On March 16, 2022 Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, reported a corporate and financial update for the fourth quarter and full year ended December 31, 2021 (Press release, Athenex, MAR 16, 2022, View Source [SID1234610163]).

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"We are pleased to announce our new vision for the future of Athenex, by deploying our resources and harnessing our expertise toward the advancement of our promising cell therapy programs. The encouraging data that we have generated so far, along with our belief that cell therapy is driving the next major innovation cycle in cancer treatment, give us confidence in our new strategy," said Johnson Lau, Chief Executive Officer of Athenex. "We are taking action to swiftly redirect our resources and have begun executing on cost savings measures to right-size the company and support our transformation to a lean, focused cell therapy company."

Corporate Developments

Key Program and Business Updates

Focus R&D resources on developing cell therapy programs
Discontinue oral discovery program apart from oral paclitaxel
Reducing operating expenses: With the objective of extending its cash runway, the Company is implementing cost cutting initiatives and workforce reduction to lower operating expenses by over 50%
Plan to monetize non-core assets consistent with new focus and strategy of company
Key Anticipated Milestones

Update on ANCHOR data previously presented at ASH (Free ASH Whitepaper) in 2021 at ASTCT on April 23-26, 2022
Update from KUR-501, our autologous program in pediatric neuroblastoma at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) annual meeting on May 16-19, 2022
Preclinical data presentation of allogeneic GPC3 CAR-NKT cell program in liver cancer at ASCO (Free ASCO Whitepaper) on June 3-7, 2022
Expand CD19 CAR-NKT ANCHOR study to a multi-center study under the newly allowed IND
Data update from KUR-502 at American Society of Hematology (ASH) (Free ASH Whitepaper) on December 10-13, 2022
Potential regulatory update from MHRA for Oral Paclitaxel in metastatic breast cancer (mBC) in UK
Data from I-SPY 2 trial of Oral Paclitaxel in combination with dostarlimab in neoadjuvant breast cancer expected in 2H22
IND filing for KUR-503, our allogeneic CAR-NKT program in liver cancer in 1H23
Fourth Quarter 2021 and Recent Business Highlights

Clinical Programs

Cell Therapy

Observed ORR of 80% with 3 CRs and 1 PR out of 5 evaluable patients in data presented from Phase 1 ANCHOR trial of KUR-502 in relapsed/refractory CD19-positive leukemia and lymphoma at the American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting in December.
Announced agreement with the National Cancer Institute (NCI) to license TCRs targeting KRAS, TP53, and EGFR to combine with our allogeneic platform for application in solid tumors
Received IND clearance from the FDA to expand Phase 1 ANCHOR study of KUR-502 to up to 12 clinical sites
Orascovery

Initiated the expansion portion of Phase 1/2 trial of Oral Paclitaxel in combination with pembrolizumab in (Non-small Cell Lung Cancer) NSCLC
Commercial Update

Klisyri (tirbanibulin)

Commercial Partner Almirall reported 3% market share the U.S. and expects gains to continue
Over 2,000 HCPs have prescribed Klisyri since its launch, and it has access to over 70 million covered lives within the commercial space
Almirall expects Medicare Part D coverage in 2022
Specialty Pharmaceutical Business

Athenex Pharmaceutical Division (APD) currently markets a total of 29 products with 54 SKUs.
Athenex Pharma Solutions (APS) currently markets 5 products with 16 SKUs
Fourth Quarter and Full Year 2021 Financial Highlights

Revenues from product sales increased to $23.5 million for the three months ended December 31, 2021, from $21.8 million for the three months ended December 31, 2020, an increase of $1.7 million or 8%. Product sales for the full year 2021 were $92.3 million, down from $105.3 million in 2020, which represents a 12% decrease. This decrease was primarily attributable to non-recurring COVID-related sales in 2020 and international supply chain disruptions caused by COVID-19.

License fees and other revenue for three months and year ended December 31, 2021 were $1.5 million and $27.9 million, respectively, compared to $28.0 thousand and $39.1 million, respectively, for the same periods in 2020.

Cost of sales for the three months ended December 31, 2021 totaled $20.7 million, an increase of $2.4 million, or 13%, as compared to $18.3 million for the three months ended December 31, 2020. Cost of sales totaled $82.4 million for the full year in 2021, a decrease of 14% as compared to $95.4 million for the full year in 2020. The decrease in our cost of specialty product sales was in-line with the decrease in revenue partially offset by corresponding increases in 503B, Contract Manufacturing, and API product sales.

