On August 1, 2022 Emergent BioSolutions Inc. (NYSE: EBS) reported financial results for the second quarter ended June 30, 2022 (Press release, Emergent BioSolutions, AUG 1, 2022, View Source [SID1234617234]).

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"Central to Emergent’s mission – to protect and enhance life – is our ability to provide quality products and services for the benefit of our patients and customers," said Robert G. Kramer, president and CEO of Emergent BioSolutions. "We continue to focus on combating critical public health threats with our core medical countermeasure and commercial businesses, executing on our growth strategy with M&A opportunities, and further strengthening our public-private partnerships, development pipeline, and manufacturing network to reinforce the durability of our diversified business."

SELECT Q2 2022 AND OTHER RECENT BUSINESS UPDATES

Executed an agreement to acquire from Chimerix its worldwide rights to TEMBEXA(R) (brincidofovir), the first FDA-approved smallpox oral antiviral for all ages; continue to anticipate transaction closing in Q3 2022.
Announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for AV7909 (Anthrax Vaccine Adsorbed, Adjuvanted), the company’s new anthrax vaccine candidate; the goal date for a decision by the FDA is in April 2023.
Announced a collaboration with Ridgeback Biotherapeutics ("Ridgeback Bio") to expand the availability of Ebanga (Ansuvimab-zykl), a monoclonal antibody therapeutic approved by the FDA in December 2020 for the treatment of Ebola.
Initiated a phase 3 safety and immunogenicity study to evaluate CHIKV VLP, the Company’s single-dose chikungunya vaccine candidate, in adults 65 and older, in addition to the current phase 3 study in individuals aged 12 to 64, for which enrollment into the study is ongoing.
Continued to repurchase the Company’s common stock under an existing authorization by the Board of Directors to management to repurchase up to $250.0 million through November 11, 2022; during the quarter ended June 30, 2022, the Company purchased an additional 0.7 million shares for $23.3 million, resulting in an aggregate of approximately 4.4 million shares for $187.9 million since initiating repurchases in Q4 2021.
Expanded the Company’s leadership with the appointment of Sujata Dayal to the board of directors; Ms. Dayal was appointed a member of both the Nominating and Governance Committee as well as the Special Committee on Manufacturing and Quality Oversight.
Product Sales, net
Anthrax vaccines
For Q2 2022, revenues from Anthrax vaccines increased $44.1 million as compared to Q2 2021. The increase is largely driven by timing of deliveries to the U.S. government (USG), specifically the Strategic National Stockpile (SNS). The Company received an AV7909 contract modification in September 2021 valued at approximately $399.0 million to deliver additional AV7909 doses through March 2023.

Nasal naloxone products
For Q2 2022, revenues from nasal naloxone products decreased $4.6 million as compared to Q2 2021. The decrease was driven by a reduction in commercial retail sales following the launch of a generic in December 2021. This decrease was offset by strong growth in unit sales of branded NARCAN (naloxone HCl) Nasal Spray to public interest customers in the U.S. and customers in Canada, as well as from sales of the authorized generic product licensed to Sandoz, which launched in December 2021.

Other (4)
For Q2 2022, revenues from other product sales increased $16.5 million as compared to Q2 2021. The increase was primarily due to sales of two of the Company’s Government/Medical Countermeasure (MCM) products: i) VIGIV [Vaccinia Immune Globulin Intravenous (Human)], driven by timing of deliveries to the SNS; and ii) BAT [Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) – (Equine)] , driven by timing of deliveries to international customers.

Contract Development and Manufacturing
CDMO Services
For Q2 2022, revenues from contract development and manufacturing services decreased $100.9 million as compared to Q2 2021. This decrease is largely due to lower combined revenues of $82.0 million from AstraZeneca and Janssen reflecting the impact of reduced production activities at the Bayview facility as a result of a cessation of manufacturing activities under the AstraZeneca contract which occurred in 2021, and a pause and eventual cessation of manufacturing activities under the Janssen contract which began in Q1 2022. Additionally for Q2 2022, the Company reversed $8.3 million of previously recognized revenue under the Janssen contract to align cumulative revenue recognized with cumulative cash collections. The decrease also reflects reduced production at the Camden facility in the quarter driven by additional investments in strengthening quality and compliance that restricted the Company’s ability to optimally utilize the existing capacity at the site. These declines in revenues were offset by an increase in contracted manufacturing activities at the Winnipeg facility.

