On March 8, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported its financial results for the fourth quarter and year ended December 31, 2020 (Press release, ERYtech Pharma, MAR 8, 2021, View Source [SID1234576210]).

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"2020 has been a year of strong execution and progress of our late-stage clinical programs despite the challenges caused by the ongoing COVID-19 global pandemic," said Gil Beyen, CEO of ERYTECH. "After more than three years of focused execution, we are looking forward to important catalysts in all four our clinical programs in 2021. Most important will be the results of our pivotal Phase 3 trial in second-line pancreatic cancer, expected in the fourth quarter of this year, as previously announced. The trial completed patient recruitment in December 2020, and is, with 512 patients enrolled, to our knowledge the largest ongoing clinical trial in this indication worldwide."

Gil Beyen added: "Pancreatic cancer is a devastating disease and remains a large unmet medical need. We are hopeful that eryaspase may provide survival benefit to these patients and look forward to reporting the results of the trial later this year. The positive results of the Phase 2 investigator-sponsored trial in acute lymphoblastic leukemia, reported at the end of last year, have the potential to be another important catalyst for the company, as we are evaluating whether this Phase 2 trial could potentially support an approval in this indication of high unmet medical need. Two other ongoing clinical trials, focused at further enlarging the scope of eryaspase, are expected to report results in 2021 as well. Our expertise in innovative red blood cell treatments as well as the excellence and dedication of our employees allow us to face the future with confidence."

Business Highlights

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

TRYbeCA-1 is a randomized controlled Phase 3 trial, evaluating eryaspase in second-line advanced pancreatic cancer in close to 90 clinical sites in the United States and 11 countries 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).

In April 2020, the U.S. Food and Drug Administration (FDA) granted eryaspase Fast Track Designation as a potential second-line treatment for patients with metastatic pancreatic cancer. Eryaspase also benefits from Orphan Drug status in pancreatic cancer, both in the United States and in Europe.
Patient enrollment was completed in December 2020. A total of 512 patients were randomized in the trial, slightly 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). The IDMC recommended the trial to 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 is expected in the fourth quarter of 2021.

§ Phase 2 trial in acute lymphoblastic leukemia, 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. Primary objectives of the trial were asparaginase enzyme activity and safety.

Patient enrollment was completed in August 2020. A total of 55 patients participated in the trial.
In December 2020, positive trial results were presented at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting. The primary endpoint was met and eryaspase demonstrated sustained asparaginase enzyme activity above the threshold of >100 U/L at trough levels, 14 days after first infusion in 54 of the 55 patients treated. Eryaspase was generally well tolerated when added to chemotherapy and almost all patients were able to receive the intended courses of asparaginase (median of 5 doses).
The Company is in dialogue with the FDA to evaluate the potential for approval based on these positive results and expects to provide an update in the first half of 2021. If the potential is confirmed, the Company expects to submit a Biologics License Application (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. Target enrollment is approximately 64 patients.

ü The trial is enrolling patients in three European countries.

First results of the trial are expected in Q4 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.

The trial was initiated in the fourth quarter of 2020 and started patient enrollment in January 2021.
Two more patients were enrolled in February, completing the first treatment cohort.
Determination of the maximum tolerable dose is expected in the second half of 2021.

Leadership and governance
In March 2020, ERYTECH strengthened the Board of Directors with the appointment of Dr Melanie Rolli, which was ratified at the General Meeting of Shareholders in June 2020. Dr. Rolli brings 17 years of experience in the global biopharmaceutical and biotechnology industry to the Board, of which 14 years at Novartis AG, where she held positions of increasing responsibilities across the Drug Development, Safety, and Medical Affairs functions both in Europe and the United States.
In October 2020, the Company reinforced its leadership team with the appointment of Dr. Stewart Craig as its Chief Technical Officer (CTO) and member of the executive team. Dr. Craig brings 35+ years of experience in development, manufacturing, technical operations, quality systems and regulatory affairs for complex biologics and cell & gene therapies worldwide for companies such as Orchard Therapeutics, Sangamo and Stem Cells Inc.

