CRSP highlights progress in clinical pipeline of gene-edited therapies

 

• CRISPR Therapeutics AG (NASDAQ:CRSP) reported a net loss of $112.2 million for Q3 2023, a significant decrease from the net loss of $174.5 million in Q3 2022.

• R&D expenses for Q3 2023 were $90.7 million, compared to $116.6 million for the same period in 2022.

• The company's cash position as of September 30, 2023, was $1,739.8 million, compared to $1,868.4 million as of December 31, 2022.

• CRSP highlighted significant progress in its clinical pipeline of gene-edited therapies, including exa-cel for the treatment of severe sickle cell disease

CRISPR Therapeutics AG (NASDAQ:CRSP), a leading biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, announced its financial results for the third quarter ended September 30, 2023, on November 6, 2023. The company reported significant progress across its broad clinical pipeline of potentially curative gene-edited therapies.

Company Performance and Challenges

CRISPR Therapeutics AG (NASDAQ:CRSP) reported a net loss of $112.2 million for the third quarter of 2023, compared to a net loss of $174.5 million for the same period in 2022. The company's cash position as of September 30, 2023, was $1,739.8 million, compared to $1,868.4 million as of December 31, 2022. The decrease in cash of $128.6 million was primarily driven by operating expenses, offset by payments received from Vertex in connection with a non-exclusive license agreement and related milestone, as well as interest income.

Financial Achievements

CRISPR Therapeutics AG (NASDAQ:CRSP) reported a decrease in R&D expenses for Q3 2023, which were $90.7 million, compared to $116.6 million for the same period in 2022. The decrease in R&D expense was primarily driven by reduced variable external research and manufacturing costs. General and administrative expenses were $18.3 million for the third quarter of 2023, compared to $27.0 million for the same period in 2022. The decrease in G&A expense was primarily driven by a decrease in external professional costs.

https://finance.yahoo.com/news/crispr-therapeutics-ag-crsp-reports-223002068.html

uniQure: Net Loss Widens, Cash Position Strengthens

 

• uniQure NV (NASDAQ:QURE) reported a net loss of $89.6 million for Q3 2023, compared to a net loss of $47.9 million in the same period in 2022.

• The company's cash position as of September 30, 2023, was approximately $660 million, expected to fund operations into the second quarter of 2027.

• uniQure NV (NASDAQ:QURE) announced a strategic reorganization expected to deliver $180 million of cost savings over the next three years.

• The company received FDA clearance of IND application for AMT-260 in refractory mesial temporal lobe epilepsy.

On November 7, 2023, uniQure NV (NASDAQ:QURE), a leading gene therapy company, announced its financial results for the third quarter of 2023. The company reported a net loss of $89.6 million, or $1.88 basic and diluted loss per ordinary share, compared to a net loss of $47.9 million, or $1.02 basic and diluted loss per ordinary share, for the same period in 2022.

Financial Highlights

As of September 30, 2023, uniQure NV (NASDAQ:QURE) held cash and cash equivalents and investment securities of $658.9 million, compared to $392.8 million as of December 31, 2022. The company's revenues for the three months ended September 30, 2023, were $1.4 million, consistent with the same period in 2022.

Research and development expenses were $65.4 million for Q3 2023, compared to $48.1 million in the same period in 2022. Selling, general and administrative expenses were $18.1 million for Q3 2023, compared to $13.3 million in the same period in 2022.

Company Progress and Achievements

During the third quarter, uniQure NV (NASDAQ:QURE) made significant progress towards advancing multiple clinical-stage programs. The company achieved FDA clearance of the IND for AMT-260 in refractory mesial temporal lobe epilepsy and is making good progress toward initiating patient screening for a Phase I/II trial of AMT-162 in SOD1-ALS and the submission of an IND for AMT-191 in Fabry disease.

https://finance.yahoo.com/news/uniqure-nv-qure-reports-q3-131228993.html

Matinas: Positive in vivo Efficacy Results of Oral LNC Docetaxel Formulation in Melanoma Model

 Study results show reduction in tumor size comparable to IV docetaxel and no systemic toxicity

Matinas BioPharma Holdings, Inc. (NYSE American: MTNB), a clinical-stage biopharmaceutical company focused on delivering groundbreaking therapies using its lipid nanocrystal (LNC) platform technology, announces positive results from an in vivo animal study of an oral LNC formulation of docetaxel, a well-known chemotherapeutic agent used in the management of multiple metastatic and unresectable tumors. Currently, docetaxel is administered intravenously and can be associated with significant side effects and toxicities.