CDMO Leases
For Q2 2022, revenues from contract development and manufacturing leases decreased $91.8 million as compared to Q2 2021. The decrease was primarily due to the completion of the Company’s public-private partnership with BARDA in November 2021, which contributed $70.4 million in Q2 2021 and a $21.9 million decrease in lease revenues related to the Janssen contract. Included in the $21.9 million decrease, the Company reversed $5.0 million of previously recognized revenue under the Janssen contract to align cumulative revenue recognized with cumulative cash collections.

Contracts and Grants
For Q2 2022, revenues from contracts and grants decreased $18.1 million as compared to Q2 2021. The decrease is primarily due to lower revenue from BARDA as a result of the completion of the Center for Innovation and Advanced Development and Manufacturing (CIADM) agreement, which occurred in November 2021, as well as decreases in development activities associated with various other externally funded R&D projects, most notably the AV7909 program, which has now completed its clinical phase and for which a BLA is currently under review by the FDA with an anticipated goal date for completion in April 2023.

Cost of Product Sales
For Q2 2022, cost of product sales increased $9.8 million as compared to Q2 2021. The increase is primarily due to the higher volume of product sales.

Cost of CDMO
For Q2 2022, cost of CDMO decreased $67.8 million as compared to Q2 2021. The decrease is primarily due to reduced production activities across our CDMO network in Q2 2022 compared to Q2 2021 resulting in decreased raw materials consumption, as well as a $41.5 million inventory write-off in Q2 2021. These decreases were partially offset by increased costs at the Company’s Winnipeg facility due to an increase in manufacturing activities and Camden facility due to additional investments in quality enhancement and improvement initiatives.

Research and Development
For Q2 2022, research and development expenses were consistent with Q2 2021 reflecting the Company’s continued commitment to investment in important pipeline programs addressing additional public health preparedness and response areas of focus.

Selling, General and Administrative
For Q2 2022, selling, general and administrative expenses decreased $10.1 million due to reduced professional services and marketing costs partially offset by higher compensation costs.

Additional Financial Information

Segment Information
During Q1 2022, the Company began assessing its operating performance by focusing on two reportable segments: 1) a products segment (Products) consisting of the MCM and Commercial products business lines; and 2) a services segment (Services) consisting of the CDMO services business line. The Company evaluates the performance of these reportable segments based on revenue and adjusted gross margin. Segment revenue includes external customer sales but does not include inter-segment services. The Company does not allocate contracts and grants, R&D, SG&A, amortization of intangible assets, interest and other income (expense) or taxes to its evaluation of the performance of these segments.

For the three and six months ended June 30, 2022, Product gross margin increased $46.2 million and $117.7 million, respectively, as compared to the three and six months ended June 30, 2021. Product adjusted gross margin for the three and six months ended June 30, 2022 increased $45.5 million and $117.8 million, respectively, as compared to the three and six months ended June 30, 2021. The increases in Product gross margin and Product adjusted gross margin are primarily due to increased volumes and changes in product mix.

For the three months ended June 30, 2022, Services gross margin and Services adjusted gross margin decreased $124.9 million as compared to the three months ended June 30, 2021. For the six months ended June 30, 2022, Services gross margin and Services adjusted gross margin decreased $276.8 million as compared to the six months ended June 30, 2021. The decreases in 2022 are primarily due to the decline in revenue at the Bayview facility as a result of the completion of the Company’s arrangement with BARDA, the pause in manufacturing activities for improvement and modifications, and an increase in professional services costs.

As of June 30, 2022, the number of CDMO customers declined by one versus the prior reported period of March 31, 2022. The mix of customers for the Company’s CDMO services remains largely a combination of small and medium sized biopharmaceutical companies.

During the three months ended June 30, 2022, the Company secured new CDMO services business of $16.0 million. This new business was on behalf of existing customers for non-COVID related work on both new and existing projects and molecules.

For the three months ended June 30, 2022, capital expenditures decreased largely due to less spending associated with the expansion project at the Company’s Rockville facility, which has progressed to a less capital intensive phase. The Company anticipates completing this expansion project by year end 2022.

2022 FINANCIAL FORECAST(1)

The Company has resumed providing financial guidance for 2022 and announces the following update to its full year 2022 forecast.

Assumptions

The Company’s 2022 financial forecast takes into consideration the following assumptions.

