On May 11, 2021 Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing precision T cell therapies to treat solid tumors, reported its financial results for the first quarter ended March 31, 2021 and recent business highlights(Press release, Achilles Therapeutics, MAY 11, 2021, View Source [SID1234584048]).

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"Achilles made significant progress in the first quarter of 2021. We reported the first clinical data from our ongoing CHIRON and THETIS trials evaluating our precision TIL cNeT therapy in patients with non-small cell lung cancer and melanoma, respectively, and priced our successful US initial public offering on Nasdaq, which closed just after quarter-end," said Dr Iraj Ali, Chief Executive Officer of Achilles. "We continue to enroll and dose patients and have opened our first clinical sites in the US and EU. This year, we expect to report interim data from a total of ten patients that have received cNeT monotherapy across the CHIRON and THETIS trials and will also begin enrolling patients to receive higher dose cNeT. In addition, we will open Cohort B in the THETIS study to evaluate the addition of a PD-1 inhibitor to our cNeT therapy."

Business Highlights

Received a recommendation from the Independent Data Safety Monitoring Committee to continue the ongoing Phase I/IIa CHIRON and THETIS trials as planned

Announced initial clinical data from the first six patients dosed with the Company’s cNeT therapy showing encouraging evidence of cNeT engraftment, an overall tolerability profile similar to that of standard TIL products, stable disease in four out of the six patients, and one patient with tumor lesion reduction

Presented data at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting detailing the Company’s comprehensive translational research program and insights into the in vivo dynamics of cNeT post-dosing, and the potential to develop a potency-based release assay

Strengthened the Board of Directors and Scientific Advisory Board with the additions of Julie O’Neill and Markwin Velders, Ph.D., respectively, and continued to build the team in the UK & US, including key appointments across manufacturing, supply chain and clinical operations, bio-processing and intellectual property

Received a Horizon 2020 grant as part of the Neoantigen Consortium, with the aim of developing a tool to predict neoantigen immunogenicity.

Financial Highlights

IPO: Priced an initial public offering of 9,750,000 ADRs at a public offering price of $18.00 per share for gross proceeds of $175.5 million. The IPO closed on April 6, 2021, after the quarter end.

Cash and cash equivalents: Cash and cash equivalents were $159.3 million as of March 31, 2021 as compared to $177.8 million as of December 31, 2020, not including $160.6 million in net proceeds from the IPO which closed on April 6, 2021. The Company anticipates that its existing cash and cash equivalents plus the IPO proceeds are sufficient to fund its planned operations into the second half of 2023, including full funding of the ongoing Phase I/IIa CHIRON and THETIS clinical trials.

Operating Expenses: Operating Expenses were $13.7 million for the quarter ended March 31, 2021, which included $8.9 million in Research & Development, and General and Administrative expenses of $4.8 million.

Net loss: Net loss attributable to ordinary shareholders was $13.8 million for the quarter ended March 31, 2021 and the basic and diluted net loss per ordinary share was $8.38 for the quarter ended March 31, 2021.

Upcoming Events

Iraj Ali, Chief Executive Officer, will participate in a fireside chat at the BofA Securities 2021 Virtual Healthcare Conference at on Thursday, May 13, 2021, at 9:30 a.m. ET / 2:30 p.m. BST.

A poster detailing abstract TPS9138 entitled, An Open-Label, Multi-Centre Phase I/IIa Study Evaluating the Safety and Clinical Activity of Clonal Neoantigen Reactive T cells in Patients with Advanced Non-Small Cell Lung Cancer (CHIRON), will be presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting taking place virtually from June 4-9, 2021. Full abstracts will be released on May 19, 2021 at ASCO (Free ASCO Whitepaper).org.

On May 11, 2021 Innate Pharma SA (Euronext Paris: IPH – ISIN: FR0010331421; Nasdaq: IPHA) ("Innate" or the "Company") reported its revenue and cash position for the three-month period ending March 31, 2021.

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"This quarter, we have continued to execute against our strategy, successfully progressing the lacutamab TELLOMAK trial in the mycosis fungoides cohort while also advancing our first NK cell engager with our partner, Sanofi," said Mondher Mahjoubi, Chief Executive Officer of Innate Pharma. "As we continue to advance our business, we look forward to delivering additional data read-outs from these programs this year, as well as leveraging our strong R&D competencies to lead the next wave of scientific innovation at Innate."

