On August 8, 2019 InnoCure Therapeutics (CEO Hye-dong Yoo) reported the company is accelerating the development of new drugs using next-generation target protein degradation mechanisms (Press release, InnoCure Therapeutics, AUG 8, 2019, View Source;nid=233623 [SID1234651740]). According to the company, they are conducting research to develop treatments for rare diseases including anticancer drugs and degenerative brain diseases by utilizing next-generation new drug development technologies such as ‘PROTAC’ and ‘AUTOTAC’, artificial intelligence (AI) to shorten drug development time and increase drug efficacy, big data analysis, and molecular modeling to induce specific protein degradation mechanisms.

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Based on this, InnoCure Therapeutics was recognized for its technological prowess when it was selected for the R&D project of the Ministry of SMEs and Startups’ TIPS program in February 2019, in which Magna Investment participated as the operator.

The company said that it has secured several new substances effective in the field of rare diseases at the research institute that moved to Pangyo, Gyeonggi-do in June, and is currently pursuing IND application for clinical trials in 2021. It is also pursuing joint research with pharmaceutical and bio companies specializing in degenerative brain diseases and rare diseases in the United States.

"InnoCure is actively utilizing PROTAC and AUTOTAC, next-generation technologies that can serve as ‘game changers’ in the new drug development field," said Bae Jun-hak, Vice President of Magna Investment. "The management team, which has experience in global bio companies and a global human network, possesses the know-how in global market entry strategies."

Meanwhile, PROTAC targeted protein degradation technology is known as a drug development technology that can increase efficacy and reduce side effects with small doses. Avinas, founded in 2014 by Professor Craig Crews of Yale University, a pioneer in PROTAC technology, became the first PROTAC technology company to enter clinical trials with a drug using PROTAC technology when its prostate cancer treatment under development received clinical approval from the FDA in March 2019.

InnoCure Therapeutics CEO Yoo Hye-dong is currently conducting clinical research on a drug for degenerative brain diseases that was jointly researched with a U.S. East Coast biotech company specializing in degenerative brain diseases while working at NCE Sciences, a San Francisco-based startup in the U.S. While working at Celgene, she participated in the development of new drugs, including the development of an alternative to the blockbuster nano-oncology drug ‘Abraxane.’

In January 2018, he founded Innocure Therapeutics.

On August 8, 2019 Cytovation AS ("Cytovation") and Specialized Medical Services-oncology BV ("SMS-oncology") reported that SMS-oncology has been selected as the CRO to conduct the phase I/II clinical trial with CyPep-1 in patients with advanced solid cancers (Press release, Cytovation, AUG 8, 2019, View Source [SID1234561561]).

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In recent months, Cytovation has progressed through preclinical and toxicology studies with its lead candidate CyPep-1, a first-in-class lytic agent. Through its unique pharmacological properties, CyPep-1 selectively targets and lyses tumor cell membranes based on their altered molecular composition. This mode of action kills cancer cells, releases tumor antigens, and potentially induces a tumor-specific immune response by in-situ immunization.

Cytovation is currently initiating a first-in-human trial with intratumoral injection of CyPep-1 and aims to reach first in patient (FPI) milestone by the end of 2019. An agreement is now concluded with the oncology dedicated CRO SMS-oncology in Schiphol, the Netherlands.

The study is designed as an open-label, dose escalation phase I/II trial to evaluate the safety, efficacy and pharmacokinetics of intratumoral CyPep-1 in patients with advanced solid cancers. The multicenter trial in Europe will enroll approximately 18 patients. SMS-oncology is developing the protocol and giving guidance regarding patient inclusion criteria and investigational sites for a best-fit and seamless transition to clinic. This will be followed by full conduct of the trial, expected to commence in Q4 2019.

Mr. Kjell-Inge, CEO of Cytovation: "We are excited CyPep-1 is now progressing to the next phase of development, which is an important step for our company. We are pleased with selecting SMS-oncology as our partner in this transition to clinic. From the initial discussions, they lived up to their reputation as experts in the field of early phase and immuno-oncology trials. Preparations of the trial are moving forward in a fast pace, and we feel confident our trial will be conducted in an optimal and valuable matter with our goals is mind."

Ms. Philine van den Tol, CEO of SMS-oncology: "We are delighted to support Cytovation and look forward to jointly make the CyPep-1 trial a great success. It is exactly these type of innovative projects SMS-oncology leverages great experience in, and beyond all, is passionate
about. The enthusiasm of Cytovation is catching and our team can’t wait to see results of this promising first-in-class lytic compound."

