Inclusion of the first patient in the TEMOkids pediatric clinical trial

On May 4, 2021 ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs, reported the inclusion of a first patient in the TEMOkids pediatric clinical study (Press release, ORPHELIA Pharma, MAY 4, 2021, View Source [SID1234579088]). The TEMOkids study aims to determine the pharmacokinetic parameters, tolerance, acceptability and response to treatment of Kimozo, the first oral suspension of temozolomide, in the pediatric population (www.temokids.eu).

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Temozolomide is an anti-cancer drug used in treatment regimens for refractory or relapsed neuroblastoma that mainly affect very young children. It is also used to treat rhabdomyosarcoma and certain brain tumors. The only oral forms of temozolomide currently available are capsules which are not suited to children. In fact, to be administered, the capsules must be opened and their content added to food. In young children, the formulation is essential to ensure correct medication intake, safety, accuracy of the delivered dose and adherence to treatment. To meet the requirements of onco-pediatricians and the needs of children, ORPHELIA Pharma, working in close collaboration with Gustave Roussy hospital teams, has developed Kimozo, the first ready-to-use drinkable suspension of temozolomide.

"The TEMOkids trial, during which our oral suspension of temozolomide will be administered to children for the first time, is a key stage in the clinical development of Kimozo," says Caroline Lemarchand, Chief Pharmaceutical Development Officer for ORPHELIA Pharma, "This study will evaluate the pharmacokinetics of temozolomide, alone or in combination with other treatments, in children from 1 year of age. It will also define the tolerance and acceptability of Kimozo within the study population, and collect data on tumor response to treatment. The first results of this clinical trial are expected mid-2022."

"We are very pleased to be involved in the clinical development of this long-awaited new pediatric formulation of temozolomide for the treatment of cancer in children," says Dr Samuel Abbou, pediatric oncologist at the Gustave Roussy hospital, and coordinating investigator of the TEMOkids study. "The study plans to recruit 40 children in 5 countries; that is France, Germany, Spain, the UK and the Netherlands, thanks to the support of a structured European-scale pediatric oncology network."

"We have received an enthusiastic response from the members of the European pediatric oncology community who have chosen to participate in this clinical trial," says Hugues Bienaymé, General Manager and Chief Scientific Officer for ORPHELIA Pharma, before adding by way of a conclusion that "TEMOkids complements our ongoing bioequivalence study in adult patients, and should support the filing of the Kimozo registration dossier at European level in 2023."

About the TEMOkids study (NCT04610736)

The clinical trial entitled "TEMOkids Study: A Phase I Pediatric Study for KIMOZO, Oral Suspension of temozolomide" is an international, open-label, non-randomized, phase 1 study involving 40 pediatric patients aged 1 year and over. The main objective of this study is to determine the pharmacokinetic parameters of Kimozo in the population concerned by the study. The secondary objectives are to evaluate its tolerance and acceptability among children, and their response to treatment.

Ten clinical centers are involved in the TEMOkids study: Gustave Roussy in Villejuif (the coordinating center); Institut Curie in Paris, France; La Timone Children’s Hospital in Marseille, France; The Institute of Pediatric Hematology and Oncology in Lyon, France; The Oscar Lambret Center in Lille, France; The Charité University Medical Hospital in Berlin, Germany; The Hopp Children’s Cancer Center in Heidelberg, Germany; The Princess Máxima Center for Pediatric Oncology in Utrecht, the Netherlands; The Vall d’Hebron Research Institute in Barcelona, Spain; and Southampton General Hospital in the United Kingdom.

For further information, please go to www.temokids.eu.

About Kimozo

Kimozo (also known as ORP-005 or Ped-TMZ) is a ready-to-use and taste-masked oral suspension of temozolomide that is currently under development to address the needs of children. Kimozo is an investigational medicinal product not yet approved for marketing.

