On May 9, 2023 Lisata Therapeutics, Inc. (Nasdaq: LSTA) ("Lisata" or the "Company"), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, reported a business update and provides financial results for the three months ended March 31, 2023 (Press release, Lisata Therapeutics, MAY 9, 2023, View Source [SID1234631233]).

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"During the first quarter of 2023, our team continued its focus on the advancement of multiple ongoing and planned clinical studies evaluating LSTA1, our lead investigational product," stated David J. Mazzo, Ph.D., Chief Executive Officer of Lisata. "We expect to report progress on several of these activities over the coming months and quarters. Just recently, we, along with our research partner, WARPNINE, announced the treatment of the first patient in the iLSTA Trial in Australia evaluating LSTA1 in combination with standard-of-care chemotherapy and immunotherapy as a first-line treatment in locally advanced non-resectable pancreatic ductal adenocarcinoma. We are hopeful that this and our other trials will continue to show the potential of LSTA1 in combination with corresponding standards-of-care as well as with emerging treatment modalities, such as immunotherapies, as an effective treatment against various solid tumors.

Dr. Mazzo continued, "Our overarching goal is to report meaningful clinical data to benefit patients and to support our development pipeline in the most expeditious manner possible. Positive data should also bring value to shareholders and encourage additional partnering opportunities."

Development Portfolio Update

LSTA1 as a treatment for solid tumor cancers in combination with other anti-cancer agents

LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to penetrate solid tumors more effectively. LSTA1 actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor, while normal tissues are not expected to be affected. LSTA1 also has the potential to modify the tumor microenvironment, with the objective of making tumors more susceptible to immunotherapies. We and our collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies, and RNA-based therapeutics. To date, LSTA1 has also demonstrated favorable safety, tolerability and activity in completed and ongoing clinical trials designed to test its ability to enhance delivery of standard-of-care chemotherapy for pancreatic cancer. Currently, LSTA1 is the subject of Phase 1b/2a and 2b clinical studies being conducted globally in metastatic pancreatic ductal adenocarcinoma in combination with each of the two standards-of-care for this disease. The combination of LSTA1 with corresponding standards-of-care in other solid tumor indications is planned for clinical study commencing before the end of the second quarter of 2023.

HONEDRA (LSTA12) for the treatment of critical limb ischemia ("CLI")

•HONEDRA is the Company’s SAKIGAKE-designated product candidate for the treatment of CLI and Buerger’s disease in Japan, which is now in the pre-consultation phase of the registration process

with the Pharmaceuticals and Medical Devices Agency ("PMDA") in Japan. Data from the follow-up of all patients completed in the registration-eligible clinical trial in Japan have been compiled and are the subject of discussions with the PMDA as part of the Japanese regulatory pre-consultation process and in preparation for the formal clinical consultation meetings which precede a Japanese new drug application. To date, the PMDA has provided advice on how to prepare for the formal consultation meeting. Concomitantly, the Company has reinforced its efforts to secure a partner to complete the remaining steps of development/registration and potential commercialization in Japan through the engagement of an advisory firm specializing in Japanese partnerships.

LSTA201 for the treatment of diabetic kidney disease ("DKD")

The Company initiated a Phase 1b, open-label, proof-of-concept trial evaluating LSTA201, a CD34+ regenerative cell therapy investigational product, for intra-renal artery administration in patients with DKD. Preclinical studies in kidney disease and injury models have demonstrated that protecting or replenishing the microcirculation of the kidney may result in improved kidney function. A key criterion for continued development of LSTA201 was determined, a priori, to be the ability of LSTA201 to regenerate kidney function as indicated by increased Glomerular Filtration Rate. The Company treated the first patient in the LSTA201 proof-of-concept study in April 2022 and completed treatment for all six subjects during the third quarter of 2022. Top line results, which were reported on February 6, 2023, showed that LSTA201 was safe and well-tolerated by patients with no serious adverse events related to the therapy. However, the study did not demonstrate a consistent improvement in kidney function among patients. Nevertheless, the Company, based on the encouragement of the study’s principal investigator/key opinion leader, believes there may still be potential for use of CD34+ cell therapy for the treatment of DKD. However, it is expected that further development of LSTA201 would require significantly larger studies and capital investment. Thus, LSTA201 development will only be continued if a strategic partner that can contribute the necessary capital for future development is identified.