R&D expenses totaled $18.3 million for the three months ended December 31, 2021, in-line with expenses for the three months ended December 31, 2020. R&D expenses totaled $80.2 million for the full year in 2021, an increase of 6% as compared to $75.9 million for the full year in 2020. This increase is primarily due to an increase in costs related to Oral Paclitaxel, drug licensing costs, and cell therapy costs.

SG&A expenses totaled $13.4 million for the three months ended December 31, 2021, a decrease of 57% as compared to $31.4 million for the three months ended December 31, 2020. SG&A expenses totaled $72.6 million for the full year in 2021, a decrease of 22%, as compared to $92.9 million for the full year in 2020. This was primarily due to a $24.8 million decrease of costs for preparing to commercialize Oral Paclitaxel as significant pre-launch activities occurred in 2020 and slowed upon receipt of the Complete Response Letter in February 2021, offset by increased operating costs, professional fees and compensation expense.

The Company recorded impairment of $69.4 million, including goodwill impairment of $67.7 million, during the year ended December 31, 2021, based on the results of a quantitative goodwill impairment test for our reporting units.

Interest expense totaled $5.1 million and $4.4 million for the three months ended December 31, 2021 and 2020, respectively. Interest expense for the year ended December 31, 2021 totaled $20.7 million, an increase of $9.5 million, as compared to $11.2 million for the year ended December 31, 2020, primarily due to increased borrowings. Interest expense in the current period was incurred from the Senior Credit Agreement with Oaktree, while interest expense in the prior period was primarily incurred from debt under a former credit agreement with Perceptive Advisors LLC and its affiliates.

Income tax benefit (expense) for the three months ended December 31, 2021 amounted to ($15.0) thousand, compared to income tax expense of ($8.0) thousand for the same period in 2020. Income tax benefit totaled $10.6 million and income tax expense totaled ($4.1) million for the full year in 2021 and 2020, respectively. The income tax benefit in the current year is primarily the result of taxable temporary difference due to the deferred tax liability recognized for the indefinite lived intangible assets acquired in connection with the acquisition of Kuur’s in-process research and development. This taxable temporary difference is considered a source of taxable income to support the realization of deferred tax assets from the acquirer which resulted in a reversal of our valuation allowance. The income tax expense in the prior year was primarily attributable to foreign income tax withholdings on our revenue earned under our out-license arrangements.

Net loss attributable to Athenex for the three months and year ended December 31, 2021 were $104.4 million and $199.8 million, respectively, or ($0.95) and ($1.92) per diluted share, respectively, as compared to a net loss of $49.5 million and $146.2 million, or ($0.53) and ($1.72) per diluted share, for the same periods in 2020.

For further details on the Company’s financial results, including the results for the full year ended December 31, 2021, refer to the Form 10K filed with the SEC.

2022 Financial Guidance

Athenex’s product sales in 2021 amounted to $92.3 million, which represents 9% growth over the prior-year period, excluding one-time international sales of $21.0 million relating to the COVID pandemic. The Company expects 2022 year-over-year product sales growth to be in the range of 15-20%. While the Company does not give quarterly guidance, product sales are expected to increase throughout the year, as new products are launched.

Cash Conservation Update

As of December 31, 2021, the company had cash and cash equivalents of $35.2 million, restricted cash of $16.5 million, and short-term investments of $10.2 million, for a total of $61.9 million. The Company is implementing cost saving programs and monetizing non-core assets, resulting in plans to extend cash runway in 2022.

Conference Call and Webcast Information

Athenex will host a conference call and live audio webcast today, Wednesday, March 16, 2022, at 4:30 p.m. Eastern Time to discuss the financial results and provide a business update.

To participate in the call, dial either the domestic or international number fifteen minutes before the conference call begins:

The live conference call and replay can also be accessed via audio webcast here and on the Investor Relations section of the Company’s website under "Events and Presentations", located at View Source

On March 16, 2022 Provectus (OTCQB: PVCT) reported that data from an ongoing clinical trial of investigational cancer immunotherapy PV-10 (rose bengal sodium) for the treatment of neuroendocrine tumors (NET) metastatic to the liver (mNET) refractory to somatostatin analogs (SSAs) and peptide receptor radionuclide therapy (PRRT) (NCT02693067) was presented at the annual conference of the European Neuroendocrine Tumor Society (ENETS), held from March 10-11, 2022 in a hybrid setting in Barcelona, Spain and online (Press release, Provectus Biopharmaceuticals, MAR 16, 2022, View Source [SID1234610179]).

