On May 5, 2022 ChemoCentryx, Inc., (Nasdaq: CCXI), reported financial results for the first quarter ended March 31, 2022 and provided an overview of recent corporate highlights (Press release, ChemoCentryx, MAY 5, 2022, View Source [SID1234613795]).

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"Strong performance was evident in the first quarter," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "The traction to-date is clear: all key performance indicators are up and Q1 revenue exceeded our model. A five-fold increase in net sales occurred versus Q4 2021; there was a nearly three-fold increase in unique prescribers, and a greater than three-fold increase of patients on drug this quarter. Further, referrals are coming from a growing number of first time as well as repeat prescribers. In my view, all of this shows that we are executing on plan – and we believe this is just the start for TAVNEOS.

Beyond TAVNEOS, compelling data now is emerging from the Phase I study of our orally administered PD-L1 immune checkpoint inhibitor, CCX559. The PK and PD data presented at the recent American Association of Cancer Research meeting show that CCX559 dosed once daily is pharmacologically active in cancer patients, and the data support the fact that 120 mg once a day is therapeutically relevant. We are on track to advance into the next phase of clinical development in the second half of 2022. A very promising start to the year in my view, one that shows both patients and shareholders alike that CCXI is fiercely dedicated to the proposition of improving lives and creating value."

Key First Quarter 2022 Highlights and Recent Developments

TAVNEOS (avacopan) Commercial Progress
Commercial Execution: TAVNEOS generated net product sales of approximately $5.4 million during the first quarter of 2022 from US commercial sales.
248 new patient start forms (PSFs) received for TAVNEOS during the first quarter of 2022.
281 unique prescribers to date.
277 patients on drug (POD) as of March 31, 2022.
Continued Traction Ex-US: TAVNEOS was approved in the European Union (EU) in January 2022, followed by an additional approval in Canada in April 2022 for the treatment of ANCA-associated vasculitis.
ChemoCentryx’s partner, Vifor Pharma, recently initiated marketing activities for TAVNEOS in Germany during February followed by Austria in March, and expects to launch in other markets in 2022. Vifor will pay ChemoCentryx royalties in the teens to the mid-20s percent on aggregate net sales from the Vifor territories outside the US.
The EU approval triggered a non-refundable $45 million milestone payment from Vifor. The cash payment has been received in full and is included in the Company’s reported cash balance. Collaboration revenue (distinct from product sales) will be recognized in the future as underlying development activities occur, in accordance with ASC606, which is currently estimated to be over a four year period, subject to adjustment from time to time.
TAVNEOS (avacopan) Clinical Development:
Lupus nephritis (LN): ChemoCentryx expects to receive feedback from the FDA mid-year 2022 regarding the clinical development plan for TAVNEOS in LN, which it plans to initiate during the second half of 2022.
Severe hidradenitis suppurativa (HS): The Company plans to meet with the FDA to discuss the Phase III development of TAVNEOS in patients with Hurley Stage III (severe) HS late in the second quarter, with the goal of initiating a Phase III clinical trial in those patients in the second half of 2022.
C3 glomerulopathy (C3G): The Company anticipates meeting with the FDA during the second half of the year to discuss the dataset from the ACCOLADE Phase II clinical trial of TAVNEOS in the very rare disorder of C3G, for which there are no FDA approved therapies.
CCX559 Clinical Development:
CCX559 is a novel, orally administered PD-L1 checkpoint inhibitor being developed for the treatment of various cancers, currently being evaluated in a Phase I dose escalation study. An orally administered small molecule inhibitor of PD-L1 could have advantageous properties compared to approved monoclonal antibodies, such as better penetration into solid tumors, reduced immunogenicity, lack of Fc-mediated side effects and convenience of oral administration.
At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 in April, ChemoCentryx reported preclinical data along with pharmacokinetic (PK) and pharmacodynamic (PD) data available from patients with advanced cancer who were enrolled in the first three dose cohorts of the ongoing Phase I study. Data show CCX559 is pharmacologically active in cancer patients and exhibits immune system activating properties.
ChemoCentryx intends to present additional clinical data, including further escalating dose level data, at major oncology conferences through 2022. The Company is on track to initiate a Phase Ib/II clinical trial to measure anti-tumor effects of CCX559 more directly during the second half of 2022.
Cash Position: The Company maintained a strong balance sheet with cash, cash equivalents and investments of approximately $371.8 million as of March 31, 2022. This includes a $45 million milestone received from Vifor Pharma in the first quarter of 2022 following the EU approval of TAVNEOS.
First Quarter 2022 Financial Results

TAVNEOS US net product sales were $5.4 million for the first quarter ended March 31, 2022. Collaboration and license revenue was $0.1 million for the quarter ended March 31, 2022, compared to $10.2 million in 2021. The decrease in collaboration revenue was attributable to the $10.0 million milestone received in 2021 for the acceptance of the Japanese NDA, for TAVNEOS in the treatment of ANCA-associated vasculitis.