Pipeline highlights:

Lacutamab (IPH4102, anti-KIR3DL2 antibody):

In February 2021, the Company announced lacutamab showed a positive early signal in the KIR3DL2-expressing mycosis fungoides cohort (‘cohort 2’) of the TELLOMAK clinical trial. This cohort reached the pre-determined number of responses needed to advance to stage 2 earlier than anticipated. The Company plans to present this preliminary data as an oral presentation at the 16th International Conference on Malignant Lymphoma, Lugano (16-ICML) being held virtually between June 18-22, 2021.
The Company expects to initiate its Phase 1b trial evaluating lacutamab as a monotherapy in KIR3DL2-expressing patients with relapsed/refractory peripheral t-cell lymphoma (PTCL) by mid-2021. In addition, the Lymphoma Study Association (LYSA) will initiate an investigator-sponsored, randomized trial to evaluate lacutamab in combination with chemotherapy GEMOX (gemcitabine in combination with oxaliplatin) versus GEMOX alone in KIR3DL2-expressing relapsed/refractory PTCL patients in the second half of 2021.
IPH6101 (NKp46-based NK cell engager), partnered with Sanofi:

Progress was made in the NKCE collaboration with Sanofi, resulting in the decision announced in January 2021 that Sanofi will transition IPH6101/SAR443579 into investigational new drug (IND)-enabling studies. IPH6101 is a NKp46-based NK cell engager (NKCE) using Innate’s proprietary multi-specific antibody format (Gauthier et al. Cell 2019). The decision triggered a €7 million milestone payment from Sanofi to Innate.
In January 2021, a GLP-tox study was initiated for the IPH6101/SAR443579 program.
The Company will share an update on its NKCE platform at the Federation of Clinical Immunology Societies (FOCIS) meeting being held virtually between June 8-11, 2021.

Monalizumab (anti-NKG2A antibody), partnered with AstraZeneca:

The Company expects to publish data this year from the Phase 2 expansion cohort (‘cohort 3’), exploring the combination of monalizumab, cetuximab and durvalumab in first-line IO naïve patients with R/M SCCHN.

Avdoralimab in COVID-19 (anti-C5aR1 antibody):

The investigator-sponsored Phase 2 clinical trial, FORCE (FOR COVID-19 Elimination), has completed enrollment and is ongoing for patient follow-up and data analysis. More information on this study can be found at clinical trials.gov.

Financial Results:

Cash, cash equivalents and financial assets of the Company amounted to €181.7 million as of March 31, 2021. At the same date, financial liabilities amounted to €18.5 million.

During the first quarter of the year 2021, a €7.0m milestone payment was received from Sanofi in February 2021. This milestone followed the decision taken by Sanofi to advance IPH6101/SAR443579 into investigational new drug (IND)-enabling studies.

Revenues for the first three months of 2021 amounted to €4.5 million (€19.3 million for the same period in 2020). For the three-month period, ended March 31, 2021, revenue from collaboration and licensing agreements mainly results from the spreading of the payments received under our agreements with AstraZeneca and Sanofi. (Press release, Innate Pharma, MAY 11, 2021, View Source [SID1234579639])

On May 11, 2021 Applied Therapeutics, Inc. (Nasdaq: APLT) ("Applied Therapeutics" or the "Company"), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the first quarter ended March 31, 2021 (Press release, Applied Therapeutics, MAY 11, 2021, View Source [SID1234579683]).

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"In the first quarter we continued to progress our late stage programs towards commercialization in Galactosemia and Diabetic Cardiomyopathy, while advancing our additional rare disease programs in SORD and PMM2-CDG," said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics. "We are looking forward to our planned NDA submission in Galactosemia in the third quarter of this year, and continue to focus on our primary objective – bringing drugs to patients in desperate need of treatment."

Recent Highlights

Initiated Phase 2 Study of AT-007 in Patients with SORD Deficiency. The Phase 2 pilot study of AT-007 in patients with Sorbitol Dehydrogenase Deficiency (SORD Deficiency), a progressive, debilitating hereditary neuropathy that affects peripheral nerves and motor neurons, has been initiated. Preclinical findings demonstrate that AT-007 has the potential to be the first disease-modifying therapy for SORD, targeting the underlying cause of disease.
Presented Data on Galactosemia Disease Progression at the 2021 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics. In April 2021, the Company presented data featuring a cross-sectional analysis of nineteen pediatric patients with Classic Galactosemia, providing meaningful insight on the progressive worsening of the central nervous system phenotype with age.
Hosted Virtual Rare Disease Forum. In March 2021, the Company hosted a virtual forum highlighting its AT-007 development programs in Galactosemia, SORD Deficiency, and PMM2-CDG. A replay of the presentation, which featured several rare disease key opinion leaders, is available at View Source
Announced Restart of Pediatric Galactosemia Study. In February 2021, the Company announced that the FDA lifted the clinical hold on the AT-007 ACTION-Galactosemia Kids pediatric clinical study, and the study resumed immediately. Applied Therapeutics worked closely with FDA to modify the trial, with the shared goal of ensuring that all patients have the opportunity to receive clinical benefit, and remains on target to submit an NDA no later than Q3 2021.

