On November 15, 2021 Pulse Biosciences, Inc. (Nasdaq: PLSE), a novel bioelectric medicine company commercializing the CellFX System powered by Nano-Pulse Stimulation (NPS) technology, reported financial results for the third quarter of 2021 (Press release, Pulse Biosciences, NOV 15, 2021, View Source [SID1234595607]).

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Recent Highlights

Achieved third quarter 2021 revenue of $574 thousand, representing 12 Controlled Launch Program participants opting to acquire their CellFX Systems and transition to commercial use
Onboarded 68 CellFX System Controlled Launch Program participants to date and anticipate the remaining to be onboarded in Q4 to complete program enrollment across the U.S., Europe and Canada
Completed enrollment of 150 patients in an FDA IDE approved pivotal comparison study to assess the treatment of cutaneous non-genital warts using the CellFX System, the corresponding 510(k) submission is on track to be completed in the first half of 2022
Completed enrollment of 30 patients as part of a feasibility study to assess the treatment of basal cell carcinoma using the CellFX System, on track for tissue analysis to be completed in Q1 2022
Strengthened the executive leadership team with the appointment of Mitchell E. Levinson as Chief Strategy Officer, to spearhead the Company’s multi-specialty expansion efforts to leverage NPS technology, the CellFX platform and CellFX CloudConnect in applications outside of dermatology
Appointed healthcare industry executive Laureen DeBuono to its Board of Directors
"We made important progress across the business in the third quarter resulting in our first quarter of revenue as the initial Controlled Launch Program participants elected to purchase their CellFX Systems and Cycle Units for commercial use," said Darrin Uecker, President and CEO of Pulse Biosciences. "On the clinical front we continue to prioritize studies to support regulatory submissions to expand the CellFX System’s indications for use in dermatology. At the same time, we are pleased about the investigational preclinical work being done with NPS across other medical specialties. We are laying a foundation to drive value creation and maximize the full potential of the CellFX platform."

Third Quarter 2021 Results

Revenue for the three months ended September 30, 2021 was $574 thousand. System revenue for the three months ended September 30, 2021 was $490 thousand recognized on a non-cash basis resulting from the Controlled Launch Participants opting to acquire CellFX Systems. Cycle units revenue for the three months ended September 30, 2021 was $84 thousand resulting from the purchase of initial cycle units to be used with commercial systems.

Total GAAP gross loss* for the three months ended September 30, 2021 was ($0.2) million. Excluding non-cash expenses for stock-based compensation and depreciation and amortization, non-GAAP gross loss for the three months ended September 30, 2021 was ($0.1) million. As the business transitioned to commercial operations in the third quarter of 2021, all uncapitalized manufacturing operations costs are now recorded in cost of revenue. Prior to commercialization, these costs were recorded in research and development expenses.

Total GAAP operating expenses representing research and development, sales and marketing and general and administrative expenses for the three months ended September 30, 2021 were $14.1 million, compared to $12.9 million for the prior year period. Non-GAAP operating expenses for the three months ended September 30, 2021 were $12.3 million, compared to $10.0 million for the same period in the prior year. The year-over-year increase in operating expenses was primarily driven by the expansion of commercial and operational infrastructure, including increased headcount, to support commercialization activities.

GAAP net loss for the three months ended September 30, 2021 was ($14.3) million compared to ($12.9) million for the three months ended September 30, 2020. Non-GAAP net loss for the three months ended September 30, 2021, was ($12.4) million compared to ($10.0) million for the three months ended September 30, 2020.

Cash, cash equivalents and investments totaled $42.0 million as of September 30, 2021 compared to $47.4 million as of June 30, 2021. Cash used in the third quarter of 2021 totaled $13.8 million excluding net proceeds received in the June 2021 private placement. This compares with $8.3 million used in the same period in the prior year and $15.0 million used in the second quarter of 2021.

* Gross loss is calculated as total revenues less cost of revenues.

