On September 17, 2021 MaxCyte, Inc., (NASDAQ: MXCT; LSE: MXCT, MXCN), a leading commercial cell engineering company focused on providing enabling platform technologies to advance innovative cell-based research as well as next-generation cell therapeutic discovery, development and commercialization, reported its financial results for its second quarter and six months ended June 30, 2021 (Press release, MaxCyte, SEP 17, 2021, View Source [SID1234587889]).

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Second Quarter & Recent Highlights

Total revenue of $7.1 million in the second quarter of 2021, representing 38% growth compared to the same period in 2020
Excluding SPL Program-related revenue, revenue from cell therapy customers was $4.8 million for the second quarter, an increase of 59% year-over-year, while revenue from drug discovery customers was $1.8 million in the second quarter, an increase of 60% year-over-year. SPL Program-related revenue was $0.5 million in the second quarter, as compared to $1.0 million for the same period in 2020
Signed two new SPL agreements with Celularity, Inc. (Q2) and Sana Biotechnology, Inc. (Q3) for the use of MaxCyte’s Flow Electroporation ExPERT platform to advance cellular research and development of cell-based therapies
Expanded Board of Directors with the appointment of Ms. Rekha Hemrajani and Dr. Yasir Al-Wakeel
Completed U.S. initial public offering on Nasdaq Global Select Stock Market, raising $201.8 million in gross proceeds
"We are pleased to report strong second quarter and half year results driven by growth in instrument revenue and disposable sales to the cell therapy market as our cell therapy partners continue to progress into and through the clinic. We also saw a resurgence of growth in drug discovery customers as new disposables introduced in 2020 have started to gain traction, driving both instrument and disposable sales growth," said Doug Doerfler, President and CEO of MaxCyte.

"Our customer base is expanding and we continue to increase the number of strategic partnerships. We now have 14 SPL agreements covering over 75 potential clinical programs, which is a testament to MaxCyte’s reputation as a leading collaborator for complex cellular engineering. With the proceeds from our IPO in the U.S., MaxCyte is well-positioned to support growing adoption of the ExpertTM platform technology for cellular-based research and next-generation therapeutic development."

Second Quarter Financial Results

Total revenue for the second quarter of 2021 was $7.1 million, compared to $5.2 million in the second quarter of 2020, representing year-over-year growth of 38%. Overall sales to the cell therapy (up 59%) and the drug discovery (up 60%) markets were each sources of strength in the quarter.

The Company recognized $0.5 million in Program-related revenue in the quarter (comprised of pre-commercial milestone revenues) as compared to $1.0 million in Program-related revenue in the second quarter of 2020.

Gross profit for the second quarter of 2021 was $6.3 million (89% gross margin), compared to $4.7 million (91% gross margin) in the same period of the prior year. The slight decline in gross margin was driven by the reduction in SPL Program-related revenues; excluding SPL Program-related revenues, gross margin was relatively unchanged.

Operating expenses for the second quarter of 2021 were $10.7 million, compared to operating expenses of $7.5 million in the second quarter of 2020. The overall increase in operating expense was principally driven by a $3.3 million increase in compensation expense associated with increased headcount and higher stock-based compensation (principally due to stock-price appreciation), as well as a $1.0 million increase in legal and professional service expenses. Partially offsetting this expense growth was a $1.9 million decline in CARMA-related expenses compared with the same period last year. As of March 2021, all pre-clinical and clinical activities related to the CARMA platform were substantially completed.

Second quarter 2021 net loss was ($4.4) million compared to net loss of ($3.0) million for the same period in 2020.

Total cash, cash equivalents and short-term investments was $73.4 million as of June 30, 2021 excluding the $201.8 million in gross proceeds from the U.S. IPO that closed in August 2021.

Preliminary 2021 Revenue

Following our recent IPO on the Nasdaq, the company is establishing an initial projection of total revenue of approximately $30 million for fiscal year 2021.

First Half 2021 Financial Results

Total revenue for the first half of 2021 was $13.6 million, compared to $10.9 million in the first half of 2020, representing year-over-year growth of 25%. Overall sales to the cell therapy (up 53%) and the drug discovery (up 22%) markets were each sources of strength in the first half.