2020 Financial Results and Cash Guidance

§ Key financial figures for the full-year 2020 compared with the same period of the previous year are summarized below:

Net loss for the full year 2020 was €73.3 million, up €10.6 million (+17%) year-over-year, with a €4.8 million increase (+7%) in operating loss and a €5.9 million decrease in financial result. The €4.8 million increase in operating loss was attributable to the €5.4 million increase in preclinical and clinical development expenses, mostly related to expenses for the Company’s Phase 3 clinical trial in pancreatic cancer, a €2.2 million decrease in general and administrative expenses, which was related to the end of expenses related to the establishment of the manufacturing capacity, mostly incurred in 2019, and a €1.6 million decrease in other income, of which €0.5 million were related to the decrease in R&D tax credits and €0.9 million consisted in the upfront payment from the June 2019 license agreement with SQZ Biotechnologies that did not recur in 2020. The €5.9 million decrease in financial result reflected a €1.1 million decrease in foreign exchange swap gains, a €3.8 million negative dollar/euro foreign exchange impact, and a €1.0 million net expense due to the IFRS accounting of the convertible notes.

As of December 31, 2020, ERYTECH had cash and cash equivalents totaling €44.4 million (approximately $54.4 million), compared with €73.2 million on December 31, 2019 and €45.4 million on June 30, 2020. The €28.7 million decrease in cash position during the twelve months of 2020, consisting of net decreases of €14.6 million in the first quarter of 2020, €13.1 million in the second quarter and €4.9 million in the third quarter, and a net cash position increase of €3.9 million in the fourth quarter, was the result of a 12-month 27.7 million net cash utilization, which was mostly comprised of a €51.7 million net cash utilization in operating activities, €1.5 million used for investing activities and €25.4 million generated in financing activities, while the depreciation at the end of the period of the U.S. dollar against the euro led to a €1.0 million negative currency exchange impact.

2020 financing activities included the draw down of five tranches of €3.0 million each under the convertible notes (OCABSA) financing agreement signed with Alpha Blue Ocean in June 2020, for net proceeds of €14.2 million, a €10.0 million non-dilutive, state-guaranteed PGE loan granted by Bpifrance and Société Générale in November 2020, and €3.0 million loan milestone payments from Bpifrance on the preclinical R&D Tedac project.

Since the beginning of 2021, ERYTECH has called a 6th OCABSA tranche for net proceeds of €2.9 million and has made a placement of 744,186 newly issued shares in the United States through its at-the-market (ATM) equity financing program for net proceeds of €6.4 million.

As of the date of this press release, all notes of the first five OCABSA tranches have been converted and, together with the shares issued under the ATM program, have resulted in the issuance of 3,175,111 new shares, representing 16.4% of the Company’s outstanding share capital to date.

The Company believes that its current cash position can fund its planned operating expenses and current programs into the fourth quarter of 2021, and together with the remaining option of potential proceeds available under the convertible bonds financing agreement, into the first quarter of 2022.

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)
Update on potential path forward for registration of eryaspase in ALL patients who developed hypersensititivities to pegylated asparaginase (1H 2021)
Potential eryaspase BLA filing for ALL (2H 2021)
First results from randomized Phase 2 TRYbeCA-2 trial of eryaspase in TNBC (Q4 2021)
Determination of the maximum tolerated dose in rESPECT, Phase 1 1L PAC IST (2H 2021)
FY 2020 Conference Call Details

ERYTECH management will hold a conference call and webcast on Tuesday, March 9, 2021 at 8:30am ET / 2:30 pm CET on the business highlights and financial results for the year ended December 31, 2020. 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#: 8861234#

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: 8861234#.

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 First Quarter of 2021: May 4, 2021 (after U.S. market close), followed by a conference call & webcast on May 5, 2021 (2:30pm CET/8:30am ET)
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:

HC Wainwright Global Life Sciences Conference, March 9-10, virtual
European SmallCap Event, April 14, virtual
Kempen Healthcare & Life Sciences Conference, April 28, virtual
Jefferies 2021 Global Healthcare Conference, June 1-3, New-York
JMP Securities Life Science Conference, June 16-17, virtual
European Midcap Event – Spring, June 24, Paris

On March 8, 2021 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported its financial results for the fourth quarter and twelve months ended Dec. 31, 2020 (Press release, Castle Biosciences, MAR 8, 2021, View Source [SID1234576209]).

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"Our execution in 2020 allowed us to successfully navigate through this unique year and further our position as the leader in genomic testing for dermatologic cancers," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "We began the year with one skin cancer test and ended the year with a suite of three tests, more than tripling our estimated in-market U.S. total addressable market (TAM) to $2 billion.

