On May 12, 2023 Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company focused on discovering and developing medicines that are transformational for patients, reported its financial results and business highlights for the first quarter ended March 31, 2023 (Press release, Centessa Pharmaceuticals, MAY 12, 2023, View Source [SID1234631591]).

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"Centessa continued strong clinical momentum in the first quarter with multiple clinical milestones planned for the year," said Saurabh Saha MD PhD, Chief Executive Officer of Centessa. "We are advancing the registration program for SerpinPC for the treatment of hemophilia B; dosing subjects in the ongoing Phase 1/2a clinical trial for LB101, our first LockBody molecule for the treatment of solid tumors; and, conducting IND enabling activities for our newest product candidate, ORX750, for the treatment of narcolepsy with potential expansion into other sleep disorders."

Dr. Saha continued, "We expect to rapidly drive our pipeline forward and anticipate a lower cash burn rate for the remainder of the year. We believe we are well positioned with a cash runway into 2026 to support multiple clinical readouts across our programs."

Recent Highlights
•In March, the first subject was dosed in the Phase 1/2a first-in-human clinical trial of LB101, a conditionally tetravalent PD-L1xCD47 bispecific monoclonal antibody from the Company’s LockBody technology platform for the treatment of solid tumors. LB101 is the Company’s first

LockBody candidate to enter the clinic. The Company announced clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) to initiate the Phase 1/2a trial in January.
•In March, the Company announced ORX750, an orally administered, selective orexin receptor 2 (OX2R) agonist, as a product candidate for the treatment of narcolepsy with potential expansion into other sleep disorders.
•In February, the Company presented additional data from the open-label extension (OLE) of the ongoing Phase 2a study of SerpinPC for the treatment of hemophilia during an oral presentation at the 16th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD). With total exposure of over 40 patient-years across multiple dosing regimens, the Phase 2a data showed a continued favorable safety and tolerability profile for SerpinPC, as well as evidence of sustained efficacy, as measured by a reduction in the all-bleeds annualized bleeding rates (ABR). To date, no thromboembolic events and no treatment-related sustained elevations of D-dimer have been observed across the Phase 2a study.

Anticipated Upcoming Program Milestones
•Hemophilia (SerpinPC)- The registrational program for hemophilia B is ongoing. PRESent-5, an observational feeder study, is enrolling subjects and the Company expects to begin dosing in the PRESent-2 and PRESent-3 studies later this year. In addition, the Company plans to share data from Part 5 of the OLE of the Phase 2a study of SerpinPC, subject to completion, at a scientific meeting this year.
•Solid Tumors (PD-L1xCD47 LockBody LB101)- The Phase 1/2a first-in-human clinical study is ongoing.
•Narcolepsy and Other Sleep Disorders (ORX750)- ORX750 is in preclinical development and undergoing IND-enabling activities. The Company plans to share preclinical data at a scientific meeting later this year.

The Company has multiple earlier-stage programs, including MGX292 and other LockBody molecules such as PD-L1xCD3, and discovery-stage programs. Where applicable, the Company plans to provide updates on preclinical programs as they advance toward clinical studies.
First Quarter 2023 Financial Results

•Cash, Cash Equivalents and Short-term Investments: $346.2 million as of March 31, 2023, which the Company expects will fund operations into 2026, without drawing on the remaining available tranches under the Oberland credit facility.
•Research & Development Expenses: $32.8 million for the first quarter ended March 31, 2023, compared to $36.9 million for the first quarter ended March 31, 2022.
•General & Administrative Expenses: $16.1 million for the first quarter ended March 31, 2023, compared to $14.4 million the first quarter ended March 31, 2022.
•Net Loss Attributable to Ordinary Shareholders: $50.7 million for the first quarter ended March 31, 2023, compared to $54.5 million for the first quarter ended March 31, 2022.

On May 12, 2023 Bio-Techne Corporation (NASDAQ: TECH) reported that Jim Hippel, Executive Vice President and Chief Financial Officer, reported that it will present at the 2023 RBC Capital Markets Global Healthcare Conference on Tuesday, May 16, 2023, at 2:35 p.m. EDT (Press release, Bio-Techne, MAY 12, 2023, View Source [SID1234631589]). A live webcast of the presentation can be accessed via the IR Calendar page of Bio-Techne’s Investor Relations website at View Source

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On May 12, 2023 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported its financial results for the first quarter ended March 31, 2023 and provided an update on recent corporate developments (Press release, Bio-Path Holdings, MAY 12, 2023, View Source [SID1234631588]).