https://finance.yahoo.com/news/matinas-biopharma-announces-positive-vivo-123000703.html

Entrada starts Phase 1 for Duchenne

 Initiated a Phase 1 clinical trial of ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy, marking Entrada’s transition to a clinical-stage company –

– Expanded the Company’s Duchenne franchise with the selection of its third clinical candidate, ENTR-601-50, for the potential treatment of people living with Duchenne who are exon 50 skipping amenable –

– Cash runway expected through 2025 with $354 million in cash, cash equivalents and marketable securities as of September 30, 2023 –

https://www.globenewswire.com/news-release/2023/11/07/2774935/0/en/Entrada-Therapeutics-Reports-Third-Quarter-2023-Financial-Results.html

J&J MedTech aims to put Ottava surgical robot up for US clinical trials in late 2024

 Johnson & Johnson MedTech has given itself a new deadline for the development of its long-awaited entrant into the globally competitive robotic surgery market. 

The company said that in the latter half of next year, it plans to request the FDA’s permission to begin U.S. clinical studies of its Ottava system, which it described as designed to fit inside any operating room around the world.

The soft-tissue surgical robot was previously slated to make its first-in-human debut in late 2022, but Ottava’s timeline was previously pushed back following technical challenges and disruptions due to the COVID-19 pandemic.

Ottava was initially born out of a joint venture between J&J and Google’s life-science-focused sister company, Verily. 

Launched in late 2015 as Verb Surgical, the somewhat secretive outfit had aimed to build the future of the field—which they often described as a digitized and data-fed “Surgery 4.0”—while keeping the details of the robot under wraps. J&J would announce in December 2019 that it was taking over the Fierce 15 winner as a whole and bought out Verily’s stake in the project.

Ottava aims to take on robotics giant Intuitive Surgical, with its mainstay da Vinci system, as well as more recent challengers from Medtronic and CMR Surgical. 

Where Intuitive’s da Vinci, with its large, towerlike frame, inspired competitors to offer somewhat smaller, modular solutions—with Medtronic’s Hugo and CMR’s Versius each separating their multiple robotic arms among rollable carts—Ottava aims to cut the footprint down to the bare minimum by building the hardware directly into a standard-sized patient bed.

Equipped with J&J’s long-running catalog of Ethicon surgical instruments, Ottava aims to offer an “invisible design,” according to the company, with individual robotic arms being stowed underneath the surgical table when they are not in use.

The adaptable system incorporates four arms—down from the previously reported maximum of six—and can link their movements to adjustments of the table, allowing clinicians to reposition the patient without interrupting the procedure.

After receiving regulatory approvals, Ottava will join J&J’s robotic portfolio alongside its Monarch platform—the flexible, snakelike lung endoscopy system developed by Auris Health, which it acquired in 2019 through a $3.4 billion deal—as well as its Velys robot for assisting in knee replacement surgeries.

Medtronic’s Hugo robot, meanwhile, collected green lights in Europe, Canada and Japan in 2022—spanning general surgery, urology and gynecology—and the company announced its first U.S. patient was enrolled in clinical trials last December.

Prior to that, CMR raised a massive $600 million venture capital round in mid-2021 and followed it up with $165 million more in September of this year. The company has also outlined plans for international commercial expansions and has worked with J&J to bundle its Versius robots with Ethicon instrumentation for minimally invasive procedures.

https://www.fiercebiotech.com/medtech/jj-medtech-aims-put-ottava-surgical-robot-us-clinical-trials-late-2024

Augmedix Delivers 50% Revenue Growth and Expanded Gross Margins for Third Quarter

 Augmedix (Nasdaq: AUGX), a healthcare technology company that delivers industry-leading ambient medical documentation and data solutions, reported today financial results for the three and nine months ended September 30, 2023.

“The third quarter marked another period of strong progress for Augmedix as we continue to be a leader in the large and rapidly growing ambient medical documentation market,” commented Manny Krakaris, Chief Executive Officer at Augmedix. “Growing adoption of Augmedix Live and Augmedix Notes drove 50% revenue growth and net revenue retention of 157%, while expanding gross margins by 390 basis points to 49.5%. With strong revenue growth and improving profitability, we are on track to achieve our financial goals and are increasing our revenue guidance for 2023 accordingly.”