Closed Approximately $75 million Public Offering. In February 2021, the Company completed an underwritten public offering of 3,450,000 shares of its common stock, including the exercise in full of the underwriters’ option to purchase 450,000 additional shares of common stock at a price of $23.00 per share, resulting in aggregate net proceeds of approximately $74.4 million.
Financial Results

Cash and cash equivalents and short-term investments totaled $148.1 million as of March 31, 2021, compared with $96.8 million at December 31, 2020.

Research and development expenses for the three months ended March 31, 2021 were $14.4 million, compared to $7.3 million for the three months ended March 31, 2020. The increase of $7.2 million was primarily related to an increase in clinical and pre-clinical expense of $1.3 million, related to the progression of the AT-007 ACTION-Galactosemia adult extension study, the AT-007 ACTION-Galactosemia Kids pediatric registrational study and the AT-001 Phase 3 ARISE-HF clinical study; an increase in drug manufacturing and formulation costs of $5.2 million related to the progression of the manufacturing campaigns and the completion and release of AT-001 and AT-007 drug product batches; an increase in personnel expenses of $0.3 million due to the increase in headcount in support of our clinical program pipeline; an increase in stock-based compensation of $0.2 million due to new stock option and restricted stock unit grants; and an increase in regulatory and other expenses of $0.2 million relating to an increase in clinical consulting fees during the three months ended March 31, 2021.

General and administrative expenses were $9.8 million for the three months ended March 31, 2021, compared to $5.2 million for the three months ended March 31, 2020. The increase of approximately $4.6 million was primarily related to an increase of $1.9 million related to the establishment of a commercial department; an increase in stock-based compensation of $1.5 million and increase in personnel expenses of $0.9 million related to an increase in headcount; an increase of $0.2 million related to increased insurance costs; an increase of $0.4 million in other expenses relating to increased costs of rent and other office expenses; and a $0.4 million decrease in legal and professional fees due to lower external legal fees.
Net loss for the first quarter of 2021 was $24.2 million, or $1.00 per basic and diluted common share, compared to a net loss of $12.4 million, or $0.59 per basic and diluted common share, for the first quarter 2020.

On May 11, 2021 MannKind Corporation (Nasdaq: MNKD), a company focused on the development and commercialization of inhaled therapeutic products for patients with endocrine and orphan lung diseases, reported that its Chief Executive Officer, Michael Castagna, PharmD, will participate in a Fireside Chat at the 2021 RBC Capital Markets Global Healthcare Conference on Tuesday, May 18, 2021 at 4:15 pm (ET) (Press release, Mannkind, MAY 11, 2021, View Source [SID1234579699]). Interested parties can access a link to the webcast from the Events & Presentations section of the Company’s website at View Source The webcast replay will remain available for 14 days following the live presentation.

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On May 11, 2021 Accelerated Biosciences, a regenerative medicine innovator, reported that new data that further demonstrates statistically significant cytolysis with induced pluripotent stem cell (iPSC)-derived natural killer (NK) cells programmed from its ethically sourced human trophoblast stem cells (hTSCs) (Press release, Accelerated Biosciences, MAY 11, 2021, View Source [SID1234579715]). Pluristyx, a Seattle-based firm supporting drug development, regenerative medicine, and cell and gene therapies, further confirmed Accelerated Biosciences’ hTSC line offers before-unrealized opportunities in cell-specific therapeutics. Along with this recent data on successful iPSC differentiation, Accelerated Biosciences has already demonstrated efficient differentiation of its pluripotent stem cells with remarkable doubling times and growth characteristics to programmed NK, cartilage, bone, fat, neuron, pancreas, liver, and secretome cells.

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"This new data validates our findings," explains Yuta Lee, President and Founder of Accelerated Biosciences. "We know the properties of our trophoblast stem cells have been long-sought by the medical science community because of the potential to speed and amplify the development of life-saving therapeutics; they’re immune privileged, chromosomally stable (not tumorigenic), pathogen free, pluripotent, easy to scale and manufacturer, and of special interest, they are ethically sourced from the chorionic villi (pre-placental tissue) of non-viable and often life-threatening tubal ectopic pregnancies." Mr. Lee’s father, Professor Jau-Nan Lee, MB, MD, PhD, an obstetrics and gynecologic physician and researcher in Taiwan, first isolated hTSC in 2003. Mr. Lee created Accelerated Biosciences to elevate the visibility of this pluripotent human trophoblast stem cell platform to those engaged in developing allogeneic cell therapeutics and has been instrumental in the filing and prosecution of intellectual property to protect the company’s hTSC platform – to date holding 34 patents.