CyPep-1, in a cream formulation for topical application, is currently being evaluated in a phase I trial in patients with HPV-induced cutaneous tumors (warts).

On August 8, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported its financial and operational results for the second quarter ended June 30, 2019 (Press release, Autolus, AUG 8, 2019, View Source [SID1234550688]).

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Operational Highlights:

In April, Autolus announced the presentation of initial data from the ongoing Phase 1/2 ALLCAR19 clinical trial of AUTO1 in adult ALL at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 in Atlanta, Georgia. As of the data cutoff date of May 18, 2019, 13 patients were leukapheresed, and products for 12 patients were manufactured, including 7 with Autolus’ semi-automated, fully enclosed manufacturing process. Using the Lee criteria, there were no patients with severe cytokine release syndrome (CRS) (≥ Grade 3), and only 2 of 10 patients (20%) with Grade 2 CRS. Tocilizumab was used in 2 of 10 patients (20%). None of the patients were admitted to intensive care due to CRS. One patient developed delayed Grade 3 neurotoxicity following high levels of CAR T expansion, which resolved promptly following administration of steroids. Four patients died while enrolled in the trial, 2 due to progression of the disease and 2 due to sepsis, a common complication in patients with advanced ALL. Nine patients were evaluable for response at 1 month with 8 (88%) achieving a molecular complete response. One patient died of sepsis before the one-month evaluation point. At a median follow-up of 5 months (range 0.62-10.6 months), 6 of 10 patients were alive and continue to be in molecular remission with continued evidence of ongoing B cell aplasia and CAR T persistence.
In April, Autolus completed an underwritten public offering of 4,830,000 American Depositary Shares ("ADSs") representing 4,830,000 ordinary shares, at a public offering price of $24.00 per ADS, which includes an additional 630,000 ADSs issued upon the exercise in full of the underwriters’ option to purchase additional ADSs. Aggregate net proceeds to Autolus, after underwriting discounts but before estimated offering expenses, were $108.8 million.
Pipeline Updates and Anticipated Milestones:

Initiation of a Phase 2 registration trial of AUTO1 in adult ALL in the fourth quarter of 2019 (pending regulatory feedback). Updated Phase 1 data from the ALLCAR19 clinical trial is expected at ASH (Free ASH Whitepaper) 2019.

Decision on Phase 2 initiation for the Alexander study of AUTO3 in DLBCL is expected for mid-2020. Interim Phase 1 data is expected at ASH (Free ASH Whitepaper) 2019.

Development of AUTO1 in pediatric ALL as part of a pediatric investigational program (PIP). Next data update from CARPALL clinical trial is expected at ASH (Free ASH Whitepaper) 2019. Development program includes a next generation version of AUTO1 (AUTO1NG), which incorporates the CD19 CAR of AUTO1 and a novel CD22 CAR. First preclinical data on CD22 CAR expected to be presented at ASH (Free ASH Whitepaper) 2019.

Additional presentations targeted for ASH (Free ASH Whitepaper) 2019 are: data from AMELIA clinical trial of AUTO3 in pediatric ALL and data from clinical trial of AUTO2 in multiple myeloma.

Next generation (NG) programs for AUTO1, AUTO2, AUTO3 and AUTO6 are expected to enter the clinic in 2020.
"This quarter we have focused on expanding operations at our clinical manufacturing site at the Catapult Cell and Gene facility in Stevenage, UK, enabling us to meet our expected clinical trial demand, including a registration trial with AUTO1. In April, we presented encouraging data on our ALLCAR19 clinical trial of AUTO1 in adult ALL. These data show that AUTO1 is well differentiated from the standard of care in ALL and other CAR T products in development, reaching a high level of clinical activity without inducing severe cytokine release syndrome," stated Dr. Christian Itin, chairman and chief executive officer of Autolus. "For the remainder of 2019, our efforts are focused on moving AUTO1 into a Phase 2 registration trial in adult ALL and advancing AUTO3 in DLBCL toward a Phase 2 decision point in Q2 2020."

Financial results for second quarter 2019:

Cash and equivalents at June 30, 2019 totaled $266.2 million, compared with $217.5 million at December 31, 2018.

Net total operating expenses for the three months ended June 30, 2019 were $37.2 million, net of grant income of $0.3 million, as compared to net operating expenses of $16.5 million, net of grant income of $0.4 million, for the same period in 2018. The increase was due, in general, to the increase in development activity; increased headcount primarily in our development and manufacturing functions; and the cost of being a public company.