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The oral presentation was made by the principal investigator of the clinical trial’s single center at The Queen Elizabeth Hospital (TQEH) in Adelaide, Australia: Tim Price, MBBS, DHlthSc (Medicine), FRACP, Head of Clinical Oncology Research and Chair of the combined Hematology and Medical Oncology Unit at TQEH, and Clinical Professor in the Faculty of Medicine at the University of Adelaide.

Highlights from the 2022 ENETS Presentation:

Patient characteristics
N = 12 patients: 50% male; median age of 66 years (range 47-79)
Primary tumor sites: 7 small bowel (58%), 3 pancreas (25%), 1 caecal (8%), and 1 unknown (8%; likely pancreas)
NET grades: 5 Grade 1 (42%) and 7 Grade 2 (58%)
All patients were refractory to SSA (100%), and 11 received PRRT (92%) as part of their prior treatment
All patients had symptomatic, progressive disease
Baseline chromogranin A (CgA): median 1,585 µg/L (range 35-10,370)
PV-10 treatment
4 patients (33%) received more than 1 dose (range 2-4)
8 patients (67%) received 1 dose
Safety
All treatment-emergent adverse events (TEAEs) were Grade 1 or 2, primarily injection site pain (75%)
Single subjects experienced Grade 3 TEAEs of photosensitivity reaction or transaminases increased
Efficacy
Median progression-free survival (mPFS): 9.4 months (range 1.0-41.8)
Median overall survival (mOS): 22.5 months (range 5.5-42.3); 4 patients alive
Subgroup analysis of primary NET histology, pancreas vs intestine: mPFS 2.7 months vs 19.7 months; mOS 11.8 months vs 25.5 months
Quality of life (QOL)
Stable or improved health status and symptoms after 3 months in most patients
A recording of Dr. Price’s presentation and a copy of his slides are available on Provectus’ website at View Source

Dominic Rodrigues, Vice Chair of the Company’s Board of Directors, said, "Study data continue to show encouraging local and systemic disease control, as well as symptom control, in a heavily pre-treated population and to support a role for monotherapy PV-10 as a treatment for neuroendocrine cancer patients who fail standard therapy."

Mr. Rodrigues added, "PV-10-led combination therapies for neuroendocrine cancer patients may represent opportunities to improve quality of life and clinical outcomes. Front-line approaches may include combining liver-directed PV-10 therapy with systemically-delivered immune checkpoint inhibitors to enhance PV-10’s immune mechanisms, or using PV-10 to enhance the activity of cytotoxic treatments such as peptide receptor radionuclide therapy."

About PV-10

Intralesional (IL) administration of PV-10 for the treatment of solid tumor cancers can yield immunogenic cell death within hours of tumor injection, and induce tumor-specific reactivity in circulating T cells within days. This PV-10-induced functional T cell response may be enhanced and boosted in combination with immune checkpoint blockade (CB). In CB-refractory disease, PV-10 may restore disease-specific T cell function. IL PV-10 has been administered to over 450 patients with melanoma and cancers of the liver in both monotherapy and combination therapy settings. IL PV-10 is administered under visual, tactile, or ultrasound guidance to superficial malignancies, and under CT or ultrasound guidance to visceral hepatic tumors.

Systemic administration of PV-10 is undergoing preclinical study as prophylactic and therapeutic treatments for high-risk and refractory adult solid tumor cancers, and as a therapeutic treatment for relapsed and refractory blood cancers.

On March 15, 2022 IDEAYA Biosciences, Inc. (Nasdaq:IDYA), a synthetic lethality focused precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported financial results for the year ended December 31, 2021 (Press release, Ideaya Biosciences, MAR 15, 2022, View Source [SID1234610108]).

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"We are encouraged by the early clinical activity and the tolerability profile in our clinical-stage programs, including our Phase 1 MAT2A inhibitor, IDE397, in MTAP-deletion patients, and our Phase 2 PKC inhibitor, darovasertib, in MUM and other GNAQ/11 patients. As these data mature, we are evaluating multiple expansion opportunities for these clinical programs, including various combination therapies. We are also aggressively advancing our preclinical programs toward the clinic – including our potential first-in-class PARG inhibitor, IDE161, for which are targeting an IND in Q4 2022, and our potential first-in-class Pol Theta helicase inhibitor, for which we are collaborating with GSK with IND-enabling studies in H1 2022," said Yujiro S. Hata, Chief Executive Officer and President of IDEAYA Biosciences.