2022 Product and Contract and Grant Revenues

Anthrax vaccines revenues are expected to continue at similar levels to 2021 under the terms of the Company’s existing contract with BARDA.
ACAM2000, (Smallpox (Vaccinia) Vaccine, Live) vaccine deliveries are expected to continue under the terms of the Company’s existing contract with the U.S. Department of Health and Human Services (HHS) at unit volume levels consistent with 2021 deliveries.
Nasal naloxone products revenues reflect the formation of a generic market and comprise revenues from a combination of NARCAN Nasal Spray and the authorized generic of NARCAN Nasal Spray, a product licensed to Sandoz and launched in late 2021 and one in which the Company retains a financial interest.
Other Products + Contracts and Grants revenues: 1) other products revenues reflect continued procurement of other products not highlighted on a standalone basis from various government customers under existing multi-year contracts; 2) contracts and grants revenues reflect continued funding of select development programs from various government and other non-dilutive sources.
CDMO

CDMO revenues are expected to re-baseline throughout the year as the Company transitions the business to a focus on non-COVID projects and on expanding its drug product capabilities as it progresses toward a higher level of capacity utilization principally at the Camden, Rockville and Winnipeg sites. CDMO revenues exclude further contribution from Janssen.
Other

Pipeline progress is expected across the R&D portfolio with the advancement of the CHIKV VLP Phase 3 clinical trials, the FDA acceptance of the Company’s BLA filing for AV7909, and anticipated advancements of a number of early-stage programs.
Capital expenditures, net of reimbursement, are expected to be approximately 10% of total revenues at the midpoint, reflecting ongoing investments in capacity and capability expansions related to the CDMO business and the Company’s R&D programs, and aligned with the average over the previous five-year period.
The updated forecasted ranges do not take into account the impact of the addition of TEMBEXA, the acquisition of which is expected to close in the third quarter of 2022.

Q3 2022 Total Revenues

The Company is also providing a forecast for Q3 2022 total revenues of $230 – $270 million.

FOOTNOTES

(1) All financial information incorporated within this release is unaudited.
(2) See "Reconciliation of Net Income (Loss) to Adjusted Net Income (Loss)," "Reconciliation of Net Income (Loss) to Adjusted EBITDA," and "Adjusted Revenues" for a definition of terms and the reconciliation tables.
(3) Product sales, net are reported net of variable consideration including returns, rebates, wholesaler fees and prompt pay discounts in accordance with U.S. generally accepted accounting principles.
(4) Other can include a combination of sales of any of the following products: BAT, VIGIV, Anthrasil, raxibacumab, RSDL, Trobigard, Vivotif, and Vaxchora.
(5) CDMO Customer is defined as a client (commercial, government, NGO) for whom the Company has performed CDMO services where there is evidence of meeting all of the following criteria: i) completion of any billable project milestones in the preceding 24-month period, indicating ongoing work; ii) secured project work planned in the future, which has not yet been invoiced, capturing future work not yet indicated in the invoice record; and, iii) neither the Company nor the client having yet to formally terminate the last remaining project, thereby removing any client for whom work has fully concluded.
(6) CDMO New Business Secured is defined as initial value of contracts secured as well as incremental value of existing contracts modified within the indicated period.

CONFERENCE CALL, PRESENTATION SUPPLEMENT AND WEBCAST INFORMATION

Company management will host a conference call at 5:00 pm (Eastern Time) today, August 1, 2022, to discuss these financial results. The conference call and presentation supplement can be accessed from the Company’s website or through the following:

Advanced registration is required to participate by phone.
Visit https://register.vevent.com/register/BIf66c9ca4b8a34754a22943c20e4a7e4d to register and receive an email with the dial-in number, passcode and registrant ID.

Live Webcast Information:
Visit View Source for the webcast.

A replay of the call can be accessed from the Investors page of the Company’s website.

On August 1, 2022 Biomea Fusion, Inc. (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing novel covalent small molecules to treat and improve the lives of patients with genetically defined cancers and metabolic diseases, reported second quarter 2022 financial results and business highlights (Press release, Biomea Fusion, AUG 1, 2022, View Source [SID1234617233]).