"DecisionDx-Melanoma, DecisionDx DiffDx- Melanoma and DecisionDx-SCC, our tests for cutaneous melanoma, suspicious pigmented lesions and high-risk cutaneous squamous cell carcinoma, respectively, are each designed to provide actionable information for dermatology clinicians, which helps to guide cancer management decisions and improve patient outcomes.

"We are now entering the next chapter of our growth story. Earlier today, we announced clinical availability of an artificial intelligence-based integrated DecisionDx-Melanoma test result – integrating meaningful clinical and pathologic features with tumor biology identified by our DecisionDx-Melanoma test – designed to provide a more precise prediction of risk. Further, we are developing several pipeline tests that are focused on answering clinical questions with high unmet need along the patient care continuum. We estimate these pipeline tests could add an additional $3.6 billion to our U.S. TAM, bringing our aggregate U.S. TAM to slightly more than $5.5 billion."

Twelve Months Ended December 31, 2020, Financial Highlights

Revenues were $62.6 million, a 21% increase compared to $51.9 million during the same period in 2019. Included in revenue for the period were positive revenue adjustments related to tests delivered in prior periods. These positive prior period revenue adjustments for the twelve months ended Dec. 31, 2020, were $0.2 million, compared to $2.5 million for the same period in 2019.
Total gene expression profile test reports delivered in 2020 were 18,185, compared to 17,055 in 2019:
DecisionDx-Melanoma test reports delivered in 2020 were 16,232, compared to 15,529 in 2019. Third-party data for 2020 suggests that diagnoses of melanoma were down more than 20% over the prior year.
DecisionDx-SCC test reports delivered in 2020 (Aug. 31, 2020 – Dec. 31, 2020) were 485.
DecisionDx DiffDx-Melanoma test reports delivered in 2020 (Nov. 2, 2020 – Dec. 31, 2020) were 73.
DecisionDx-UM test reports delivered in 2020 were 1,395, compared to 1,526 in 2019.
Gross margin for the twelve months ended Dec. 31, 2020, was 85%.
Operating cash flow was $9.9 million, compared to $7.0 million for the same period in 2019.
Adjusted operating cash flow, excluding the effects of certain relief payments described below, was $1.5 million, compared to $7.0 million for the same period in 2019.
Cash and Cash Equivalents

As of Dec. 31, 2020, the Company’s cash and cash equivalents totaled $410 million.

Fourth Quarter Ended December 31, 2020, Financial Highlights

Revenues were $17.3 million in the fourth quarter of 2020, compared to $17.6 million in the fourth quarter of 2019. Included in revenue for the quarters were positive revenue adjustments related to tests delivered in prior periods. These positive prior period revenue adjustments for the three months ended Dec. 31, 2020, were $3.5 million, compared to $4.3 million for the same period in 2019.
Delivered 5,157 total gene expression profile test reports in the fourth quarter of 2020, compared to 4,914 in the same period in 2019:
DecisionDx-Melanoma test reports delivered in the quarter were 4,246, compared to 4,480 in the same period in 2019.
DecisionDx-SCC test reports delivered in the quarter were 428.
DecisionDx DiffDx-Melanoma test reports delivered from Nov. 2 through Dec. 31, 2020, were 73.
DecisionDx-UM test reports delivered in the quarter were 410, compared to 434 in the same period in 2019.
Gross margin in the fourth quarter of 2020 was 85%.
Operating cash flow was $(0.4) million in the fourth quarter of 2020, compared to $4.5 million in the fourth quarter of 2019.
Adjusted operating cash flow, excluding the effects of certain relief payments described below, was $1.5 million in the fourth quarter of 2020, compared to $4.5 million in the fourth quarter of 2019.
Recent Clinical Evidence Highlights

