On May 9, 2023 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported financial results for the first quarter ended March 31, 2023 (Press release, Repare Therapeutics, MAY 9, 2023, View Source [SID1234631291]).

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"We continue to execute clinically and across our pipeline programs, including presenting initial clinical data of camonsertib in combination with various PARP inhibitors from the ongoing TRESR and ATTACC trials at this year’s AACR (Free AACR Whitepaper) conference," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "These findings are consistent with our preclinical data demonstrating that low, intermittent dosing of the camonsertib and PARP inhibitor combinations appears well tolerated and exhibited antitumor activity, most notably in advanced ovarian cancer. In addition, we are on track to present initial Phase 1 monotherapy data for RP-6306 in June."

First Quarter 2023 Review and Operational Updates:

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Announced initial clinical data from the Phase 1/2 TRESR and ATTACC trials evaluating camonsertib (RP-3500/RG6526, partnered with Roche), a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase), in combination with three poly (ADP-ribose) polymerase (PARP) inhibitors in a Clinical Trials Plenary Session at the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
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Camonsertib combinations appear to be well tolerated. Dose limiting toxicity in 68 patients treated with the proposed combination doses were related to myelotoxicity (Grade 3+ anemia 3%, thrombocytopenia 6%, neutropenia 7%, and febrile neutropenia 3%). No prophylactic growth factors were required when administering the PARP inhibitors at evaluated doses.
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Camonsertib combination resulted in durable clinical benefit across tumor types and genomic alterations, regardless of choice of PARP inhibitor and presence of platinum resistance. Overall clinical benefit rate (CBR) for all patients was 48%. Patients with platinum-resistant tumors had an overall response rate (ORR) of 12% and CBR of 49% and benefited similarly to non-platinum-resistant tumors (ORR 13%, CBR 46%).

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Compelling results were observed particularly in patients with advanced ovarian cancer (n = 19). In these patients, overall response was 32%, CBR was 58% and median progression-free survival was approximately 7 months with treatment greater than 16 weeks and ongoing in 9 patients.
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Early circulating tumor DNA molecular responses in 66% (31/47) of evaluable patients confirm antitumor activity of low dose, intermittent PARP inhibitor + ATR inhibitor therapy. The molecular response rate (MRR) was significantly higher in patients with clinical benefit (83%) compared to those without (48%; p=0.015) and significantly higher than camonsertib monotherapy that was also administered at higher doses (43% or 27/63; p=0.02). Molecular responses were also observed in patients with prior PARP inhibitor exposure (57%) and platinum resistance (64%).
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Repare is conducting dose optimization and efficacy assessments in tumor specific expansions in the ATTACC study in collaboration with Roche to support future clinical development plans for camonsertib combinations with PARP inhibitors.
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Evaluating RP-6306, a first-in-class, oral PKMYT1 inhibitor as a monotherapy and in combinations in multiple early clinical studies.
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Repare presented two poster presentations for RP-6306 at the 2023 AACR (Free AACR Whitepaper) Annual Meeting regarding the co-mutation landscape in CCNE1 amplifications and the tumor heterogeneity of copy number in ovarian and uterine cancers. Additionally, several collaborators presented preclinical findings on the potential benefits of combining a Wee1 inhibitor with RP-6306 and the effect of RP-6306 in triple negative breast cancer.
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Repare expects to report initial Phase 1 monotherapy clinical data for RP-6306 for the treatment of molecularly selected advanced solid tumors (MYTHIC) in June 2023. The Company expects to report initial Phase 1 combination therapy clinical data for RP-6306 for the treatment of molecularly selected advanced solid tumors in the fourth quarter of the year.
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Repare is working with clinical investigators to initiate clinical testing, as part of an investigator-sponsored trial (IST), of a fourth RP-6306 combination with carboplatin, with first patient dosing expected this year.
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Repare is collaborating with the Canadian Cancer Trials Group for a basket Phase 2 IST to evaluate RP-6306 in patients with selected, advanced cancers receiving standard agents that is expected to begin this year. A sub-study under the master clinical trial protocol will evaluate RP-6306 in combination with gemcitabine in patients with CD4/6i-resistant ER+/HER2- metastatic breast cancer.
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Advancing preclinical programs into clinical development.
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Repare initiated IND-enabling studies in the first half of this year for a small molecule, now designated RP-1664, against an undisclosed target with potential to enter the clinic in late 2023 or early 2024.
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Repare is also pursuing development of an inhibitor of polymerase theta (Polθ) that is expected to enter the clinic in 2024.
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In April 2023, Repare announced the appointment of Susan Molineaux, Ph.D., to its Board of Directors, effective as of the date of the Company’s upcoming annual meeting of shareholders in June 2023. Concurrent with Dr. Molineaux’s appointment as of the date of the annual meeting, Jerel