Reconciliations of GAAP to non-GAAP operating expenses and net loss have been provided in the tables following the financial statements in this press release. An explanation of these measures is also included below under the heading "Non-GAAP Financial Measures."

Webcast and Conference Call Information

Pulse Biosciences’ management will host a conference call today, November 15, 2021, beginning at 1:30pm PT. Investors interested in listening to the conference call may do so by dialing 1-877-705-6003 for domestic callers or 1-201-493-6725 for international callers. A live and recorded webcast of the event will be available at View Source

On November 15, 2021 Kaleido Biosciences, Inc. (Nasdaq: KLDO), a clinical-stage biotech company with a differentiated, small-molecule approach to treating inflammatory conditions and diseases by selectively targeting the resident microbiome to restore gut-immune homeostasis, reported that CEO Dan Menichella will participate in a fireside chat at the Piper Sandler 33rd Annual Virtual Healthcare Conference (Press release, Kaleido Biosciences, NOV 15, 2021, View Source [SID1234595623]). The presentation will be available on-demand starting at 10:00AM ET on Monday, November 22.

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The webcast of the presentation will be made available in the Investors & Media section of Kaleido’s website at View Source An archived replay will be available for 90 days following the event.

On November 15, 2021 AOP Orphan reported in-licensed the exclusive rights for clinical development and commercialization of Ropeginterferon alfa-2b in Polycythaemia Vera (PV), a rare disease, and other myeloproliferative neoplasms (MPNs) for European, Commonwealth of Independent States (CIS), and Middle Eastern markets. Based on a pivotal study development program, which included the studies PEGINVERA, PROUD-PV and CONTINUATION-PV the European Medicines Agency (EMA) authorized BESREMi for the treatment of PV in February 2019 (Press release, AOP Orphan Pharmaceuticals, NOV 15, 2021, View Source [SID1234595637]). The whole clinical development program was designed and conducted by AOP Orphan in Europe.

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PharmaEssentia Corp., AOP Orphan’s licensor, subsequently submitted the clinical data generated in the clinical development program for marketing authorisation of BESREMi by the FDA in the United States. On March 12, 2021, according to PharmaEssentia, the FDA issued a Complete Response Letter for BESREMi (Ropeginterferon alfa-2b) confirming the safety and efficacy of BESREMi in compliance with FDA regulations. Now, after PharmaEssentia’s manufacturing sites in Taiwan have successfully passed the FDA’s GMP pre-approval inspection in September 2021, the FDA approved BESREMi for sale in the United States.

"FDA’s confirmation of BESREMi’s safety and efficacy, based on AOP Orphan’s clinical study program and scientific knowledge, attests to the quality of work at our company" says Dr. Rudolf Widmann, Member of the Governing Board of AOP Orphan International. "We are extremely happy that now patients in the US have access for treating this rare blood cancer disease. This is the first of AOP Orphan’s ambitious projects to get medicinal products for rare and special diseases approved in the US. This achievement speaks not only to the scientific expertise and clinical development know-how of AOP Orphan, but also supports our company strategy to make drugs for rare and special diseases available worldwide."

Clinical studies conducted by AOP Orphan

Ropeginterferon alfa-2b is a specifically modified pegylated Interferon alpha 2b invented by PharmaEssentia’s CEO KC Lin.

The application of this third-generation interferon to the treatment of PV was invented by Dr. Rudolf Widmann of AOP Orphan: The company has continually invested in its hematology and drug development know-how to get an injection pen for patients’ at-home self-administration approved. Several clinical studies have been conducted by the company leading to a successful marketing authorization of BESREMi for the treatment of Polycythaemia Vera in the EU in February 2019, in Switzerland and in Taiwan in 2020, in Israel and Korea in 2021.