Program Updates
Key highlights for IDEAYA’s pipeline programs include:

IDE397 (MAT2A)
IDEAYA is evaluating IDE397, a potent and selective small molecule inhibitor targeting methionine adenosyltransferase 2a (MAT2A), in patients having solid tumors with methylthioadenosine phosphorylase (MTAP) deletion, a patient population estimated to represent approximately 15% of solid tumors. IDEAYA is leading early clinical development of IDE397. Subject to exercise of its option, GlaxoSmithKline (GSK) will lead later stage global clinical development. Highlights:

Actively enrolling patients into Cohort 6 of the Phase 1 clinical trial IDE397-001 (NCT04794699)
Patients are being identified by next generation sequencing (NGS) or by MTAP immunohistochemistry (IHC) assay with confirmatory NGS
Evaluating IDE397 in patients with MTAP deletion across multiple solid tumor types, including non-small cell lung cancer, pancreatic cancer, thymic cancer, adenoid cystic carcinoma, esophagogastric cancer and bladder cancer
IDE397 has been generally well tolerated, with no observed drug-related serious adverse events, no observed dose limiting toxicities and without observing the maximum tolerated dose through Cohort 5
Observed dose-proportional pharmacokinetic exposures across dose ranges of Cohort 1 through Cohort 5 of the Phase 1 dose escalation; achieved active exposure targets established from preclinical models at doses of Cohort 4 and Cohort 5
Observed exposure-dependent pharmacodynamic modulation of S-adenosyl methionine (SAM) in evaluable plasma samples across dose ranges of Cohort 1 through Cohort 5
Observed exposure-dependent pharmacodynamic modulation of symmetric dimethyl arginine (SDMA) in evaluable tumor biopsies from Cohort 4 and Cohort 5
Observed preliminary signals of clinical activity in MTAP-deletion patients in early dose escalation cohorts, including pharmacodynamic modulation and tumor shrinkage
Submitted a clinical protocol amendment to the FDA to support monotherapy cohort expansion in NSCLC, esophagogastric cancer, as well as one or more basket cohorts, and to support potential combinations, including taxane and other combination agents
Targeting IDE397 monotherapy cohort expansion and initiation of combination cohorts mid-year 2022, with an aggregate of 150 or more patients across expansion cohorts; the timing of the monotherapy expansion cohorts and combination cohorts may be influenced by the timing of when a MTD is observed
Targeting delivery of IDE397 option data package to GSK mid-year 2022, subject to initiation of expansion cohorts or establishing the MTD; the option data package will trigger an evaluation period for GSK to make an opt-in decision; subject to GSK election to opt-in and HSR clearance, the company is entitled to receive a $50 million opt-in payment from GSK, ongoing development costs will be shared as 80% GSK / 20% IDEAYA, and IDEAYA is entitled to potential development and regulatory milestones aggregate up to $465 million; upon commercialization, IDEAYA is entitled to 50% of U.S. net profits and tiered royalties on global non-U.S. net sales ranging from high single digit to sub-teen double digit percentages, as well as certain commercial milestones of up to $475 million
Demonstrated robust preclinical PK / PD in in vivo models, with sustained target inhibition and biological impact, as evidenced for example, by modulation of alterations to pre-mRNA splicing profile
Observed preclinical in vivo efficacy of IDE397 in combination with standard of care agents, including with taxanes showing enhanced TGI in pancreatic cancer PDX models, and with novel combination agents; evaluating additional IDE397 combination strategies
PARG
IDEAYA is advancing preclinical research for an inhibitor of poly (ADP-ribose) glycohydrolase (PARG) in patients having tumors with a defined biomarker based on genetic mutations and/or molecular signature. PARG is a novel target in the same clinically validated biological pathway as poly (ADP-ribose) polymerase (PARP). IDEAYA owns or controls all commercial rights in its PARG program. Highlights:

Ongoing IND-enabling studies for IDE161, a potential first-in-class PARG inhibitor development candidate for patients having tumors with homologous recombination deficiencies (HRD), including BRCA1 and BRCA2, and potentially other genetic alterations
Targeting IND for IDE161 in the fourth quarter of 2022
Exercised option for an exclusive worldwide license from Cancer Research Technology Ltd., also known as Cancer Research UK (CRUK), and University of Manchester; following the option exercise, IDEAYA holds an exclusive worldwide license to patent rights covering a broad class of PARG inhibitors
Demonstrated preclinical in vivo efficacy as monotherapy, including dose-dependent efficacy with tumor regression or stasis in ovarian, gastric and breast cancer CDX models, and tumor regressions in multiple breast cancer PDX models with defined genetic and subtyping profiles
Observed in vivo efficacy with enhanced TGI or tumor regressions relative to niraparib, a PARPi, in multiple CDX models, including in a niraparib-resistant CDX model, as well as in niraparib resistant breast cancer PDX models
Showed pharmacological inhibition of PARG in a panel of homologous recombination deficient cell lines and in CDX and PDX models; study data reported at AACR (Free AACR Whitepaper) 2021
Pol Theta
IDEAYA’s DNA Polymerase Theta, (Pol Theta) program targets tumors with BRCA or other homologous recombination deficiency, or HRD, mutations. IDEAYA and GSK are collaborating on ongoing preclinical research, including small molecules and protein degraders, and GSK will lead clinical development for the Pol Theta program. Highlights:

Demonstrated in vivo efficacy with tumor regression in BRCA2 -/- xenograft model with IDEAYA Pol Theta Helicase inhibitor in combination with niraparib, a GSK PARP inhibitor
Targeting IND-enabling studies for a Pol Theta helicase inhibitor in the first half of 2022 in collaboration with GSK
Potential for up to $20 million in aggregate milestone payments from GSK for advancing a Pol Theta Helicase inhibitor from preclinical to early Phase 1 clinical
Werner Helicase
IDEAYA is advancing preclinical research for an inhibitor targeting Werner Helicase for tumors with high microsatellite instability (MSI). IDEAYA and GSK are collaborating on ongoing preclinical research, and GSK will lead clinical development for the Werner Helicase program. Highlights:

Observed dose-dependent cellular viability effect and dose-dependent cellular PD response in multiple endogenous MSI high cell lines
Demonstrated efficacy and PD response in relevant MSI high in vivo models
Targeting selection of a Werner Helicase development candidate in 2023
Potential for up to $20 million in aggregate milestone payments from GlaxoSmithKline for advancing a Werner Helicase inhibitor from preclinical to early Phase 1 clinical
Other Synthetic Lethality Pipeline Programs
IDEAYA is advancing additional preclinical research programs to identify small molecule inhibitors for an MTAP-synthetic lethality target, as well as for multiple potential first-in-class synthetic lethality programs for patients with solid tumors characterized by proprietary biomarkers or gene signatures.

Darovasertib (IDE196)
IDEAYA continues to execute on its clinical trial strategy to evaluate darovasertib (IDE196), a potent and selective PKC inhibitor.

IDEAYA is evaluating darovasertib in combination with crizotinib, a cMET inhibitor, in metastatic uveal melanoma (MUM). The company is also clinically evaluating darovasertib as a combination with crizotinib in GNAQ/11 mutant skin melanoma in an ongoing arm of the current clinical trial, and in adjuvant primary uveal melanoma (UM) as monotherapy through an investigator sponsor clinical trial (IST). IDEAYA is also evaluating other potential darovasertib expansion opportunities, including in cMET driven tumors and in KRAS-mutation tumors.

Darovasertib / Crizotinib Combination Therapy
IDEAYA is continuing patient enrollment into the darovasertib / crizotinib combination arm of the Phase 1/2 clinical trial under clinical trial collaboration and supply agreements with Pfizer. Highlights:

As of March 1, 2022, the company has enrolled 53 MUM patients into the darovasertib/crizotinib combination arm, and is continuing patient enrollment in the dose expansion cohort of this combination arm
IDEAYA presented darovasertib and crizotinib clinical combination data in December 2021. As of data and analyses cutoff on November 25, 2021, twenty-two heavily pre-treated MUM patients (91% with prior therapies, and 59% with 2 or more prior therapies) had enrolled in the darovasertib and crizotinib combination arm at the expansion dose, with sixteen evaluable patients who had received one or more tumor scans and six patients who were awaiting their 1st tumor scan. Thirteen patients had received two or more tumor scans for evaluation of potential response. The reported preliminary data, based on an unlocked database, showed robust clinical activity with manageable side effect profile:
100% Disease Control Rate (DCR): 16 of 16 evaluable patients with >1 post-baseline scan showed tumor shrinkage as determined by target lesion size reduction
31% Overall Response Rate (ORR): 4 of 13 patients with > 2 post-baseline scans had a confirmed partial response (PR) as determined by RECIST 1.1 based on investigator or central review; and no patients have come off-treatment prior to the 2nd scan
46% of patients (6 of 13) with > 2 post-baseline scans observed >30% tumor reduction, including one patient with an unconfirmed PR as determined by RECIST 1.1 is awaiting follow-on tumor scan
Observed side effect profile in MUM patients (n=22) showed a low rate of drug-related serious adverse events (SAE’s) and predominantly Grade 1 or 2 drug-related adverse events; eighteen patients experienced a drug-related AE, of which six patients observed Grade 3, and no patients observed Grade 4 or Grade 5
These data provide clinical proof-of-concept for the darovasertib and crizotinib synthetic lethal combination treatment, and are consistent with the company’s translational research discovery that Phase 1 clinical response to darovasertib monotherapy associated with low cMET activity, as measured by gene signature score
The company is targeting a clinical data update for darovasertib and crizotinib combination in mid-2022, including tolerability and clinical efficacy. IDEAYA is also planning to seek FDA regulatory guidance for potential registration-enabling trial design to evaluate darovasertib and crizotinib combination in MUM in mid-2022. The timing of the clinical data and FDA regulatory guidance may be influenced by data maturity, including observation of median duration of response (DOR) or median progression free survival (mPFS).
Expanded relationship with Pfizer under a clinical collaboration and supply agreement to support clinical evaluation of darovasertib and crizotinib combination in a potential registration-enabling clinical trial in MUM, subject to FDA feedback and guidance
Associated cMET expression and activation to observed clinical response based on a retrospective analysis of human clinical biopsies from the Novartis darovasertib Phase 1 clinical trial, supporting cMET expression / activation as potential combination agent
Observed preclinical synergies between darovasertib and crizotinib in relevant cellular models under conditions simulating a tumor microenvironment in the liver, the site of approximately 90% of uveal melanoma metastases; study data reported at AACR (Free AACR Whitepaper) 2021
Darovasertib Monotherapy
IDEAYA has completed enrollment into its ongoing Phase 1/2 clinical trial evaluating darovasertib as monotherapy in MUM patients.

IDEAYA is planning to initiate an Investigator Sponsored Trial, with St. Vincent’s Hospital Sydney Limited to evaluate IDE196 as monotherapy in a neo-adjuvant / adjuvant setting in (non-metastatic) uveal melanoma (UM) patients. Data from this clinical trial may offer proof of concept on its hypothesis that earlier treatment of UM patients with IDE196, prior to tumor metastasis, may lead to improved patient outcomes.

Darovasertib – Other Potential Indications
IDEAYA is evaluating the potential for darovasertib in other oncology indications, including in cMET-driven tumors and in KRAS-mutation tumors. The company is also evaluating darovasertib for potential treatment of GNAQ mutation-mediated rare diseases, including Sturge-Weber Syndrome (SWS) and Port Wine Stains (PWS), neurocutaneous disorders characterized by capillary malformations and associated with mutations in GNAQ. Highlights:

Expanded its relationship with Pfizer under a clinical collaboration and supply agreement for clinical evaluation of darovasertib and crizotinib combination therapy in cMET-driven tumors, such as NSCLC or HCC, subject to preclinical validation studies
Evaluating darovasertib in combination with a KRAS inhibitor in preclinical studies in KRAS-driven solid tumors
General

IDEAYA continues to monitor Covid-19 and its potential impact on clinical trials and timing of clinical data results. Initiation of clinical trial sites, patient enrollment and ongoing monitoring of enrolled patients, including obtaining patient computed tomography (CT) scans, may be impacted for IDEAYA clinical trials evaluating IDE397 and darovasertib; the specific impacts are currently uncertain.

Corporate Updates

IDEAYA’s net losses were $49.8 million and $34.5 million for the years ended December 31, 2021 and December 31, 2020, respectively. As of December 31, 2021, the company had an accumulated deficit of $176.7 million.

As of December 31, 2021, IDEAYA had cash, cash equivalents and marketable securities of $368.1 million. IDEAYA believes that its cash, cash equivalents and marketable securities will be sufficient to fund its planned operations into 2025. These funds will support the company’s efforts through potential achievement of multiple preclinical and clinical milestones across multiple programs.

Our updated corporate presentation is available on our website, at our Investor Relations page: View Source

Financial Results

As of December 31, 2021, IDEAYA had cash, cash equivalents and short-term and long-term marketable securities totaling $368.1 million. This compared to cash, cash equivalents and short-term and long-term marketable securities of $283.6 million at December 31, 2020. The increase was primarily due to $86.0 million in net proceeds received from issuance of common stock in an underwritten public offering on July 12, 2021 and $57.3 million in net proceeds under the ATM Program received through December 31, 2021, offset by cash used in operations and purchases of property and equipment.

Collaboration revenue for the three months ended December 31, 2021 totaled $3.0 million compared to $10.6 million for the same period in 2020. Collaboration revenue was recognized for the performance obligations satisfied through December 31, 2021 under the GSK Collaboration Agreement.

