itziar Canamasas, Ph.D., to its Board of Directors (the "Board"). With more than 20 years of biopharmaceutical industry experience, Dr. Canamasas brings expertise in driving business growth and operational excellence.
First Quarter 2022 Financial Results:

Cash, Cash Equivalents and Investments: Cash, cash equivalents and investments as of March 31, 2022 were $341.7 million. ALX Oncology believes its cash, cash equivalents and investments are sufficient to fund planned operations through mid-2024.
Research and Development ("R&D") Expenses: R&D expenses consist primarily of pre-clinical, clinical and manufacturing expenses related to the development of ALX Oncology’s current lead product candidate, evorpacept, and R&D employee-related expenses. These expenses for the three months ended March 31, 2022, were $17.1 million, compared to $9.8 million for the prior-year period. The increase in expenses during the three months ended March 31, 2022 compared to the three months ended March 31, 2021 were primarily attributable to an increase of $2.6 million in clinical and development costs primarily due to manufacturing of clinical trial materials to support a higher number of active clinical trials and future expected patient enrollment related to the advancement of evorpacept, as well as an increase of $0.9 million related to the collaboration with Tallac Therapeutics, Inc., an increase of $2.1 million in personnel related costs driven by headcount growth, an increase of $1.5 million in stock-based compensation expense due to additional awards granted since March 31, 2021 and an increase of $0.6 million in other research costs due primarily to an increase in facility costs related to the expansion of our new laboratory space.
General and Administrative ("G&A") Expenses: G&A expenses consist primarily of administrative employee-related expenses, legal and other professional fees, patent filing and maintenance fees, and insurance. These expenses for the three months ended March 31, 2022, were $7.7 million, compared to $4.4 million for the prior-year period. The expense increases during the three months ended March 31, 2022 compared to the three months ended March 31, 2021 were primarily attributable to an increase of $2.2 million in stock-based compensation expense due to additional awards granted since March 31, 2021 and an increase of $0.8 million in other costs primarily driven by an increase in corporate legal fees, regulatory related filing fees and facility and information technology costs.
Net loss: GAAP net loss was $24.5 million for the first quarter ended March 31, 2022, or $0.60 per basic and diluted share, as compared to a net loss of $14.2 million for the first quarter ended March 31, 2021, or $0.35 per basic and diluted share. Non-GAAP net loss was $19.0 million for the first quarter ended March 31, 2022, as compared to a net loss of $12.4 million for the first quarter ended March 31, 2021. A reconciliation of GAAP to non-GAAP financial results can be found at the end of this news release.

On May 9, 2022 Enara Bio, a biotechnology company advancing novel T-cell receptor (TCR) directed immunotherapies against unconventional, shared, cancer-specific antigens, reported that three abstracts from the Company and its academic collaborators have been selected for presentation at the 12th International CD1-MR1 European Molecular Biology Organization (EMBO) Meeting, which will be held in Gothenberg, Sweden, 22-26 May 2022 (Press release, Enara Bio, MAY 9, 2022, View Source [SID1234613975]).

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The three abstracts cover pioneering research being conducted by Enara Bio in collaboration with leading immunology and MR1 research groups at Monash University and the University of Oxford towards the identification and validation of cancer-specific MR1 ligands and the development of MR1-targeted T-cell receptor cell therapies (TCR-T).

MR1 is an unconventional antigen-presenting molecule that presents metabolites to the immune system in the context of cancer and infection. The monomorphic nature of MR1 strongly suggests that therapies based on MR1-displayed antigens could be effective across the entire human population. These unique properties make MR1 a promising target for off-the-shelf, HLA-independent, TCR-based immunotherapies for solid tumors.

The first presentation, from Enara scientists, highlights the progress the Company is making with its lead autologous MR1-targeting TCR-T program. The two other presentations, led by scientists from Jamie Rossjohn’s and Tony Purcell’s groups at the Monash Biomedicine Discovery Institute (BDI), and Nicola Ternette’s group at the University of Oxford’s Nuffield Department of Medicine, describe novel immunopeptidomic and metabolomic methods to identify and characterize the cancer-specific MR1 ligandome.

Joe Dukes, Vice President, Head of Research at Enara Bio, commented: "We are very pleased to be presenting important abstracts at the upcoming CD1-MR1 EMBO meeting, which showcase the continued developments we are making in partnership with our world-class collaborators to advance the next generation of cancer immunotherapies designed to treat a broad patient population. We have made remarkable progress in our mission to characterize unconventional immunotherapy targets, such as ligands presented by MR1, which we hope will enable development of a pipeline of product candidates that can be advanced into clinical development."