The Company recognized $0.5 million in Program-related revenue in the first half (comprised of pre-commercial milestone revenues) as compared to $1.8 million in Program-related revenue in the first half of 2020.

Gross profit for the first half of 2021 was $12.1 million (89% gross margin), compared to $9.8 million (90% gross margin) in the same period of the prior year. The slight decline in gross margin was driven by the reduction in SPL Program-related revenues; excluding SPL Program-related revenues, gross margin was relatively unchanged.

Operating expenses for the first half of 2021 were $22.9 million, compared to operating expenses of $15.6 million in the first half of 2020. The overall increase in operating expense was principally driven by a $5.8 million increase in compensation expense associated with increased headcount and higher stock-based compensation (principally due to stock-price appreciation), as well as a $1.4 million increase in legal and professional service expenses. Partially offsetting this expense growth was a $0.8 million decline in CARMA-related expenses compared with the same period last year. As of March 2021, all pre-clinical and clinical activities related to the CARMA platform were substantially completed.

First half 2021 net loss was ($11.5) million compared to net loss of ($6.1) million for the same period in 2020.

Webcast and Conference Call Details

MaxCyte will host a conference call today, September 13, 2021, at 4:30 p.m. Eastern Time. Interested parties may access the live teleconference by dialing (844) 679-0933 for domestic callers or (918) 922-6914 for international callers, followed by Conference ID: 3199124. A live and archived webcast of the event will be available on the "Events" section of the MaxCyte website at View Source

On September 17, 2021 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported that AstraZeneca (LSE/STO/Nasdaq: AZN) presented results from the randomized COAST Phase 2 trial during the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 on September 17, 2021 (Press release, Innate Pharma, SEP 17, 2021, View Source [SID1234587888]).

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In particular, the results of the interim analysis showed monalizumab in combination with durvalumab improved progression-free survival (PFS) and objective response rate (ORR) compared to durvalumab alone in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who had not progressed after concurrent chemoradiation therapy (CRT). Monalizumab, Innate’s lead partnered asset, is a potentially first-in-class immune checkpoint inhibitor targeting NKG2A receptors expressed on tumor infiltrating cytotoxic CD8+ T cells and NK cells.

To read more about the Phase 2 COAST results, please see AstraZeneca’s press release here.

"We’re pleased to see the monalizumab COAST results, particularly the improved clinical outcomes for patients with unresectable, Stage III non-small cell lung cancer," said Mondher Mahjoubi, Chief Executive Officer of Innate Pharma. "Monalizumab is one of the first checkpoint inhibitors targeting a NK cell receptor, and today’s results further support the role it can play in treating certain cancers by blocking the inhibitory receptor, NKG2A. We look forward to continuing to invest in NK cell science and further advancing the next wave of scientific innovation at Innate."

Based on these results, AstraZeneca informed Innate that it plans to start a registrational study with monalizumab in combination with durvalumab in patients with unresectable, Stage III non-small cell lung cancer (NSCLC).

Stage III NSCLC:
In 2020, an estimated 2.2 million people were diagnosed with lung cancer worldwide1. Lung cancer is broadly split into NSCLC and small cell lung cancer, with 80-85% classified as NSCLC.2,3,4 Stage III NSCLC represents approximately one quarter of NSCLC incidence5.

Stage III (locally advanced) NSCLC is commonly divided into three subcategories (IIIA, IIIB and IIIC), defined by how much the cancer has spread locally. In contrast to Stage IV, when cancer has spread (metastasised), the majority of Stage III patients are currently treated with curative intent2,6.

The majority of Stage III NSCLC patients are diagnosed with unresectable tumours2,5.

About Monalizumab:

Monalizumab is a potentially first-in-class immune checkpoint inhibitor targeting NKG2A receptors expressed on tumor infiltrating cytotoxic CD8+ T cells and NK cells.