Davis, Ph.D., Managing Director at Versant Ventures and a founding member of Repare’s Board of Directors, will step down from the Board. Additionally, Repare has expanded the senior leadership team with the appointment of Daniel Belanger as EVP Human Resources in May 2023.
First Quarter 2023 Financial Results:

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Cash and cash equivalents and marketable securities: Cash and cash equivalents and marketable securities as of March 31, 2023 were $314.1 million.
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Revenue from collaboration agreements: Revenue from collaboration agreements were $5.7 million and $0.4 million for the three months ended March 31, 2023 and 2022, respectively. The year-over-year increase in revenue was due to revenue recognized from our collaboration and license agreement with Roche.
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Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $31.8 million and $26.5 million for the three months ended March 31, 2023 and 2022, respectively. The year-over-year increase in Net R&D expenses was primarily due to higher personnel related costs from headcount in support of our development activities, and direct external costs related to the advancement of preclinical programs into IND-enabling studies.
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General and administrative (G&A) expenses: G&A expenses were $8.6 million and $8.8 million for three months ended March 31, 2023 and 2022, respectively. The year-over-year decrease in G&A was primarily due to lower professional fees associated with our collaboration and license agreement with Roche and lower D&O insurance premiums, offset by higher personnel related costs.
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Net loss: Net loss was $34.9 million, or $0.83 per share, in the three months ended March 31, 2023, and $34.8 million, or $0.83 per share in the three months ended March 31, 2022.
About Repare Therapeutics’ SNIPRx Platform

Repare’s SNIPRx platform is a genome-wide CRISPR-based screening approach that utilizes proprietary isogenic cell lines to identify novel and known synthetic lethal gene pairs and the corresponding patients who are most likely to benefit from the Company’s therapies based on the genetic profile of their tumors. Repare’s platform enables the development of precision therapeutics in patients whose tumors contain one or more genomic alterations identified by SNIPRx screening, in order to selectively target those tumors in patients most likely to achieve clinical benefit from resulting product candidates.

On May 9, 2023 Quanterix Corporation (NASDAQ: QTRX), a company fueling scientific discovery through ultrasensitive biomarker detection, reported financial results for the three months ended March 31, 2023 (Press release, Quanterix, MAY 9, 2023, View Source [SID1234631290]).

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"Our corporate transformation initiated in August 2022 to maximize Quanterix’s full potential is on track. We remain confident that we have taken the right steps to build a strong foundation in preparation for future growth," said Masoud Toloue, President and Chief Executive Officer of Quanterix. "This progress takes on added importance with the FDA’s recent decision to approve tofersen for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis, in large part based on strong surrogate neurofilament light chain biomarker data. This decision adds significant momentum to the use of biomarkers for predicting disease severity and clinical benefit in neurodegenerative diseases, and we believe Quanterix will continue to be at the forefront of these developments."

First Quarter 2023 Financial Highlights

● Q1 total revenue was $28.5 million versus prior year Q1 of $29.6 million, a decrease of 4% driven by a decline in instrument revenue. Q1 total revenue increased 10% from Q4 2022 driven by increased consumable revenue.
● Gross margin, a key success indicator of the strategic realignment, saw strong quarter over quarter improvement. Q1 2023 GAAP gross margin was 59.5% versus Q1 2022 GAAP gross margin of 49.3% and Q4 2022 GAAP gross margin of 48.8%. Q1 2023 non-GAAP gross margin was 53.1% versus Q1 2022 non-GAAP gross margin of 43.2% and Q4 2022 non-GAAP gross margin of 41.3%.
● Q1 net loss was $6.1 million versus prior year Q1 of $18.2 million, a decrease of 66%.
● Cash burn for Q1 was $9.1 million, leaving us with $329.4 million of unrestricted cash as of March 31, 2023.
For additional information on the non-GAAP financial measures included in this press release, please see "Use of Non-GAAP Financial Measures" and "Reconciliation of GAAP to Non-GAAP Financial Measures" below.

