On August 8, 2022 CTI BioPharma Corp. (Nasdaq: CTIC) repView Source [SID1234617777]).

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"CTI continues to make substantial headway with the U.S. commercial launch of VONJO, as we work to become the market leader in the treatment of cytopenic myelofibrosis. In the second quarter, we have performed well generating net product revenue of $12.3 million," said Adam Craig, President and Chief Executive Officer of CTI BioPharma. "With a differentiated profile, VONJO is a simple, safe and effective treatment that is a meaningful alternative to existing therapies. We look forward to extending the reach of VONJO over the coming months and years."

Recent Accomplishments and Updates

VONJO listed as recommended treatment in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for Myeloproliferative Neoplasms, as a first-line treatment for high-risk patients with myelofibrosis with platelet counts <50 x 109/L who are not candidates for transplant, and as a second-line treatment for lower-risk and higher-risk patients with myelofibrosis with platelet counts ≥50 x 109/L who are not candidates for transplant.
Planned data presentations at international medical meetings by end of year that demonstrate pacritinib’s activity as a potent ACVR1/ALK2 inhibitor as well as data on pacritinib’s substantial anemia benefit in myelofibrosis.
Data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Congress demonstrating that full dose pacritinib achieved higher response rates and a similar, manageable safety profile compared to lower-dose ruxolitinib in patients with myelofibrosis who have moderate or severe thrombocytopenia.
Data presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting demonstrating the safety profile of pacritinib 200 mg twice a day is comparable to best available therapy, including ruxolitinib, and that pacritinib 200 mg twice daily could be a full-dose therapeutic option for patients with myelofibrosis, including those who experience severe thrombocytopenia.
Filing of patent term extension application for the composition of matter U.S. Patent No. 8,153,632, with a requested five-year extension, which, if granted, would extend the expiration date of that patent from January 2029 to January 2034.
Second Quarter Financial Results

Net product sales of $12.3 million and $14.6 million for the three and six months ended June 30, 2022, respectively, were entirely attributable to VONJO product sales in the United States. There were no product sales for the comparable periods in 2021. Operating loss was $18.9 million and $19.5 million for the three months ended June 30, 2022 and 2021, respectively, and $54.0 million and $36.6 million for the six months ended June 30, 2022 and 2021, respectively. The decrease in operating loss between the three-month periods ended June 30, 2022 and 2021 was primarily attributable to net product sales, offset by an increase in selling, general and administrative activities related to the commercial launch of VONJO. The increase in operating loss between the six-month periods ended June 30, 2022 and 2021 resulted primarily from an increase in selling, general and administrative activities related to the commercial launch of VONJO, as well as a $10.3 million milestone expense related to FDA approval of VONJO, which was included in other operating expenses.

Net loss for the three months ended June 30, 2022 was $22.7 million, or $0.21 for basic and diluted loss per share, compared to net loss of $19.7 million, or $0.21 for basic and diluted loss per share, for the same period in 2021. Net loss for the six months ended June 30, 2022 was $59.8 million, or $0.57 for basic and diluted loss per share, compared to net loss of $36.9 million, or $0.44 for basic and diluted loss per share, for the same period in 2021.

As of June 30, 2022, our cash, cash equivalents and short-term investments totaled $95.9 million. We expect our present financial resources, along with expected cash receipts from net product sales of VONJO and up to $25.0 million in contractual funding commitments receivable upon achievement of minimum net product sales of VONJO under the terms of the previously-announced debt and royalty transactions with DRI Healthcare Trust, will enable us to fund our operations for at least one year.

Conference Call and Webcast

CTI will host a conference call and webcast to review its second quarter 2022 financial results and provide an update on business activities today, August 8, 2022, at 4:30 p.m. ET. To access the live call by phone please dial (888) 317-6003 (domestic), (855) 669-9657 (Canada) or (412) 317-6061 (international); the conference ID is 0078819. A live audio webcast of the event may also be accessed through the "Investors" section of CTI’s website at www.ctibiopharma.com. A replay of the webcast will be available for 30 days following the event.

About VONJO (pacritinib)

Pacritinib is an oral kinase inhibitor with activity against wild type Janus Associated Kinase 2 (JAK2), mutant JAK2V617F form and FMS-like tyrosine kinase 3 (FLT3), which contribute to signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. Myelofibrosis is often associated with dysregulated JAK2 signaling. Pacritinib has higher inhibitory activity for JAK2 over other family members, JAK3 and TYK2. At clinically relevant concentrations, pacritinib does not inhibit JAK1. Pacritinib exhibits inhibitory activity against additional cellular kinases (such as CSF1R and IRAK1), the clinical relevance of which is unknown.

VONJO is indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Important VONJO Safety Information

Hemorrhage:

Serious (11%) and fatal (2%) hemorrhages have occurred in VONJO-treated patients with platelet counts <100 × 109/L. Serious (13%) and fatal (2%) hemorrhages have occurred in VONJO-treated patients with platelet counts <50 × 109/L. Grade ≥3 bleeding events (defined as requiring transfusion or invasive intervention) occurred in 15% of patients treated with VONJO compared to 7% of patients treated on the control arm. Due to hemorrhage, VONJO dose reductions, dose interruptions, or permanent discontinuations occurred in 3%, 3%, and 5% of patients, respectively.