First Quarter 2023 Financial Highlights

Research and development expenses were approximately $3.2 million for the three months ended March 31, 2023, compared to $3.3 million for the three months ended March 31, 2022, representing a decrease of $0.1 million or 3.2%. This was primarily due to expenses associated with our XOWNA Phase 2b study (the FREEDOM Trial) in the prior year, partially offset by study start up activities in the current year associated with the planned LSTA1 Phase 2 proof-of-concept basket trial in various solid tumors in combination with the corresponding standards of care, enrollment activities for the LSTA1 Phase 2b ASCEND study and chemistry, manufacturing and control activities for LSTA1.

General and administrative expenses were approximately $3.7 million for the three months ended March 31, 2023, compared to $3.3 million for the three months ended March 31, 2022, representing an increase of $0.3 million or 9.8%. This was primarily due to the addition of one employee acquired through the Company’s merger with Cend Therapeutics, Inc., an increase in external legal fees and an increase in accounting and tax-related fees.
Overall, net losses were $6.2 million for the three months ended March 31, 2023, compared to $4.2 million for the three months ended March 31, 2022.

Balance Sheet Highlights

As of March 31, 2023, the Company had cash, cash equivalents and marketable securities of approximately $61.1 million. These figures do not include the recently announced $2.2 million in non-dilutive funding received as an approved participant of the Technology Business Tax Certificate Transfer Program (the "Program") sponsored by the New Jersey Economic Development Authority, which will be recorded in the second quarter of 2023. The Program enables qualifying New Jersey-based biotechnology or technology companies to sell a percentage of their New Jersey net operating losses and research and development tax credits to unrelated qualifying corporations. The Company is confident that its projected capital will fund its operations into the first quarter of 2026 encompassing anticipated data milestones from all of its ongoing and planned clinical trials.

Conference Call Information

Lisata will hold a live conference call today, May 9, 2023, at 4:30 p.m. Eastern time to discuss financial results, provide a business update and answer questions.
Those wishing to participate must register for the conference call by way of the following link: https://register.vevent.com/register/BI0ea7c01f84154b159cddca509bd575cd. Registered participants will receive an email containing conference call details with dial-in options. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time.
A live webcast of the call will also be accessible under the Investors & News section of Lisata’s website and will be available for replay beginning two hours after the conclusion of the call for 12 months.

On May 9, 2023 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the first quarter ended March 31, 2023, including recent commercial and operational progress, and regulatory updates (Press release, bluebird bio, MAY 9, 2023, View Source [SID1234631232]).

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"The first quarter of 2023 saw us continue to establish and scale the commercial model for ex-vivo gene therapy in the US through the launches of ZYNTEGLO and SKYSONA and lay the foundation for revenue generation for years to come for bluebird," said Andrew Obenshain, chief executive officer, bluebird bio. "Additionally, with the submission of the lovo-cel BLA for sickle cell disease in April, bluebird has taken a pivotal step towards realizing its most significant opportunity yet — bringing a transformative gene therapy to individuals living with sickle cell disease."