2023 Business Highlights

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In January 2023, the Company expanded its lab developed test (LDT) menu with the launch of an NfL LDT, which can be used as an aid in the evaluation of individuals for possible neurodegenerative conditions or other causes of neuronal or central nervous system damage. Quanterix’s Simoa NfL is the most widely published NfL test with hundreds of research papers, demonstrating its validity for assessing neuronal damage, and Simoa NfL has become widely adopted in therapeutic clinical trial designs. We believe the recent FDA approval of tofersen, which relied on neurofilament light chain biomarker data, also validates the importance of this test.

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The Company participated in the 2023 Alzheimer’s Disease/Parkinson’s Disease conference in Gothenburg Sweden. Simoa-based biomarkers were highlighted in more than 11 panel discussions and over 30 poster presentations.

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Published discoveries enabled through Quanterix’s Simoa technology continue to illustrate industry reliance on the Company’s ultra-sensitive technology for breakthrough discovery in research and clinical applications. The technology was highlighted in more than 140 new publications in the first quarter 2023, bringing total Simoa-specific inclusions to over 2,200 as of March 31, 2023.

Full Year Business Outlook

We have made a modest increase to our guidance for the full year 2023. We expect revenues to be in the range of $104 to $111 million. We expect GAAP gross margin percentage to be in the high 40s and non-GAAP gross margin percentage to be in the mid 40s. Expected cash burn for 2023 is unchanged, and we expect an approximately 10% improvement over 2022.

Conference Call

In conjunction with this announcement, Quanterix Corporation will host a conference call on May 9, 2023 at 4:30 p.m. Eastern Time. Individuals interested in listening to the conference call may do so by pre-registering here and obtaining a dial-in number and passcode.

A live webcast will also be available at: View Source You may also access the live webcast by visiting the News & Events page within the Investors section of the Quanterix website at www.quanterix.com. The webcast will be available on the Company’s website for one year following completion of the call.

On May 9, 2023 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, reported financial results for the first quarter ended March 31, 2023 and provided a business update (Press release, Precision Biosciences, MAY 9, 2023, View Source [SID1234631289]).

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"Our progress in the first quarter of 2023 makes it clear that we are focused on the generation of meaningful clinical data from our CAR T programs while working in parallel to advance our broad in vivo portfolio into the clinic as soon as possible," said Michael Amoroso, Chief Executive Officer at Precision BioSciences. "We continue to collect data from our latest patient cohorts for both our azer-cel and PBCAR19B programs and anticipate providing an update once we have sufficient follow up from these cohorts. We expect that these data will inform key next steps for development and potential regulatory interactions."

"From our in vivo portfolio, we continue to leverage the core features of ARCUS that we believe drive high efficiency gene insertion and the ability to make large gene excisions while advancing programs where we see a clear and rapid path to the clinic. Preclinical data presentations at the Global Hepatitis Summit 2023 on hepatitis B virus (HBV) and an upcoming, late breaker oral presentation on Duchenne muscular dystrophy (DMD) at the American Society of Gene & Cell Therapy Meeting highlight key execution steps that have brought ARCUS closer to the clinic. We look forward to providing further updates across our wholly owned and partnered gene editing programs at our upcoming in vivo gene editing R&D Day in the middle of the year," added Mr. Amoroso.

Ex Vivo Allogeneic CAR T Portfolio:

Azer-cel: Precision continues to progress its Phase 1/2a clinical trial of azer-cel, the Company’s lead investigational anti-CD19 allogeneic CAR T candidate, in adult subjects with non-Hodgkin lymphoma (NHL) who have relapsed following CAR T treatment. Precision plans to provide an update on the expansion cohort as well as additional long-term follow up from the previously presented azer-cel cohorts in May 2023. As of May 1, 2023, seven patients have been treated in the expansion/Phase 2a cohort with a lower dose lymphodepletion regimen, in addition to the 12 patients who were treated in previous cohorts.

PBCAR19B: Precision continues to progress the ongoing Phase 1 study of PBCAR19B, its second generation, anti-CD19 targeting allogeneic CAR T candidate designed to evade immune rejection by host T cell and natural killer (NK) cells with a single-gene edit to knock-down beta-2 microglobulin and insert an HLA-E transgene. The Company also expects to provide a program update on seven patients treated at Dose Level 2 in May 2023.