Collaboration revenue is recognized ratably in proportion to actual costs incurred as a percentage of total program budgeted costs as the Company completes its performance obligations under its alliance agreements. The $45.0 million non-refundable regulatory milestone received upon TAVNEOS approval in Europe during the first quarter of 2022 is currently estimated to be recognized over a four year period, subject to adjustment from time to time.

Cost of sales for the first quarter ended March 31, 2022, was $0.2 million. Costs incurred for manufacturing campaigns initiated prior to the October 2021 FDA approval of TAVNEOS were recorded as research and development expense. Accordingly, cost of sales in the near term will likely be lower than in later periods given the sales of pre-approval inventory will carry little to no manufacturing costs since such costs were previously expensed to research and development expense.

Research and development expenses were $17.5 million for the first quarter of 2022, compared to $23.4 million for the same period in 2021. Based on the accounting treatment of pre-approval manufacturing campaigns described above, the decrease was primarily attributable to manufacturing costs of commercial drug supply in the first quarter of 2021. In addition, the completion of the TAVNEOS AURORA Phase IIb clinical trial in patients with HS in 2021 contributed to lower Phase II related expenses. These decreases were partially offset by higher research and drug discovery expenses, including those associated with the development of CCX559, our orally available small molecule checkpoint (PD-L1/PD-1) inhibitor.

Selling, general and administrative expenses were $26.0 million for the first quarter of 2022, compared to $16.3 million for the same period in 2021. The increase was primarily attributable to higher employee-related expenses and professional fees, including those associated with the Company’s launch and commercialization of TAVNEOS.

Net loss for the first quarter of 2022 was $38.6 million, compared to net loss of $29.7 million for the same period in 2021.

Total shares outstanding as of March 31, 2022, were approximately 71.1 million shares.

Cash, cash equivalents and investments totaled approximately $371.8 million as of March 31, 2022.

Conference Call and Webcast

The Company will host a conference call and webcast today, May 5, 2022, at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. To participate by telephone, please dial (877) 303-8028 (Domestic) or (760) 536-5167 (International). The conference ID number is 6597218. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the call.

INDICATION

TAVNEOS (avacopan) is indicated as an adjunctive treatment of adult patients with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (granulomatosis with polyangiitis [GPA] and microscopic polyangiitis [MPA]) in combination with standard therapy including glucocorticoids. TAVNEOS does not eliminate glucocorticoid use.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS
Serious hypersensitivity to avacopan or to any of the excipients

WARNINGS AND PRECAUTIONS
Hepatotoxicity: Serious cases of hepatic injury have been observed in patients taking TAVNEOS, including life-threatening events. Obtain liver test panel before initiating TAVNEOS, every 4 weeks after start of therapy for six months and as clinically indicated thereafter. Monitor patients closely for hepatic adverse reactions and consider pausing or discontinuing treatment as clinically indicated (refer to section 5.1 of the Prescribing Information). TAVNEOS is not recommended for patients with active, untreated and/or uncontrolled chronic liver disease (e.g., chronic active hepatitis B, untreated hepatitis C, uncontrolled autoimmune hepatitis) and cirrhosis. Consider the risk and benefit before administering this drug to a patient with liver disease.

Serious Hypersensitivity Reactions: Cases of angioedema occurred in a clinical trial, including one serious event requiring hospitalization. Discontinue immediately if angioedema occurs and manage accordingly. TAVNEOS must not be re-administered unless another cause has been established.

Hepatitis B Virus (HBV) Reactivation: Hepatitis B reactivation, including life threatening hepatitis B, was observed in the clinical program. Screen patients for HBV. For patients with evidence of prior infection, consult with physicians with expertise in HBV and monitor during TAVNEOS therapy and for six months following. If patients develop HBV reactivation, immediately discontinue TAVNEOS and concomitant therapies associated with HBV reactivation and consult with experts before resuming.