RECENT HIGHLIGHTS

lovo-cel (lovotibeglogene autotemcel) BLA Submitted

On April 24, 2023, bluebird bio announced it submitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for lovo-cel for patients with sickle cell disease (SCD) who are 12 years and older and have a history of vaso-occlusive events. The Company has requested Priority Review, which, if granted, would shorten the FDA’s review of the application to six months from the time of filing, versus a standard review timeline of 10 months.
ZYNTEGLO (betibeglogene autotemcel) Commercial Launch

In the weeks since the Company’s last commercial update, bluebird continues to build on the launch of ZYNTEGLO for beta-thalassemia. To date, there have been six patient starts (cell collections) for ZYNTEGLO.
The first commercial ZYNTEGLO infusion has been completed and the Company anticipates recognizing revenue in Q2 2023.
Payer uptake continues to be positive and to date, the Company has received zero ultimate denials for ZYNTEGLO; prior authorization approvals for drug product remain consistent at approximately two weeks.
As previously communicated, patient starts remain the key commercial metric during the first year of the ZYNTEGLO launch. bluebird does not expect to provide ZYNTEGLO revenue projections for 2023.
SKYSONA (elivaldogene autotemcel) Commercial Launch

First commercial infusion with SKYSONA was completed in March 2023. In total, cell collection has been completed for three patients.
Since approval, bluebird has activated three qualified treatment centers (QTCs) to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD). Two additional QTCs on the West Coast are anticipated in 2023.
UPCOMING ANTICIPATED MILESTONES

LOVO-CEL

The Company anticipates BLA acceptance of lovo-cel for SCD in Q2 2023.
The Company continues to anticipate commercial launch in early 2024, if approved. bluebird estimates approximately 20,000 individuals living with sickle cell disease (or 1/5 of the SCD population in the US) may be eligible for gene therapy.
ZYNTEGLO

The Company is on track to scale to 40-50 QTCs by the end of 2023. bluebird’s QTC network is designed to maximize its commercial opportunity in beta-thalassemia and to prioritize proximity to individuals with living with SCD in anticipation of a 2024 commercial launch for lovo-cel, if approved by the FDA.
SKYSONA

The Company continues to anticipate 5-10 patient starts this year as previously guided.
FIRST QUARTER 2023 FINANCIAL RESULTS

Cash Position: The Company’s cash, cash equivalents, marketable securities and restricted cash balance was approximately $364 million, as of March 31, 2023. As bluebird bio launches two first-in-class gene therapies and readies its third investigational gene therapy for SCD for the commercial setting, full-year 2023 cash burn is expected to be in the range of $270-$300 million, as previously guided. Based on current operating plans, bluebird expects its cash, cash equivalents, marketable securities and restricted cash will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the fourth quarter of 2024. This runway includes approximately $45 million of restricted cash, which is currently unavailable for use. Please see our Quarterly Report on Form 10-Q for the quarter ended March 31, 2023 for further information regarding our cash runway guidance and other financial results.

Revenue, net: Total revenue, net was $2.4 million for the three months ended March 31, 2023, compared to $1.9 million for the three months ended March 31, 2022. The increase of $0.4 million was primarily due to SKYSONA product revenue.

SG&A Expenses: Selling, general and administrative expenses were $37.4 million for the three months ended March 31, 2023, compared to $36.1 million for the three months ended March 31, 2022. SG&A includes lease expense related to the 50 Binney Street; however, sublease income is presented in other income (expense), net. Excluding the lease expense for 50 Binney St., SG&A expenses were $27.8 million for the three months ended March 31, 2023, compared to $36.1 million for the three months ended March 31, 2022. This decrease is mainly attributable to costs related to employee compensation, benefits, and other head-count related expenses.

R&D Expenses: Research and development expenses were $46.1 million for the three months ended March 31, 2023, compared to $77.9 million for the three months ended March 31, 2022. The decrease of $31.7 million was primarily due to decreased employee compensation, benefit and other headcount-related expenses and a decrease in R&D production costs in 2023.

Gain from sale of priority review voucher: Operating expenses were offset by the one-time gain from sale of the Company’s second priority review voucher, for $92.9 million, net, in the first quarter of 2023.
Net income (loss): Net income was $21.2 million for the three months ended March 31, 2023, compared to a net loss of $122.2 million for the three months ended March 31, 2022.