In Vivo Gene Editing Portfolio:

Chronic Hepatitis B Virus (HBV): Precision is prioritizing nomination of a development candidate for its wholly owned PBGENE-HBV in vivo program, with the goal of submitting a Clinical Trial Application (CTA) and/or Investigational New Drug Application (IND) in 2024. An abstract titled "Targeting Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo" was presented in April 2023 at the Global Hepatitis Summit 2023. Data showed that ARCUS demonstrated a high degree of on-target antiviral activity, as measured by substantial reductions of both intracellular cccDNA and secretion of HBsAg, with no detectable translocations in ARCUS-treated primary human hepatocytes. These data support the further development of PBGENE-HBV with the goal of developing a functional cure for HBV. The Company plans to present additional data in 2023.

Novartis Partnered Program: Precision continues to advance its gene editing program with Novartis to develop a custom ARCUS nuclease for patients with sickle cell disease and beta thalassemia. The collaborative intent is to insert, in vivo, a therapeutic anti-sickling gene as a potential one-time transformative treatment administered directly to the patient that would overcome many of the hurdles present today with other therapeutic technologies, including those targeting an ex vivo gene editing approach.

Prevail, a wholly-owned subsidiary of Lilly, Partnered Programs: Precision continues its in vivo gene editing collaboration with Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company (Lilly), in applying ARCUS nucleases to three initial targets, including DMD in muscle, a central nervous system directed target, and a liver directed target. The goal of the PBGENE-DMD program is to utilize a pair of ARCUS nucleases, delivered by a single AAV, that are designed to excise an approximately 500,000 base pair mutation "hot spot" region from the dystrophin gene to generate a variant of the dystrophin protein that is functionally competent.

In April 2023, Precision announced that a late-breaking abstract featuring preclinical data from its PBGENE-DMD program for the potential treatment of DMD was selected for an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting being held May 16-20, 2023 in Los Angeles, CA.

Ornithine Transcarbamylase (OTC) Deficiency: Led by iECURE, an ARCUS-mediated gene insertion approach is being pursued as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. Non-human primate (NHP) data presented by researchers from the University of Pennsylvania’s Gene Therapy Program demonstrated sustained gene insertion of a therapeutic OTC transgene one-year post-dosing in newborn and infant NHP with high efficiency. iECURE is targeting to submit a CTA and/or IND in the second half of 2023.

Other ARCUS Research: Preclinical data presented at the Keystone Symposium in March 2023 demonstrated the unique potential of ARCUS nucleases to achieve high frequency gene insertion in nondividing primary hepatocytes. These data highlight the potential advantage of ARCUS for use in developing therapeutics for gene addition, for which Precision believes ARCUS is highly differentiated given the need for extremely low levels of off-target editing and high insertion efficiency.

Corporate:

Intellectual Property (IP) Update: In February 2023, the U.S. Patent and Trademark Office issued Precision a Notice of Allowance for an application with composition of matter claims covering a PCSK9-specific ARCUS nuclease. This ARCUS nuclease has been used preclinically for both gene deletion approaches for cardiovascular diseases such as familial hypercholesterolemia as well as a viable and safe site to insert a functional copy of a specific gene to restore function addressing other diseases requiring gene addition. This patent further builds on the Company’s IP portfolio that cover the ARCUS platform and its use developing novel ex vivo and in vivo gene editing therapies.

Quarter Ended March 31, 2023 Financial Results:

Cash and Cash Equivalents: As of March 31, 2023, Precision had approximately $158 million in cash and cash equivalents. In line with the same period for 2022, net cash used in operating activities for the quarter ended March 31, 2023 are expected to be higher in the first fiscal quarter than the remainder of 2023 primarily due to the timing of the Company’s annual compensation cycle. The Company expects that existing cash and cash equivalents, expected operational receipts, and available credit will be sufficient to fund its operating expenses and capital expenditure requirements through the first quarter of 2025.

Revenues: Total revenues for the quarter ended March 31, 2023 were $8.8 million, as compared to $3.3 million for the same period in 2022. The increase of $5.5 million in revenue during the quarter ended March 31, 2023 was primarily driven by revenue recognized under the Novartis agreement as work thereunder began in the third quarter of 2022.

Research and Development Expenses: Research and development expenses were $22.2 million for the quarter ended March 31, 2023, as compared to $20.0 million for the same period in 2022. The increase of $2.2 million was primarily due to an increase in direct research and development expenses related to our azer-cel and PBCAR19B product candidates as well as consulting fees related to our increased focus on quality management, partially offset by a decrease in expenses related to manufacture of materials for use in preclinical studies, PBCAR20A external development costs, and share-based compensation expense from recent forfeitures.