Details of the poster presentations (which will be available on the Company website at the time of the meeting) are as follows:

Enara Bio

– Abstract Title: A novel TCR-T cell therapy targeting a cancer-specific antigen presented by monomorphic MR1 to overcome the challenge of HLA restricted TCR therapies.
– Presenter: Dr. Jonathan Silk

Monash University

– Abstract Title: Design and testing of cleavable constructs for identification and functional validation of MR1 ligands.
– Presenter: Dr. Patricia Illing

The University of Oxford

– Abstract Title: Development of a metabolomics-based MR1 ligand discovery platform using proteomics instrumentation
– Presenter: Dr. Thierry Schmidlin

On May 9, 2022 Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx plant cell-based protein expression system, reported that it will release its financial results for the first quarter 2022 and provide a business update on Monday, May 16, 2022 (Press release, Protalix, MAY 9, 2022, View Source [SID1234613974]).

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Management will host a conference call with investors to discuss the financial results and provide an update on recent corporate and clinical developments at 8:30 a.m. Eastern Daylight Time (EDT).

The conference will be webcast live from the Company’s website and will be available via the following links:

Please access the websites at least 15 minutes ahead of the conference to register, download and install any necessary audio software.

The conference call will be available for replay for two weeks on the Events Calendar of the Investors section of the Company’s website, at the above link.

On May 9, 2022 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that it has completed the divestiture of Sunosi (solriamfetol) in the U.S. to Axsome Therapeutics, Inc. (Nasdaq: AXSM) (Press release, Jazz Pharmaceuticals, MAY 9, 2022, View Source [SID1234613973]).

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Under the terms of the agreement, Jazz received an upfront payment of $53 million, and will receive a high single-digit royalty on Axsome’s U.S. net sales of Sunosi in current indications and a mid-single-digit royalty on Axsome’s U.S. net sales of Sunosi in future indications. Subject to the satisfaction of conditions to closing, the ex-U.S. transaction is expected to close within 60 days.

"The divestiture of Sunosi enables us to sharpen our focus on the strategic areas where we see the most opportunity for sustainable growth and enhanced shareholder value," said Bruce Cozadd, chairman and CEO of Jazz Pharmaceuticals. "Looking forward, Jazz will continue to invest in our highest strategic priorities, driving the transformation of Jazz to an innovative, global biopharmaceutical leader, and delivering improved top- and bottom-line growth through operational excellence."

About Sunosi (solriamfetol)

Sunosi is a dual-acting dopamine and norepinephrine reuptake inhibitor shown to improve wakefulness in adults living with excessive daytime sleepiness (EDS) due to narcolepsy or obstructive sleep apnea (OSA). Sunosi received U.S. Food and Drug Administration approval on March 20, 2019, to improve wakefulness in adult patients with EDS associated with narcolepsy or OSA and was designated a Schedule IV medicine by the U.S. Drug Enforcement Agency on June 17, 2019. In 2014, Jazz Pharmaceuticals acquired a license to develop and commercialize solriamfetol from Aerial Biopharma LLC. Jazz Pharmaceuticals has worldwide development, manufacturing, and commercialization rights to solriamfetol, excluding certain jurisdictions in Asia. SK Biopharmaceuticals Co., Ltd., the discoverer of the compound, maintains rights in 12 Asian markets, including Korea, China and Japan. Sunosi has orphan drug designation for narcolepsy in the United States.

Important Safety Information for Sunosi

SUNOSI (solriamfetol) is available in 75 mg and 150 mg tablets and is a federally controlled substance (C-IV) because it contains solriamfetol that can be a target for people who abuse prescription medicines or street drugs. Keep SUNOSI in a safe place to protect it from theft. Never give or sell your SUNOSI to anyone else, because it may cause death or harm them and it is against the law. Tell your doctor if you have ever abused or been dependent on alcohol, prescription medicines, or street drugs.

Before taking SUNOSI, tell your doctor about all of your medical conditions, including if you:

have heart problems, high blood pressure, kidney problems, diabetes, or high cholesterol
have had a heart attack or a stroke
have a history of mental health problems (including psychosis and bipolar disorders), or of drug or alcohol abuse or addiction
are pregnant or planning to become pregnant. It is not known if SUNOSI will harm your unborn baby
are breastfeeding or plan to breastfeed. It is not known if SUNOSI passes into your breast milk. Talk to your doctor about the best way to feed your baby if you take SUNOSI.
What are the possible side effects of SUNOSI?

SUNOSI may cause serious side effects, including:

Increased blood pressure and heart rate. SUNOSI can cause blood pressure and heart rate increases that can increase the risk of heart attack, stroke, heart failure, and death. Your doctor should check your blood pressure before and during treatment with SUNOSI. Your doctor may decrease your dose or tell you to stop taking SUNOSI if you develop high blood pressure that does not go away during treatment with SUNOSI.
Mental (psychiatric) symptoms including anxiety, problems sleeping (insomnia), irritability, and agitation. Tell your doctor if you develop any of these symptoms. Your doctor may change your dose or tell you to stop taking SUNOSI if you develop side effects during treatment with SUNOSI.
The most common side effects of SUNOSI include:

headache
decreased appetite
problems sleeping
nausea
anxiety
These are not all the possible side effects of SUNOSI. Call your doctor for advice about side effects.