Besides achieving high rates of complete hematologic responses including freedom of phlebotomy in 8 out of 10 patients, BESREMi offers the possibility of disease modification and eventually operational cure in a subset of patients. This is exemplified by a decrease of mutant JAK2 allele burden (the disease-causing oncogene) from 37.3% at baseline to 7.3% in patients receiving Ropeginterferon alfa-2b, while an increase from 38.1% to 42.6% in the control group receiving hydroxyurea/best available therapy was observed (p<0.0001).1

Polycythaemia Vera patients in approximately 20 European countries are already being treated with Ropeginterferon alfa-2b. AOP Orphan is continuously working on increasing access for many more patients and continues the clinical development of BESREMi.

About BESREMi
BESREMi is a long-acting, mono-pegylated proline interferon (ATC L03AB15). Its unique pharmacokinetic properties offer a new level of tolerability. BESREMi is designed to be conveniently self-administered subcutaneously with a pen once every two weeks, or monthly after stabilization of hematological parameters. This treatment schedule is expected to lead to overall better safety, tolerability and adherence compared to conventional pegylated interferons. Ropeginterferon alfa-2b was discovered by PharmaEssentia, a long-term partner of AOP Orphan.

For the EMA Summary of Product Characteristics please visit: View Source

On November 15, 2021 StacheStrong, a non-profit devoted to raising funds and awareness for brain cancer research, reported the launch of the Glioma Connectome Project (GCP), a newly-established consortium of neurosurgery centers to advance clinical research and treatment for patients with glioblastoma, an aggressive form of brain cancer (Press release, StacheStrong, NOV 15, 2021, View Source [SID1234595653]). The mission is to generate groundbreaking brain connectomics research to drive clinical and practice changes at neurosurgical centers across the U.S.

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The nascent field of connectomics is a global interdisciplinary effort to study brain connectivity which has helped identify and understand individual brain networks responsible for functions such as language, emotion, and cognition. This new consortium will translate breakthrough neuroscience into real-world applications and potential new therapies for patients with glioblastoma and other types of brain cancer.

"We are excited to launch this consortium of leading institutions in the United States, to provide hope for patients diagnosed with brain cancer, and serve as a catalyst for change," said Colin Gerner, President and Co-Founder of StacheStrong. "This promising new study brings together top neurosurgeons and neuroradiologists to better learn about brain connectomics to perform more successful glioma surgeries, as well as how to better treat and rehabilitate after surgery."

The GCP consortium, which includes the University of Pennsylvania, University of Miami, Mount Sinai, Henry Ford Health System, Northwestern University and University of Nebraska Medical Center, will launch a series of prospective observational studies that harness large-scale multi-institution clinical data produced in the routine care of glioma patients. The study will collect patient data from MRI scans, to produce personalized brain maps. Harnessing machine learning techniques and cutting-edge software, the project will be structured to maximize the quality and volume of data, while minimizing the time and resources needed from physicians and patients.

"The GCP is a critical effort to translate the breakthrough findings of connectomics into neurological care. It embodies the common cause of these leading institutions to properly equip physicians fighting this devastating disease," said Dr. Michael Sughrue, a global thought leader in connectomics and Chief Medical Officer of Omniscient Neurotechnology.

About the Glioma Connectome Project (GCP)

The Glioma Connectome Project is a consortium of leading brain tumor centers dedicated to studying and exploring the wiring of the human brain, or "connectome", to further our understanding of the origins and progression of glioblastomas as well as developing and evaluating surgical, radiation, medical and immunological therapies. The consortium’s objectives include improving current treatment paradigms, developing new biomarkers and endpoints in glioma therapy, measuring the benefits and risks of glioma therapy including surgery, radiation, chemotherapy, electrical field therapy, and others. The consortium includes the University of Pennsylvania, University of Miami, Mount Sinai, Henry Ford Health System, University of Nebraska, and Northwestern University.

On November 15, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported a business update and an update on its cash position at the end of September 2021 (Press release, ERYtech Pharma, NOV 15, 2021, View Source [SID1234595692]).