General and Administrative Expenses: General and administrative expenses were $11.1 million for the quarter ended March 31, 2023, as compared to $10.7 million for the same period in 2022. The increase of $0.4 million was primarily related to consulting fees related to in vivo program competitive landscape analyses, partially offset by a decrease in insurance expense.

Net Loss: Net loss was $25.1 million, or $(0.23) per share (basic and diluted), for the quarter ended March 31, 2023, as compared to a net loss of $28.2 million, or $(0.46) per share (basic and diluted), for the same period in 2022. Weighted average shares of common stock outstanding were approximately 111.3 million for the quarter ended March 31, 2023, as compared to approximately 61.0 million for the quarter ended March 31, 2022. The increase in weighted average shares of common stock outstanding was primarily due to a $50 million underwritten offering of common stock and Novartis’ $25 million equity investment in 2022.

On May 9, 2023 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, reported updates and financial results for the first quarter ended March 31, 2023 (Press release, Poseida Therapeutics, MAY 9, 2023, View Source [SID1234631288]).

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"In the first quarter, we continued to advance our clinical and research stage efforts as well as enhanced our leadership with the recent appointment of Kristin Yarema, Ph.D., as President, Cell Therapy," said Mark Gergen, Chief Executive Officer of the Company. "In our allogeneic CAR-T portfolio, we are actively enrolling patients in the Phase 1 studies for P-MUC1C-ALLO1 and P-BCMA-ALLO1 and remain on track for an IND for P-CD19CD20-ALLO1 mid-year. These initial allogeneic clinical programs are designed to evaluate many aspects of our unique high-TSCM allogeneic platform including dosing strategies, pre-conditioning regimens, potential biomarkers and many other factors. In our gene therapy portfolio, we are in discussions with Takeda about our collaboration following the news that they have made some strategic decisions in their research priorities and will update when appropriate. In the meantime, we remain excited about the strong progress made on all gene therapy programs and look forward to giving six preclinical presentations at the American Society of Gene and Cell Therapy annual meeting next week."

Program Updates

CAR-T Programs

In cell therapy, the Company is focused on three allogeneic CAR-T programs with two programs currently progressing in Phase 1 clinical trials and one expected IND during the year:

MUC1-C Program

P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate targeting solid tumors derived from epithelial cells, including breast and ovarian cancers. The Company is currently evaluating P-MUC1C-ALLO1 in a Phase 1 clinical trial and presented early clinical data in December 2022 at the European Society for Medical Oncology Immuno-Oncology 2022 Annual Congress (ESMO I-O) in Geneva, Switzerland. The Company currently expects to present further clinical updates for the program at a medical meeting in 2023.

BCMA Program

P-BCMA-ALLO1 is an allogeneic CAR-T product candidate being developed to target relapsed/refractory multiple myeloma (R/R MM) in partnership with Roche. The Company is currently evaluating P-BCMA-ALLO1 in a Phase 1 clinical trial and shared early clinical data from the

program at ESMO (Free ESMO Whitepaper) I-O in December 2022. The Company currently expects to present further clinical updates for the program at a medical meeting in 2023, subject to clearance with Roche.

CD19CD20 Program

P-CD19CD20-ALLO1 is a preclinical allogeneic CAR-T product being developed to target B-cell malignancies in partnership with Roche. P-CD19CD20-ALLO1 is the Company’s first dual CAR program and contains two fully functional CAR molecules to target cells that express either CD19 or CD20. The Company believes that by targeting both CD19 and CD20, there is potential to overcome potential antigen escape that has been observed by others. The Company expects to file an IND for P-CD19CD20-ALLO1 in mid-2023.

Gene Therapy Programs

The Company is advancing multiple preclinical gene therapy programs in liver-directed diseases:

OTC Program

P-OTC-101 is an in vivo program for the treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene. The Company is developing the P-OTC-101 program utilizing a hybrid delivery system and working on an updated timeline for the program. The Company presented data at its R&D Day in February 2023, highlighting continued advancements in preclinical models leading towards a potential functional cure of OTC Deficiency.

FVIII Program

The Company is advancing its P-FVIII-101 preclinical program partnered with Takeda, which is in development for the in vivo treatment of Hemophilia A. P-FVIII-101 utilizes piggyBac gene modification delivered via lipid nanoparticle that has demonstrated stable and sustained Factor VIII expression in animal models. The Company is presenting preclinical data from this program at the upcoming American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting being held in Los Angeles on May 16-20, 2023.

