On May 5, 2023 FibroGen, Inc. (NASDAQ: FGEN) reported that MATTERHORN, a Phase 3 clinical study of roxadustat for treatment of anemia in patients with transfusion-dependent lower risk myelodysplastic syndromes (MDS) did not meet its primary efficacy endpoint (Press release, FibroGen, MAY 5, 2023, View Source [SID1234631093]). The proportion of patients who achieved red blood cell transfusion independence in the first 28 weeks was 47.5% for the roxadustat arm compared to 33.3% for placebo (p=0.217). The adverse event profile of roxadustat that was observed in the preliminary safety analysis was generally consistent with previous findings. Safety will be further evaluated at study completion.

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A total of one-hundred forty (140) patients were enrolled in MATTERHORN, a Phase 3, double-blind placebo-controlled study investigating the safety and efficacy of roxadustat for treatment of anemia in patients with lower risk transfusion-dependent myelodysplastic syndromes. The primary endpoint of the study is transfusion independence for ≥ 56 consecutive days during the first 28 weeks of treatment, and patients are followed for up to 52 weeks. The MATTERHORN study is sponsored and conducted by FibroGen and is part of FibroGen’s co-development collaborations with AstraZeneca and with Astellas Pharma Inc.

About Myelodysplastic Syndromes Anemia
Myelodysplastic syndromes (MDS) are a group of disorders characterized by poorly formed or dysfunctional blood cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S1. Approximately 80% of MDS patients have anemia at the time of diagnosis2 and around 60% of MDS patients will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease3. Lower-risk MDS patients represent approximately 77% of the total diagnosed MDS population4. Anemia in MDS patients is associated with increased risk of cardiovascular complications and the need for blood transfusion5. Transfusion dependent MDS patients suffer higher rates of cardiac events, infections and transformation to acute leukemia, and a decreased overall survival rate when compared with non-transfused patients with MDS, and decreased survival compared to an age-matched elderly population6. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Currently, there are few options available for treating anemia in MDS. Patients with MDS typically rely on repeated blood transfusions and administration of ESAs.

About Roxadustat
Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is in clinical development for anemia of chronic kidney disease (CKD) and anemia associated with myelodysplastic syndromes (MDS), and for chemotherapy-induced anemia (CIA).

Roxadustat is approved in China, Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). Several other licensing applications for roxadustat have been submitted by partners, Astellas and AstraZeneca to regulatory authorities across the globe, and are currently under review.

Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in the U.S., China, and other markets not licensed to Astellas.

On May 5, 2023 Enochian Biosciences presented its corporate presentation (Presentation, Enochian BioSciences, MAY 5, 2023, View Source [SID1234631092]).

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On May 5, 2023 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that the Board of Directors of Curis approved the grant of inducement stock options to its newly appointed Chief Development Officer, Jonathan B. Zung, Ph.D. to purchase a total of 500,000 shares of Curis common stock, with a grant date of May 1, 2023 (the "Inducement Grant") (Press release, Curis, MAY 5, 2023, View Source [SID1234631091]).

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The Inducement Grant has an exercise price per share equal to the closing price of the Company’s common stock on May 1, 2023. The Inducement Grant has a 10 year term and vests over four years, with 25% of the original number of shares underlying the award vesting on the first anniversary of Dr. Zung’s start date and an additional 6.25% of the original number of shares underlying the award vesting on each successive three-month period thereafter, subject to Dr. Zung’s continued service with the Company through the respective vesting dates. Dr. Zung’s stock option was granted as an inducement equity award outside of the Company’s Fourth Amended and Restated 2010 Stock Incentive Plan and was made as an inducement material to his acceptance of employment with the Company.

On May 4, 2023 AbCellera (Nasdaq: ABCL) reported financial results for the first quarter of 2023 (Press release, AbCellera, MAY 5, 2023, View Source [SID1234631073]). All financial information in this press release is reported in U.S. dollars, unless otherwise indicated.

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"In the first quarter we have continued to execute on our strategy of building the industry’s preferred engine for the discovery and development of antibody therapeutics. Notably, new data on our T-cell engager platform demonstrates that we can generate TCEs with superior properties on a highly accelerated timeline, and against challenging tumor targets," said Carl Hansen, Ph.D., founder and CEO of AbCellera. "As we progress further into 2023, we remain steadfast in our strategy of building best-in-world capabilities for the discovery and development of antibody therapeutics."

Q1 2023 Business Summary

Earned $12.2 million in total revenue.
Generated a net loss of $40.1 million, compared to net earnings of $168.6 million in Q1 2022.
Reached a cumulative total of 177 programs under contract with 41 different partners.
Maintained a cumulative total of 101 partnered program starts.
Reporting the advancement of one additional molecule into the clinic, bringing the cumulative total to nine molecules in the clinic.
Key Business Metrics

Cumulative Metrics

March 31, 2022

March 31, 2023

Change %

Number of discovery partners

36

41

14

%

Programs under contract

158

177

12

%

Partnered program starts

84

101

20

%

Molecules in the clinic

6

9

50

%

AbCellera added three partnered programs in Q1 2023 to reach a cumulative total of 177 programs under contract (up from 158 on March 31, 2022) that are either completed, in progress, or under contract with 41 different partners as of March 31, 2023 (up from 36 on March 31, 2022). AbCellera maintained a cumulative total of 101 partnered program starts in Q1 2023 (up from 84 on March 31, 2022). AbCellera’s partners advanced an additional molecule into the clinic in Q1 2023, bringing the cumulative total of molecules in the clinic to nine (up from six on March 31, 2022).