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"While the outcome of our TRYbeCA-1 trial in second-line pancreatic cancer did not achieve its primary endpoint of overall survival, we remain encouraged by the observed improvement of overall survival in a subset of patients treated with FOLFIRI and will continue analyzing the sizeable TRYbeCA-1 data set in order to distill the reasons for this disappointing outcome," said Gil Beyen, CEO of ERYTECH. "We are also encouraged by the progress we are making towards seeking an approval for eryaspase for the treatment of ALL patients who experienced hypersensitivities to pegylated asparaginase. The dialogue with the FDA is continuing and we are hopeful we can submit our first BLA around year end. We were pleased with the granting of the Fast Track designation for this high unmet need indication in July."

Business Highlights

Path to BLA in hypersensitive ALL, based on results of NOPHO-sponsored Phase 2 trial

The NOPHO trial evaluated the safety and pharmacological profile of eryaspase in acute lymphoblastic leukemia (ALL) patients who had previously experienced hypersensitivity reactions to pegylated asparaginase therapy. 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 hypersensitivity reactions.

The Company continued its interactions with the U.S. Food and Drug Administration (FDA) regarding a potential regulatory approval in this indication based on the NOPHO-sponsored trial. A pre-BLA meeting to discuss the submission of a Biologics License Application (BLA) took place in June after which the Company confirmed its intention to submit a BLA subject to successful completion of remaining activities.
In July, the Company announced that the FDA had granted eryaspase Fast Track designation for the treatment of ALL patients who have developed hypersensitivity reactions to E. coli-derived pegylated asparaginase.
Subject to review of remaining information requests, the Company intends to submit a BLA around year-end.

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

As reported in late October, the Phase 3 TRYbeCA-1 trial did not meet the primary efficacy endpoint of overall survival (OS). The median OS for patients treated with eryaspase plus chemotherapy was 7.5 months, compared to 6.7 months for chemotherapy alone, with an OS hazard ratio (HR) of 0.92 in the intent-to-treat (ITT) population (p-value 0.375).

The prespecified subgroup of patients treated with eryaspase and FOLFIRI, an irinotecan-based chemotherapy, demonstrated a nominal increase in median OS of 2.3 months, from 5.7 to 8 months (HR = 0.77; per protocol population), which the Company believes merits further investigation.
Patients treated with eryaspase demonstrated improved disease control compared to patients treated with chemotherapy only. Other secondary endpoints showed nominal improvement.
The safety profile of eryaspase was consistent with earlier clinical trials results and safety reviews.

Final data are being analyzed and will be presented at an upcoming medical conference.

rESPECT, Phase 1 investigator-sponsored trial (IST) 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 locally advanced and metastatic pancreatic cancer in approximately 18 patients.

Patient enrollment started in January 2021, and the first dose cohort (75 U/kg) of three patients was enrolled by the end of February. No dose-limiting toxicity (DLT) was observed, and the trial was escalated to the next dosing cohort (100 U/kg).
After review of the safety data in the first two dose cohorts, the dose escalation committee concluded that the novel combination of mFOLFIRINOX plus eryaspase was well tolerated with no DLT. Consequently, the maximum tolerated dose (MTD) was determined at a dose of 100 U/kg eryaspase.
Additionally, all six patients evaluated for response achieved disease control; three patients with objective response and three with stable disease.

The trial will continue enrolling up to approximately 18 patients. Reporting of final data is expected in the first half of 2022.

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 the disappointing results of eryaspase in combination with a gemcitabine-based chemotherapy in the TRYbeCA-1 trial in second-line pancreatic cancer, the Company has, in consultation with the trial’s Steering Committee, decided to stop further enrollment in the TRYbeCA-2 trial.

The results of the patients enrolled in the TRYbeCA-2 trial to date are expected to be reported in the first half of 2022.

Process to review strategic options and partnering alternatives launched

The Company launched a process and appointed a specialized advisor to evaluate its strategic and partnering alternatives, including for the further development and commercialization of eryaspase. Gil Beyen, the CEO of ERYTECH, will lead these partnering efforts and take on the role of acting Chief Business Officer (CBO), as Jean-Sebastien Cleiftie, current CBO of ERYTECH will leave the Company at the end of this month.