On March 8, 2021, the Company announced clinical availability of an artificial intelligence-based integrated DecisionDx-Melanoma test result. The Company validated the integration of clinicopathologic features with the tumor biology insights provided by the DecisionDx-Melanoma test. The integrated test result (ITR) is designed to provide a more precise risk prediction to further improve the clinical actionability by clinicians and their patients in helping to guide cancer management decisions. For more information, see the Company’s news release from earlier today.
In January 2021, the Company presented data on DecisionDx-Melanoma and DecisionDx DiffDx-Melanoma at the 18th Annual Winter Clinical Dermatology Conference:
The virtual poster for DecisionDx-Melanoma was entitled, "Identifying predictors of sentinel lymph node metastasis in cutaneous melanoma patients using molecular and clinicopathologic high-risk features." For 3,093 patients with T1-T4 cutaneous melanoma, authors used decision tree analysis to determine which molecular and clinicopathologic features best stratify sentinel lymph node (SLN) positivity risk and demonstrated that DecisionDx-Melanoma was the most important feature in distinguishing between high and low SLN-positivity rates (p<0.001).
The virtual poster for DecisionDx DiffDx-Melanoma was entitled, "Performance of a 35-gene expression profile test in suspicious pigmented lesions of the head and neck." The study evaluated DecisionDx DiffDx-Melanoma’s accuracy in classifying pigmented lesions on the head and neck. The data demonstrated that DecisionDx DiffDx-Melanoma has the ability to be an effective tool for refining melanoma diagnoses on the head and neck and therefore improving downstream management decisions, as indicated by its high sensitivity and specificity in the study.
Also in January 2021, the Company presented data at the Maui Derm for Dermatologists 2021 conference:
The virtual poster for DecisionDx-SCC was entitled, "Clinical utility of the 40-gene expression profile (40-GEP) for improved patient management decisions and disease related outcomes when combined with current clinicopathological risk factors for cutaneous squamous cell carcinoma (cSCC): Case Series." Two SCC cases were presented that highlight DecisionDx-SCC’s utility in stratifying risk in SCC. The cases had very similar risk of metastasis at diagnosis as both presented with a history of immunosuppression and had identical staging (T2a per Brigham and Women’s Hospital staging; T1 per American Joint Committee on Cancer staging), but had divergent outcomes:
Case 1 did not recur, despite incomplete resection. This case had a low-risk (Class 1) DecisionDx-SCC result, consistent with the clinical outcome of no clinical progression.
Case 2 developed local recurrence and regional metastasis, and eventually died from SCC, despite clear surgical margins. This case had a highest-risk (Class 2B) DecisionDx-SCC result, consistent with clinical progression. The study authors concluded that incorporating DecisionDx-SCC as a prognostic factor with traditional clinicopathologic risk factors can improve stratification of high-risk SCC patients with at least one risk factor, thereby informing risk-appropriate management strategies.
In February 2021, the Company presented data on DecisionDx-Melanoma at the 19th Annual South Beach Symposium:
The first poster was entitled, "31-Gene expression profiling improves risk stratification in patients with T1 cutaneous melanoma." Univariate analysis of the study data showed DecisionDx-Melanoma to be a stronger predictor of recurrence-free survival (RFS) than SLN status. Additionally, multivariable analysis showed DecisionDx-Melanoma to be a strong, independent predictor of RFS. With Class 2B RFS status similar to SLN positive status, Class 2B patients warrant follow-up strategies similar to SLN positive patients.
The second DecisionDx-Melanoma poster was entitled, "The clinical and financial impact of the 31-gene expression profile testing on sentinel lymph node biopsy patients selection in patients with T1b cutaneous melanoma." The authors analyzed all clinical DecisionDx-Melanoma tests that were reported from Jan. 3, 2019 through Sept. 4, 2020. The data showed that 75% of eligible patients with T1b tumors had a Class 1A result and could potentially forego sentinel lymph node biopsy (SLNB). The authors estimate that foregoing SLNB in these patients could reduce healthcare expenditures by up to $120 million in SLNB-related costs.
For a summary of Castle’s 2020 business and clinical evidence highlights, please see the Company’s news release from Jan. 13, 2021. Find the release here: Castle Biosciences Announces Preliminary Fourth Quarter and Full Year 2020 Results.
Conference Call and Webcast Details

Castle Biosciences will hold a conference call on Monday, March 8, 2021, at 4:30 p.m. Eastern time to discuss its fourth quarter and full-year 2020 results and provide a corporate update.

A live webcast of the conference call can be accessed here: View Source or via the webcast link on the Investor Relations page of the Company’s website (www.castlebiosciences.com). Please access the webcast at least 10 minutes before the conference call start time. An archive of the webcast will be available on the Company’s website until March 29, 2021.

To access the live conference call via phone, please dial 877-282-2581 from the United States and Canada, or +1 470-495-9479 internationally, at least 10 minutes prior to the start of the call, using the conference ID 3834669.

There will be a brief Question & Answer session following management commentary.