Research and development (R&D) expenses for the three months ended December 31, 2021 totaled $16.1 million compared to $12.1 million for the same period in 2020. The increase was primarily due to higher personnel-related expenses, laboratory supplies expenses and consulting fees.

General and administrative (G&A) expenses for the three months ended December 31, 2021 totaled $5.2 million compared to $3.8 million for the same period in 2020. The increase was primarily due to higher personnel-related expenses, software expenses and consulting fees.

The net loss for the three months ended December 31, 2021 was $18.2 million compared to $5.1 million for the same period in 2020. Total stock compensation expense for the three months ended December 31, 2021 was $2.1 million compared to $1.0 million for the same period in 2020.

The net loss for the year ended December 31, 2021 was $49.8 million compared to $34.5 million for the same period in 2020. Total stock compensation expense for the year ended December 31, 2021 was $8.2 million compared to $3.6 million for the same period in 2020.

On March 15, 2022 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage biopharmaceutical company, reported financial results for the fourth quarter and full year ended December 31, 2021 and provided a corporate update (Press release, Altimmune, MAR 15, 2022, View Source [SID1234610124]).

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"We expect the next 12 months to be a period of intense execution with value-creating data readouts from multiple clinical trials. We are extremely pleased with the progress of our pemvidutide (ALT-801) program as we showed double-digit weight loss after 12 weeks of treatment, good tolerability without the use of dose titration, and pronounced decreases in serum lipids commonly associated with cardiovascular disease. We also observed a remarkable reduction in liver fat content to undetectable levels after only 6 weeks of pemvidutide treatment in subjects with hepatic steatosis, or fatty liver," said Vipin K. Garg, Ph.D., President and Chief Executive Officer at Altimmune. "In addition to our ongoing 12-week Phase 1b NAFLD trial, we expect to initiate a 48-week Phase 2 trial of pemvidutide in obesity, the MOMENTUM trial, in the next few weeks and look forward to sharing data from both of these trials later this year. In addition, we expect to read out our HepTcell trial in the first half of 2023."

Recent Highlights and Anticipated Milestones:

Pemvidutide1 (ALT-801)

Enrollment in Phase 1b nonalcoholic fatty liver disease (NAFLD) trial is over 90% complete, and data readout is expected in Q3 2022

The 12-week trial is being conducted at 15 sites in the U.S., with Dr. Stephen A. Harrison serving as Principal Investigator. The trial will be comprised of 72 non-diabetic and diabetic subjects across four treatment arms (pemvidutide 1.2, 1.8, 2.4 mg and placebo).
The primary efficacy readouts of this trial are liver fat reduction and weight loss.
A 52-week biopsy driven Phase 2 non-alcoholic steatohepatitis (NASH) trial is expected to follow the conclusion of the NAFLD trial.

Received U.S. Food and Drug Administration (FDA) clearance of pemvidutide investigational new drug application (IND) for obesity – Initiation of the Phase 2 MOMENTUM trial of pemvidutide in obesity expected in the first quarter of 2022

The trial is expected to enroll approximately 320 non-diabetic subjects with either obesity or overweight with at least one obesity-related complication. Subjects will be randomized 1:1:1:1 to receive either 1.2 mg, 1.8 mg, 2.4 mg pemvidutide or placebo administered weekly for 48 weeks.
The primary endpoint of the MOMENTUM trial is the relative (percent) change in body weight at 48 weeks compared to baseline, with additional readouts including metabolic and lipid profiles, cardiovascular measures and glucose homeostasis.
An interim analysis is planned to assess changes in body weight after 24 weeks of treatment, with an expected readout in Q4 2022.

Initiated a double-blind, placebo-controlled 12-week extension to the ongoing NAFLD Phase 1b Trial

This extension will allow subjects to receive up to a total of 24 weeks of pemvidutide or placebo and provide a read out on weight loss at 24 weeks.
Topline data from the extension trial expected in Q4 2022.
Completed 6-month and 9-month toxicology studies of pemvidutide in rats and non-human primates

No significant findings, including no ALT or blood glucose elevations, were reported.
Toxicology results support 24-week NAFLD extension and 48-week obesity studies.
Initiated a 12-week Phase 1 trial to characterize effects of pemvidutide on glucose control in diabetic population
This represents a follow-on to the evaluation of a pre-diabetic population in our first-in-human trial in which reductions of insulin resistance and maintenance of glucose control were observed.
Results of a drug-drug interaction trial of pemvidutide expected in the first half of 2022
HepTcell

