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Feliza Mirasol

Roche announced results on Dec. 19, 2024 from a Phase IIb study (PADOVA) where prasinezumab, an investigational monoclonal antibody (mAb), failed to achieve statistical significance, missing the study’s primary endpoint. The study involved 586 patients with early stage Parkinson’s disease (PD) who were treated for a minimum of 18 months while on stable symptomatic treatment. Prasinezumab demonstrated potential clinical efficacy in the primary endpoint, which was time to confirmed motor progression, with a hazard ratio (HR)=0.84 [0.69-1.01] and p=0.0657, missing statistical significance, according to a company press release (1). The effect of prasinezumab, as evaluated in a pre-specified analysis, was more pronounced in the patients (75% of the participants) who were treated with levodopa, with results showing a HR=0.79 [0.63-0.99]. It was also observed that consistent positive trends occurred across multiple secondary endpoints as well as exploratory endpoints.

Prasinezumab also remains well tolerated and no new safety signals were observed in the study. Roche is evaluating the data further and will work together with health authorities to determine what steps to take next.

“Parkinson’s is complex and devastating with no disease modifying treatment options available for the millions of people impacted,” said Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development, Roche, in the press release (1). “We believe the consistent efficacy trends from the Phase IIb study of prasinezumab merit further exploration. We will continue our close collaboration with the Parkinson’s community as we further evaluate the data to determine next steps.”

Both a Phase II study (PASADENA) and this Phase IIb study (PADOVA) will continue to give Roche a chance to explore the observed effects in both studies. Full results from the Phase IIb (PADOVA) study are expected to be presented at an upcoming medical meeting.

Prasinezumab is part of a licensing, development, and commercialization agreement that Roche entered into with Prothena, an Ireland-based late-stage clinical biotechnology company with expertise in protein dysregulation. Under the agreement, formed in December 2013 (1), the companies aim to develop and commercialize mAbs that target α-synuclein, a presynaptic neuronal protein that is genetically and neuropathologically linked to PD (2). Prasinezumab targets the build-up of α-syn protein in the brain, which can potentially prevent further accumulation and spreading between cells. This action can slow down the progression of the disease (1).

“The results from the Phase [IIb] PADOVA study are a significant step forward to potentially bring the first disease-modifying treatment option to the millions of individuals living with Parkinson’s disease and their families,” said Gene Kinney, PhD, president and chief executive officer, Prothena, in that company’s press release (3). “As pioneers in developing the first anti-alpha synuclein targeting antibody, we look forward to Roche presenting the results from the PADOVA study at an upcoming medical conference and sharing with health authorities to determine the most appropriate path forward.”

1. Roche. Roche’s Phase IIb Study of Prasinezumab Missed Primary Endpoint, but Suggests Possible Benefit in Early-Stage Parkinson’s Disease. Press Release. Dec. 19, 2024.
2. Stefanis, L. α-Synuclein in Parkinson's Disease. 

 (2), a009399. DOI: 10.1101/cshperspect.a009399.
3. Prothena. Roche’s Phase IIb Study of Prasinezumab Missed Primary Endpoint, but Suggests Possible Clinical Benefit in Early-Stage Parkinson’s Disease. Press Release. Dec. 19, 2024.

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Results from a Phase IIb study missed the primary endpoint by failing to achieve statistical significance. 

Prasinezumab Misses Primary Endpoint in Roche’s Phase IIb Study, but Has Potential in Treating Early Stage Parkinson’s Disease

Nicolas Pivet Cytiva discusses automation benefits process development analytics

Process Development Benefits from Automated Analytical Technologies

In an interview with the PharmTech Group, Nicolas Pivet, vice-president and general manager, Technology Solutions at Cytiva, notes that among the challenges in developing a biomanufacturing process is the process analytical technology (PAT) used in that process. Pivet discusses how automation benefits process development, especially when applied to analytics.

“I would say digital documentation is always helping with fast regulatory filing and tech transfer,” Pivet says, adding that, “What we've been experiencing in Cytiva is that many of our customers actually use a platform approach for process development, which is great for speed, but sometimes is not optimized for everything.”

“The introduction of recipe-driven equipment in process development has really enabled 24/7 utilization of labs, so, it's really enhancing the throughput of your lab staff,” Pivet says. The scientist, therefore, with the help of automation, can program lab equipment to rapidly execute experiments and collect samples. “What we've been seeing as well is that SCADA—the supervisory control and data acquisition system—is a layer that allows equipment information to be viewed together. That's why, in Cytiva over the past seven years, we've been developing our Figurate automation offering, which is a wide range of automation solutions and libraries, from historian to control systems with Unicorn [a control software] to SCADA with Wonderware [an automation platform] and to DCS [distributed control system] with Delta V and PlantPAx [types of DCS].”

In the area of PAT, Pivet feels that the bio/pharma industry needs to move faster. In the sensors space, for example, P80 sensors have been seeing an emergence of improved sensing technology. “Measuring CQAs [critical quality attributes], for instance, inline or online is really the holy grail of the biopharma industry right now,” Pivet says. “…The industry needs to get there in being able to measure CQAs inline and helping with real-time release, or right-time release, of the drug product.”