Use of Non-GAAP Financial Measures (UNAUDITED)

In this release, we use the metric of Adjusted Operating Cash Flow, which is a non-GAAP financial measure and is not calculated in accordance with generally accepted accounting principles in the United States (GAAP). This non-GAAP financial measure reflects adjustments to net cash provided by operating activities to remove the effects of two payments we received associated with government aid to healthcare providers due to COVID-19, which we believe are not indicative of our ongoing operations.

We use Adjusted Operating Cash Flow internally because we believe this metric provides useful supplemental information in assessing our cash flow performance from our core ongoing business activities by removing the effects of these items on our operating cash flows. We believe this metric is also useful to investors as a supplement to GAAP measures in analyzing the performance of our business. However, this non-GAAP financial measure may be different from non-GAAP financial measures used by other companies, even when the same or similarly titled terms are used to identify such measures, limiting their usefulness for comparative purposes. This non-GAAP financial measure is not meant to be a substitute for net cash provided by (used in) operating activities reported in accordance with GAAP and should be considered in conjunction with our financial information presented on GAAP basis. Accordingly, investors should not place undue reliance on non-GAAP financial measures. Reconciliations of this non-GAAP financial measure to the most directly comparable GAAP financial measure are presented in the table at the end of this press release.

On March 8, 2021 Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of treatments for rare and orphan diseases, reported recent business progress and year-end financial results for 2020 (Press release, Cerecor, MAR 8, 2021, View Source [SID1234576208]).

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"Looking back, 2020 was a very productive and transformative year for Cerecor highlighted by swift transition post the merger with Aevi Genomic Medicine, improving the financial position of the company and initiating clinical trials across our pipeline," said Mike Cola, Chief Executive Officer of Cerecor. "2021 started strong with positive Phase 2 data from CERC-002, our novel anti-LIGHT drug candidate in development for the treatment of COVID-19 ARDS. We have recently filed both Breakthrough Therapy and Fast Track Designations and anticipate meeting with the FDA to discuss next steps in this program soon. Overall, we believe 2021 will be a breakout year for Cerecor and look forward to building on early momentum with important pipeline updates across our immunology, oncology, and rare disease assets."

Business Updates:

•Cerecor announced CERC-002 significantly reduced respiratory failure and mortality in Phase 2 clinical trial in Patients hospitalized with COVID-19 ARDS.
•The Company closed an underwritten public offering for net proceeds of approximately $37.6 million in January.
•Dr. Gilla Kaplan was appointed to the Board of Directors in October of 2020 bringing decades of experience in rare diseases and immune-inflammatory disorders.
•Schond Greenway was appointed as Chief Financial Officer, with an established focus on investor relations to further help capitalize the company.
Program Updates:

•CERC-002: Anti-LIGHT monoclonal antibody in clinical development for COVID-19 ARDS and severe pediatric onset Crohn’s disease.
°Completed double-blinded, placebo-controlled Phase 2 proof-of-concept study of CERC-002 in cytokine storm-induced COVID-19 ARDS.
°Final analysis inclusive of the 60-day safety update in the randomized placebo-controlled study demonstrated CERC-002 was statistically significant in reducing respiratory failure and mortality at Day 28 in patients hospitalized with COVID-19-associated pneumonia and mild to moderate acute respiratory distress syndrome (ARDS), the primary endpoint, (n=62, p=0.044)
°At both the 28-day and the 60-day final timepoints, an approximately 50% trend in mortality reduction (22.5% vs 10.8%) was observed. CERC-002 appeared safe and well-tolerated on top of standard of care including high dose steroids (>90%) and remdesivir (>65%).
°Cerecor has submitted applications to the FDA for Breakthrough Therapy and Fast Track Designations and plans to meet with FDA to discuss the path to Emergency Use Authorization (EUA) and full approval.
°The company is continuing to enroll patients in its Phase 1b trial in severe pediatric-onset Crohn’s disease with initial data expected in the second quarter and is exploring the applicability of CERC-002 in non-COVID-19 ARDS.

•CERC-007: Anti-IL-18 monoclonal antibody for the treatment of multiple myeloma (MM) and Still’s disease (AOSD and sJIA).
°In December 2020, announced FDA Acceptance of two Investigational New Drug Applications for CERC-007 for the treatment of Still’s disease and for the treatment of relapsed or refractory multiple myeloma (MM).
°Following the first patient dosed in the Phase 1b clinical trial in patients with relapsed or refractory MM in December, the Company has successfully completed enrollment of the first patient cohort and looks to begin dosing cohort two.
°Initial data anticipated from Phase 1b clinical trial in adult onset Still’s disease in the second quarter of 2021.