Enrollment ongoing for the Phase 2 clinical trial in chronic hepatitis B subjects, with study readout expected H1 2023
Readouts from this trial are expected to include virological markers of hepatitis B infection and functional cure.
Financial Results for the Three Months Ended December 31, 2021

Altimmune had cash, cash equivalents, short-term investments and restricted cash totaling $190.3 million at December 31, 2021.
Revenue was $3.3 million for the three months ended December 31, 2021 compared to $2.3 million in the same period in 2020. The increase in revenue quarter over quarter was primarily due to the receipt of prior period rate adjustments under the Company’s U.S. government contract for NasoShield, partially offset by the discontinuation of development work under prior programs.
Research and development expenses were $20.2 million for the three months ended December 31, 2021, compared to $9.0 million in the same period in 2020. The change was primarily the result of the increased costs related to the development of pemvidutide and an increase in the contingent liability for stock-based milestone payments associated with the acquisition of pemvidutide, partially offset by the discontinuation of development work for prior clinical programs.
General and administrative expenses were generally consistent period-over-period with $3.8 million recognized for the three months ended December 31, 2021 and $4.1 million in the same period in 2020.
Net loss for the three months ended December 31, 2021 was $23.9 million, or $0.57 net loss per share, compared to $10.6 million in the same period in 2020, or $0.29 net loss per share, due to the factors noted above.
Conference Call Information

Date: Tuesday, March 15
Time: 8:30 am Eastern Time
Domestic Dial-in: (844) 615-6509
International Dial-in: (918) 922-3148
Conference ID: 4557398
Webcast: View Source
Following the conclusion of the call, the webcast will be available for replay on the Investor Relations page of the Company’s website at www.altimmune.com. The Company has used, and intends to continue to use, the IR portion of its website as a means of disclosing material non-public information and for complying with disclosure obligations under Regulation FD.

About Pemvidutide
Pemvidutide is a novel, investigational, peptide-based GLP-1/glucagon dual receptor agonist in development for the treatment of obesity and NASH. Activation of the GLP-1 and glucagon receptors is believed to mimic the complementary effects of diet and exercise on weight loss, with GLP-1 suppressing appetite and glucagon increasing energy expenditure. By combining GLP-1 and glucagon activity in a single peptide, pemvidutide has the potential to achieve weight loss comparable to bariatric surgery. Pemvidutide incorporates the EuPortTM domain, a proprietary technology that increases its serum half-life for weekly dosing while slowing the entry of pemvidutide into the bloodstream, which may improve its tolerability. In a 12-week Phase 1 clinical trial, pemvidutide-treated subjects demonstrated striking reductions in body weight, liver fat and serum lipids commonly associated with cardiovascular disease.

About HepTcell
HepTcell is a novel, investigational, immunotherapeutic comprised of nine synthetic peptides representing conserved hepatitis B (HBV) sequences formulated with IC31, a TLR9-based adjuvant from Valneva SE. The HBV-directed peptides are designed to drive T cell responses against all HBV genotypes towards a functional cure for chronic HBV in patients of diverse genetic backgrounds.

On March 15, 2022 PharmaJet, a biotech company that has developed a more effective way of administering drugs and biologics with their innovative, needle-free injection technology, reported that its partner Immunomic Therapeutics will be starting a phase 1 clinical study of their plasmid DNA vaccine ITI-3000 in patients with Merkel cell carcinoma (MCC), a rare but aggressive form of skin cancer (Press release, Immunomic Therapeutics, MAR 15, 2022, View Source [SID1234610140]). The study will be conducted at the University of Washington School of Medicine and the Fred Hutchinson Cancer Research Center in Seattle, Washington and will exclusively use the Stratis Needle-free injection System for delivery of the vaccine.

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"This therapeutic vaccine trial is the first of its kind in the world and may help address the fact that MCC recurs in 40% of cases after initial treatment, but no adjuvant therapy is approved for these patients," noted Dr. Paul Nghiem, co-lead of the clinical study, and Head of Dermatology at University of Washington. The PharmaJet Needle-free System was chosen due to its ability to precisely deliver the vaccine to the intramuscular tissue layer.

Chris Cappello, President and CEO, PharmaJet commented, "We are pleased to be collaborating with our partner in this important therapeutic DNA vaccine clinical trial for this aggressive skin cancer. Oncology applications are a very important part of our R&D pipeline. Our PharmaJet Needle-free Systems have been successfully used for the prevention or treatment of HPV-related lesions, Lymphoma and solid tumors, among other oncology indications."1