Discussion of Q1 2023 Financial Results

Revenue – Total revenue was $12.2 million, compared to $316.6 million in Q1 2022. The partnership business generated research fees of $10.6 million, compared to $9.3 million in Q1 2022. Milestone payments were $1.3 million and licensing revenue was $0.4 million.
Research & Development (R&D) Expenses – R&D expenses were $52.6 million, compared to $26.4 million in Q1 2022, reflecting continuing investments in the capacity and capabilities of AbCellera’s engine for antibody discovery and development, and in co-development programs.
Sales & Marketing (S&M) Expenses – S&M expenses were $3.8 million, compared to $2.4 million in Q1 2022. The increase reflects continued investments in business development.
General & Administrative (G&A) Expenses – G&A expenses were $15.1 million, compared to $14.3 million in Q1 2022, with the increase driven by investments to support the growth of the company.
Net Loss – Net loss of $40.1 million, or $(0.14) per share on a basic and diluted basis, compared to net earnings of $168.6 million, or $0.59 per share on a basic and $0.54 on a diluted basis in Q1 2022.
Liquidity – $821.5 million of total cash, cash equivalents, and marketable securities.
Conference Call and Webcast

AbCellera will host a conference call and live webcast to discuss these results today at 2:00 p.m. Pacific Time (5:00 p.m. Eastern Time).

The live webcast of the earnings conference call can be accessed on the Events and Presentations section of AbCellera’s Investor Relations website. A replay of the webcast will be available through the same link following the conference call.

On May 4, 2023 Celyad Oncology (Euronext & Nasdaq: CYAD) (the "Company"), a biotechnology company focused on innovative technologies for chimeric antigen receptor (CAR) T-cell therapies, reported its financial results for the first quarter of 2023 and provides an update on recent business developments (Press release, Celyad, MAY 5, 2023, View Source [SID1234631023]).

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"Celyad Oncology is now fully committed to leveraging its expertise, know-how and intellectual property (IP) portfolio with the goal of providing innovative solutions to overcome the current limitations of CAR T-cell approaches. We have generated exiting data from our short hairpin RNA (shRNA)-based multiplexing platform, which highlights the versatility and adaptability of the technology. We also have made significant progress with our dual CAR program, for which we anticipate sharing an update at international conferences in the upcoming months,"

commented Georges Rawadi, Chief Executive Officer of the Company.
CORPORATE HIGHLIGHTS
Georges Rawadi was appointed as Chief Executive Officer of the Company
OPERATIONAL HIGHLIGHTS
shRNA multiplexing platform: Data validating our shRNA multiplexing approach, which allows to down-regulate several genes simultaneously, were presented at the World Oncology Cell Therapy Congress in Boston, US (April 25-26, 2023):
We developed a microRNA (miRNA)-based multiplex shRNA platform designed for easy, efficient, and tunable knock-down regulation of up to four target genes simultaneously;
Furthermore, we showed that the down-regulation of each target gene could be fine-tuned, from a moderate down-regulation up to achieving a functional knock-out, all without the need of gene editing and thus avoiding associated potential safety issues;
The plug-and-play design of our platform is designed to allow for swapping each target sequence without affecting performance, streamlining the generation of engineered adoptive T-cell therapies; and
To demonstrate the effectiveness of our approach, we have been able to simultaneous knock down in CAR T-cells several genes involved in different cellular processes such as alloreactivity (CD3ζ), cell persistence (β2M, CIITA), T-cell exhaustion (PD-1, LAG-3), or ligand-induced apoptosis (CD95).
NKG2D-based CAR T-cells: Results from the hematological arm of the Phase I THINK trial have been published in The Lancet Haematology journal (Lancet Haematol. 2023 Mar;10(3):e191-e202). Data from the 16 patients treated in the dose-escalation segment provided proof-of-concept of targeting NKG2D ligands by CAR T-cell therapy. Further development of NKG2D-based CAR T-cells are warranted, potentially via combinatorial approaches or further CAR optimization to improve anti-tumor efficacy; and
We continue to progress on the development of NKG2D-based dual CARs and B7-H6-targeting CAR T-cells, with the aim of broadening the landscape of CAR T-cell therapies.
FINANCIAL HIGHLIGHTS – FIRST QUARTER 2023 FINANCIAL REVIEW
As of March 31, 2023, the Company had cash and cash equivalents of €9.2 million ($10.0 million). Net cash burn during the first quarter of 2023 amounted to €3.2 million, in line with expectations.

The Company projects that its existing cash and cash equivalents should be sufficient to fund operating expenses and capital expenditure requirements into the fourth quarter of 2023.
After due consideration of detailed budgets and estimated cash flow forecasts for the years 2023 and 2024, the Company continues to project that its existing cash and cash equivalents will not be sufficient to fund its estimated operating and capital expenditures over at least the next 12 months from the date that this release is issued.

The Company is currently evaluating different financing options to obtain the required funding to extend the Company’s cash runway beyond 12 months from the date this release is issued.

FINANCIAL CALENDAR 2023

August 3rd, 2023 – First Half 2023 Interim Results
November 9th, 2023 – Third Quarter 2023 Business Update
The financial calendar is communicated on an indicative basis and may be subject to change.

UPCOMING ANTICIPATED MILESTONES

The Company will provide an update on its dual CAR platform and business development in the first half of 2023; and
The Company anticipates fundraising in the first half of 2023.
UPCOMING CONFERENCES
The Company will take part in the Immuno-Oncology summit in London (June 20-22, 2023) and the BIO International Convention in Boston (June 5-8, 2023).