Research and development expenses increased to $26.2 million for the three months ended June 30, 2019 from $8.9 million for the three months ended June 30, 2018. Cash costs, which exclude depreciation as well as share-based compensation, increased to $20.2 million from $7.1 million. The increase in research and development cash costs of $13.1 million consisted primarily of an increase of compensation-related costs of $5.6 million primarily due to an increase in headcount to support the advancement of our product candidates in clinical development, an increase of $2.3 million in facilities costs supporting the expansion of our research and translational science capability and investment in manufacturing facilities and equipment, an increase of $3.3 million in research and development program expenses related to the activities necessary to prepare, activate, and monitor clinical trial programs, an increase of $0.8 million in professional fees, an increase of $0.5 million in telecom and software costs, and other additional costs in the amount of $0.4 million.

General and administrative expenses increased to $11.4 million for the three months ended June 30, 2019 from $8.1 million for the three months ended June 30, 2018. Cash costs, which exclude depreciation as well as share-based compensation, increased to $7.3 million from $6.9 million. The increase of $0.4 million consisted primarily of an increase in commercial costs of $1.2 million, compensation-related expense of $0.5 million due to an overall increase in headcount, offset by a decrease in legal and professional fees of $1.3 million related to certain corporate reorganization costs that were incurred in the three months ended June 30, 2018.

Net loss attributable to ordinary shareholders was $28.5 million for the three months ended June 30, 2019, compared to $7.7 million for the same period in 2018.

The basic and diluted net loss per ordinary share for the three months ended June 30, 2019 totaled $(0.65) compared to a basic and diluted net loss per ordinary share of $(0.26) for the three months ended June 30, 2018.

Autolus anticipates that cash on hand provides a runway into the second half of 2021.
Conference Call and Presentation Information

Autolus management will host a conference call today, August 8, at 8:30 a.m. EDT/ 1:30pm BST, to discuss the company’s financial results and operational update.

To listen to the webcast and view the accompanying slide presentation, please go to: View Source

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 2763978. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 2763978.

On August 8, 2019 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to developing and commercializing next-generation medicines to improve the lives of patients with immune-mediated diseases, reported quarter ended June 30, 2019 financial results and provided a corporate update (Press release, Aldeyra Therapeutics, AUG 8, 2019, View Source [SID1234539799]).

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"With Part 1 of our first Phase 3 trial in dry eye disease expected to be completed later this year, Aldeyra continues to advance reproxalap, the lead product candidate in our novel RASP inhibitor platform, towards commercialization," commented Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. "In addition, in the fourth quarter of 2019, we expect to confirm the design of our second Phase 3 trial of reproxalap in allergic conjunctivitis and initiate Phase 3 clinical testing of ADX-2191 in our first retinal indication, proliferative vitreoretinopathy, a rare and potentially blinding inflammatory disorder with no approved therapies."

Recent Highlights and Corporate Updates

Dry Eye Disease – Part 1 of the Adaptive Phase 3 RENEW Trial Expected to be Completed in Fourth Quarter of 2019. Aldeyra announced completion of enrollment in Part 1 of the two-part adaptive Phase 3 RENEW Trial of topical ocular reproxalap in dry eye disease. Following completion of Part 1 of RENEW, expected in the fourth quarter of 2019, Aldeyra plans to report the endpoints, dosing regimen, and sample size for Part 2 of the trial. Top-line results of the RENEW Trial are expected to be announced following completion of Part 2.

Allergic Conjunctivitis – FDA Meeting Scheduled for Fourth Quarter to Confirm Design of Second Phase 3 Clinical Trial. In March 2019, Aldeyra reported positive top-line results from the Phase 3 ALLEVIATE Trial of topical ocular reproxalap in allergic conjunctivitis, and plans to present full results of ALLEVIATE at the American Academy of Ophthalmology 2019 Annual Meeting in October 2019. In addition, in June of 2019, Aldeyra announced results of topical ocular reproxalap in an allergen chamber trial, which demonstrated that ocular itching and redness in reproxalap-treated patients were statistically lower than that of vehicle-treated patients. A meeting with the U.S. Food and Drug Administration (FDA) has been scheduled in the fourth quarter of 2019 to confirm the design of a second Phase 3 trial.