On May 9, 2023 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage precision oncology medicine company developing MasterKey therapies designed to overcome limitations of existing therapies by targeting families of oncogenic driver mutations in patients with genetically defined cancers, reported financial results for the first quarter ended March 31, 2023, and provided a corporate update (Press release, Black Diamond Therapeutics, MAY 9, 2023, View Source [SID1234631231]).

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"We are incredibly pleased with the pace of execution since the start of 2023 as we continue to advance our pipeline programs, BDTX-1535 and BDTX-4933 in addition to our newest program, BDTX-4876. Having three investigational new drug (IND) acceptances in three years is exemplary of our breadth of expertise in precision oncology drug discovery and development. We are confident that our momentum will continue to bring tremendous progress across our pipeline as we head further into this transformational year for Black Diamond," said David Epstein, Ph.D., President and Chief Executive Officer of Black Diamond Therapeutics. "2023 brings several key inflection points, including a clinical update from the dose-escalation portion of our Phase 1 trial of BDTX-1535 in patients with non-small cell lung cancer (NSCLC) and glioblastoma multiforme (GBM), the initiation of a Phase 1 clinical trial for BDTX-4933, and today’s announcement of a development candidate nomination for our fibroblast growth factor receptor (FGFR) 2/3 selective program. We remain acutely focused on bringing our next-generation precision medicines to patients in need, particularly as we work to expand the addressable patient population within the broader oncogenic mutation landscape. Our sophisticated drug design and our deep-rooted commitment to effectively targeting shared, activated conformations used by oncogenic drivers for tumor growth are all enabled by our Mutation Allostery Pharmacology (MAP) drug discovery engine and ultimately realized by our MasterKey therapies."

Recent Developments & Upcoming Milestones:

BDTX-1535:

BDTX-1535, an epidermal growth factor receptor (EGFR) MasterKey inhibitor, is being developed to treat genetically defined cancer in patients whose tumors are positive for EGFR MasterKey mutations in NSCLC, as well as in GBM. BDTX-1535 is a fourth generation EGFR inhibitor designed to be irreversible (covalent) and brain-penetrant and is unique in how it targets a family of driver and resistance mutations arising from the use of third generation EGFR inhibitors to treat EGFR-positive NSCLC.
In April 2023, Black Diamond presented two posters highlighting BDTX-1535’s preclinical development as well as the ongoing Phase 1 clinical trial at the 2023 AACR (Free AACR Whitepaper) Annual Meeting. Key highlights from the preclinical poster included an outline of the unmet need for next generation EGFR inhibitors that target classical driver mutations as well as acquired and intrinsic resistance mutations expressed in the context of EGFR driver mutations in NSCLC, and EGFR alterations expressed in GBM. Black Diamond also outlined its ongoing Phase 1, open-label, multicenter clinical trial to assess the safety, tolerability, pharmacokinetics, central nervous system (CNS) penetrance and preliminary antitumor activity of BDTX-1535 in locally advanced or metastatic NSCLC with or without CNS disease or in recurrent GBM (rGBM).
Enrollment in the Phase 1 clinical trial was initiated in 2022 and dose escalation is ongoing. Dose Expansion cohorts are expected to open in 2023. Black Diamond remains on track to provide a clinical update on BDTX-1535 in the second half of 2023.
BDTX-4933:

BDTX-4933 is designed as a brain-penetrant, oral MasterKey inhibitor of oncogenic BRAF Class I, II and III and RAS mutations, while also avoiding paradoxical activation.
In the first quarter of 2023, Black Diamond received allowance of its IND application from the U.S. Food and Drug Administration (FDA).
In April 2023, Black Diamond presented a poster at the 2023 AACR (Free AACR Whitepaper) Annual Meeting, outlining its approach to characterizing RAF, RAS and MAPK pathways in addition to the design and preclinical development of BDTX-4933. Based on preclinical data, BDTX-4933 has a potential best-in-class profile to treat cancer patients harboring oncogenic BRAF Class I, II, III and RAS mutations, with or without brain disease.
Black Diamond expects to dose its first patient in a Phase 1 clinical trial of BDTX-4933 in patients with tumors harboring all-class BRAF or RAS mutations in the second quarter of 2023.
Discovery-Stage Pipeline and MAP Drug Discovery Engine:

Today, Black Diamond announced that it selected a development candidate, BDTX-4876, for its FGFR program. BDTX-4876 is selective against MasterKey mutations and alterations in FGFR 2 and 3, while sparing FGFR 1 and 4.
Black Diamond plans to evaluate strategic alternatives for the FGFR program as it deepens focus on its two clinical stage assets.
Black Diamond continues to leverage its MAP drug discovery engine to advance its discovery-stage pipeline to bring therapies to underserved patients.
Corporate:

In March 2023, the Company promoted Fang Ni, Pharm.D., from interim Chief Financial Officer to full-time Chief Financial Officer, in addition to his role as Chief Business Officer.
Financial Highlights

Cash Position: Black Diamond ended the first quarter of 2023 with approximately $103.4 million in cash, cash equivalents, and investments compared to $122.8 million as of December 31, 2022. Net cash used in operations was $20.0 million for the first quarter of 2023 compared to $28.6 million for the first quarter of 2022.
Research and Development Expenses: Research and development (R&D) expenses were $14.8 million for the first quarter of 2023, compared to $17.8 million for the same period in 2022. The decrease in R&D expenses was primarily due to reduced clinical trial activities stemming from the discontinuation of the development of BDTX-189 to focus on advancement of the Company’s pipeline programs, BDTX-1535 and BDTX-4933.
General and Administrative Expenses: General and administrative (G&A) expenses were $6.8 million for the first quarter of 2023, compared to $7.9 million for the same period in 2022. The decrease in G&A expenses was primarily due to a decrease in legal and other professional fees.
Net Loss: Net loss for the first quarter of 2023 was $20.9 million, as compared to $25.5 million for the same period in 2022.
Financial Guidance

Black Diamond ended the first quarter of 2023 with approximately $103.4 million in cash, cash equivalents and investments, which the Company believes is sufficient to fund its anticipated operating expenses and capital expenditure requirements into the third quarter of 2024.

On May 9, 2023 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for severe unmet medical needs, reported that it will hold a business update conference call on Monday, May 15, 2023, at 10 a.m. CEST (Press release, BerGenBio, MAY 9, 2023, View Source [SID1234631230]). BerGenBio’s senior management team will provide a briefing via a webcast presentation on the Company’s website, followed by a Q&A session.

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Topics to be addressed during the presentation include:

Proposed Rights Issue: The Company announced on April 25, 2023, its intentions to carry out a partially underwritten rights issue to raise gross proceeds of up to NOK 250 million.

A Q&A document pertaining to the rights issue can from today be found in the General Meetings portion of the Investors section of www.bergenbio.com.

BGBC008 Additional Data: Additional results of pre-planned analyses from the Phase 2 BGBC008 trial evaluating bemcentinib in combination with pembrolizumab in 2L+ NSCLC patients will be presented.

BGBC003 Topline Data: Topline data will be presented from the Phase 2 BGBC003 trial evaluating bemcentinib in combination with chemotherapy in patients with AML and as a single agent in patients with MDS.

Financial Update: An update on the Company’s key financial numbers will be provided as part of the business update.

Full first quarter 2023 financial results report will currently not be prioritized and release will be delayed to 22 June 2023.

The webcast link will be available at www.bergenbio.com in the Investors/Financial Reports section.

A recording will be available shortly after the webcast has finished.

On May 9, 2023 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the first quarter ended March 31, 2023 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAY 9, 2023, View Source [SID1234631229]). Avidity ended the first quarter of 2023 with cash, cash equivalents and marketable securities totaling $586.3 million.