PAH Program

Announced at the Company’s R&D Day in February 2023, P-PAH-101 is a liver-directed gene therapy partnered with Takeda to treat Phenylketonuria (PKU), an inherited genetic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene resulting in buildup of phenylalanine in the body. If left untreated, PKU can affect a person’s cognitive development. P-PAH-101 utilizes piggyBac technology combined with its hybrid adeno-associated virus (AAV) and nanoparticle delivery system. The Company’s preclinical data has demonstrated the potential to resolve phenylalanine to normal levels following a single treatment in juvenile and adult mice. P-PAH-101 is currently in preclinical development.

Leadership Updates

Kristin Yarema, Ph.D., Appointed President, Cell Therapy

In April 2023, the Company announced the appointment of Kristin Yarema, Ph.D., as President, Cell Therapy, to lead the Company’s drug development programs in cell therapy, including its collaboration with Roche. She joined the Company after most recently serving as Chief Commercial Officer at Atara Biotherapeutics, where she led the commercialization of EBVALLO, which became the world’s first marketed allogeneic T cell therapy after receiving regulatory approval in Europe for the treatment of a rare lymphoma. Prior to Atara, she held a series of U.S. and global commercial leadership roles at Amgen and Novartis in hematology-oncology among other therapeutic areas.

Rafael G. Amado, M.D., Appointed to Board of Directors

In April 2023, Rafael G. Amado, M.D., was appointed to the Company’s Board of Directors. Dr. Amado is currently President, Head of Global Oncology Research and Development at Zai Lab, a public biopharmaceutical company. Prior to Zai Lab, Dr. Amado served as Executive Vice President, Head of Research and Development and Chief Medical Officer from September 2019 to December 2022 at Allogene Therapeutics, Inc. and President of Research and Development from August 2018 to August 2019 and Chief Medical Officer from March 2015 to August 2018 at Adaptimmune, LLC.

Financial Results for the First Quarter 2023

"We continue to be disciplined on spend and evaluate plans and options to help us further manage our cash burn rate," said Johanna Mylet, Chief Financial Officer at Poseida. "We have been operating at the low end of our guided cash utilization range and our current base plan should allow us to preserve our runway into at least mid-2024, and possibly further depending on other decisions, including business development opportunities."

Revenues

Revenues were $10.3 million for the first quarter ended March 31, 2023, compared to $1.4 million for the same period in 2022. The increase of revenues was primarily due to revenues earned from the collaboration and license agreement with Roche, which became effective in the third quarter of 2022.

Research and Development Expenses

Research and development expenses were $38.1 million for the first quarter ended March 31, 2023, compared to $48.9 million for the same period in 2022. The decrease was primarily driven by the wind-down of the Company’s clinical development activities associated with our autologous programs and related contract termination expense in the prior year and the transition of manufacturing to the Company’s internal pilot plant for P-BCMA-ALLO1, partially offset by increases in the number of ongoing clinical trials, including enrollment and manufacturing for the P-MUC1C-ALLO1 and P-BCMA-ALLO1 Phase 1 clinical trials, and increases related to the Company’s preclinical stage programs due to an increase in research collaboration activity and personnel and facilities expenses as a result of increased headcount.

General and Administrative Expenses

General and administrative expenses for the first quarter ended March 31, 2023 were $11.8 million compared to $9.5 million for the same period in 2022. The increase was primarily related to an increase in stock-based compensation expense due to a one-time modification associated with the retirement of the Company’s former Executive Chairman.

Net Loss

Net loss was $38.8 million for the first quarter ended March 31, 2023, compared to $58.1 million for the same period of 2022.

Cash Position

As of March 31, 2023, the Company’s cash, cash equivalents and short-term investments balance was $247.2 million. The Company expects that its cash, cash equivalents and short-term investments together with the remaining near-term milestones and other payments from Roche will continue to be sufficient to fund operations into at least mid-2024.

On May 9, 2023 Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, reported a corporate update and announced first quarter 2023 financial results (Press release, Pliant Therapeutics, MAY 9, 2023, View Source [SID1234631287]).