Continued Mr. Krakaris, “We are delivering strong financial results while expanding our product portfolio and building out the foundations of our data and platform strategies. We recently launched early access to Augmedix Go, our fully automated scalable AI medical documentation solution, and we continue to collaborate closely with HCA Healthcare to launch Augmedix Go for the emergency room setting. We have also forged strategic relationships with three innovative digital health companies as part of our open network and platform strategy. These vendor partners will be able to use our delivery platform through application programming interfaces (APIs) so that they and our health system partners can efficiently benefit and generate incremental ROI from our structured data and bi-directional communication channel to the point of care.”

https://www.globenewswire.com/news-release/2023/11/06/2774476/0/en/Augmedix-Delivers-50-Revenue-Growth-and-Expanded-Gross-Margins-for-Third-Quarter-of-2023.html

Vertex drops phase 2 lung therapy, rethinks DMD drug's journey toward clinic

Vertex Pharmaceuticals may be looking forward to a “milestone-rich period coming up,” but one candidate will no longer be joining the biotech on its journey while another will be delayed into the clinic.

The company is discontinuing its alpha-1 antitrypsin deficiency (AATD) therapy, dubbed VX-864, “due to non-serious rash events in some patients,” Vertex revealed in its third-quarter earnings release.

Patients with AATD have a mutation that causes them to produce abnormal forms of the AAT protein. These misfolded proteins get trapped in the liver instead of being released into the blood like normal AAT proteins. The buildup of abnormal AAT in the liver, coupled with low levels of AAT in the blood, can result in liver and lung diseases.

VX-864 is a “corrector molecule” designed to prevent the misfolding of AAT and increase plasma levels of AAT. Vertex had already decided against moving the therapy into late-stage development back in 2021. Despite all three dose levels of VX-864 beating placebo when it came to boosting patients’ plasma AAT levels in a phase 2 study, the company decided that the drug’s effect was so small that it was unlikely to “translate into substantial clinical benefit.”

However, that didn’t stop Vertex from launching a fresh phase 2 study the following year to assess the impact of longer-term treatment on polymer clearance from the liver. The study was sparked by an analysis of the previous midstage trial, which had revealed that VX-864 reduced levels of Z-polymer in the blood, Vertex explained at the time. Researchers have investigated Z-polymer as a potential biomarker for ATTD outcomes.

But it seems that this last remaining avenue of exploration for VX-864 has now also been closed due to the reports of rash. In yesterday’s release, Vertex highlighted that it would continue to enroll and dose healthy volunteers in its two other AAT correctors, VX-634 and VX-668. This “next wave” of investigational therapies for the autosomal recessive inherited disease have “significantly improved potency and drug-like properties compared with the first-generation AATD correctors,” the company added.

Vertex’s plans to enter the notoriously tricky Duchenne muscular dystrophy (DMD) space also seem to be stalled for the time being. A week after Sarepta Therapeutics saw its stock tank when its approved DMD gene therapy Elevidys failed to hit the primary goal of a pivotal study, Vertex said it won’t be rushing into the clinic in this indication anytime soon.

Vertex has been investigating whether the muscle degenerative condition can be treated by delivering CRISPR/Cas9 gene editing technology to the muscle cells with a virus, AAV9. The aim is to change the targeted DNA sequence to restore dystrophin protein expression.

But the company said today that after considering the preclinical data generated so far, it’s decided that additional in vitro and animal studies of the delivery system for the therapy are needed before it moves into human testing.

“Consistent with its portfolio approach to research and development, Vertex is also using the learnings from its first-generation vectors to design next-generation delivery systems for in vivo gene editing in DMD,” the company added.

Still, the slow pace of the DMD work and the discontinuation of Vertex’s original AATD bet are small fry compared to the FDA’s upcoming decision on exa-cel. Should the agency give a positive verdict on the CRISPR Therapeutics-partnered prospect in sickle cell disease Dec. 8—which analysts seem pretty confident of—it will mark the first-ever CRISPR gene-editing-based therapy to make it to market.

https://www.fiercebiotech.com/biotech/vertex-drops-lung-therapy-rethinks-dmd-drugs-journey-towards-clinic