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"We continue to make important progress advancing our clinical programs as we await topline results from several key cohorts in our Phase 1/1b study of BP1001-A in solid tumors, Phase 1/1b study of BP1002 in relapsed/refractory AML, and Phase 2 study of prexigebersen in AML," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. "Despite advances in the field, cancer deaths continue to rise. We believe our DNAbilize platform can overcome the challenges with current treatment options to address the urgent need for safe and effective new treatments."

Important Near-Term Clinical Milestones

BP1001-A Phase 1/1b Clinical Trial in Solid Tumors

● Important trial with advanced or recurrent solid tumors, including ovarian and uterine, pancreatic and breast cancer with initial cohort completion and data readout currently expected mid-year.

BP1002 Phase 1/1b Clinical Trial in Relapsed/Refractory AML

● Focus on patients who relapsed on venetoclax treatment with initial cohort completion and readout expected mid-year.

Prexigebersen (BP1001) Phase 2 Clinical Trial in AML

● Two of the three cohorts in the clinical trial already exceed the minimum efficacy required for enrollment expansion.
● Assess safety and efficacy of each cohort treatment combination therapy with potential to qualify for expedited program status after cohort’s initial interim analysis, which are expected to commence by cohort in the second quarter of 2023.

Financial Results for the First Quarter Ended March 31, 2023

● The Company reported a net loss of $5.3 million, or $0.66 per share, for the three months ended March 31, 2023, compared to a net loss of $3.4 million, or $0.47 per share, for the three months ended March 31, 2022.

● Research and development expense for the three months ended March 31, 2023 increased to $4.0 million, compared to $2.1 million for the three months ended March 31, 2022, primarily due to manufacturing expenses related to drug product releases during the quarter.

● General and administrative expense for both the three months ended March 31, 2023 and March 31, 2022 was $1.3 million.

● As of March 31, 2023, the Company had cash of $6.7 million, compared to $10.4 million as of December 31, 2022. Net cash used in operating activities for the three months ended March 31, 2023 was $3.7 million, compared to $2.5 million for the comparable period in 2022.

Conference Call and Webcast Information

Bio-Path Holdings will host a conference call and webcast today at 8:30 a.m. ET to review these first quarter 2023 financial results and to provide a general update on the Company. To access the conference call please dial (833) 630-1956 (domestic) or (412) 317-1837 (international). A live audio webcast of the call and the archived webcast will be available in the Media section of the Company’s website at www.biopathholdings.com.

On May 12, 2023 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a leader in cell therapy to treat cancer, reported its financial results for the first quarter ended March 31, 2023 and provided a business update (Press release, Adaptimmune, MAY 12, 2023, View Source [SID1234631580]).

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Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer: "We have entered 2023 at pace with the announcement of the strategic combination with TCR2, the finalization of the return of lete-cel from GSK and continued excellent progress on the rolling BLA submission to make afami-cel a transformative therapy for people with synovial sarcoma. In addition, we are deploying ADP-A2M4CD8 in ovarian cancer with the SURPASS-3 clinical trial. Following the strategic combination with TCR2, the combined company will have the most robust and advanced pipeline in the solid tumor cell therapy field and will position us as a preeminent integrated cell therapy company with a cash runway into early 2026."

Afami-cel – Adaptimmune’s first potential commercial product for the treatment of synovial sarcoma

BLA update

In Q1 2023, Adaptimmune completed submission of the clinical module (Part 2) of the afami-cel BLA, which is targeted for completion in mid-2023. This BLA is supported by data from Cohort 1 of the pivotal trial SPEARHEAD-1, which met its primary endpoint for efficacy. The Company has Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for afami-cel for the treatment of synovial sarcoma. Cohort 2 of the SPEARHEAD-1 trial has completed recruitment.

Data presentations

ASGCT: Translational analyses for afami-cel will be showcased on May 18th at 4:00 p.m. PST during the Cell Therapy Product Engineering and Development oral session in Room 502AB at the upcoming American Society of Cell and Gene Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting in a presentation entitled "Mechanistic Characterization of Afamitresgene Autoleucel." These analyses seek to correlate in-vitro T-cell effector functions with patient response.

ASCO: Data from SPEARHEAD-1 will be presented on June 3rd at 1:15 p.m. CDT during the Sarcoma Track Poster Session in Hall A at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in a presentation entitled: "The SPEARHEAD-1 trial of afamitresgene autoleucel ("afami-cel"): Analysis of overall survival in advanced synovial sarcoma."

AACR: Translational data from patients with sarcoma who received afami-cel were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April. Data demonstrated engagement of the broader immune system after a single dose of afami-cel correlating with durable antitumor activity (poster can be accessed here). These data further support the use of afami-cel for the treatment of synovial sarcoma.