•CERC-006: Dual mTORC1 and mTORC2 small molecule inhibitor for complex lymphatic malformations.
°Initial data anticipated from proof-of-concept study in the second quarter of 2021.

•CERC-800 programs (CERC-801, CERC-802, and CERC-803): Therapeutic doses of monosaccharide therapies for congenital disorders of glycosylation (CDGs).
°CERC-801 – In collaboration with the Frontiers in Congenital Disorders of Glycosylation Consortium clinical program, data are anticipated from the pivotal trial evaluating the safety and efficacy of D-galactose in patients suffering from Phosphoglucomutase-1 deficiency related congenital disorders of glycosylation (PGM1-CDG) in second half of 2021.
°CERC-802 – Data anticipated from the pivotal trial evaluating the safety and efficacy of D-mannose in patients suffering from Mannose phosphate isomerase deficiency related CDG (MPI-CDG) in second half of 2021.
°CERC-803 – Clearance to proceed on the Investigational New Drug Application and received Fast Track Designation from the FDA in the fourth quarter 2020. Data anticipated from the pivotal trial evaluating the safety and efficacy of L-fucose in patients suffering from Leukocyte Adhesion Deficiency II (LAD II) in second half of 2021.
2020 Financial Update:

As of December 31, 2020, Cerecor had $18.9 million in cash and cash equivalents which is a significant increase over the prior year balance of $3.6 million. Furthermore, in January 2021, the Company closed an underwritten public offering for net proceeds of approximately $37.6 million.

There were significant increases to most operating expenses, net loss and net loss per share related to the merger with Aevi Genomic Medicine that occurred in February 2020 (the Aevi Merger). Notably, research and development expense for the year ended December 31, 2020 significantly increased to $32.2 million, which was driven by activities to advance the Company’s expanded pipeline as a result of the Aevi Merger. There was a $25.5 million acquired in-process research and development (IPR&D) charge in 2020 directly related to the Aevi Merger. General and administrative expense increased to $17.4 million; the largest driver of such increase was stock-based compensation and other expenses related to leadership changes as a result of the Aevi Merger.

We believe these significant investments in our expanded pipeline and leadership team will lead to value driving milestones as our pipeline progresses toward commercialization.

On March 8, 2021 Avid Bioservices, Inc. (NASDAQ:CDMO) (NASDAQ:CDMOP), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported financial results for the third quarter and first nine months of fiscal 2021, ended January 31, 2021 (Press release, Avid Bioservices, MAR 8, 2021, View Source [SID1234576207]).

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Highlights Since October 31, 2020

"The third quarter was exceptional on all fronts," stated Nicholas Green, president and chief executive officer of Avid Bioservices. "Top line revenues were strong, contributing to a significant improvement in margins and other key financial metrics, and we continued to generate cash from operations during the quarter.

"Avid’s business development team signed $74 million in new business during the quarter, resulting in a backlog of $120 million, the largest in our history. As a result, we are raising our revenue guidance for the second time in fiscal 2021 to between $88 million and $91 million.

"Finally, to support this increase in demand, we are currently executing a two-phased expansion plan of our Myford facility that, when complete, is expected to provide a total revenue capacity of up to $270 million annually. During the quarter we also raised funds to support this expansion. The expansions represent critical developments in the business as we continue to provide capacity to onboard new clients as well as capacity to accommodate the successful clinical development and commercial growth of our existing clients. Furthermore, we look forward to incorporating a higher level of automation and digitization into the second phase as we add further focus on commercial manufacture.

"The team’s strong performance across all areas of the business is not only driving growth today, but also establishing the foundation necessary for continued growth in the future."

Financial Highlights and Guidance

The company is increasing revenue guidance for the full fiscal year 2021 from between $84 million and $88 million to between $88 million and $91 million.

Revenues for the third quarter of fiscal 2021 were $21.8 million, a 61% increase compared to revenues of $13.6 million recorded during the third quarter of fiscal 2020. For the first nine months of fiscal 2021, revenues were $68.3 million, a 45% increase as compared to revenues of $47.2 million in the prior year period. The increases in revenue for both the third quarter and first nine months of fiscal 2021 were primarily attributable to the growth in the number and scope of in-process and/or completed manufacturing runs, as well as an increase in process development projects. Additionally, as previously disclosed, prior year manufacturing revenue for the third quarter and first nine months was impacted by a production interruption.