Serious Infections: Serious infections, including fatal infections, have been reported in patients receiving TAVNEOS. The most common serious infections reported in TAVNEOS group were pneumonia and urinary tract infections. Avoid use of TAVNEOS in patients with active, serious infection, including localized infections. Consider the risks and benefits before initiating TAVNEOS in patients with chronic infection, at increased risk of infection or who have been to places where certain infections are common.

ADVERSE REACTIONS
The most common adverse reactions (≥5% of patients and higher in the TAVNEOS group vs. prednisone group) were: nausea, headache, hypertension, diarrhea, vomiting, rash, fatigue, upper abdominal pain, dizziness, blood creatinine increased and paresthesia.

DRUG INTERACTIONS
Avoid coadministration of TAVNEOS with strong and moderate CYP3A4 enzyme inducers. Reduce TAVNEOS dose when co-administered with strong CYP3A4 enzyme inhibitors to 30 mg once daily. Monitor for adverse reactions and consider dose reduction of certain sensitive CYP3A4 substrates.

Please see Full Prescribing Information and Medication Guide for TAVNEOS.

About TAVNEOS (avacopan)

TAVNEOS (avacopan), approved by the FDA as an adjunctive treatment of ANCA-associated vasculitis, is a first-in-class, orally administered small molecule that employs a novel, highly targeted mode of action in complement-driven autoimmune and inflammatory diseases. While the precise mechanism in ANCA vasculitis has not been definitively established, TAVNEOS, by blocking the complement 5a receptor (C5aR) for the pro-inflammatory complement system fragment known as C5a on destructive inflammatory cells such as blood neutrophils, is presumed to arrest the ability of those cells to do damage in response to C5a activation, which is known to be the driver of ANCA vasculitis. TAVNEOS’s selective inhibition of only the C5aR is believed to leave the beneficial C5a pathway through the C5L2 receptor functioning normally.

ChemoCentryx is also developing TAVNEOS for the treatment of patients with C3 glomerulopathy (C3G), severe hidradenitis suppurativa (HS) and Lupus Nephritis (LN). The US Food and Drug Administration granted TAVNEOS orphan drug designation for ANCA-associated vasculitis and C3G. The European Commission has granted orphan medicinal product designation for TAVNEOS for the treatment of two forms of ANCA-associated vasculitis: microscopic polyangiitis and granulomatosis with polyangiitis (formerly known as Wegener’s granulomatosis), as well as for C3G. TAVNEOS has not been approved for indications discussed as in development, and the safety and efficacy of those uses has not been established.

About ANCA-Associated Vasculitis

ANCA-associated vasculitis is a systemic disease in which over-activation of the complement pathway further activates neutrophils, leading to inflammation and destruction of small blood vessels. This results in organ damage and failure, with the kidney as the major target, and is fatal if not treated. Currently, treatment for ANCA-associated vasculitis consists of courses of non-specific immuno-suppressants (cyclophosphamide or rituximab), combined with the administration of daily glucocorticoids (steroids) for prolonged periods of time, which can be associated with significant clinical risk including death from infection.

On May 5, 2022 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the first quarter ended March 31, 2022 and reiterated full year 2022 product revenue guidance (Press release, Vertex Pharmaceuticals, MAY 5, 2022, View Source [SID1234613793]).

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"Following upon our success in transforming the treatment of cystic fibrosis, Vertex’s unique and differentiated R&D strategy continues to deliver with positive Phase 2 proof-of-concept studies in multiple disease areas, and another wave of therapies set to enter the clinic in the second half of this year," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "With yet another quarter of strong revenue performance characterized by 22% year-over-year growth, we are well positioned for continued innovation and sustained growth as we work to bring additional transformative medicines to more patients around the globe."

Starting in the first quarter of 2022, Vertex no longer excludes research and development charges resulting from upfront or contingent milestone payments in connection with collaborations, asset acquisitions and/or licensing of third-party intellectual property rights from its Non-GAAP financial measures. Non-GAAP financial measures for the first quarter of 2021 have been recast to reflect this change.

Product revenues increased 22% to $2.10 billion compared to the first quarter of 2021, primarily driven by the strong launches of TRIKAFTA/KAFTRIO in multiple countries internationally and the strong performance of TRIKAFTA in the U.S., including the June 2021 launch of TRIKAFTA in children 6-11 years old in the U.S. Net product revenues in the first quarter of 2022 increased 9% to $1.37 billion in the U.S. and increased 55% to $729 million outside the U.S., compared to the first quarter of 2021.

GAAP and Non-GAAP net income increased compared to the first quarter of 2021, driven by strong product revenue growth.