ADP-A2M4CD8 – Adaptimmune’s next-generation product with responses in multiple solid tumor indications

● Initiating the Phase 2 SURPASS-3 trial in combination with nivolumab for platinum resistant ovarian cancer. This trial has the potential to become registrational. ADP-A2M4CD8 has been granted FDA RMAT designation for treatment of patients with platinum resistant ovarian cancer.
● Initiating additional cohorts in the Phase 1 SURPASS trial in combination with pembrolizumab to treat patients in the first-line treatment setting for head & neck cancer and second-line setting for urothelial cancer.

Additional pipeline updates

● Adaptimmune and GSK agreed terms for transfer of PRAME and NY-ESO target programs back to Adaptimmune. Adaptimmune will receive ~$37 million from GSK in relation to the transition of the ongoing NY-ESO clinical trials
● Adaptimmune and GSK will work collaboratively to ensure continuity for patients in ongoing clinical trials for lete-cel and next generation TCR T-cells targeting NY-ESO
● Adaptimmune will continue to focus on its MAGE-A4 franchise while determining the optimal development path for complementary PRAME and NY-ESO programs
● Partnered programs with Genentech continue with the allogeneic pipeline

Strategic combination with TCR2

● Adaptimmune announced a strategic combination with TCR² Therapeutics Inc.
● As a result, and following the closing of the transaction, it is anticipated that the combined company’s cash runway will extend into 2026.
● The Adaptimmune General Meeting will be held on May 30, 2023 and, subject to receipt of approvals of Adaptimmune shareholders and TCR² stockholders and satisfaction or waiver of other closing conditions, the transaction is expected to close in Q2 2023, following which Adaptimmune shareholders will own approximately 75% of the combined company and TCR2 stockholders will own approximately 25% of the combined company.
● Further information about the combined pipeline, catalysts and changes to the Board of Directors was provided in the March Press Release.

Financial Results for the first quarter ended March 31, 2023

● Cash / liquidity position: As of March 31, 2023, Adaptimmune had cash and cash equivalents of $119.9 million and Total Liquidity1 of $165.6 million, compared to $108.0 million and $204.6 million, respectively, as of December 31, 2022.
● Revenue: Revenue for the first quarter ended March 31, 2023 was $47.6 million, compared to $3.6 million for the same period in 2022. Revenue has increased primarily due to the termination of the Astellas collaboration, resulting in a release of the remaining deferred income for the collaboration being released as revenue in March 2023.
● Research and development (R&D) expenses: R&D expenses for the first quarter ended March 31, 2023 were $25.5 million, compared to $36.8 million for the same period in 2022. R&D expenses decreased due to a decrease in the average number of employees engaged in research and development, decreases in subcontracted expenditures, a decrease in share-based compensation expenses and a decrease in offsetting reimbursements receivable for research and development tax and expenditure credits.
● General and administrative (G&A) expenses: G&A expenses for the first quarter ended March 31, 2023 were $20.4 million, compared to $16.8 million for the same period in 2022 due to restructuring charges recognised in the quarter and an increase in other corporate costs due to an increase in accounting, legal and professional fees incurred in relation to the TCR2 Therapeutics Inc merger agreement, offset by a decrease in share-based compensation expenses.
● Net profit/(loss): Net profit attributable to holders of the Company’s ordinary shares for the first quarter ended March 31, 2023 was $1.0 million ($0.00 profit per ordinary share), compared to a net loss of $50.3 million ($(0.05) loss per ordinary share), for the same period in 2022.

Financial Guidance

The Company believes that its existing cash, cash equivalents and marketable securities, together with the additional payments under the Strategic Collaboration and License Agreement with Genentech and payments under the Termination and Transfer Agreement with GSK, will fund the Company’s current operations into early 2025, as further detailed in the Company’s Quarterly Report on Form 10-Q for the first quarter ended March 31, 2023, to be filed with the Securities and Exchange Commission following this earnings release.

On March 6, 2023 the Company announced entry into a merger agreement under which the Company will combine with TCR² Therapeutics Inc in an all-stock transaction. Following the closing of the transaction, we currently estimate that the cash runway of the combined company will extend into early 2026.

Webcast Information

The Company will host a live webcast to provide additional details at 8:00 a.m. EDT (1:00 p.m. BST) today, May 12, 2023. A live webcast of the conference call and replay can be accessed at View Source Call in information is as follows: (800)-319-4610 (US or Canada) or +1 (416)-915-3239 (International and additional options available HERE). Callers should dial in 5-10 minutes prior to the scheduled start time and simply ask to join the Adaptimmune call.

On May 12, 2023 Pulmatrix Inc (NASDAQ: PULM), a clinical-stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and central nervous system disease using its patented iSPERSE technology, reported its first quarter financial results for 2023 and provided a corporate update on its development programs (Press release, Pulmatrix, MAY 12, 2023, View Source [SID1234631574]).