Proliferative Vitreoretinopathy – the Adaptive Phase 3 GUARD Clinical Trial of ADX-2191 Expected to Initiate in the Fourth Quarter of 2019. Later this year, Aldeyra expects to initiate the adaptive Phase 3 GUARD Trial of ADX-2191 for the prevention of proliferative vitreoretinopathy (PVR). The trial will compare recurrence rates across patients treated with ADX-2191 or standard of care following surgical repair of retinal detachment due to PVR. PVR is expected to be the first indication in Aldeyra’s retinal disease platform to begin clinical testing.

Sjögren-Larsson Syndrome – Part 1 of the Phase 3 RESET Trial Completed. In Part 1 of the two-part adaptive Phase 3 RESET trial in Sjögren-Larsson Syndrome, investigator-assessed dermal scaling scores in the six patients treated with 1% reproxalap topical dermatologic formulation were statistically lower than pre-treatment values over six months of therapy, an improvement that was numerically greater than that observed in the three patients treated with vehicle, when adjusted for baseline score. Prior to initiating subsequent clinical testing, Aldeyra plans to discuss the RESET Part 1 results with regulatory authorities. The RESET Trial is a randomized, multi-center, double-masked Phase 3 clinical trial of 1% topical dermal reproxalap for the treatment of ichthyosis, a severe skin disease associated with Sjögren-Larsson Syndrome.

Programs in Systemic Immune-Mediated Diseases Expected to Begin Clinical Testing in 2019. As Aldeyra expands to the development of therapies for the treatment of systemic immune-mediated disease, a Phase 2 clinical trial of ADX-1612 in post-transplant lymphoproliferative disorder and a Phase 1 clinical trial of ADX-629 for the treatment of systemic autoimmune and metabolic disease remain on track to be initiated in the second half of 2019.

Quarter Ended June 30, 2019 Financial Review

For the quarter ended June 30, 2019, Aldeyra reported a net loss of approximately $13.3 million, compared to a net loss of approximately $9.1 million for the quarter ended June 30, 2018. Basic and diluted net loss per share was $0.49 for the quarter ended June 30, 2019, compared to $0.46 per share for the same period in 2018. Losses have resulted from the costs of research and development programs, as well as from general and administrative expenses.

Research and development expenses were $10.7 million for the quarter ended June 30, 2019, compared to $6.8 million for the same period in 2018. The increase of $3.9 million is primarily related to an increase in manufacturing, preclinical, and clinical development costs; an increase in personnel costs; and non-cash compensation costs related to a portion of upfront acquisition consideration that is subject to vesting based on continued service.

General and administrative expenses were $3.1 million for the quarter ended June 30, 2019, compared to $2.4 million for the quarter ended June 30, 2018. The increase of $0.7 million is primarily related to an increase in personnel costs.

For the quarter ended June 30, 2019, total operating expenses were approximately $13.7 million, compared to total operating expenses of approximately $9.2 million for the same period in 2018.

Cash, cash equivalents, and marketable securities were $69.5 million as of June 30, 2019.

Conference Call & Webcast Information

Aldeyra will hold a conference call on Thursday, August 8, 2019, at 8:00 a.m. Eastern Time. The dial-in numbers are 1-877-211-4098 for domestic callers and 1-647-689-6613 for international callers. The conference ID number for the live call will be 9329328. A live webcast of the conference call will also be available on the investor relations page of the Aldeyra Therapeutics corporate website at www.aldeyra.com. After the live webcast, the event will remain archived on the Aldeyra Therapeutics website for one year.

On August 8, 2019 LineaRx reported that it has acquired the assets and intellectual property of Vitatex, a developer of invasive circulating tumor cell (iCTC) analysis technologies (Press release, Vitatex, AUG 8, 2019, View Source [SID1234538579]).

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According to LineaRx — an Applied DNA Sciences subsidiary formed around technologies for DNA design, manufacture, and chemical modification by large scale PCR — Vitatex’s technologies allows iCTCs to be isolated from standard blood samples. These cells can then be cultured and challenged against panels of cancer therapies to help personalize treatments, such as CAR T immunotherapies, for specific patients.

Following the acquisition, LineaRx said that it will work with other companies to enhance their cancer assays, generate personalized redirected cell therapies against cancers with high mutation burdens and metastatic potential, out-license Vitatex’s technologies for use in early cancer diagnostics and prognostics, and design synthetic genes to redirect immune cells against antigens that are recognized by leukocytes that comigrate with iCTCs. It also aims to develop therapeutics that use its proprietary DNA manufacturing methods.