Invectys, Inc appoints Biopharma Veteran, Praveen Tyle, PhD as President & CEO

On May 4, 2021 Invectys, Inc., a clinical-stage immunotherapy company headquartered in Houston and dedicated to the development of a new generation of products for cancer patients, reported the appointment of Praveen Tyle, PhD, as President & CEO and election to its Board of Directors (Press release, Invectys, MAY 4, 2021, View Source [SID1234579087]). Dr. Tyle brings over 35 years of experience in both large and small pharma and biotech companies, most recently serving as Executive V.P. for Research and Development of the public company, Lexicon Pharmaceuticals, Inc. Earlier, Dr. Tyle served as the Corporate Senior Vice President and Chief Scientific Officer of Bausch and Lomb and then as Senior Vice President and Global Head of Business Development and R&D at Novartis OTC. Subsequently, Dr. Tyle joined Osmotica Pharmaceutical Corp. serving as President and CEO where he led the successful merger of the Company into Avista Partners in 2016.

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Cary McNair, Chairman of the Board of Invectys and Chairman/CEO of the Houston-based McNair Interests stated: "We are delighted to welcome Praveen as CEO of Invectys. Having served over 3 decades at the intersection of pharma deep science and biopharma business development, Praveen is well poised to lead the commercial development of Invectys’ innovative immunotherapy platforms. His proven management skills and past commercial success bodes well for his new leadership role to steer Invectys into a new stage of commercial development."

Founding investor Shannon Fairbanks, Chair of the Fairbanks Investment Fund Holdings LLC noted: "This is an exciting period producing an explosion of new ideas in the health care sector which is starting to deliver innovative products to cancer patients. Praveen’s vision and leadership will help to deliver a next generation of breakthrough advances in immunotherapy".

Dr Tyle noted, "I am excited to take the helm at Invectys at this propitious time and I look forward to working with the talented team at Invectys to deliver products based on the Company’s strong scientific promise. I intend to apply my biotech and pharma management and product development experience to bring exciting new immunotherapy solutions to market."

In 2020, Invectys was awarded a $14.5 million product development research grant from the prestigious Cancer Prevention and Research Initiative of Texas ("CPRIT") for clinical development of its pathbreaking CAR-T treatment for solid tumors.

Novo Nordisk A/S – Share repurchase programme

On May 4, 2021 Novo Nordisk reported that initiated a share repurchase programme in accordance with Article 5 of Regulation No 596/2014 of the European Parliament and Council of 16 April 2014 (MAR) and the Commission Delegated Regulation (EU) 2016/1052 of 8 March 2016 (the "Safe Harbour Rules") (Press release, Novo Nordisk, MAY 4, 2021, View Source [SID1234579086]). This programme is part of the overall share repurchase programme of up to DKK 17 billion to be executed during a 12-month period beginning 3 February 2021.

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Under the programme initiated 3 February 2021, Novo Nordisk has repurchased B shares for an amount up to DKK 3.0 billion in the period from 3 February 2021 to 3 May 2021. The programme is now concluded.

Since the announcement as of 26 April 2021, the following transactions have been made:

With the transactions stated above, Novo Nordisk owns a total of 5,657,389 B shares of DKK 0.20 as treasury shares, corresponding to 0.2% of the share capital. The total amount of A and B shares in the company is 2,310,000,000 including treasury shares.

Novo Nordisk expects to repurchase B shares for an amount up to DKK 17 billion during a 12- month period beginning 3 February 2021. As of 3 May 2021, Novo Nordisk has since 3 February 2021 repurchased a total of 6,754,777 B shares at an average share price of DKK 444.12 per B share equal to a transaction value of DKK 2,999,924,849.

Genprex Initiates Site Recruitment for Acclaim-2 Clinical Trial for the Treatment of Non-Small Cell Lung Cancer

On May 4, 2021 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported it has commenced clinical trial site recruitment for its upcoming Acclaim-2 clinical trial for the treatment of non-small cell lung cancer (NSCLC) (Press release, Genprex, MAY 4, 2021, View Source [SID1234579085]).