Avoid use of VONJO in patients with active bleeding and hold VONJO seven days prior to any planned surgical or invasive procedures. Assess platelet counts periodically, as clinically indicated. Manage hemorrhage using treatment interruption and medical intervention.

Diarrhea:

VONJO causes diarrhea in approximately 48% of patients compared to 15% of patients treated on the control arm. The median time to resolution in VONJO-treated patients was two weeks. The incidence of reported diarrhea decreased over time, with 41% of patients reporting diarrhea in the first eight weeks of treatment, 15% in weeks 8 through 16, and 8% in weeks 16 through 24. Diarrhea resulted in treatment interruption in 3% of VONJO-treated patients. None of the VONJO-treated patients reported diarrhea that resulted in treatment discontinuation. Serious diarrhea adverse reactions occurred in 2% of patients treated with VONJO compared to no such adverse reactions in patients in the control arm.

Control pre-existing diarrhea before starting VONJO treatment. Manage diarrhea with antidiarrheal medications, fluid replacement, and dose modification. Treat diarrhea with antidiarrheal medications promptly at the first onset of symptoms. Interrupt or reduce VONJO dose in patients with significant diarrhea despite optimal supportive care.

Thrombocytopenia:

VONJO can cause worsening thrombocytopenia. VONJO dosing was reduced due to worsening thrombocytopenia in 2% of patients with pre-existing moderate to severe thrombocytopenia (platelet count <100 × 109/L). VONJO dosing was reduced due to worsening thrombocytopenia in 2% of patients with pre-existing severe thrombocytopenia (platelet count <50 × 109/L).

Monitor platelet count prior to VONJO treatment and as clinically indicated during treatment. Interrupt VONJO in patients with clinically significant worsening of thrombocytopenia that lasts for more than seven days. Restart VONJO at 50% of the last given dose once the toxicity has resolved. If toxicity recurs hold VONJO. Restart VONJO at 50% of the last given dose once the toxicity has resolved.

Prolonged QT interval:

VONJO can cause prolongation of the QTc interval. QTc prolongation of >500 msec was higher in VONJO-treated patients than in patients in the control arm (1.4% vs 1%). QTc increase from baseline by 60 msec or higher was greater in VONJO-treated patients than in control arm patients (1.9% vs 1%). Adverse reactions of QTc prolongation were reported for 3.8% of VONJO-treated patients and 2% of control arm patients. No cases of torsades de pointes were reported.

Avoid use of VONJO in patients with a baseline QTc of >480 msec. Avoid use of drugs with significant potential for QTc prolongation in combination with VONJO. Correct hypokalemia prior to and during VONJO treatment. Manage QTc prolongation using VONJO interruption and electrolyte management.

Major Adverse Cardiac Events (MACE):

Another JAK)-inhibitor has increased the risk of MACE, including cardiovascular death, myocardial infarction, and stroke (compared to those treated with TNF blockers) in patients with rheumatoid arthritis, a condition for which VONJO is not indicated.

Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with VONJO, particularly in patients who are current or past smokers and patients with other cardiovascular risk factors. Patients should be informed about the symptoms of serious cardiovascular events and the steps to take if they occur.

Thrombosis:

Another JAK-inhibitor has increased the risk of thrombosis, including deep venous thrombosis, pulmonary embolism, and arterial thrombosis (compared to those treated with TNF blockers) in patients with rheumatoid arthritis, a condition for which VONJO is not indicated.

Patients with symptoms of thrombosis should be promptly evaluated and treated appropriately.

Secondary Malignancies:

Another JAK-inhibitor has increased the risk of lymphoma and other malignancies, excluding non-melanoma skin cancer (NMSC) (compared to those treated with TNF blockers), in patients with rheumatoid arthritis, a condition for which VONJO is not indicated. Patients who are current or past smokers are at additional increased risk.

Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with VONJO, particularly in patients with a known malignancy (other than a successfully-treated NMSC), patients who develop a malignancy, and patients who are current or past smokers.

Risk of Infection:

Another JAK-inhibitor has increased the risk of serious infections compared to best available therapy (BAT) in patients with myeloproliferative neoplasms. Serious bacterial, mycobacterial, fungal and viral infections may occur in patients treated with VONJO. Delay starting therapy with VONJO until active serious infections have resolved. Observe patients receiving VONJO for signs and symptoms of infection and manage promptly. Use active surveillance and prophylactic antibiotics according to clinical guidelines.

Interactions with CYP3A4 Inhibitors or Inducers:

Co-administration of VONJO with strong CYP3A4 inhibitors or inducers is contraindicated. Avoid concomitant use of VONJO with moderate CYP3A4 inhibitors or inducers.

Drug interruptions due to an adverse reaction occurred in 27% patients who received VONJO 200 mg twice daily compared to 10% of patients treated with BAT. Dosage reductions due to an adverse reaction occurred in 12% of patients who received VONJO 200 mg twice daily compared to 7% of patients treated with BAT. Permanent discontinuation due to an adverse reaction occurred in 15% of patients receiving VONJO 200 mg twice daily compared to 12% of patients treated with BAT.

Please visit View Source for full Prescribing Information and the Medication Guide.