As of January 31, 2021, revenue backlog was $120 million, an increase of 78% compared to $67 million at the end of the second quarter of fiscal 2021, and an increase of 85% compared to $65 million at the end of the prior fiscal year. The company expects to recognize most of this backlog by the end of the next fiscal year.

Gross margin for the third quarter of fiscal 2021 was 28%, a significant increase compared to a gross margin of 6% for the third quarter of fiscal 2020. Gross margin for the first nine months of fiscal 2021 was 31%, a significant increase compared to 11% in the prior year period. The increase in the third quarter and first nine months of fiscal 2021 was primarily due to the growth in manufacturing and process development revenues. Additionally, the prior year period gross profit was impacted by certain costs associated with the production interruption noted above, which costs were not incurred during the current year period.

Selling, general and administrative expenses ("SG&A") for the third quarter of fiscal 2021 were $4.0 million, an increase compared to $3.0 million recorded for the third quarter of fiscal 2020. For the first nine months of fiscal 2021, SG&A expenses were $12.0 million, an increase compared to $11.0 million recorded for the prior year period. The increases during both the third quarter and first nine months of 2021 were due primarily to increases in payroll related costs, including stock-based compensation.

For the third quarter of fiscal 2021, the company recorded a consolidated net income attributable to common stockholders of $0.8 million or $0.01 per basic and diluted share, as compared to a consolidated net loss attributable to common stockholders of $3.5 million or $0.06 per basic and diluted share, for the third quarter of fiscal 2020. For the first nine months of fiscal 2021, the company recorded a consolidated net income attributable to common stockholders of $5.6 million or $0.10 per basic and diluted share, compared to a consolidated net loss attributable to common stockholders of $9.3 million or $0.17 per basic and diluted share, for fiscal 2020.

Avid reported $70.9 million in cash and cash equivalents as of January 31, 2021. This balance includes approximately $32.1 million in net proceeds raised in the third quarter follow-on underwritten equity offering. This balance represents an increase of $35.2 million from the end of the second quarter and an increase of $34.6 million compared to $36.3 million as of the end of the prior fiscal year. The company also generated cash flows from operating activities of $5.2 million during the third quarter and $13.3 million during the nine months ended January 31, 2021.
More detailed financial information and analysis may be found in Avid Bioservices’ Quarterly Report on Form 10-Q, which will be filed with the Securities and Exchange Commission today.

Recent Corporate Developments

Signed orders for $74 million during the quarter with new and existing customers, driving Avid’s backlog to an all-time high.

Completed a follow-on underwritten public offering resulting in net proceeds of approximately $32.1 million, after deducting underwriting discounts and commissions and other offering related expenses. The company intends to use the net proceeds from the offering for the expansion of its manufacturing capabilities.

Continued to advance the two-phased expansion of the Myford facility. The first phase of our expansion plan, which was initiated during the second quarter of fiscal 2021, expands the production capacity of the company’s existing Myford North facility by adding a second downstream processing suite. The second phase, which was initiated during February, will further expand capacity through the build out of a second manufacturing train, including both upstream and downstream processing suites within Myford South.

The company estimates the first phase will take approximately 12 to 15 months to complete at an estimated cost of approximately $15 million and may increase the company’s annual revenue generating capacity by up to $50 million, bringing the combined annual revenue generating capacity of our Franklin and Myford North facilities to up to $170 million. The company estimates that the Myford South expansion will take approximately 18 to 24 months to complete at a cost of approximately $45 to $55 million and may increase the company’s annual revenue generating capacity by an additional $100 million for a total revenue generating capacity of up to $270 million annually.
Conference Call

Avid will host a conference call and webcast this afternoon, March 8, 2021, at 4:30 PM EST (1:30 PM PST).

To listen to the conference call, please dial (877) 312-5443 or (253) 237-1126 and request the Avid Bioservices conference call. To listen to the live webcast, or access the archived webcast, please visit: View Source

On March 8, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the fourth quarter ended December 31, 2020 (Press release, Syndax, MAR 8, 2021, View Source [SID1234576206]). In addition, the Company provided a clinical and business update.