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"We are pleased with the positive AOC 1001 topline data reported at AAN demonstrating directional functional improvements across multiple functional measurements. We are focused on discussions with the FDA to define a path forward for AOC 1001 as soon as possible," said Sarah Boyce, president and chief executive officer at Avidity. "We have a number of important milestones from our pipeline expected this year including a first look at data from the MARINA-OLE trial and data from healthy volunteers in the Phase 1/2 EXPLORE44 study. We are executing on our clinical and pipeline programs to develop much needed therapies for people living with devastating rare diseases."
"We ended Q1 with $586 million which puts us in a strong financial position with a runway to mid-2025. We continue to invest in our advancing and expanding pipeline in skeletal muscle and cardiology rare disease programs while also expanding the broad utility of our AOC platform through our internal discovery efforts and collaborations," said Mike MacLean, chief financial officer and chief business officer at Avidity
Recent Highlights

•Presented top-line data for MARINA at American Academy of Neurology (AAN) 2023 Annual Meeting in April 2023:
◦The MARINA trial concluded with the 38 participants with myotonic dystrophy type 1 (DM1). The company will continue to dose the participants at both 2 mg/kg and 4 mg/kg of AOC 1001 in the MARINA open-label extension study (MARINA-OLE). AOC 1001 MARINA topline data demonstrated:
▪Directional improvement in multiple functional assessments including measures of myotonia, strength and mobility

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▪Meaningful DMPK reduction and splicing changes in participants treated with AOC 1001 followed by directional improvements in functional measures at 2 mg/kg and 4mg/kg doses of AOC 1001
▪AOC 1001 demonstrated broad splicing improvements in more than a thousand genes impacted by DM1, confirming activity in the nucleus
▪Favorable safety and tolerability profile of AOC 1001 with most adverse events mild or moderate

•The company provided a regulatory update on the AOC 1001 partial clinical hold on new participant enrollment and provided more information on the rare serious adverse event in a single participant that led to the partial clinical hold. Avidity continues to work diligently with the FDA and remains very confident in the benefit/risk profile of AOC 1001.
•Avidity announced that it advanced and expanded its wholly-owned early stage AOC pipeline including adding a rare cardiology program and an additional program in rare skeletal muscle disease. Ongoing collaborations with Eli Lilly and Company (Lilly) and Bristol Myers Squibb (BMS) continue to advance beyond skeletal muscle in immunology and cardiology, respectively.
•The FDA granted AOC 1020 Fast Track Designation in January 2023 and in February 2023, the FDA and the European Medicines Agency (EMA) granted AOC 1020 Orphan Designation.
•The FDA also recently granted AOC 1044 Fast Track Designation in April 2023.
Upcoming Milestones:
•The company continues to advance three distinct rare disease clinical programs: AOC 1001 for DM1, AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping (DMD44). Upcoming milestones include:
◦A first look at data from the MARINA-OLE trial is anticipated in late 2023.
◦Data from healthy volunteers in the EXPLORE44 trial is anticipated in the second half of 2023.
◦Data from a preliminary assessment in approximately half of participants in the FORTITUDE trial is anticipated in the first half of 2024.

First Quarter 2023 Financial Results
Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $586.3 million as of March 31, 2023, compared to $610.7 million as of December 31, 2022.

•Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Lilly and totaled $2.2 million for the first quarter of 2023 compared with $1.8 million for the first quarter of 2022.

•Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $47.8 million for the first quarter of 2023 compared with $27.7 million for the first quarter of 2022. The increases were primarily driven by the advancement of AOC 1001, AOC 1020 and AOC 1044, as well as internal and external costs related to the expansion of the company’s overall research capabilities.
•General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $12.1 million for the first quarter of 2023 compared with $8.6 million for the first quarter of 2022. The increases were primarily due to higher personnel costs and professional fees to support the company’s expanded operations.