Cash, cash equivalents and marketable securities as of March 31, 2022 were $8.2 billion, an increase of approximately $0.7 billion compared to December 31, 2021. The increase was primarily driven by strong revenue growth and operating cash flow.

Starting in the first quarter of 2022, Vertex no longer excludes research and development charges resulting from upfront or contingent milestone payments in connection with collaborations, asset acquisitions and/or licensing of third-party intellectual property rights from its Non-GAAP financial measures.

Key Business Highlights

Cystic Fibrosis (CF) Marketed Products

Vertex anticipates the number of CF patients treated with our medicines will continue to grow as the uptake of TRIKAFTA in the U.S. and the launches of KAFTRIO outside the U.S. continue, and as we enter into additional reimbursement agreements and achieve new approvals for the treatment of younger patients. Recent progress includes:

Health Canada granted marketing authorization for TRIKAFTA in children 6 to 11 years of age. With this approval, approximately 500 children with CF are newly eligible for treatment with a CFTR modulator.
Vertex signed a reimbursement agreement with the Australian Pharmaceutical Benefits Scheme for TRIKAFTA (ivacaftor/tezacaftor/elexacaftor) for the treatment of patients with cystic fibrosis 12 years and older with at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. With this agreement, approximately 700 people in Australia will have access to a CFTR modulator therapy for the first time.
Vertex completed enrollment in the Phase 3 study of TRIKAFTA/KAFTRIO in children 2 to 5 years old. The Company anticipates filing a supplementary new drug application (sNDA) with the FDA before the end of 2022.
In March, Vertex filed an sNDA with the FDA for ORKAMBI for the use of ORKAMBI in children 12 months to less than 24 months old. Vertex intends to submit regulatory filings in Europe in Q2 2022.
In January, the European Commission and the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) approved a label extension for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of CF in children ages 6 through 11 years old who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. With these approvals, approximately 1,900 children are newly eligible for KAFTRIO.
In 2021, Vertex presented the first long-term follow-up data for TRIKAFTA, demonstrating no loss in mean lung function at 96 weeks, a first for any CFTR modulator to date in people with F/F and F/MF mutations. Vertex has now completed a comparison of long-term data in TRIKAFTA patients to matched untreated controls, and will present these data at an upcoming medical congress.
TRIKAFTA/KAFTRIO is now approved and reimbursed or accessible in more than 25 countries.

R&D pipeline

Vertex is delivering on a diversified pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is summarized below.

Cystic Fibrosis

Vertex continues to pursue next-in-class, small molecule CFTR modulator therapies as well as new treatment options for the approximately 5,000 patients who cannot benefit from CFTR modulators alone.

Vertex is conducting two Phase 3 global, randomized, double-blind, active-controlled clinical trials (SKYLINE 102 and SKYLINE 103) evaluating Vertex’s new once-daily investigational triple combination of VX-121/tezacaftor/VX-561 in patients with CF. The SKYLINE 102 and SKYLINE 103 trials will compare the efficacy and safety of VX-121/tezacaftor/VX-561 to TRIKAFTA. More than 180 sites across both studies are open and enrolling, and enrollment in both trials is expected to be completed in late 2022 or early 2023.
In collaboration with Moderna, Vertex is developing CF mRNA therapeutics designed to treat the underlying cause of CF by programming cells in the lungs to produce functional CFTR protein for the treatment of the approximately 5,000 people with CF who do not produce any CFTR protein. IND-enabling studies have been completed, and Vertex is on track to submit an IND for this program in 2H 2022.
Beta Thalassemia and Sickle Cell Disease (SCD)

The CTX001 program employs a non-viral ex vivo CRISPR gene-editing therapy, which is being developed as a potential functional cure for transfusion-dependent thalassemia (TDT) and severe sickle cell disease (SCD). Vertex is developing CTX001 in collaboration with CRISPR Therapeutics.

Enrollment is complete in the ongoing Phase 3 clinical trials in TDT and SCD, with more than 75 patients dosed to date. Vertex anticipates presenting updated data from the clinical trials, with more patients and longer follow-up, at medical conferences in 2022.
Two new Phase 3 studies of CTX001 were initiated in pediatric patients with TDT and SCD.
Vertex plans to submit global regulatory filings for CTX001 in TDT and SCD in late 2022.
APOL1-Mediated Kidney Disease (AMKD)

Vertex has discovered multiple oral, small molecule inhibitors of APOL1 function, pioneering a new class of medicines that target an underlying genetic driver of kidney disease.