About Myelofibrosis

Myelofibrosis is bone marrow cancer that results in formation of fibrous scar tissue and can lead to thrombocytopenia and anemia, weakness, fatigue and an enlarged spleen and liver. Within the United States, there are approximately 21,000 patients with myelofibrosis, 7,000 of which have severe thrombocytopenia (defined as blood platelet counts of less than 50 x109/L). Severe thrombocytopenia is associated with poor survival and high symptom burden and can occur as a result of disease progression or from drug toxicity with other JAK2 inhibitors, such as JAKAFI and INREBIC.

On August 8, 2022 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results and provided a business update for the second quarter ended June 30, 2022 (Press release, Syndax, AUG 8, 2022, View Source [SID1234617776]).

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"The coming quarters are poised to be transformational for Syndax with topline data from both the revumenib and axatilimab pivotal programs expected starting in the first half of 2023," said Michael A. Metzger, Chief Executive Officer. "As we continue to advance these programs in areas of significant unmet need, we are focused on executing a broad clinical development plan that fully realizes the potential of both compounds and builds upon the robust datasets presented to date."

"For revumenib, which we believe is positioned to serve as a first-to-market and best-in-class menin inhibitor for patients with mNPM1 and MLLr acute leukemias, we look forward to sharing updated data from the Phase 1 portion of the AUGMENT-101 trial, enrolling patients with relapsed/refractory (R/R) disease, in the fourth quarter of this year. Beyond the R/R setting, we are committed to creating additional value for the revumenib program by expanding into newly diagnosed and maintenance settings in mNPM1 and MLLr acute leukemias, as well as into colorectal cancer (CRC), our first assessment of revumenib in solid tumors. We also expect similarly broad utility with axatilimab, which we believe could have a meaningful impact in multiple fibrotic diseases. Building on the data in chronic graft versus host disease (cGVHD), we are looking forward to initiating a 52-week Phase 2b trial of axatilimab in patients with idiopathic pulmonary fibrosis (IPF) in the fourth quarter of this year."

Recent Pipeline Progress and Anticipated Milestones

Revumenib

The pivotal Phase 2 portion of AUGMENT-101 is ongoing and the Company continues to expect completion of enrollment in one of the three pivotal cohorts by year-end. The trials are enrolling a total of 64 adult and up to 10 pediatric patients across each of three distinct trial populations: patients with NPM1 mutant acute myeloid leukemia (AML), patients with MLLr AML, and patients with MLLr acute lymphocytic leukemia (ALL). Based on discussions with the U.S. Food and Drug Administration, AUGMENT-101 may serve as the basis for regulatory filings in each of the three distinct populations. The Company expects to report topline data from the trials starting in the first half of 2023, with the potential for the first New Drug Application filing later in 2023. The Company also anticipates announcing updated data from the Phase 1 portion of the AUGMENT-101 trial in the fourth quarter of 2022.
Two trials, BEAT-AML and AUGMENT-102, are ongoing and will assess the safety, tolerability, and preliminary anti-leukemic efficacy of revumenib and establish an appropriate Phase 2 dose when used in combination with other approved agents. BEAT-AML is a front-line combination trial of revumenib with venetoclax and azacitidine being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial. AUGMENT-102 is a trial assessing revumenib in combination with chemotherapy in patients with R/R mNPM1 or MLLr acute leukemias.
The Company expects the Australasian Leukaemia and Lymphoma Group (ALLG) to initiate the INTERCEPT trial of revumenib as monotherapy in patients with AML who are minimal residual disease-positive (MRD+) following initial treatment, in the fourth quarter of 2022. The trial is a part of the INTERCEPT AML Master Clinical Trial, a collaborative clinical trial investigating novel therapies to target early relapse and clonal evolution as pre-emptive therapy in AML. Revumenib is the first menin inhibitor to be included in the INTERCEPT AML Master Clinical Trial.
The Company previously announced it intends to initiate a proof-of-concept clinical trial of revumenib in patients with unresectable metastatic microsatellite stable CRC in the fourth quarter of 2022.
Axatilimab

Enrollment is ongoing in the Company’s global pivotal Phase 2 AGAVE-201 trial of axatilimab in patients with cGVHD. The trial is evaluating the safety and efficacy of three dosing regimens of axatilimab. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for cGVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score. The Company remains on track to report topline data in the first half of 2023, with the potential for a Biologics License Application filing later in 2023.
The Company plans to initiate a Phase 2b trial to assess the efficacy, safety and tolerability of axatilimab in patients with IPF in the fourth quarter of 2022. This 52-week, randomized, double-blind and placebo-controlled trial is expected to enroll approximately 170 patients. The primary endpoint will assess the change from baseline in forced vital capacity, which is the current registrational endpoint in IPF.
The Company is working with its partner, Incyte, to plan additional trials of axatilimab in earlier lines of cGVHD, and expects that Incyte will initiate a Phase 1 trial of axatilimab in combination with Jakafi in patients with steroid-refractory cGVHD in the fourth quarter of 2022.
Corporate Updates

In June 2022, the Company announced the appointment of Keith A. Goldan as Chief Financial Officer. Mr. Goldan brings to Syndax nearly thirty years of leadership and operational experience at several pharmaceutical, biotechnology, and medical technology companies.
Second Quarter 2022 Financial Results

As of June 30, 2022, Syndax had cash, cash equivalents, short-term and long-term investments of $378.9 million and 60.4 million shares outstanding, that included 4.0 million pre-funded warrants.