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Acclaim-2 is an open-label, multi-center Phase 1/2 clinical trial that combines Genprex’s lead drug candidate, REQORSA immunogene therapy, with Merck & Co, Inc’s Keytruda in patients with late-stage NSCLC who are low expressors (1% to 49%) of the protein programmed death-ligand 1 ("PD-L1").

"Our clinical team remains focused on engaging with leading clinical investigators and esteemed research institutions in order to further our mission of bringing hope to lung cancer patients who are in need of new treatment options," said Rodney Varner, President and Chief Executive Officer of Genprex. "We look forward to selecting the most optimal study sites, which will help to further fuel the success of our clinical trials."

The Company plans to conduct the Acclaim-2 clinical trial in approximately 15 sites with about 150 patients in an adaptive Phase 1/2 design. The final protocol is subject to change based on input from investigators.Additional information on the Acclaim-2 clinical trial will be posted on ClinicalTrials.gov.

ERYTECH Provides Business Update and Reports Financial Results for the First Quarter of 2021

On May 4, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, today provided a business update and reported its financial results for the first quarter of 2021 (Press release, ERYtech Pharma, MAY 4, 2021, View Source(U.S.)%20and,its%20financial%20results%20for%20the [SID1234579076]).

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"The first quarter of 2021 has again been one of tremendous progress, and has further set the stage for a catalyst-rich second half of this year," said Gil Beyen, CEO of ERYTECH. "All four of our clinical programs are expected to report significant defining events before the end of the year, two of which potentially supporting the application for regulatory approvals within the next twelve months. Our 512-patient pivotal Phase 3 trial in second-line pancreatic cancer, TRYbeCA-1, is on track for final results in the fourth quarter. Subject to positive results, we expect to submit our applications for approval in this indication in the United States and Europe in the first half of 2022. The second potentially pivotal track of eryaspase is for the treatment of ALL patients who developed hypersensitivities to pegylated asparaginase. We recently requested a pre-BLA meeting with the FDA to discuss the path to approval in this indication and we are targeting the potential submission of a Biologics License Applicaton (BLA) in the second half of this year. We are also continuing work to further broaden the indication scope of eryaspase. The safety profile and encouraging clinical activity observed in the first dose cohort of the ongoing Phase 1 IST in first line pancreatic cancer add to our conviction that eryaspase can be a meaningful contributor to helping cancer patients live longer, better. "

Business Highlights

TRYbeCA-1, pivotal Phase 3 clinical trial in second-line advanced pancreatic cancer

TRYbeCA-1 is a randomized, controlled Phase 3 pivotal trial, evaluating eryaspase in second-line advanced pancreatic cancer at approximately 90 sites in the United States and in Europe. Eryaspase, in combination with standard chemotherapy (gemcitabine/nab paclitaxel or an irinotecan-based regimen), is compared with standard chemotherapy alone in a 1 to 1 randomization. The primary endpoint is overall survival (OS).

Enrollment was completed in January 2021. A total of 512 patients were randomized in the trial, above the target enrollment of 482 patients.

In February 2021, an interim efficacy and safety analysis was performed by an independent data monitoring committee (IDMC), which recommended the trial continue without modification to its final analysis. As with the three previous IDMC reviews, no safety issues have been identified and the Company remains blinded to the primary and secondary endpoint efficacy data.

Reporting of the final results from this trial is expected in the fourth quarter of 2021.

Phase 2 trial in acute lymphoblastic leukemia (ALL), sponsored by the Nordic Society of Pediatric Hematology and Oncology (NOPHO)
The NOPHO trial evaluated the safety and pharmacological profile of eryaspase in ALL patients who had previously experienced hypersensitivity reactions to pegylated asparaginase therapy. The primary objective of the trial was to assess asparaginase enzymatic activity. In December 2020, positive trial results were presented at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting. Eryaspase in combination with chemotherapy, administered every two weeks, provided a sustained asparaginase enzyme activity level, and was generally well tolerated with few hypersentivity reactions.