In March, Vertex initiated pivotal development of VX-147 in a single Phase 2/3 study in patients with AMKD with two APOL1 mutations and proteinuric kidney disease.
This Phase 2/3 adaptive study will first evaluate two doses of VX-147 to select a dose for Phase 3 and subsequently evaluate the efficacy and safety of the single, selected dose in the Phase 3 portion of the study. The primary efficacy endpoint for the final analysis is eGFR slope in patients receiving the VX-147 selected dose compared to placebo at two years. The study is designed to have a pre-planned interim analysis at Week 48 evaluating eGFR slope, supported by a percent change from baseline in proteinuria in the VX-147 arm versus placebo. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval of VX-147 in the U.S. for patients with AMKD.
Pain (NaV1.8)

Vertex has discovered multiple selective small molecule inhibitors of NaV1.8 with the objective of creating a new class of pain medicines that have the potential to provide effective pain relief, without the limitations of opioids.

In March, Vertex reported positive data from two Phase 2 dose ranging acute pain studies with VX-548, one following bunionectomy surgery and the other following abdominoplasty surgery. Both studies met their primary endpoint and established proof of concept for VX-548.
Vertex plans to advance VX-548 into pivotal development in acute pain in the second half of 2022, following discussions with regulators.
Type 1 Diabetes (T1D)

Vertex is evaluating cell therapies using stem cell-derived islets to replace the endogenous insulin-producing islet cells that are destroyed in people with T1D with the goal of developing a potential functional cure for this disease.

VX-880 is a stem cell-derived, fully differentiated islet replacement therapy, used in combination with standard immunosuppression to protect the implanted cells. VX-880 is being evaluated in a Phase 1/2 clinical trial for the treatment of T1D.
This program has been placed on clinical hold in the U.S. by the FDA, based on their determination of insufficient information for dose escalation with the product. Vertex is working collaboratively and with urgency to understand and address the FDA’s questions.
Vertex previously announced:
The first patient, who received a half dose of VX-880, is insulin-independent with an HbA1C of 5.2% at Day 270.
The second patient, who also received a half dose of VX-880, demonstrated restoration of glucose-responsive insulin production and significant improvement in glycemic control with reductions in exogenous insulin requirements.
Taken together, results in the first two patients establish proof-of-concept for VX-880 in the treatment of T1D. Per protocol, the Independent Data Monitoring Committee reviewed the totality of the safety data from the first two patients dosed and recommended advancement to Part B of the study, and treatment with the full target dose.
The third patient treated with VX-880 received the full target dose and has reached the Day 29 follow-up milestone.
Across the program, VX-880 has been generally well tolerated to date. There have been no serious adverse events (SAEs) considered related to VX-880. The majority of adverse events (AEs) were mild or moderate in all patients treated to date. The safety profile was generally consistent with the immunosuppressive regimen used in the study and the perioperative period.
Vertex is continuing to advance additional programs in T1D, in which these same stem cell-derived islets are encapsulated and implanted in an immunoprotective device or modified to produce hypoimmune stem cell islets with the goal of eliminating the need for immunosuppression.
Vertex is on track to submit an IND for the cells plus device program in 2022.
Vertex expects to share additional data for VX-880 at medical conferences this year.

Alpha-1 Antitrypsin (AAT) Deficiency

Vertex is working to address the underlying genetic cause of alpha-1 antitrypsin (AAT) deficiency by developing novel small molecule correctors of Z-AAT protein folding, with a goal of increasing the secretion of functional AAT into the blood and addressing both the lung and the liver aspects of AAT deficiency.

Vertex is on track to advance one or more novel small molecule Z-AAT correctors into the clinic in 2022.
Duchenne Muscular Dystrophy (DMD)

Vertex is investigating a novel approach to treating DMD by delivering CRISPR/Cas9 gene-editing technology to muscle cells with the goal of restoring near-full length dystrophin protein expression by targeting specific mutations in the dystrophin gene that cause the disease.

Vertex has advanced its first in vivo gene editing therapy for DMD into IND-enabling studies.
Consistent with its overall strategy, Vertex takes a portfolio approach to all of its programs, with additional assets in CF, SCD, Beta Thalassemia, AMKD, T1D, Pain, and AATD in earlier stages of development.