Second quarter 2022 research and development expenses increased to $29.7 million from $16.9 million for the prior year period. The increase was primarily due to increased clinical and manufacturing activities for revumenib and axatilimab.

General and administrative expenses for the second quarter 2022 increased to $8.0 million from $5.8 million for the prior year period. The increase is primarily due to increased employee related expenses and professional fees.

For the three months ended June 30, 2022, Syndax reported a net loss attributable to common stockholders of $37.6 million, or $0.62 per share, compared to a net loss attributable to common stockholders of $22.9 million, or $0.44 per share, for the prior year period.

Financial Update and Guidance

For the third quarter of 2022, the Company expects research and development expenses to be $25 to $30 million, and total operating expenses to be $35 to $40 million. For the full year of 2022, the Company continues to expect research and development expenses to be $130 to $140 million and total operating expenses to be $160 to $170 million.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, August 8, 2022.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.

On August 8, 2022 (GLOBE NEWSWIRE) — Chinook Therapeutics, Inc. (Nasdaq: KDNY), a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, reported financial results for the quarter and six months ended June 30, 2022 (Press release, Aduro Biotech, AUG 8, 2022, View Source [SID1234617775]).

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"During the second quarter of 2022, we executed well on advancing our pipeline of clinical and preclinical programs for rare, severe chronic kidney diseases. We continue to ramp up enrollment of patients in the phase 3 ALIGN trial for atrasentan, and the data we presented at ERA from the ongoing phase 2 AFFINITY trial of atrasentan demonstrated consistent and clinically meaningful proteinuria reductions in patients with IgAN," said Eric Dobmeier, president and chief executive officer of Chinook Therapeutics. "For BION-1301, the additional data presented at ERA from the ongoing phase 1/2 trial reaffirms its disease-modifying potential in IgAN by demonstrating durable reductions in mechanistic biomarkers and corresponding impressive proteinuria reductions. We look forward to advancing BION-1301 into a phase 3 study for patients with IgAN in 2023. We are also continuing to make progress with dose escalation in the ongoing phase 1 trial of CHK-336 in healthy volunteers and expect to report data in the first half of 2023."

Recent Accomplishments and Updates

Atrasentan
Atrasentan is a potent and selective endothelin A (ETA) receptor antagonist that has the potential to provide benefit in multiple chronic kidney diseases by reducing proteinuria and having direct anti-inflammatory and anti-fibrotic effects to preserve kidney function. The phase 3 ALIGN trial of atrasentan is currently enrolling patients with IgAN, and the phase 2 AFFINITY basket trial of atrasentan is currently enrolling patients with proteinuric glomerular diseases.

Enrollment of the phase 3 ALIGN trial of atrasentan continues to advance with the activation of new trial sites and expansion into additional countries. Chinook expects to report topline data from the six-month interim proteinuria endpoint analysis in 2023 to support an application for accelerated approval under Subpart H in the United States.

Chinook presented data from the IgAN patient cohort of the phase 2 AFFINITY trial in an oral presentation at the 59th ERA Congress in May 2022, demonstrating consistent and clinically meaningful proteinuria reductions at weeks six, 12 and 24 of treatment in patients with IgAN already on a maximally tolerated and stable dose of a RAS inhibitor. Specifically, atrasentan demonstrated a 38.0% geometric mean reduction in 24-hour urine protein creatinine ratio (UPCR) in 20 patients at six weeks of treatment, 49.9% geometric mean reduction in 24-hour UPCR in 18 patients at 12 weeks of treatment and 58.5% geometric mean reduction in 24-hour UPCR in 11 patients at 24 weeks of treatment. After 24 weeks of treatment, ten of the 11 patients (91%) who had completed this visit had greater than a 40% cumulative reduction in UPCR. There were no meaningful changes in blood pressure or acute eGFR effects, suggesting proteinuria reductions were not primarily due to hemodynamic effects of atrasentan. There were no increases in BNP or mean bodyweight, suggesting minimal fluid retention. As of the April 22, 2022 data cutoff, atrasentan was well-tolerated in patients with IgAN, with no treatment-related serious adverse events.

Chinook has completed enrollment of the IgAN patient cohort of the AFFINITY trial, and continues to enroll the other three cohorts, including patients with focal segmental glomerulosclerosis (FSGS), Alport syndrome and diabetic kidney disease in combination with SGLT2 inhibitors. Chinook plans to report additional data from the AFFINITY trial in the second half of 2022, as well as during 2023.

Chinook delivered a mini-oral presentation at the 59th ERA Congress on preclinical mechanistic data describing atrasentan’s effect to block mesangial cell injury and the pathogenic transcriptional networks driving IgAN progression in a model system.
BION-1301
BION-1301 is a novel anti-APRIL monoclonal antibody currently in phase 1/2 development for patients with IgAN. BION-1301’s potentially disease-modifying approach to treating IgAN by reducing circulating levels of galactose-deficient IgA1 (Gd-IgA1) to prevent the formation of pathogenic immune complexes has been demonstrated preclinically as well as clinically in both healthy volunteers and patients with IgAN.

Enrollment of up to 30 patients in Cohort 2 of Part 3 of the ongoing phase 1/2 trial of BION-1301 is ongoing. Patients in Cohort 2 receive a SC dose of 600 mg of BION-1301 every two weeks. Chinook expects to report initial data from Cohort 2 in the second half of 2022.