Benjamin Fryer, PhD, Co-founder and CEO of Pluristyx, worked closely with Accelerated Biosciences to prepare much of its key hTSC data. Dr. Fryer, a trophoblast expert who was previously a research scientist at Janssen Research & Development of Johnson & Johnson, now serves on Accelerated Biosciences’ Scientific Advisory Board. "Initially I was skeptical these cells were what they said they were. If we hadn’t grown and characterized them in our lab, I might have remained skeptical. These are indeed trophoblast stem cells," explained Dr. Fryer. "The potential of these cells is enormous. One of the industry’s largest challenges is that it’s almost impossible to scale primary cells. These cells are scalable. With these cells you can make the amount required for millions of patients and they’re sourced compliant to regulatory requirements. We’ve made IPS cells (induced pluripotent stem cells) and NK (natural killer) cells from them, which is the next wave of cells for cell therapies. For therapeutic developers, because these cells are not sourced from a person or viable embryo, these cells deliver the trifecta of legal, ethical, and IP advantages."

"As the biotechnology industry works toward developing therapies that target only diseased cells without harming healthy cells and tissues, cell-based therapies draw increasing interest," explains industry expert, Martina Molsbergen, CEO of C14 Consulting, who has partnered with Accelerated Biosciences in a business development role. "With all the promise that cell therapies hold, the biotechnology industry also remains concerned that the therapeutics are derived in a socially and ethically responsible manner. Accelerated Biosciences has discovered and is now offering what scientists see as the holy grail of stem cell sources."

Prominent biosciences experts have been drawn to Accelerated Biosciences’ cell breakthrough. Protein chemist and molecular biologist Igor Fisch, PhD, former President and CEO of Selexis, Geneva, Switzerland, recognizes the impact that Accelerated Biosciences’ hTSCs will have on human health: "Not only are these cells ‘politically correct’, but they can also differentiate. Because they are sourced from pre-placenta material, they’re immune privileged, which means that are not seen as foreign by the human body. With these cells, we can create a cell bank – a single source for a wide range of patients."

Peter Hudson, FTSE, BSc Hons, PhD, Chief Scientist and a senior advisor to Avipep P/L in Melbourne, Australia, and an adjunct professor at the University of Queensland, led a large oncology consortium to complete the first Phase 1 clinical trial of a novel engineered antibody targeting prostate and ovarian cancer. Hudson’s interest in Accelerated Biosciences’ hTSCs has evolved into a role on its Scientific Advisory Board. "Trophoblast stem cells are likely to be the next wave of cancer-targeting therapeutics," explains Dr. Hudson. "The ability to ethically source trophoblast stem cells and program them to target only diseased, cancerous cells is very powerful technology."

Why are scientists so interested in stem cell-based therapies?

The human body constantly produces specialized cells from its own stem cells (undifferentiated cells) to renew and repair itself. Current therapies harness this power in autologous cell therapies in which the patient’s own cells are removed, differentiated into disease-fighting cells, and reinserted.

What makes the human trophoblast stem cell so important to medical science?

The human trophoblast stem cell (hTSC) comes from placental tissue and has special properties that make it extremely desirable to therapeutic developers. The hTSC is such an early stem cell that it has much more capacity for growth than a stem cell taken from an adult, for example. This means that one cell can become millions. The hTSC also carries with it the same immune-privilege that a growing embryo has inside its mother: it’s not seen as foreign although it’s genetically different than its mother. Unlike other foreign materials, the hTSC is not rejected by the human body, which means that it can be used with many different patients (allogeneic cell therapy). With these benefits, the scientific community holds a high regard for hTSCs, but it also faces socio-ethical concerns about how those stem cells are typically sourced.

Accelerated Biosciences sidesteps hTSC sourcing concerns in a profoundly elegant way. Dr. Jau-Nan Lee, an OB-GYN in Taiwan, found inspiration in what was considered medical waste. When surgical intervention was necessary to remove an ectopic pregnancy that would otherwise risk the woman’s life, the non-viable embryo and pre-placental tissue lodged in the fallopian tube was removed, sent to pathology, and discarded. Gaining permission from institutional colleagues and sampling the pre-placental tissue, Dr. Lee isolated hTSC that offered all the benefits of hTSC – pluripotency, immune privilege, and scalability – without pathogens and without ethical compromises.