NKG2A is an inhibitory checkpoint receptor for HLA-E. By expressing HLA-E, cancer cells can protect themselves from killing by NKG2A+ immune cells. HLA-E is frequently overexpressed in the cancer cells of many solid tumors and hematological malignancies. Monalizumab may reestablish a broad anti-tumor response mediated by NK and T cells, and may enhance the cytotoxic potential of other therapeutic antibodies7.

AstraZeneca obtained full oncology rights to monalizumab in October 2018 through a co-development and commercialization agreement initiated in 2015. The ongoing development for monalizumab is focused on investigating monalizumab in various combination strategies in different malignancies.

About COAST Trial:

COAST is a Phase 2, multi-arm, randomised trial investigating durvalumab alone or in combination with either monalizumab, an anti-NKG2A monoclonal antibody, or oleclumab, an anti-CD73 monoclonal antibody, in 189 patients with locally advanced, unresectable Stage III NSCLC who had not progressed after concurrent CRT.

COAST is being conducted by AstraZeneca in 82 centers across nine countries in North America, Europe and Asia. The primary endpoint of the trial is ORR as a measure of anti-tumor activity. Secondary endpoints include safety, duration of response, overall survival and PFS.

On September 17, 2021 Greenfire Bio portfolio company Pacylex Pharmaceuticals, Inc. reported that it initiated a Phase 1 clinical trial of PCLX-001, a first-in-class, orally bioavailable small molecule inhibitor of N-myristoyltransferase 1 and 2 (NMT1, NMT2), at the University of Alberta Cross Cancer Institute (Press release, Greenfire, SEP 17, 2021, View Source [SID1234587886]). This open label, dose escalation study will examine the safety and tolerability of PCLX-001 and determine the dose to be used in initial efficacy studies. Pacylex received a No Objection Letter from Health Canada on March 8, 2021, authorizing the Phase 1 Trial of PCLX-001 in relapsed/refractory B-cell Non-Hodgkin’s Lymphoma and advanced solid malignancies. PCLX-001 is believed to be the first NMT inhibitor that will be clinically tested.

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PCLX-001 is the first clinical candidate sponsored by Pacylex Pharmaceuticals to reach the clinical stage. "We are excited to see PCLX-001 transition to helping serious refractory cancer patients," said Michael Weickert, CEO of Pacylex Pharmaceuticals. "Our mission to bring this new potential cancer breakthrough to patients is finally being realized."

"Pacylex is the first investment based on our strategy of finding game changing assets that are close to initiating clinical studies," said Ajit Gill, the CEO of Greenfire Bio. "We are delighted to see Pacylex enter the clinic so soon after becoming part of our portfolio and look forward to seeing the benefits we hope this will provide to cancer patients."

Patient enrollment and dosing will also begin soon at Princess Margaret Hospital in Toronto and the BC Cancer Agency in Vancouver. The study will enroll 20-30 patients in the initial phase. Three principal investigators will oversee the clinical study at the three clinical sites in Canada: Dr. John Kuruvilla at Princess Margaret Cancer Centre in Toronto, Dr. Randeep Sangha at the Cross Cancer Institute in Edmonton, and Dr. Laurie Sehn at the British Columbia Cancer Center in Vancouver.

This study is registered at ClinicalTrials.gov Identifier: NCT04836195.

About Non-Hodgkin’s Lymphoma
Non-Hodgkin’s Lymphoma (NHL) is the most common hematologic malignancy and the eleventh most common cancer worldwide, with nearly 510,000 new cases diagnosed in 2018. It accounted for nearly 249,000 deaths worldwide in 2018. The most prevalent form of NHL, accounting for about 40% of newly diagnosed NHL cases, is an aggressive form called diffuse large B-cell lymphoma (DLBCL), that comes with a life expectancy of weeks or months if left untreated.