Based on the data generated to date in the ongoing phase 1/2 study and after consulting with an expert advisory panel, Chinook has decided to advance BION-1301 into phase 3 utilizing the current Cohort 2 dose of 600 mg SC every two weeks, and will not move forward with the optional Cohort 3. Chinook is currently finalizing trial design, conducting site and country feasibility and pursuing regulatory interactions to enable initiation of a global phase 3 trial of BION-1301 in 2023.

Chinook presented additional interim data from Cohort 1 of Part 3 in a mini-oral presentation at the ERA Congress in May 2022, further demonstrating its disease-modifying potential in IgAN by generating durable reductions in mechanistic biomarkers and corresponding impressive proteinuria reductions within three months of initiating treatment. After at least 24 weeks of treatment, patients in Cohort 1 transitioned from IV dosing at 450 mg every two weeks to SC dosing at 600 mg every two weeks. BION-1301 treatment resulted in steady-state reductions in Gd-IgA1 in the range of 70–80%, demonstrating depletion of the pathogenic IgA variant, and establishing the potentially disease-modifying mechanism of BION-1301 by directly targeting the initial pathway in the pathogenesis of IgAN. Additionally, BION-1301 demonstrated a 48.8% geometric mean reduction in 24-hour UCPR in all eight patients at six months of treatment, a 70.9% geometric mean reduction in 24-hour UPCR in six patients at one year of treatment and a 69.1% geometric mean reduction in 24-hour UPCR in two patients at 1.5 years of treatment. As of the May 6, 2022 data cutoff, BION-1301 was well-tolerated, with no serious adverse events or treatment discontinuations due to adverse events.

BION-1301 was granted orphan drug designation for the treatment of primary IgAN by the European Commission in July 2022.
CHK-336
CHK-336 is an oral small molecule lactate dehydrogenase A (LDHA) inhibitor with liver-targeted tissue distribution that Chinook is developing for the treatment of patients with primary hyperoxaluria (PH), secondary hyperoxaluria due to increased endogenous oxalate production and idiopathic stone formation.

In April 2022, Chinook initiated dosing in a phase 1 clinical trial evaluating CHK-336 in healthy volunteers. Data from this trial is expected in the first half of 2023.
Precision Medicine Research & Discovery
Chinook is focused on the discovery and development of novel precision medicines for rare, severe chronic kidney diseases (CKDs) with defined genetic or molecular drivers of disease initiation and progression, and efficient development paths. Chinook has multiple preclinical programs across the discovery, target validation, lead identification and lead optimization stages to generate future clinical pipeline candidates. Chinook is leveraging its ongoing strategic collaboration with Evotec to identify and validate novel targets and enable patient stratification strategies through access to the NURTuRE CKD Patient Biobank, which provides comprehensive PANOMICS characterization of thousands of CKD patients with prospective clinical follow-up and retained bio-samples of urine and blood for exploratory biomarker analysis.

Chinook delivered an oral presentation at the 59th ERA Congress in May 2022 on the approach used in collaboration with Evotec to leverage the NURTuRE CKD biobank to generate mechanistic disease understanding for patient-centric, integrated target and biomarker discovery that will enable the development of novel precision treatments for CKD patient subsets.
Corporate

In May 2022, Chinook completed an underwritten public offering of 7.6 million shares of common stock at a price to the public of $14.00 per share, including the exercise in full of the underwriters’ option to purchase an additional 1.1 million shares of common stock. As part of the offering, Chinook sold to certain investors pre-funded warrants to purchase up to an aggregate of 1.1 million shares of common stock at a purchase price of $13.9999 per pre-funded warrant. The underwritten public offering resulted in gross proceeds to Chinook of $120.7 million.

In April 2022, Chinook announced an outreach initiative in collaboration with the IgA Nephropathy Foundation and Komodo Health, leveraging data and technology to drive awareness of IgAN and engage key medical providers at nephrology practices across the U.S., with the goal of ensuring patients have access to optimal support and treatment options earlier in their disease journey.
Quarter and Six Months Ended June 30, 2022 Financial Results

Cash Position – Cash, cash equivalents and marketable securities totaled $405.2 million at June 30, 2022, compared to $355.1 million at December 31, 2021.

Revenue – Revenue for the quarter and six months ended June 30, 2022 was $0.4 million and $3.1 million, respectively, compared to less than $0.1 million and $0.4 million for the same periods in 2021. The increase was primarily due to revenue recognized under Chinook’s license agreement with SanReno.

Expenses –

Research and development expenses for the quarter and six months ended June 30, 2022 were $30.0 million and $56.3 million, respectively, compared to $22.8 million and $48.5 million, respectively, for the same periods in 2021. The increase was primarily due to higher employee-related costs, including stock-based compensation expense, consulting and outside services fees, as well as facilities and other costs to continue progression of our research and clinical programs. The increase in the three months ended June 30, 2022 also included an increase in licensing and contract research and manufacturing costs. The increase in the six months ended June 30, 2022, was partially offset by a decrease resulting from an upfront fee of $3.3 million to Evotec International GmbH recognized in the same period in 2021.
General and administrative expenses for the quarter and six months ended June 30, 2022 were $8.6 million and $16.5 million, respectively, compared to $7.8 million and $17.3 million, respectively, for the same periods in 2021. The increase during the quarter ended June 30, 2022 was primarily due to higher consulting and outside services costs to support our operations and an increase in stock-based compensation expense, partially offset by lower facilities and other costs. The decrease during the six months ended June 30, 2022 was primarily due to lower employee-related costs and facilities and other costs, partially offset by an increase in stock-based compensation expense.