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"The team continues to deliver against critical corporate goals as we look to build tremendous value potential for the company in the near and long-term. During the second quarter, we focused specifically on key clinical operations and clinical science activities to support COVALENT-101. Additionally, we continued to design and execute key experiments to support our diabetes effort as we march towards the clinic," said Thomas Butler, Biomea’s Chief Executive Officer and Chairman of the Board. "The scaffold protein menin has many more functional responsibilities than was previously characterized and, thus, is central to multiple disease settings. Moreover, we believe our approach of covalent menin inhibition with BMF-219 offers potential safety, tolerability, efficacy and durability benefits to patients. We are now well positioned to execute a robust clinical development plan for BMF-219, which includes many liquid and solid tumor indications, as well as type 2 diabetes. Beyond menin inhibition, we are advancing toward the clinic with our second IND candidate, BMF-500, a covalent FLT3 inhibitor with best-in-class potential."

Second Quarter 2022 Pipeline Highlights

Cancer

Enrolled first patient with MM in the ongoing first-in-human Phase I clinical trial (COVALENT-101) evaluating BMF-219 in patients with relapsed or refractory acute leukemias, DLBCL, and MM
Presented Trial in Progress posters for COVALENT-101 at multiple prominent oncology conferences in the United States and in Europe
Presented preclinical data in two posters at AACR (Free AACR Whitepaper) 2022 highlighting the impact of BMF-219 on MYC- and KRAS-driven solid tumors. Building on prior data, BMF-219 exhibited robust anti-tumor effect in cell lines and PDX models in DLBCL and MM cell lines
On track to submit IND for BMF-219 in KRAS mutant NSCLC, CRC, and pancreatic ductal adenocarcinoma (PDAC) in the fourth quarter of 2022
Presented preclinical data at ASCO (Free ASCO Whitepaper) 2022 demonstrating BMF-219’s potency in multiple ex vivo tumor models of Chronic Lymphocytic Leukemia (CLL) with varying cytogenetic risk profiles, Rai stages, and resistance to standard-of-care agents indicating broad activity across these models
Announced IND candidate selection, BMF-500, a potential best-in-class oral covalent inhibitor of FLT3, one of the most frequently altered genes in AML and associated with poor prognosis
Diabetes

Presented new preclinical data in two posters at the ADA Scientific Sessions 2022, demonstrating BMF-219’s strong, prolonged glycemic control, insulin sensitization, and hemoglobin A1C (HbA1c) reduction in two preclinical rat models of diabetes while on drug and after washout, outperforming standard-of-care agents
Announced upcoming oral presentations of BMF-219 preclinical data from two animal models of diabetes at the EASD 2022 annual meeting which will include additional data not previously presented at the ADA Scientific Sessions 2022
On track to submit IND for BMF-219 in type 2 diabetes in the second half of 2022
Second Quarter 2022 Financial Results

Net Income/Loss: Biomea reported a net loss attributable to common stockholders of $33.6 million for the six months ended June 30, 2022, compared to a net loss of $14.3 million for the same period in 2021.
R&D Expenses: Research and development expenses were $23.9 million for the six months ended June 30, 2022, compared to $9.0 million for the same period in 2021. The increase of $14.9 million was primarily due to an increase in personnel-related expenses, as well as an increase in preclinical and clinical development costs, including manufacturing and external consulting, related to the Company’s product candidates, BMF-219 and BMF-500.
G&A Expenses: General and administrative expenses were $9.9 million for the six months ended June 30, 2022, compared to $5.3 million for the same period in 2021. The increase of $4.7 million was primarily due to higher personnel-related expenses and other corporate costs to support the Company’s expanding operations as well as additional costs incurred as a public company.
Cash, Cash Equivalents, Restricted Cash, and Investments: As of June 30, 2022, the Company had cash, cash equivalents, restricted cash, and investments of $150.2 million, compared to $175.7 million as of December 31, 2021.

On August 1, 2022 Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company, reported up to a US$45 million investment from Petrichor Healthcare Capital Management ("Petrichor") (Press release, Fennec Pharmaceuticals, AUG 1, 2022, View Source [SID1234617232]). Under the terms of the investment agreement:

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Subject to customary closing conditions, Petrichor will purchase $5 million of senior secured promissory notes which are convertible into Fennec common shares at a price per share of $8.11 based upon a 20% premium to the 5-day VWAP immediately prior to announcement of the investment. The Company intends to repay in full its existing secured indebtedness with Bridge Bank before the first closing;
An additional $20 million to be funded upon the potential U.S. Food and Drug Administration (FDA) approval of PEDMARKTM by September 30, 2022 and satisfaction of other closing conditions. Fennec upon mutual agreement with Petrichor may draw up to $20 million of additional financing under the investment agreement; and
The notes have a five-year term and bear interest at a rate equal to the US Prime Rate plus 4.5%, with 3.5% of the interest paid in kind for the first twenty-four months.
Rosty Raykov, chief executive officer of Fennec, said, "Petrichor is a premier healthcare-dedicated investment firm who has worked with us to structure this transaction, which provides substantial capital and flexibility to support the launch and commercialization of PEDMARKTM, if approved. We appreciate the confidence that Petrichor is demonstrating in Fennec and the potential market opportunity for PEDMARKTM as we await a decision from the FDA by the September 23, 2022 PDUFA target action date."

Tadd Wessel, Founder and Managing Partner of Petrichor, said, "We are pleased to partner with Fennec as they seek to commercialize PEDMARKTM. The Fennec team has a clear strategic vision and the potential to bring a transformative medication to the pediatric cancer community, as PEDMARKTM stands to be the first FDA-approved therapy to reduce the risk of ototoxicity associated with cisplatin in pediatric patients. Our investment strengthens Fennec’s balance sheet and provides management with the financial flexibility to potentially capitalize on near-term growth opportunities for PEDMARKTM."

Further information surrounding the investment agreement is set forth in the Current Report on Form 8-K filed by the Company with the SEC on or about August 1, 2022. The offer and sale of the notes and the shares of common stock issuable upon conversion of the notes, if any, have not been registered under the Securities Act of 1933, as amended, or the securities laws of any other jurisdiction, and the notes and such shares may not be offered or sold absent registration with the U.S. Securities and Exchange Commission (the "SEC") or an applicable exemption from registration requirements, or in a transaction not subject to, such registration requirements. We intend to rely upon the exemption set forth in Section 602.1 of the TSX Company Manual, which provides that the TSX will not apply its standards to certain transactions involving eligible interlisted issuers on a recognized exchange, such as Nasdaq.

No regulatory authority has either approved or disapproved the contents of this press release. This press release is neither an offer to sell nor a solicitation of an offer to buy the notes or the shares of common stock issuable upon conversion of the notes, if any, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

About PEDMARK

Cisplatin and other platinum compounds are essential chemotherapeutic agents for many pediatric malignancies. Unfortunately, platinum-based therapies cause ototoxicity, or hearing loss, which is permanent, irreversible and particularly harmful to the survivors of pediatric cancer.

In the U.S. and Europe, it is estimated that, annually, over 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children that suffer ototoxicity at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

PEDMARK has been studied by cooperative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, The Clinical Oncology Group Protocol ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.

The U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s resubmitted New Drug Application (NDA) for PEDMARKTM (a unique formulation of sodium thiosulfate (STS)) for the prevention of platinum-induced ototoxicity in pediatric patients with localized, non-metastatic, solid tumors. The PDUFA target action date for the NDA is September 23, 2022. The Marketing Authorization Application (MAA) for sodium thiosulfate (tradename PEDMARQSI) is currently under evaluation by the European Medicines Agency (EMA). PEDMARK has received Breakthrough Therapy and Fast Track Designation by the FDA in March 2018.

On August 1, 2022 Gracell Biotechnologies Inc. ("Gracell" or the "Company",NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, reported the appointment of Wendy Li, M.D., as its Chief Medical Officer (CMO) (Press release, Gracell Biotechnologies, AUG 1, 2022, View Source [SID1234617227]). In this role, Dr. Li will oversee Gracell’s clinical development activities, including the advancement of its pipeline of autologous and allogeneic product candidates across the Company’s multiple proprietary technology platforms.

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Dr. Li brings to Gracell over 20 years of experience leading all critical aspects of clinical and medical operations at early-stage and large pharmaceutical organizations in the U.S. and China. Her expertise spans both clinical development and medical affairs, including leading early- and late-stage clinical trials for several therapeutic candidates for the treatment of hematological malignancies and solid tumors, and overseeing over 30 successful Investigational New Drug applications (INDs), New Drug Applications (NDAs) and Biologics License Applications (BLAs) that led to several multi-billion-dollar blockbuster drugs.