Non-GAAP Financial Measures

In this press release, Vertex’s financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex’s pre-tax income (i) stock-based compensation expense, (ii) gains or losses related to the fair value of the company’s strategic investments, (iii) increases or decreases in the fair value of contingent consideration, (iv) acquisition-related costs and (v) other adjustments. The company’s non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company’s business, are important in comparing current results with prior period results and provide additional information regarding the company’s financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company’s business and to evaluate its performance. The company’s calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information.

The company provides guidance regarding combined R&D and SG&A expenses and effective tax rate on a non-GAAP basis. The guidance regarding combined GAAP and non-GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development transactions, including collaborations, asset acquisitions and/or licensing of third-party intellectual property rights. The company does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material.

On MAY 5, 2022 INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, reported its financial results for the quarter ended March 31, 2022 and provided a business update (Press release, INmune Bio, MAY 5, 2022, View Source [SID1234613788]).

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Q1 2022 and Recent Corporate Highlights

DN-TNF Platform Highlights (XPro and INB03):

Dosed the first patient in the Phase II trial using XPro to treat patients with mild Alzheimer’s Disease (AD). The primary endpoint will examine cognition using the Early AD/MCI Alzheimer’s Cognitive Composite (EMACC). Data is anticipated in the second half of 2023.
Delivered multiple oral and poster presentations at AD/PD 2022 (International Conference on Alzheimer’s and Parkinson’s Disease).
Presented breast cancer data at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, which suggested the use of INmune Bio’s DN-TNF candidate, INB03, may help reverse resistance to immunotherapy patients with HER2+ cancers.
INKmune Platform Highlights:

Planned expansion of INKmune program to include AML and new site in EU.
2 of 3 patients with MDS/AML remain stable more than 6 months after the last treatment with INKmune.
INKmune shows best-in-class therapeutic persistence measured by activated tumor killing NK cells (NKG2D+ NK cells) in the peripheral blood or bone marrow more than 100 days after INKmune treatment in the two patients with stable disease
Highlighted the potential of INKmune primed NK cells to treat solid tumors by improving the ability of natural killer (NK) cells to survive hostile tumor microenvironments and by increased avidity, a necessary step for cancer cell killing, compared to NK cells primed with cytokines (IL-2, IL15).
Upcoming Milestones:

Initiate Xpro Phase 2 program for Mild Cognitive Impairment (MCI) in patients 1H 2022.
Initiate XPro Phase 2 program for treatment resistant depression (TRD), funded in part by a $2.9 million NIH grant, by 2H 2022.
Continued enrollment and opening of sites for XPro Phase 2 program for mild AD.
Initiate INKmune Phase 1 program in ovarian cancer or solid tumor in 2H 2022.
Additional open-label Phase 1 trial data of INKmune in high-risk MDS/AML.
Report top-line data from Phase 2 trial of Xpro in MCI patients in mid-2023.
Report top-line data from Phase 2 trial of XPro in Mild AD patients in 2H 2023.
Report pre-clinical INKmune data in renal cell carcinoma and nasopharyngeal carcinoma.
"Our scientific platforms continue to showcase our unique approach to repairing dysfunction of the innate immune system," stated RJ Tesi, M.D., CEO of INmune Bio. "In April, we announced the dosing of our first patient treated with XPro1595 ("XPro) in the treatment of neuroinflammation as a cause of mild Alzheimer’s disease (AD) in a Phase II clinical trial AD02, a blinded, randomized, placebo-controlled multicenter study in Australia, in Canada and in the United States that will assess the cognitive and functional impact of XPro in 201 mild AD patients. A second blinded, randomized, placebo-controlled Phase 2 trial in patients with mild cognitive impairment (MCI) is also planned. It appears that targeting amyloid and tau have little therapeutic benefit. Newer treatment strategies such as targeting glial dysfunction with XPro is supported by pre-clinical and epidemiologic studies. We expect top-line results from both clinical trials in 2023."

"The impact of inflammation and in particular TNF is increasingly apparent as evinced by recent genetic (link) studies," stated CJ Barnum, VP of CNS Development at INmune Bio. "These Phase 2 studies are the first step in determining the extent to which neutralizing solTNF will benefit MCI and AD patients. Our expectations are high, and we look forward to sharing the results next year.

"The clinical and scientific framework around INKmune, our NK cell targeting platform continues to grow. We have already observed that two of the three patients treated with INKmune for MDS/AML remain off therapy with their disease controlled for more than 6 months since their last dose of INKmune," said Dr. Mark Lowdell, Chief Scientific Officer of INmune Bio. "We are demonstrating the effectiveness of INKmune in treatment of hematologic malignancies but believe the most promising application will be using INKmune to treat residual disease in solid tumors."