The change in fair value of contingent consideration and contingent value rights liabilities for the quarter and six months ended June 30, 2022 was a benefit of $2.0 million and $3.0 million, respectively, compared to expense of $19.6 million and $21.4 million for the same periods in 2021. The decrease in these non-cash expenses was primarily due to a change in the fair value in the second quarter of 2021 to include the impact of Merck intending to evaluate MK-5890 in a phase 2 clinical study for a new indication and the sale of certain of our non-renal assets in exchange for preferred shares in Sairopa B.V.

Other –

A $10.0 million development milestone under the Merck collaboration was earned in the fourth quarter of 2021 and received in the first quarter of 2022. We paid this milestone, net of taxes and expenses, to the CVR holders in the second quarter of 2022.

Net Loss – Net loss for the quarter and six months ended June 30, 2022 was $37.6 million, or $0.61 per share, and $69.3 million, or $1.15 per share, respectively. Net loss for the quarter and six months ended June 30, 2021 was $42.6 million, or $0.97 per share, and $79.8 million, or $1.86 per share, respectively.

On August 8, 2022 (GLOBE NEWSWIRE) — Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported financial results for the second quarter ended June 30, 2022 and provided a corporate update (Press release, Celldex Therapeutics, AUG 8, 2022, View Source [SID1234617774]).

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"In late June, Celldex reported positive interim data from our ongoing Phase 1b study of barzolvolimab in chronic spontaneous urticaria, where multiple doses demonstrated rapid, marked and durable responses, including in patients with prior omalizumab treatment," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "We are very encouraged by these results which we believe demonstrate barzolvolimab’s potential to provide meaningful symptom relief to patients suffering from diseases driven by mast cells. These data, together with our previously reported positive results in chronic inducible urticaria, support the continued clinical development of barzolvolimab, including our recently initiated Phase 2 chronic urticaria studies."

Mr. Marucci continued, "We are pleased with our recent advances and expansion of the barzolvolimab program into later stage studies and additional indications, along with the continued development of our bispecific platform. We also recently made the decision to transition development of CDX-1140 to investigator-led studies, allowing us to focus our resources on the programs we believe hold the most value for patients and shareholders. We look forward to providing updates on progress across our programs, with additional significant key milestones expected in the upcoming quarters."

Recent Program Highlights

Barzolvolimab (also referred to as CDX-0159) – KIT Inhibitor Program

Barzolvolimab is a humanized monoclonal antibody developed by Celldex that binds the KIT receptor with high specificity and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells, which mediate inflammatory responses such as hypersensitivity and allergic reactions. KIT signaling controls the differentiation, tissue recruitment, survival and activity of mast cells.

On June 30, Celldex reported interim data from the barzolvolimab multiple dose Phase 1b study in Chronic Spontaneous Urticaria (CSU) which were presented as a late-breaking electronic poster presentation as part of the European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress 2022.

Barzolvolimab was well tolerated with a favorable safety profile; effects of multiple dose administration were consistent with observations in single dose studies. Barzolvolimab results in rapid, marked and durable responses in patients with moderate to severe CSU refractory to antihistamines, including patients with prior omalizumab treatment.
Mean reduction from baseline in urticaria activity (UAS7) of 66.6% in all patients in the 1.5 mg/kg dose group (n=8) at week 12 and 75.1% in all patients in the 3 mg/kg dose group (n=9) at week 8 (reflects only one dose), demonstrating clinically meaningful symptom improvements for patients.
Complete response (UAS7=0) of 57.1% in the 1.5 mg/kg dose group at week 12 and 44.4% at week 8 (reflects only one dose) in the 3 mg/kg dose group which is a key therapeutic goal.
75% well-controlled disease by Urticaria Control Test (UCT) in the 1.5 mg/kg dose group at week 12 and 83.3% in the 3 mg/kg dose group at week 8 (reflects only one dose).
Tryptase suppression paralleled symptom improvement, demonstrating the impact of mast cell depletion on CSU disease activity.

In June and July 2022, Celldex announced that the first patients have been dosed in the Phase 2 clinical studies of barzolvolimab for the treatment of CSU and the two most common forms of chronic inducible urticaria (CIndU) – cold urticaria (ColdU) and symptomatic dermographism (SD). These randomized, double-blind, placebo-controlled, parallel group Phase 2 studies are evaluating the efficacy and safety profile of multiple dose regimens of barzolvolimab in patients who remain symptomatic despite antihistamine therapy, to determine the optimal dosing strategies.

Celldex continues to enroll patients in the barzolvolimab Phase 1b open label study in chronic inducible urticaria in a third cohort (single dose, 3 mg/kg) in cholinergic urticaria and a fourth cohort at a lower dose (single dose, 1.5 mg/kg) in cold urticaria.

Celldex continues to enroll patients in the barzolvolimab Phase 1b multi-center, randomized, double-blind, placebo-controlled study in patients with prurigo nodularis (PN), a chronic skin disease characterized by the development of hard, intensely itchy (pruritic) nodules on the skin.

