On October 14, 2020 Vericel Corporation (NASDAQ:VCEL), a leader in advanced therapies for the sports medicine and severe burn care markets, reported preliminary financial results for the quarter ended September 30, 2020 (Press release, Vericel, OCT 14, 2020, View Source [SID1234568468]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Preliminary total net revenues for the third quarter are expected to be approximately $32 million, including approximately $24.2 million of MACI (autologous cultured chondrocytes on porcine collagen membrane) net revenue, approximately $6.7 million of Epicel (cultured epidermal autografts) net revenue, and approximately $1.2 million of revenue related to the procurement of NexoBrid (concentrate of proteolytic enzymes enriched in bromelain) by the U.S. Biomedical Advanced Research and Development Authority (BARDA) for emergency response preparedness.

The company generated approximately $4.6 million of operating cash flow in the third quarter. As of September 30, 2020, the company had approximately $85.5 million in cash and investments and no debt, compared to $79.0 million as of December 31, 2019.

"We are very pleased with our third quarter results as we generated consistent double-digit growth in revenue, implants and biopsies for MACI and achieved a record monthly high for biopsies in September," said Nick Colangelo, President and CEO of Vericel. "Moreover, the robustness of our business model was demonstrated as we generated positive operating cash flow for the quarter. While considerable uncertainties related to COVID-19 remain, given the strength of our patient pipeline, we expect to maintain strong MACI growth in the fourth quarter. We look forward to providing further updates to investors during our upcoming virtual Analyst and Investor Day webcast and our third quarter earnings call."

As previously announced, the company will host a virtual Analyst and Investor Day on October 16, 2020, at 9:00am Eastern Time. The company also will host a webcast and conference call to discuss its third quarter 2020 financial results and business highlights on November 5, 2020, at 8:30am Eastern Time. Webcast information can be found on the events and presentation section of the Investor Relations website at View Source

On October 14, 2020 PierianDx, the leading clinical genomics informatics company, and Pillar Biosciences, a leading next-generation sequencing clinical oncology company, reported a partnership that enables Pillar Biosciences to sell directly the PierianDx clinical genomic report in combination with its cancer profiling assays (Press release, PierianDx, OCT 14, 2020, View Source [SID1234568467]). Under the agreement, PierianDx will integrate the Pillar Biosciences PiVAT bioinformatics pipeline in the PierianDx environment to provide users with an accelerated implementation, including assay-specific variant filters, to produce best-in-class clinical genomics reports and enable a complete sample-to-answer solution.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Laboratories worldwide use Pillar Biosciences’ assays to detect pivotal genetic variants for cancer and other diseases. Pillar Biosciences’ targeted panels utilize a proprietary enrichment chemistry, SLIMamp, that enables rapid, automated amplification of overlapping amplicons in a single tube using minimal sample inputs. Pillar Biosciences’ complementary bioinformatics pipeline, PiVAT, is optimized for efficiency, reducing reflex testing and enabling low-frequency variant calling for every type of variant while consuming limited computing resources. PierianDx provides the Clinical Genomics Workspace, which consists of intuitive software and a robust clinical Knowledgebase that transforms variant information into a highly-structured and comprehensive physician-ready report.

Streamlining NGS along with clinical genomic interpretation and reporting, especially for sophisticated panels, remains time-consuming and challenging. By packaging the Pillar Biosciences panels with the PierianDx report, the result will be an out-of-the-box clinical genomic report that is optimized for each assay. Furthermore, the solution will be available to customers globally through secure, and in the case of European regions, Global Data Protection Regulation (GDPR)-compliant, cloud instances. As a result, laboratories and healthcare organizations worldwide will have a highly reliable method for providing these tests to enable precision diagnostics and treatments for their patients.

"The assays from Pillar Biosciences provide robust detection for all variant types in a simplified workflow," states Gang Song, PhD, CEO of Pillar Biosciences. "By optimizing variant filters and integrating with the PiVAT pipeline, PierianDx is streamlining interpretation and reporting, which adds substantial value for our customers."

Rakesh Nagarajan, MD, PhD and Founder and Chief Technical and Visionary Officer of PierianDx, adds, "We are thrilled to provide this support for the Pillar Biosciences assays. As the market shifts to more sensitive assays in the LDT space and leading assay manufacturers seek in vitro diagnostic (IVD) approval, we are committed to addressing challenges that arise from even greater complexity in the interpretation and reporting process."

On October 14, 2020 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported platform updates and disclosed five additional programs from its preclinical pipeline (Press release, Arvinas, OCT 14, 2020, View Source [SID1234568465]). Arvinas’ portfolio encompasses a range of validated and undruggable targets in oncology, immuno-oncology, and neuroscience.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to expand our pipeline and further our leadership position in targeted protein degradation by leveraging the PROTAC Discovery Engine, our integrated platform that we’ve been advancing since 2013," said John Houston, Ph.D., President and Chief Executive Officer of Arvinas. "With the programs introduced today, and the important breakthroughs we’ve made over the years – such as achieving oral bioavailability in human patients and successfully penetrating the blood-brain barrier in preclinical studies – we make it clear that we have the ability to rapidly progress Arvinas’ deep pipeline in order to benefit patients in multiple areas of high unmet need."

"The targets we announced today represent a mix of oncology, immuno-oncology and neuroscience programs," said Ian Taylor, Ph.D., Chief Scientific Officer of Arvinas. "Our progress with classic ‘undruggable’ targets like KRAS reinforces our commitment to finding solutions for patients and demonstrates the power of Arvinas’ PROTAC Discovery Engine in generating novel therapies."