"Separately, we presented additional pre-clinical breast cancer data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, which further demonstrated the use of our DN-TNF oncology drug candidate, INB03, to potentially help reverse resistance to immunotherapy for women with HER2+ breast cancer. With this robust collection of data in multiple indications, we are optimistic for the coming quarters and look forward to sharing our continued results with shareholders and clinicians alike," stated Dr. Tesi.

"We continue to move forward on our planned three Phase II programs this year and believe we are well positioned to deliver on potential value creating milestones in both our DN-TNF and INKmune platforms," concluded Dr. Tesi.

Financial Results for the Quarter Ended March 31, 2022:

Net loss attributable to common stockholders for the quarter ended March 31, 2022 was approximately $6.9 million, compared to approximately $4.6 million for the quarter ended March 31, 2021.

Revenues totaled approximately $0.2 million for the first quarter 2022 compared to approximately $0.0 million for the first quarter 2021.

Research and development expense totaled approximately $4.3 million for the first quarter 2022 compared to approximately $2.5 million during the first quarter 2021.

General and administrative expense was approximately $2.3 million for the first quarter 2022 compared to approximately $2.1 million during the first quarter 2021.

Other expense was approximately $0.4 million for the first quarter 2022 compared to approximately $0.0 million during the first quarter 2021.

As of March 31, 2022, the Company had cash and cash equivalents of approximately $66.7 million.

During the quarter, RJ Tesi, Mark Lowdell, David Moss and Kelly Ganjei bought 82,900 shares for approximately $700,000 as previously reported in an 8-K filed with the SEC. As of May 5, 2022, the Company had approximately 17.9 million common shares outstanding.

Earnings Call Information

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call.

Date: May 5, 2022
Time: 4:30 PM Eastern Time
Participant Dial-in: 1-877-407-0784
Participant Dial-in (international): 1-201-689-8560
Conference ID: 13728543

A live audio webcast of the call can be accessed using this link: View Source

A transcript will follow approximately 24 hours from the scheduled call. A replay will also be available through May 12, 2022 by dialing 1-844-512-2921 or 1-412-317-6671 (international) and entering PIN no. 13728543.

About XPro

XPro is a next-generation inhibitor of tumor necrosis factor (TNF) that is currently in clinical trial and acts differently than currently available TNF inhibitors in that it neutralizes soluble TNF (sTNF), without affecting trans-membrane TNF (tmTNF) or TNF receptors. XPro could have potential substantial beneficial effects in patients with neurologic disease by decreasing neuroinflammation. For more information about the importance of targeting neuroinflammation in the brain to improve cognitive function and restore neuronal communication visit this section of the INmune Bio’s website.

About INKmune

INKmune is a pharmaceutical-grade, replication-incompetent human tumor cell line which conjugates to resting NK cells and delivers multiple, essential priming signals akin to treatment with at least three cytokines in combination. INKmune is stable at -80oC and is delivered by a simple IV infusion. The INKmune:NK interaction ligates multiple activating and co-stimulatory molecules on the NK cell and enhances its avidity of binding to tumor cells; notably those resistant to normal NK-mediated lysis. Tumor-primed NK (TpNK) cells can lyse a wide variety of NK-resistant tumors including leukemias, lymphomas, myeloma, ovarian cancer, breast cancer.

On May 5, 2022 Celyad Oncology SA (Euronext & Nasdaq: CYAD) (the "Company"), a clinical-stage biotechnology company focused on the discovery and development of chimeric antigen receptor T cell (CAR T) therapies for cancer, reported an update on its financial results and recent business developments for the fiscal quarter ended March 31, 2022 (Press release, Celyad, MAY 5, 2022, View Source [SID1234613782]).

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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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"The first quarter of 2022 brought us both challenges and opportunities that we are facing head-on. While we continue to investigate the recent developments in the CYAD-101 Phase 1b trial, we are making great progress with our shRNA-based allogeneic programs, including CYAD-211, for which we anticipate announcing additional data during the second half of the year," commented Filippo Petti, Chief Executive Officer of the Company. "We are truly thankful for our hardworking team and the support of our shareholders while we advance towards our milestones for the year and further enhance our allogeneic CAR T investigational therapies with our proprietary non gene edited technologies."