Celldex remains on track with the development of barzolvolimab in eosinophilic esophagitis (EoE), the most common type of eosinophilic gastrointestinal disease, and plans to initiate a Phase 2 trial in the fourth quarter of 2022.
CDX-527 – Bispecific Antibody Program

CDX-527 is the first candidate developed by Celldex from its bispecific platform which utilizes the Company’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway.

In the Phase 1 dose-escalation study of CDX-527 in patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy, enrollment to the dose escalation portion of the study has been completed and an expansion cohort in ovarian cancer is currently enrolling patients.
CDX-1140 – CD40 Agonist Program

CDX-1140 is a potent CD40 human agonist antibody developed by Celldex that the Company believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

Recently, Celldex reviewed updated data from the CDX-1140 Phase 1 expansion cohorts in combination with KEYTRUDA (pembrolizumab) in patients with squamous cell head and neck cancer (SCCHN) and non-small cell lung cancer who had progressed on checkpoint therapy. Evidence of clinical benefit was most evident in patients with SCCHN, all of whom had progressive disease on prior anti-PD-1/L1 based therapies. Despite evidence of clinical benefit, questions remain to be answered about CDX-1140, and the broader CD40 agonist class, regarding the best clinical settings, regimens, and possible combinations before advancing into additional Celldex sponsored studies. Given our pipeline priorities and resource requirements, Celldex will not progress further Company-sponsored studies at this time and is exploring alternative means of answering these questions, including through investigator sponsored studies.
Recent Operational Highlights

In June 2022, Cheryl L. Cohen and Dr. Garry Neil were appointed to the Celldex Board of Directors. Ms. Cohen currently serves as President of CLC Consulting, a pharmaceutical and biotechnology consulting firm that specializes in new product start-ups and commercialization. Dr. Neil currently serves as Chief Executive Officer at Avalo Therapeutics (formerly Cerecor, Inc.), a publicly held biotechnology company.

In July 2022, Celldex executed a settlement agreement with Shareholder Representative Services (SRS), the representative of the former stockholders of Kolltan Pharmaceuticals, Inc. Celldex believes this is a favorable financial outcome for the Company and its shareholders and it achieves certainty and avoids the distraction and further cost of litigation. Total potential contingent milestones (payable in cash or stock at Celldex’s discretion) from the merger agreement were reduced from $172.5 million to $80.0 million of which $15.0 million was paid in July.
Second Quarter 2022 Financial Highlights and 2022 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2022 were $356.8 million compared to $380.5 million as of March 31, 2022. The decrease was primarily driven by second quarter cash used in operating activities of $22.2 million. At June 30, 2022, Celldex had 46.8 million shares outstanding.

Revenues: Total revenue was $0.2 million in the second quarter of 2022 and $0.3 million for the six months ended June 30, 2022, compared to $3.5 million and $4.2 million for the comparable periods in 2021. The decrease in revenue was primarily due to a decrease in services performed under our manufacturing and research and development agreements with Rockefeller University and Gilead Sciences.

R&D Expenses: Research and development (R&D) expenses were $20.7 million in the second quarter of 2022 and $37.8 million for the six months ended June 30, 2022, compared to $12.4 million and $25.1 million for the comparable periods in 2021. The increase in R&D expenses was primarily due to an increase in clinical trial and personnel expenses.

G&A Expenses: General and administrative (G&A) expenses were $7.2 million in the second quarter of 2022 and $14.1 million for the six months ended June 30, 2022, compared to $4.3 million and $8.4 million for the comparable periods in 2021. The increase in G&A expenses was primarily due to higher legal costs related to reaching a binding settlement term sheet ("the Term Sheet") with SRS, commercial planning and stock-based compensation expenses.

Changes in Fair Value Remeasurement of Contingent Consideration: The gain on fair value remeasurement of contingent consideration was $6.3 million for the second quarter of 2022 and $6.9 million for the six months ended June 30, 2022, primarily due to the Company’s decision to deprioritize the CDX-1140 program.

Litigation Settlement Related Loss: The Company recorded a one-time loss of $15.0 million in the second quarter of 2022 related to the initial payment due under the Term Sheet entered with SRS.

Net Loss: Net loss was $36.0 million, or ($0.77) per share, for the second quarter of 2022, and $59.1 million, or ($1.26) per share, for the six months ended June 30, 2022, compared to a net loss of $13.4 million, or ($0.34) per share, for the second quarter of 2021 and $29.9 million, or ($0.76) per share, for the six months ended June 30, 2021. The litigation settlement related loss had a ($0.32) impact on net loss per share in the three and six months ended June 30, 2022. The gain on fair value remeasurement of contingent consideration had a $0.14 and $0.15 impact on net loss per share in the three and six months ended June 30, 2022, respectively.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at June 30, 2022 are sufficient to meet estimated working capital requirements and fund planned operations through 2025.

On August 8, 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 second quarter ended June 30, 2022 and provided clinical development and operational highlights (Press release, ALX Oncology, AUG 8, 2022, View Source [SID1234617773]).

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"The second quarter of 2022 was marked by substantial progress advancing our lead program, evorpacept, through multiple clinical trials," said Jaume Pons, Ph.D., Founder, President and Chief Executive Officer of ALX Oncology. "Notable accomplishments included the U.S. Food and Drug Administration ("FDA") granting Fast Track designation to evorpacept for the first-line treatment of adult patients with PD-L1 positive advanced head and neck squamous cell carcinoma ("HNSCC") in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, and Orphan Drug Designation ("ODD") to evorpacept for the treatment of patients with acute myeloid leukemia ("AML")."