PCLX-001
PCLX-001 is a small molecule, first-in-class N-myristoyltransferase (NMT) inhibitor, originally developed by the University of Dundee Drug Discovery Unit as part of a program to treat African sleeping sickness funded by Wellcome Trust. Pacylex is developing PCLX-001, which has excellent oral bioavailability, to treat leukemia and lymphoma. PCLX-001 selectively kills cancer cells and completely regresses (eliminates) tumors in animal models of acute myeloid leukemia (AML), diffuse large B-cell lymphoma (DLBCL) and Burkitt lymphoma (BL). PCLX-001 has also been shown to strongly inhibit the growth of lung and breast cancer tumors in animal models. In leukemia, lymphoma and breast cancer patients, the level of NMT2 is correlated with survival, suggesting an important biological role in these cancers. In tests using cultured cancer cells in vitro, PCLX-001 is at least ten times as potent as ibrutinib (Imbruvica) and dasatinib (Sprycel), two clinically approved drugs currently used to treat hematologic malignancies.

On September 17, 2021 Protagonist Therapeutics, Inc. (Nasdaq: PTGX) ("Protagonist" or "the Company") reported the receipt of a verbal communication from the U.S. Food and Drug Administration (FDA) that Protagonist’s clinical studies for rusfertide, an investigational product candidate currently in development, have been placed on a clinical hold (Press release, Protagonist, SEP 17, 2021, View Source [SID1234587885]).

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The clinical hold follows Protagonist’s notification to the FDA of a recent non-clinical finding in a 26-week rasH2 transgenic mouse model study. The rasH2 model is designed to detect signals related to tumorigenicity, and benign and malignant subcutaneous skin tumors were observed in this study.

The Company is working with the FDA and will be prepared to make all appropriate updates to clinical study documents and determine the next steps in consultation with the FDA. In particular, we will provide additional clinical safety reports, update the investigator brochures and patient informed consent forms, and make necessary modifications to study protocols. Dosing of patients in all ongoing clinical trials with rusfertide will be put on hold, and study investigators have been contacted to facilitate patient notification.

"Patient safety is our absolute top priority," said Dinesh Patel, President and Chief Executive Officer of Protagonist. "We are fully committed to working closely with the FDA in understanding and evaluating potential clinical risks and determining next steps for the development of rusfertide."

On September 17, 2021 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Gavreto (pralsetinib) as a monotherapy for the treatment of adult patients with rearranged during transfection (RET) fusion-positive advanced non-small cell lung cancer (NSCLC) not previously treated with a RET inhibitor (Press release, Hoffmann-La Roche, SEP 17, 2021, View Source [SID1234587883]).

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"This positive CHMP opinion for Gavreto represents another important step towards our goal of providing effective therapeutics that target genomic drivers of disease for as many cancer patients as possible," said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. "Advances in personalised medicine also underscore the importance of tumour genomic profiling to identify patients who may benefit from targeted therapies."

RET alterations are key disease drivers in many cancer types, including NSCLC and multiple types of thyroid cancer. RET fusion-positive NSCLC affects approximately 37,500 people worldwide each year and the disease often affects those who least expect it;[1,2,3] RET fusion-positive NSCLC is often identified in younger people with a minimal to no history of smoking.[3] These cancers also typically represent a high unmet need, due to limitations associated with standard therapies.[4,5,6] Biomarker testing for these fusions is the most effective way to identify people with advanced NSCLC who are eligible for treatment with Gavreto. Gavreto is a highly selective, potent, and CNS-penetrant RET inhibitor and, together with Alecensa (alectinib) and Rozlytrek (entrectinib), is part of Roche’s portfolio of targeted treatments for NSCLC. Together, they offer personalised treatment options for almost one in ten people with advanced NSCLC.[7]

The CHMP recommendation is based on the results of the phase I/II ARROW study, in which Gavreto demonstrated rapid, potent, and durable clinical activity in patients with advanced RET fusion-positive NSCLC.[8] A final decision regarding the approval of Gavreto is expected from the European Commission in the coming months.

Gavreto has also shown activity across multiple solid tumour types, reflecting tumour-agnostic potential.[9] In September 2020, the U.S. Food and Drug Administration (FDA) approved Gavreto for the treatment of adults with metastatic RET fusion-positive NSCLC, and in December 2020 it was approved for the treatment of adult and paediatric patients 12 years of age and older with advanced RET-altered thyroid cancers. Gavreto has since been approved in Canada, mainland China and Switzerland. In the European Union, the MAA for Gavreto for the treatment of adults with RET fusion-positive NSCLC is ongoing, and a submission for RET-altered thyroid cancers is planned. Regulatory submissions for these indications are underway in multiple countries worldwide.