Update on Clinical and Preclinical Programs

CYAD-211 – Allogeneic shRNA-based, anti-BCMA CAR T candidate for r/r MM

The dose-escalation Phase 1 IMMUNICY-1 trial is evaluating the tolerability and clinical activity of a single infusion of CYAD-211 following preconditioning with CyFlu (cyclophosphamide and fludarabine) in patients with relapsed / refractory multiple myeloma (r/r MM).
The current segment of the IMMUNICY-1 study is evaluating CYAD-211 following enhanced lymphodepleting (eLD) regimens with the aim to improve cell expansion and persistence and potentially maximize the clinical activity of CYAD-211. In addition, the IMMUNICY-1 protocol allows for redosing of CYAD-211 in certain patients.
Enrollment in the eLD cohorts of the IMMUNICY-1 trial continues with additional data expected from the program in the second half of 2022.
CYAD-101 – Allogeneic TIM-based, NKG2D CAR T Candidate for Metastatic Colorectal Cancer (mCRC)

In February 2022, the Company voluntarily paused the Phase 1b trial of CYAD-101 after two fatalities occurred that presented with similar pulmonary findings. Subsequently, in March 2022, the Company was informed by the U.S. Food and Drug Administration that the CYAD-101-002 Phase 1b trial had been placed on clinical hold.
The Company continues to investigate these findings in the CYAD-101-002 Phase 1b trial and is evaluating any similar events in additional patients treated in the study, while also working with appropriate regulatory authorities. The Company expects to provide additional updates on the trial in the future.
shRNA Armored CAR (shARC) Franchise

Research continues in multiple discovery programs focused on the co-expression of Interleukin-18 (IL-18) in conjunction with our short hairpin RNA (shRNA) technology platform, also known as our shARC (shRNA Armored CAR) franchise.
In April, the Company decided to stop the development of CYAD-203, an allogeneic shRNA-based, IL-18-armored NKG2D CAR T candidate following the analysis of preclinical data from multiple investigational new drug application (IND)-enabling studies. The Company continues to explore back-up allogeneic NKG2D receptor CAR T candidates currently in discovery stage that leverage the Company’s shARC platform.
First Quarter 2022 Financial Review

As of March 31, 2022, the Company had cash and cash equivalents of €20.5 million ($22.9 million). Net cash burn during the first quarter of 2022 amounted to €9.5 million ($10.6 million), in line with expectations. The Company confirms its previous guidance that its existing cash and cash equivalents, combined with the remaining access to the equity purchase agreement established with Lincoln Park Capital Fund, LLC, should be sufficient to fund operating expenses and capital expenditure requirements until mid-2023.

On May 5, 2022 Aravive, Inc. (Nasdaq: ARAV), a late clinical-stage oncology company developing targeted therapeutics to treat metastatic disease, reported that it will host a Key Opinion Leader (KOL) Symposium on the GAS6-AXL signaling pathway and the Company’s lead drug candidate, batiraxcept (Press release, Aravive, MAY 5, 2022, View Source [SID1234613756]). Senior management will also provide updates on Aravive’s clinical development programs. The event will take place on May 11, 2022, from 8:00 am ET to 12:00 pm ET and will include a live and archived webcast at: View Source

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The anticipated KOL Symposium schedule (all times are ET):

8:50 am – 9:00 am Opening Remarks
Gail McIntyre, Ph.D.; Aravive CEO and Director

9:00 am – 9:30 am Overview of the TAM Kinases:
Amato Giaccia, Ph.D.; Aravive Founder and Director Director, MRC Oxford Institute for Radiation Oncology Jack, Lulu and Sam Wilson Professor of Radiation Oncology, Emeritus, Stanford University

9:30 am – 10:00 am Batiraxcept in PROC:
Katherine Fuh, M.D., Ph.D.; Gynecologic Oncologist & Associate Professor Division of Gynecologic Oncology, Washington University, Siteman Cancer Center

10:00 am – 10:30 am Batiraxcept in ccRCC:
Brian Rini, M.D.; Chief of Clinical Trials, Vanderbilt-Ingram Cancer Center & Professor of Medicine, Division of Hematology/Oncology, Vanderbilt University Medical Center

10:30 am – 11:00 am Batiraxcept in Pancreatic Adenocarcinoma:
Paul Oberstein, M.D., M.S.; Associate Professor of Medicine and Director of Gastrointestinal Medical Oncology, Perlmutter Comprehensive Cancer Center, NYU Langone Health

11:00 am – 11:15 am Program Updates

11:15 am – 12:00 pm Panel Discussion and Q&A