"We continue to expand our clinical development efforts with the introduction of a new clinical study, ASPEN-07, a Phase 1 trial of evorpacept for the treatment of patients with urothelial carcinoma ("UC") and anticipate initiation in the fourth quarter of 2022. We are continuing enrollment in ASPEN-03 and ASPEN-04, two distinct Phase 2 studies for the treatment of patients with advanced HNSCC. In addition, we continue to advance ASPEN-06, our Phase 2/3 study testing evorpacept 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 cancer or gastroesophageal junction ("GEJ") cancer," Dr. Pons continued.

Recent Clinical Developments for Evorpacept

FDA Granted Fast Track Designation as First-Line Treatment for HNSCC
In August 2022, ALX Oncology announced that the FDA granted Fast Track designation to evorpacept, a next-generation CD47 blocker, in combination with KEYTRUDA (pembrolizumab) for the first-line treatment of patients with PD-L1 expressing metastatic or unresectable, recurrent HNSCC.
ALX Oncology continues to advance ASPEN-03 and ASPEN-04, which are two distinct randomized Phase 2 studies for the treatment of patients with advanced HNSCC in combination with KEYTRUDA (pembrolizumab) with or without chemotherapy. Patient enrollment for ASPEN-03 and ASPEN-04 continues as planned with results expected to be presented by mid-2024.
FDA Granted ODD for Evorpacept for the Treatment of Patients with AML
In June 2022, ALX Oncology announced that the FDA granted ODD to evorpacept for the treatment of patients with AML.
The Phase 1 dose escalation portion of ASPEN-05, a Phase 1/2 clinical trial of evorpacept in combination with venetoclax and azacitidine for the treatment of patients with AML, has successfully completed enrollment with no safety concerns to date up to the highest protocol defined dose level of 60 mg/kg evorpacept once every four weeks.
Patient enrollment was paused before proceeding into the Phase 1 dose optimization portion of ASPEN-05 pending completion of the Phase 1 portion of ASPEN-02, a Phase 1/2 study of evorpacept in combination with azacitidine in patients with myelodysplastic syndrome ("MDS"). Data from ASPEN-02 will be used to inform the optimal dose(s) of evorpacept to be studied in the ASPEN-05 study in combination with venetoclax and azacitidine. Ongoing patients in ASPEN-05 will continue to be treated and followed per protocol.
Additional Evorpacept Clinical Program Updates
In June 2022, ALX Oncology announced expected initiation of ASPEN-07, a Phase 1 trial of evorpacept for the treatment of patients with UC. ASPEN-07 will investigate evorpacept in combination with an antibody-drug conjugate ("ADC"), PADCEV (enfortumab vedotin-ejfv), for the treatment of patients with UC in the fourth quarter of 2022.
ALX Oncology continues to advance ASPEN-06, a randomized Phase 2 (open-label) / Phase 3 (double-blind), international, multi-center study to evaluate the efficacy of evorpacept and CYRAMZA (ramucirumab) added to trastuzumab and paclitaxel for the treatment of patients with HER-positive gastric/GEJ cancer whose tumors have progressed following treatment with HER2-targeted therapy and chemotherapy. ASPEN-06 is being conducted in collaboration with Eli Lilly and Company. Patient enrollment continues to progress and results from the Phase 2 portion of ASPEN-06 are expected to be presented in 2023.
Second Quarter 2022 Financial Results:

Cash, Cash Equivalents and Investments: Cash, cash equivalents and investments as of June 30, 2022 were $324.2 million. ALX Oncology recently updated its cash forecast and believes its cash, cash equivalents and investments are sufficient to fund planned operations through the fourth quarter of 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 June 30, 2022, were $26.7 million, compared to $11.2 million for the prior-year period. The increase in expenses during the three months ended June 30, 2022 compared to the three months ended June 30, 2021 were primarily attributable to an increase of $10.3 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 our lead product candidate, as well as an increase of $1.6 million related to the Tallac Collaboration for work related to the IND filing planned for 2023; an increase of $0.4 million in preclinical costs primarily related to development of new targets; an increase of $1.8 million in personnel and related costs due primarily to an increase driven by headcount growth and a portion of a retention bonus payable to ScalmiBio stockholders; an increase of $2.0 million in stock-based compensation expense due to additional awards granted since June 30, 2021; and an increase of $1.1 million in other research costs.
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 June 30, 2022, were $7.0 million, compared to $5.1 million for the prior-year period. The expense increases during the three months ended June 30, 2022 compared to the three months ended June 30, 2021 were primarily attributable to an increase of $1.6 million in stock-based compensation expense due to additional stock option awards granted since June 30, 2021 and an increase of $0.3 million in personnel and related costs primarily driven by headcount growth.
Net loss: GAAP net loss was $32.9 million for the second quarter ended June 30, 2022, or $0.81 per basic and diluted share, as compared to a net loss of $16.3 million for the second quarter ended June 30, 2021, or $0.40 per basic and diluted share. Non-GAAP net loss was $27.1 million for the second quarter ended June 30, 2022, as compared to a net loss of $14.0 million for the second quarter ended June 30, 2021. A reconciliation of GAAP to non-GAAP financial results can be found at the end of this news release.