The Company continued its interactions with the FDA regarding a potential regulatory approval in this indication based on the NOPHO-sponsored trial. In April 2021, ERYTECH requested a pre-BLA meeting to discuss the submission of a Biologics License Application.

Subject to the feedback from this meeting, the Company plans to potentially submit the BLA in the second half of 2021.

TRYbeCA-2, randomized Phase 2 clinical trial in triple-negative breast cancer (TNBC)

The TRYbeCA-2 trial is evaluating eryaspase in combination with gemcitabine and carboplatin chemotherapy, compared to chemotherapy alone, in metastatic TNBC. Target enrollment is approximately 64 patients. The primary end point of the trial is objective response rate.

Following a recommendation of the trial’s investigators, the trial’s inclusion criteria were modified in January 2021 to include second-line patients.

The TRYbeCA-2 Steering Committee met in April 2021 and recommended the trial continue without modification after review of the safety data of the first 19 patients.

Initial data from the TRYbeCA-2 trial are expected to be reported in the fourth quarter of 2021.

rESPECT, Phase 1 investigator-sponsored trial in first-line pancreatic cancer

rESPECT is a Phase 1 trial, sponsored by the Georgetown Lombardi Comprehensive Cancer Center, evaluating the safety of eryaspase in combination with mFOLFIRINOX as a first-line treatment for advanced and locally advanced pancreatic cancer in approximately 18 patients.

Patient enrollment started in January 2021, and the first dose cohort of three patients was enrolled by the end of February.

After review of the safety data, the Dose Escalation Committee concluded that no dose-limiting toxicity (DLT) had been observed in the first cohort treated at a therapeutic dose of 75 U/kg eryaspase, and that the treatment was well tolerated in the cohort thus far.

After the first dose cycle of treatment, two of the three patients had a partial response and significantly decreased levels of CA19-9, a pancreatic cancer tumor marker; the third patient had stable disease after the first cycle of treatment.
The trial has been escalated to the next dosing cohort, where the dose level will be increased to 100 U/kg (the same dose as in TRYbeCA-1) eryaspase. This will be the highest dose level cohort in the trial and the presumed maximum tolerable dose (MTD) assuming no dose limiting toxicity is observed.

Determination of the MTD is expected in the second half of 2021.

First Quarter 2021 Financial Results and Cash Guidance

Key financial figures for the first quarter of 2021 compared with the same period of the previous year are summarized below:

Net loss for the first quarter of 2021 was €11.9 million, down €5.6 million (-32%) year-over-year, with a €5.8 million decrease (-31%) in operating loss and a €0.2 million decrease in financial income. The €5.8 million decrease in operating loss was attributable to the €4.8 million decrease in preclinical and clinical development expenses, concurrent with the completion of patient enrollment in the Company’s Phase 3 clinical trial in pancreatic cancer, a €0.3 million decrease in general and administrative expenses, and a €0.7 million increase in other income, mostly related to R&D tax credits. The €0.2 million decrease in financial result was mostly related to the IFRS accounting of the convertible notes.

As of March 31, 2021, ERYTECH had cash and cash equivalents totaling €37.4 million (approximately $43.9 million), compared with €44.4 million on December 31, 2020. The €7.0 million decrease in cash position during the first quarter of 2021 was the result of a 3-month €7.6 million net cash utilization, which was mostly comprised of a €16.3 million net cash utilization in operating activities, €0.1 million used for investing activities and €8.8 million generated in financing activities, while the variation of the U.S. dollar against the euro led to a €0.6 million positive currency exchange impact.