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"We expect 2021 will be a year of immense progress across our two highly promising programs aimed at addressing key areas of unmet need, coupled with a sharp focus on pipeline expansion," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Notably, we plan to present data from the Phase 1 portion of our ongoing AUGMENT-101 trial of SNDX-5613, our selective menin inhibitor, in patients with acute leukemias. We presented promising initial clinical data at the 2020 AACR (Free AACR Whitepaper) Annual Meeting that provided the first clinical evidence that disrupting the interaction between menin and MLL1 can induce rapid responses in difficult to treat acute leukemias. We look forward to sharing updated results which further expand on SNDX-5613’s potential to meaningfully alter the treatment paradigm in genetically-defined acute leukemias."

"Additionally, on the heels of positive data presented at the ASH (Free ASH Whitepaper) Annual Meeting in December from our Phase 1 trial of axatilimab, our anti-CSF-1R monoclonal antibody, in patients with cGVHD, we are pleased to announce that our pivotal Phase 2 AGAVE-101 trial is now underway. Through inhibition of monocyte derived macrophages, axatilimab has demonstrated important clinical benefits in multiple organ systems. We believe axatilimab could represent a meaningful therapeutic option for additional fibrotic diseases where macrophages have been shown to play a significant role, and we are actively exploring additional indications."

Recent Progress and Anticipated Milestones

SNDX-5613

Syndax plans to share data from the Phase 1 AUGMENT-101 trial of SNDX-5613, its highly selective, oral menin inhibitor, in patients with mixed lineage leukemia rearranged (MLL-r) and nucleophosmin (NPM1) mutant acute leukemias late in the first quarter or early in the second quarter of 2021. Additional details regarding the presentation, which will feature the trial’s principal investigator, Eytan M. Stein, M.D. Assistant Attending Physician and Director, Program for Drug Development in Leukemia, Department of Medicine at Memorial Sloan Kettering Cancer Center, will be announced in the coming weeks. The Company remains on track to initiate the Phase 2 portion of the trial in the second quarter of 2021, which could potentially serve as the basis for a regulatory filing.

Axatilimab

At the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2020, Syndax reported updated data from its Phase 1 trial of axatilimab, its anti-CSF-1R monoclonal antibody, in patients with chronic graft versus host disease (cGVHD). Data demonstrated deep, durable responses and multiorgan clinical benefit in patients refractory to multiple therapeutic agents.

The Company announced today that the pivotal Phase 2 AGAVE-201 trial, which will evaluate the safety and efficacy of three doses and schedules of axatilimab in patients with cGVHD, began enrolling patients earlier this quarter. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for GVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score. The Company expects to report topline data in 2023.

Fourth Quarter 2020 Financial Results

As of December 31, 2020, Syndax had cash, cash equivalents and short-term investments of $293.1 million and 51.4 million shares and share equivalents issued and outstanding. This includes 3.6 million pre-funded warrants.

Fourth quarter 2020 research and development expenses increased to $15.5 million from $9.5 million, and for the full year increased to $50.4 million compared to $43.0 million for 2019. The fourth quarter and full year increases were primarily due to increased clinical trial activities, increased CMC activities and professional fees.

General and administrative expenses for the fourth quarter 2020 decreased to $4.7 million from $5.1 million, and, for the year ended December 31, 2020, increased to $22.5 million compared to $16.1 million for the prior year. The fourth quarter decrease is primarily due to decreased pre-commercialization expenses. The increase for the full year was primarily due to increased professional fees and employee related expenses.

For the three months ended December 31, 2020, Syndax reported a net loss attributable to common stockholders of $20.4 million or $0.44 per share compared to $14.0 million or $0.44 per share for the prior year period. For the year ended December 31, 2020, Syndax reported a net loss attributable to common stockholders of $77.8 million or $1.88 per share, compared to $56.0 million or $1.84 per share for the prior year. For the year ended December 31, 2020, this includes a deemed dividend of $3.9 million.

Financial Update and Guidance

In December 2020, Syndax issued 6,250,000 shares of its common stock at $23.00 per share. As a result of the offering, Syndax received gross proceeds of approximately $143.8 million.

For the first quarter of 2021, research and development expenses are expected to be $25 to $30 million, and total operating expenses are expected to be $30 to $35 million. For the full year of 2021, research and development expenses are expected to be $90 to $100 million, and total operating expenses are expected to be $110 to $120 million.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, March 8, 2021.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the followin