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"So far in 2023 we have made significant progress in advancing the portfolio including positive 12- and 24-week data readouts from our INTEGRIS-IPF trial and a successful follow-on offering that extended our expected operating runway into the second half of 2026," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant Therapeutics. "With clinical data and development milestones expected throughout the remainder of 2023, including Phase 2a data from INTEGRIS-PSC and initiation of our BEACON-IPF Phase 2b and our oncology Phase 1 trials, we look forward to building upon an already productive year."

First Quarter and Recent Highlights

Bexotegrast (PLN-74809) Highlights

•INTEGRIS-IPF Phase 2a 24-week 320 mg clinical data showed bexotegrast was well tolerated and demonstrated durable improvements in exploratory endpoints. This 24-week Phase 2a data from the 320 dose group showed bexotegrast was well tolerated up to 40 weeks, displayed a favorable pharmacokinetic profile and in exploratory efficacy endpoints showed improvements in forced vital capacity (FVC), Quantitative Lung Fibrosis (QLF) imaging, patient reported cough severity and biomarkers in patients with idiopathic pulmonary fibrosis (IPF) to Week 24. In January, 12-week data from the 320 mg dose group showed bexotegrast demonstrated statistically significant FVC increases at all time points, was well tolerated with no drug-related severe or serious adverse events and showed dose-proportional increases in plasma concentrations, consistent with prior dose groups.
•Initiation of BEACON-IPF, a Phase 2b trial of bexotegrast in patients with IPF, expected in mid-2023. BEACON-IPF is a 52-week, multinational, randomized, placebo-controlled trial evaluating bexotegrast at doses of 160 mg or 320 mg in approximately 270 patients with IPF.
•INTEGRIS-PSC Phase 2a 12-week clinical data expected in the third quarter of 2023. INTEGRIS-PSC is a 12-week randomized, dose-ranging, double-blind, placebo-controlled trial evaluating safety, tolerability and pharmacokinetics of bexotegrast at doses of 40, 80 or 160 mg in approximately 84 patients with primary sclerosing cholangitis (PSC). Exploratory efficacy endpoints include fibrosis biomarkers such as PRO-C3 and enhanced liver fibrosis (ELF) score, changes in alkaline phosphatase (ALP), and liver imaging. INTEGRIS-PSC is fully enrolled.
•First patient dosed in the INTEGRIS-PSC Phase 2a 320 mg dose cohort, evaluating bexotegrast for at least 24 weeks. This cohort is evaluating the safety, tolerability and pharmacokinetics of bexotegrast at a dose of 320 mg versus placebo over at least 24 weeks of treatment in approximately 28 patients with PSC. The trial is also evaluating exploratory efficacy endpoints including fibrosis biomarkers such as PRO-C3 and ELF score, changes in ALP and liver imaging.

Early-Stage Development Programs

•Initiation of a Phase 1 first-in-human trial of PLN-101095 in oncology expected in the second quarter of 2023. PLN-101095 is an oral, small molecule, dual selective inhibitor of αvβ8 and αvβ1 integrins designed to block TGF-β activation in the tumor microenvironment. Initiation of a Phase 1 trial in patients with solid tumors that are resistant to immune checkpoint inhibitors is expected in the second quarter of 2023.
•Muscular dystrophy program progressing through IND enabling activities. PLN-101325 is a monoclonal antibody designed to act as an allosteric agonist of the integrin α7β1. An IND submission for Duchenne muscular dystrophy (DMD) is expected in 2023.

Corporate Highlights

•Closing of underwritten public offering of $287.5 million in common stock. The Company closed a public offering in January 2023, yielding $269.9 million in net proceeds to the Company, which included the underwriter’s exercise in full of their option to purchase additional shares.

First Quarter 2023 Financial Results

•Research and development expenses were $29.3 million, as compared to $20.9 million for the prior-year quarter. The increase was due primarily to employee related expenses and increased clinical and manufacturing related costs associated with our lead program, bexotegrast, partially offset by a decrease in preclinical manufacturing costs for our pipeline product candidates.
•General and administrative expenses were $14.2 million, as compared to $8.6 million for the prior-year quarter. The increase was due to higher personnel-related and professional services expenses.
•Net loss of $37.5 million as compared to $28.1 million for the prior-year quarter due to an increase in operating expenses coupled with a slight decrease in collaboration revenues under the Novartis collaboration during the quarter.
•As of March 31, 2023, the Company had cash, cash equivalents and short-term investments of $577.3 million, which the Company expects to be sufficient to fund operations into the second half of 2026.