Update on Q3 2021 Cash Position

As of September 30, 2021, ERYTECH had cash and cash equivalents totaling €38.0 million (approximately $43.9 million), compared with €44.4 million as of December 31, 2020 and €46.3 million on June 30, 2021. The €6.5 million decrease in cash position during the first nine months of 2021 was the result of a €7.8 million net cash utilization, which was mostly comprised of a €46.2 million net cash utilization in operating activities, €0.3 million used for investing activities and €38.8 million generated in financing activities, while the variation of the U.S. dollar against the euro led to a €1.3 million positive currency exchange impact.

Financing activities in the first nine months of 2021 included a $8 million placement in the United States through the Company’s at-the-market (ATM) equity financing program for net proceeds of €6.4 million, a $30 million Registered Direct offering for net proceeds of €22.4 million, and the drawdown of four tranches under the convertible notes (OCABSA) financing agreement signed with Alpha Blue Ocean, for net proceeds of €11.4 million.

At the date of this press release, nine OCABSA tranches have been called since the initiation of the program in June 2020. During the last 12 months, the OCABSA converted notes, together with the shares issued under the ATM program, have resulted in the issuance of 4,690,904 new shares and 235,690 warrants, representing 17.6% of the Company’s outstanding share capital.

The Company believes that its current cash position can fund its planned operating expenses and current programs into the second quarter of 2022. Further, the Company has engaged in cash preservation measures and believes that these measures, together with further utilization of the OCABSA agreement, subject to the regulatory limit of 20% dilution, could extend its cash horizon into the third quarter of 2022. The Company is currently exploring potential financing and partnering options to further extend its cash horizon beyond key 2022 development milestones.

The release on October 25, 2021 of the TRYbeCA-1 Phase 3 trial in pancreatic cancer, which did not meet its primary endpoint, is considered a triggering event for impairment analysis, which will require the Company to test tangible and intangible assets for possible impairment. The Company is therefore not in a position to announce full financial results for the third quarter of 2021 until current uncertainties on business assumptions are clarified. The Company will conduct an impairment analysis in light of its new business situation, which may potentially lead to an impairment of some of its assets.

Key News Flow and Milestones Expected Over the Next 12 Months

Planned BLA submission of eryaspase in hypersensitive ALL (around year end 2021)
Results from the Phase 1 rESPECT Trial of eryaspase in combination with mFOLFIRINOX in first-line pancreatic cancer (1H 2022)
Presentation of full dataset of TRYbeCA-1 trial at a medical meeting (1H 2022)
Data from the randomized Phase 2 TRYbeCA-2 trial of eryaspase in TNBC (1H 2022)
Third Quarter 2021 Conference Call Details

ERYTECH management will hold a conference call and webcast on Tuesday, November 16, 2021 at 8:30am EST / 2:30 pm CET to discuss the recent business and financial updates. Gil Beyen, CEO, Eric Soyer, CFO/COO, and Iman El-Hariry, CMO, will deliver a brief presentation, followed by a Q&A session.

The audio call is accessible via the below registering link:

View Source (Conference ID : 6425429)

Once registered, participants will receive a unique access code and the call number details to join the teleconference.

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: 6425429#.

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 Fourth Quarter and Full Year 2021: March 11, 2022 (after U.S. market close), followed by a conference call & webcast on March 14, 2022 (2:30pm CET/8:30am ET)

ERYTECH plans on attending the following upcoming investor conferences:

Jefferies 2021 Global Healthcare Conference, November 16-19, London
LifeSci Partners 11th Annual Corporate Access Event, January 5-7, 2022
H.C. Wainwright, BioConnect Conference, January 10-13, 2022
JPMorgan HealthCare Conference, January 10-13, 2022, San Francisco