"Dr. Li is a distinguished and enthusiastic clinical leader and shares our commitment to discovering and developing breakthrough cell therapies. We are excited to add her experience to Gracell’s global leadership team," said Dr. William (Wei) Cao, Founder, Chairman and CEO of Gracell. "Dr. Li’s decades of experience in leading clinical development and strategy across many types of cancers and treatment modalities, as well as expertise in working with regulatory agencies, will be invaluable as we continue to advance our pipeline of breakthrough cell therapies and approach the filing of our U.S. IND application for GC012F later this year."

Prior to joining Gracell, Dr. Li served as CMO of EXUMA Biotech, where she provided strategic medical and clinical leadership for the advancement of its cell therapy pipeline in the U.S. and Asia. She has also held clinical development and medical affairs leadership positions at Pfizer, Sanofi, Genentech, and Sihuan Pharmaceutical. Dr. Li holds an M.D. from Sun Yat-Sen University of Medical Sciences.

"With years of experience in CAR-T, I recognize this therapy’s potential to fulfill patients’ unmet needs and am eager to join a company with a pipeline as promising as Gracell’s," Dr. Li said. "I believe the company’s FasTCAR and TruUCAR platforms have vast potential to solve some of the greatest challenges facing CAR-T therapy. The lead candidate, GC012F, is a highly differentiated CAR-T therapy that potentially affords competitive efficacy, combined with a favorable safety profile and faster delivery to patients. I look forward to joining Gracell’s leadership team and bringing the company to its next stage of growth."

On August 1, 2022 Biosight Ltd., a pharmaceutical development company focused on the development of innovative therapeutics for hematological malignancies and disorder, reported that the United States Food & Drug Administration (FDA) has granted Orphan Drug Designation to aspacytarabine (BST-236), an investigational novel antimetabolite, for the treatment of myelodysplastic syndromes, in addition to the Orphan Drug Designation granted in 2019 for aspacytarabine for the treatment of acute myeloid leukemia (AML) (Press release, BioSight, AUG 1, 2022, View Source [SID1234617226]).

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Following the encouraging single-agent efficacy and safety profile of aspacytarabine in the recently completed Phase 2b trial in front-line treatment of AML patients unfit for standard induction chemotherapy, aspacytarabine development program is being expanded to second-line treatment of AML and MDS in two ongoing Phase 2 clinical trials, one in the US and Israel and one in France in collaboration with the European cooperative group, GFM. In addition to the monotherapy development programs, a Phase 1/2 trial of aspacytarabine in combination with the Bcl2 inhibitor, venetoclax, for the treatment of AML patients unfit for standard chemotherapy will be launched in the coming weeks.

"We are very pleased to have received from the FDA the Orphan Drug Designation for aspacytarabine for the treatment of MDS, which adds to the designation granted already for the treatment of AML" said Dr. Ruth Ben Yakar, CEO of Biosight. "The accumulating clinical data from more than a hundred patients treated to date with aspacytarabine monotherapy in four clinical trials, suggest that aspacytarabine, with a differentiated mechanism that enables high-dose chemotherapy with reduced toxicity, has the potential to transform standard of care for AML and MDS patients".

Orphan Drug Designation by the FDA entitles Biosight to seven years of market exclusivity for the use of aspacytarabine for the treatment of MDS, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

About Aspacytarabine (BST-236)

Aspacytarabine is a novel proprietary anti-metabolite. It is composed of cytarabine covalently bound to asparagine, acting as a pro-drug of cytarabine. Cytarabine serves as the backbone of AML and MDS therapy for over 45 years due to its superior efficacy, however, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities, which significantly limit its use, especially in older and medically compromised patients. Due to its unique pharmacokinetics and metabolism, aspacytarabine enables high-dose therapy with lower systemic exposure to free cytarabine and relative sparing of normal tissues. As such, aspacytarabine may serve as a superior therapy for AML, MDS, and other hematological malignancies and disorders, including for older adults who are unfit for intensive therapy.

Aspacytarabine was granted FDA Fast Track Designation for the treatment of AML in adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, and Orphan Drug designations from the FDA and EMA, which entitles Biosight to seven and ten years of market exclusivity upon aspacytarabine marketing approval for the treatment of AML in the US and Europe, respectively.

Results from the recently completed Phase 2b study evaluating aspacytarabine as a single-agent first-line AML therapy demonstrate safety and single-agent activity, and additional studies are ongoing to evaluate aspacytarabine as a second line treatment for patients with relapsed or refractory MDS or AML. For more information regarding the Phase 2b clinical study of aspacytarabine, please visit www.clinicaltrials.gov.