In addition to progressing its platform and preclinical pipeline, Arvinas is testing two PROTAC protein degraders in human clinical trials: ARV-110 for the treatment of men with metastatic castrate-resistant prostate cancer and ARV-471 for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer. Arvinas plans to share updated data for these programs later in the fourth quarter of 2020.

Newly Announced Programs

BCL6 (Oncology)

B-cell lymphoma 6 protein (BCL6) is a transcriptional repressor implicated in B cell lymphomas and facilitates B cell tolerance of rapid proliferation and somatic gene recombination via repressing cell cycle checkpoints, terminal differentiation, apoptosis, and the DNA damage response. PROTAC-mediated degradation would address the scaffolding function of BCL6. Arvinas anticipates filing an IND for this program in 2022.
KRAS (Oncology)

Kirsten rat sarcoma (KRAS) is a classic "undruggable" target, due to its lack of deep "pockets," and is associated with poor prognosis and resistance to standards of care in several tumor types. Arvinas is developing pan-KRAS mutant and mutant-specific KRAS degraders, e.g., G12D and G12V. Arvinas anticipates filing an IND for this program in 2023.
Myc (Oncology)

Myelocytomatosis (Myc) proteins are implicated in up to 70% of all human cancers. Targeting Myc indirectly, such as by inhibiting transcription modulators, has not been successful, but PROTAC-mediated degradation has the potential to directly target and degrade Myc. This is an Exploratory-stage program.
HPK1 (Immuno-oncology)

Hematopoietic progenitor kinase 1 (HPK1) is a suppressor of T cell activation and targeting HPK1 can enhance anti-tumor immune responses. PROTAC-mediated degradation has the potential to address the proposed scaffolding component of HPK1’s activity. This is an Exploratory-stage program.
mHTT (Neuroscience)

Huntington’s disease is caused by a mutation in the huntingtin (HTT) gene. PROTAC degradation has the potential to allow the selective targeting of mutant HTT protein without impacting wild-type HTT protein. This is an Exploratory-stage program.

On October 14, 2020 Nimbus Therapeutics, a biotechnology company designing breakthrough medicines through structure-based drug discovery and development, reported the close of a $60 million private financing round from life science investors RA Capital Management and BVF Partners L.P. ("RA" and "BVF", respectively) (Press release, Nimbus Therapeutics, OCT 14, 2020, View Source [SID1234568464]). The funds support the acceleration of Nimbus’ Phase 1 allosteric tyrosine kinase 2 (TYK2) inhibitor into Phase 2 early next year, its hematopoietic progenitor kinase 1 (HPK1) inhibitor into the clinic next year, as well as the advancement of its preclinical portfolio of new medicines.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Today’s financing evidences the remarkable success of our structure-based drug discovery approach and the exciting data our pipeline has generated," said Jeb Keiper, M.S., MBA, Chief Executive Officer of Nimbus. "This financing also defines a pathway for our future, and while we could not welcome all the investors with interest in Nimbus into this round, we expect with continued success to build on this financing by bringing on additional new investment in 2021."

"Nimbus’ compelling TYK2 clinical data clearly positions us for a Phase 2b trial, which will generate data on the same endpoints as BMS’ TYK2 program," said Bruce Booth, D.Phil., co-founder and Chairman of the Board of Nimbus. "We are fortunate to have RA and BVF join Nimbus, and we welcome Laura Stoppel from RA to the Board."

"We’re proud to join Nimbus’ circle of investors and to lead this most recent financing round," said Peter Kolchinsky, Ph.D., a founder and Managing Partner of RA Capital Management. "We see tremendous potential for Nimbus’ lead program, one of only two clinical allosteric approaches to TYK2 inhibition, and for the company’s newly added preclinical programs to address important therapeutic needs in oncology, immunology, and metabolism."

On October 14, 2020 NOXXON Pharma N.V. (Euronext Growth Paris: ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), reported that all three patients of the second dose cohort have been enrolled into the brain cancer clinical trial testing CXCL12 inhibitor, NOX-A12, and have already received the planned initial treatment (Press release, NOXXON, OCT 14, 2020, View Source [SID1234568428]). The Phase 1/2 clinical study investigates three dose regimens of NOX-A12 (200, 400 and 600 mg/week), each combined with external beam radiotherapy in newly diagnosed brain cancer patients.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Once the last patient in the second cohort completes four weeks of therapy of NOX-A12 and radiotherapy, the independent Data Safety Monitoring Board (DSMB) will determine whether it is safe to proceed from the middle to the highest dose level of NOX-A12. The approved protocol plans for each patient to be treated with NOX-A12 for up to six months.

"The combination of NOX-A12, at both low and middle doses, and radiotherapy has been well tolerated by the patients participating in this clinical trial. Recruitment of the last cohort could start as early as November once the next safety analysis confirms benign safety profile of NOX-A12," said Dr. Frank Giordano, Chairman of the Department of Radiation Oncology at the University Hospital Bonn.

"Completing patient recruitment is an important step in the continued clinical assessment of this novel therapy for patients with difficult-to-treat and highly aggressive brain cancer. As a measure to ensure the timely completion of the study under the current challenging conditions posed by the COVID-19 pandemic, we will soon open additional clinical sites in Germany to increase recruitment capacity," commented Aram Mangasarian, CEO of NOXXON.