Financing activities in the first quarter of 2021 included the placement of 744,186 newly issued ordinary shares underlying American Depositary Shares (ADSs) in the United States through the Company’s at-the-market (ATM) equity financing program for net proceeds of €6.4 million, and the draw down of one tranche under the convertible notes (OCABSA) financing agreement signed with Alpha Blue Ocean, for net proceeds of €2.9 million.

As of the date of this press release, all notes of the six OCABSA tranches called to date have been converted and, together with the shares issued under the ATM program, have resulted in the issuance of 3,677,676 new shares and 202,020 warrants, representing 18.3% of the Company’s outstanding share capital to date.

Financing Round of $30 million on April 29, 2021

On April 29, 2021, the Company announced that it has entered into definitive agreements with several health-care focused institutional and accredited investors for the purchase and sale of 1,034,483 units ("Units"), each Unit consisting of four ordinary shares in the form of ADSs and three warrants, each to purchase one ordinary share, in a registered direct offering to specified categories of investors.

The subscription price for one Unit is corresponding to $7.25 (€6.01) per ADS and associated 0.75 warrant. The warrants have an exercise price of €7.50 ($9.05) per share, will be immediately exercisable upon issuance and will expire two years from the issuance date.

The aggregate gross proceeds to the Company from the sale of the Units, excluding any exercise of the warrants, is expected to be approximately $30.0 million. The Company intends to use the net proceeds from this offering to fund further development of its products candidates, including through the completion of the Phase 3 TRYbeCA-1 trial and the costs to prepare the associated regulatory filings, and for associated working capital and other general corporate purposes.

The Company believes that its current cash position and the net proceeds from the April 29, 2021 offering can fund its planned operating expenses and current programs into the first quarter of 2022. Further, the Company believes that it would be able to fund operations into the third quarter of 2022 if it utilizes the OCABSA agreement, subject to the regulatory limit of 20% dilution.

Key News Flow and Milestones Expected Over the Next 12 Months

Final results from TRYbeCA-1 Phase-3 trial of eryaspase in 2L PAC (Q4 2021)

Potential eryaspase BLA filing for ALL (2H 2021)

Initial data from the randomized Phase 2 TRYbeCA-2 trial of eryaspase in TNBC (Q4 2021)

Determination of the maximum tolerated dose in rESPECT, Phase 1 IST in 1L PAC (2H 2021)

First Quarter 2021 Conference Call Details

ERYTECH management will hold a conference call and webcast on Wednesday, May 5, 2021 at 8:30am ET / 2:30 pm CET on the business highlights and financial results for the quarter ended March 31, 2021. Gil Beyen, CEO, Eric Soyer, CFO/COO, and Iman El-Hariry, CMO, will deliver a brief presentation, followed by a Q&A session.

The call is accessible via the below teleconferencing numbers, followed by the Conference ID#: 3887923#

USA/Canada: +1 (833) 818-6807 France: +33 1 70 80 71 53
International Dial-In Number: +1 (409) 350-3501 United-Kingdom: +44 2031070289

The webcast can be followed live online via the link: View Source

An archived replay of the call will be available for 7 days by dialing + 1 855 859 2056, Conference ID: 3887923#.

An archive of the webcast will be available on ERYTECH’s website, under the "Investors" section at investors.erytech.com

Financial Calendar 2021

Business Update and Financial Highlights for the Second Quarter of 2021: September 20, 2021 (after U.S. market close), followed by a conference call & webcast on September 21, 2021 (2:30pm CET/8:30am ET)

Business Update and Financial Highlights for the Third Quarter of 2021: November 15, 2021 (after U.S. market close), followed by a conference call & webcast on November 16, 2021 (2:30pm CET/8:30am ET)

ERYTECH plans on attending the following upcoming investor conferences:

Jefferies 2021 Global Healthcare Conference, June 1-4, New-York (Virtual)

JMP Securities Life Science Conference, June 16-17, New York (Virtual)

European Midcap Event – June 24, Paris (Virtual)

BTIG Biotechnology Conference – August 9-10, New York (Virtual)