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Ted Raad, Chief Executive Officer of Pulmatrix commented, "Our focus in the first quarter was to advance both PUR1900 and PUR3100. We initiated patient dosing for the PUR1900 Phase 2b study in allergic bronchopulmonary aspergillosis (ABPA). This is an important milestone for PUR1900 which has the potential to be the first product approved for treatment of ABPA, a disease with high unmet need that affects more than 300,000 patients in the United States." Mr. Raad continued, "We also announced topline results for a Phase 1 study of PUR3100, our orally inhaled formulation of dihydroergotamine (DHE) being developed for the treatment of acute migraine. Based on these Phase 1 results, we are eager to advance PUR3100 into Phase 2 and plan to file an Investigational New Drug Application (IND) in mid-2023. We anticipate that PUR3100 will be Phase 2 ready by mid-year so that we are well positioned for potential partnership discussions. While we advance all of our programs clinically, we have been keenly focused on driving operational efficiencies and have extended our projected cash runway from second quarter 2024 into the fourth quarter of 2024."

First Quarter 2023 and Recent Program and Corporate Highlights

PUR1900

PUR1900 is currently in a Phase 2 trial for the treatment of ABPA in patients with asthma (). In February 2023, Pulmatrix began dosing patients for its proof-of-concept Phase 2b study of PUR1900 (itraconazole, administered as a dry powder for inhalation). This Phase 2b trial is a randomized, double-blind, multi-center, placebo-controlled study to evaluate PUR1900’s efficacy and safety. The multi-center study is being conducted in the United States, United Kingdom, Australia and France. Endpoints include safety, tolerability, and potential efficacy outcomes to identify potential registrational endpoints in adult patients with asthma and ABPA. Pulmatrix expects to report topline data from this study in mid-2024.
PUR3100

PUR3100 is under development as an orally inhaled dihydroergotamine (DHE) engineered with iSPERSE for the acute treatment of migraine. On January 4, 2023, Pulmatrix announced PUR3100 was well-tolerated and there was a lower incidence of nausea and no vomiting was observed in PUR3100 dose groups compared to intravenously (IV) administered DHE. The study also showed that PUR3100 achieved peak exposures in the targeted therapeutic range and time to maximum concentration occurred at five minutes after dosing at all dosing levels. Pulmatrix plans to present the data at an appropriate upcoming scientific conference.
Based on the rapid systemic exposure in the therapeutic range and the improved side effect profile compared to IV dosing, Pulmatrix believes the PUR3100 formulation of DHE will be differentiated from other products approved or in development. Pulmatrix believes that PUR3100’s potential for convenient self-administration and a pharmacokinetic profile demonstrated in studies to date could address unmet needs for patients with acute migraine.
Pulmatrix plans to submit an IND in mid-2023 for a randomized, placebo-controlled, Phase 2 clinical study of PUR3100 in patients with acute migraine. The Phase 2 study would assess the safety and effectiveness of two dose levels of PUR3100, selected based on the initial Phase 1 clinical study. Initiation of the Phase 2 study is pending potential financing or partnership.
PUR1800

In February 2023, Pulmatrix presented complete results from a Phase 1b study of PUR1800 for acute exacerbations of chronic obstructive pulmonary disease (AECOPD). The topline data was initially announced in March 2022, at the American Academy of Allergy, Asthma & Immunology annual conference. The completed data analysis is expected to inform the study design of a potential Phase 2 study in patients with AECOPD. Pulmatrix plans to pursue partnership opportunities to advance PUR1800 into a potential Phase 2 clinical trial.
First Quarter 2023 Financial Results

Revenues increased $0.3 million to $1.5 million for the three months ended March 31, 2023 compared to $1.2 million for the three months ended March 31, 2022. The increase is related to the Company’s revenues recognized in accordance with the Cipla Agreement during the period.

Research and development expenses decreased $0.2 million to $3.9 million for the three months ended March 31, 2023 compared to $4.1 million for the three months ended March 31, 2022. The decrease was primarily due to decreased spend of $0.6 million in costs related to the Company’s PUR3100 program and $0.3 million in costs related to the Company’s PUR1800 program, partially offset by increased spend of $0.6 million in costs related to the Company’s PUR1900 program.

General and administrative expenses increased $0.2 million to $2.2 million for the three months ended March 31, 2023, as compared to $2.0 million for the three months ended March 31, 2022. The increase was primarily due to increased legal and professional services costs.

Pulmatrix’s total cash and cash equivalents balance as of March 31, 2023 was $30.8 million. The Company anticipates that its cash position is sufficient to fund operations into the fourth quarter of 2024.