Blueprint Medicines and Roche are co-developing Gavreto globally, with the exception of certain territories in Asia, including China.* Blueprint Medicines and Genentech, a wholly owned member of the Roche Group, are commercialising Gavreto in the US and Roche has exclusive commercialisation rights for Gavreto outside of the US, with the exception of certain territories in Asia, including China.*

*CStone Pharmaceuticals retains all rights to the development and commercialisation of Gavreto in these territories under its existing collaboration with Blueprint Medicines.

About the ARROW study[10]
ARROW is an ongoing phase I/II, open-label, first-in-human study designed to evaluate the safety, tolerability and efficacy of Gavreto, administered orally in people with rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer, RET fusion-positive thyroid cancer and other RET-altered solid tumours. ARROW is being conducted at multiple sites across the United States, Europe and Asia.

An update from the ARROW study was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Virtual Scientific Programme, 4-8 June.[11] In 126 patients with RET fusion-positive NSCLC who previously received platinum-based chemotherapy, Gavreto demonstrated an overall response rate (ORR) of 62% (95% CI: 53%, 70%), a clinical benefit rate (CBR) of 74% (95% CI: 65%, 81%), and a disease control rate (DCR) of 91% (95% CI: 85%, 96%). Median progression-free survival (PFS) was 16.5 months (95% CI: 10.5 months, 24.1 months). In 68 treatment-naïve patients, the confirmed ORR was 79% (95% CI: 68%, 88%), the CBR was 82% (95% CI: 71%, 91%), and the DCR was 93% (95% CI: 84%, 98%). Median PFS was 13.0 months (95% CI: 9.1 months, not reached (NR)). In 25 treatment-naïve patients who were enrolled after eligibility criteria were revised, to allow candidates for platinum-based therapy, the confirmed ORR was 88% (95% CI: 69%, 98%), the CBR was 88% (95% CI: 69%, 98%), and the DCR was 96% (95% CI: 80%, 100%). Median PFS was not reached. In addition, Gavreto was well-tolerated; of the 471 ARROW trial patients across RET-altered tumour types, the most common (≥ 25%) treatment-related adverse events were neutropenia, increased liver enzymes (aspartate aminotransferase [AST] and alanine aminotransferase [ALT]), anaemia, white blood cell count decrease, high blood pressure (hypertension) and lack of energy (asthenia).

About rearranged during transfection (RET)-altered cancers
RET gene alterations, such as fusions and mutations, are key disease drivers in many types of cancer, including non-small cell lung cancer (NSCLC) and several types of thyroid cancers. There are approximately 2.21 million cases of lung cancer diagnosed each year worldwide,[1] of which approximately 1.8 million are NSCLC and RET fusions are present in approximately 1-2% of these patients,[2,3] meaning RET fusion-positive NSCLC affects up to 37,500 people each year. Additionally, approximately 10-20% of people with papillary thyroid cancer (the most common type of thyroid cancer) have RET fusion-positive tumours,[12] and roughly 90% of people with advanced medullary thyroid cancer (a rare form of thyroid cancer) carry RET mutations.[13] Oncogenic RET fusions also are observed at low frequencies in cholangiocarcinoma, colorectal, neuroendocrine, ovarian, pancreatic and thymus cancers.

About Gavreto (pralsetinib)
Gavreto is a once-daily, oral targeted treatment designed to selectively target rearranged during transfection (RET) alterations, including fusions and mutations, regardless of the tissue of origin. Preclinical data have shown that Gavreto inhibits primary RET fusions and mutations that cause cancer in subsets of patients, as well as secondary RET mutations predicted to drive resistance to treatment. Blueprint Medicines and Roche are co-developing Gavreto for the treatment of people with various types of RET-altered cancers.

About Roche in lung cancer
Lung cancer is a major area of focus and investment for Roche, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have six approved medicines to treat certain kinds of lung cancer and more than ten medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.