Nicolas Pivet, Vice-President and General Manager, Technology Solutions, Cytiva

Nicolas Pivet is vice-president and general manager, Technology Solutions, at Cytiva, where his ability to find the right creative solution—whether for customers, or when writing songs—is at the root of his delivery-focused mindset. Innovation is second nature to Pivet, which allows him to bring fresh approaches to meeting customer needs. He is a seasoned leader, with laser-sharp customer focus and experience across software and hardware R&D, program management, strategy, service operations, and commercial. Since 2023, Pivet has led Cytiva’s global Automation, Digital and Learning Solutions Business Unit, developing digital solutions enabling fast, flexible, and reliable process development and manufacturing for the biopharma industry. He also leads the Manufacturing Capacity Solutions unit, where Cytiva supports customers with powerful and innovative end-to-end solutions that transform biomanufacturing.

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Cytiva's Nicolas Pivet, vice-president and general manager, Technology Solutions, emphasizes how automation benefits process development, particularly in the analytical space.

Orexo, a Sweden-based commercial-stage pharmaceutical company, announced on Dec. 17, 2024, that it has entered into a collaboration with Abera Bioscience (Abera), a Sweden-based platform and vaccine developer. Under the agreement, the companies will use AmorphOX, Orexo’s powder-based drug delivery technology, to develop mucosal vaccine candidates based on Abera's vaccine platform. This collaboration will be funded by Abera through grants it has previously received, mainly from the Coalition for Epidemic Preparedness Innovation (CEPI).

The AmorphOX technology is designed to improve properties of new or existing drugs. The technology aims to develop formulations that aid in significantly stabilizing active ingredients to enable alternative and more effective routes of administration. AmorphOX-based formulations are applicable for a range of active ingredients, from small molecules and peptides to larger biomolecules, such as vaccines, according to a company press release (1).

Orexo and Abera’s partnership will develop powder-based intranasal vaccine candidates with a focus on Abera's influenza vaccine candidate. Because an intranasal influenza vaccine has potential to easily and effectively reduce the spread of infections and prevent disease, they can play a significant role in controlling potential future pandemics.

“Influenza is a family of viruses that could mutate and cause the next pandemic, but seasonal influenza is already a major problem where vaccines are often not effective enough. Orexo has an impressive knowledge and technology for powder formulations of medicines that perfectly complements our expertise in vaccine development. Their innovative technology, AmorphOX, has shown to improve the properties of a wide range of different active ingredients. There is therefore great potential in this area, and we will be able to take many important development steps in the coming year,” said Maria Alriksson, CEO, Abera Bioscience, in the press release (1).

In a historical context, an influenza pandemic in 1918 caused at least 20 million deaths (2). Controlling another influenza pandemic will depend on innovations in vaccine production, according to researchers (3). A long-term goal for influenza vaccination should rely on a more universal vaccine, but there remains debate as to what exact traits a universal vaccine should have. A universal vaccine against all epidemic and pandemic viruses is not realistic, however, but having a vaccine that covers more drift variants may mean there could be less need for vaccine updates and a reduced need for yearly vaccinations, researchers note (4).

“Abera has an innovative and promising vaccine platform for the development of mucosal vaccines, as confirmed by their recent funding from CEPI. The collaboration is fully in line with our strategic development of the AmorphOX platform, and, through collaborations, we can showcase the potential and value of our technology. We look forward to working with Abera, where our experience and technologies are highly complementary,” said Robert Rönn, senior vice-president and head of R&D at Orexo, in the press release (1).

1. Orexo. Orexo Enters into Collaboration with Abera to Develop Nasal Powder Vaccines Based on the Amorphox Technology. Press Release. Dec. 17, 2024.
2. Crosby, A.W. 

America’s Forgotten Pandemic: The Influenza of 1918

; Cambridge University Press, 1989.
3. Kilbourne, E. D. Influenza Pandemics: Can We Prepare for the Unpredictable? 

 (3). DOI: 10.1089/vim.2004.17.350
4. Kim, H.; Webster, R. G.; Webby, R. J. Influenza Virus: Dealing with a Drifting and Shifting Pathogen. 

The companies will use Orexo’s powder-based drug delivery technology to develop mucosal vaccines in an inhaled formulation.

Orexo and Abera Partner to Develop Nasal Powder Vaccines

Scinai Immunotherapeutics (Scinai), an Israel-based biopharmaceutical company focused on developing inflammation and immunology biological products, announced on Dec. 16, 2024 the establishment of a US-based subsidiary for its contract development and manufacturing organization (CDMO) services. The company provides CDMO services through its Scinai Bioservices business unit. This US subsidiary, incorporated in Delaware, will operate under the name Scinai Bioservices Inc.

The Scinai Bioservices CDMO business unit was launched in 2024 and operates from the company's site in Jerusalem, Israel. The Jerusalem facility comprises 1850 square meters (approximately 20,000 square feet) of clean rooms and laboratories, which operate under current good manufacturing practice (CGMP) conditions in compliance with European Medicines Agency (EMA) and FDA requirements, according to a company press release (1).

"We are thrilled and energized by the opening of our new subsidiary in the [United States]. Last week, we signed our first contract with US customer, Serpin Pharma, to support their clinical manufacturing,” said Amir Reichman, CEO of Scinai, in the press release (1). “I believe that our expanded presence in the US will significantly enhance the prospects of our CDMO unit."

From its launch, the CDMO unit has executed drug development projects for nine different biotech companies, which included upstream and downstream process development, scale-up, analytical methods development, and drug manufacturing for pre-clinical and clinical supplies. The company has also partnered with Ayana Pharma, an Israel-based pharmaceutical company specializing in liposomal therapeutics for treating cancer, under which Scinai will provide liposomal encapsulated drug development services.

The Scinai Bioservices unit operates on projects at the early stage, where the company noted in its press release capacity is strained because of the complex manufacturing processes associated with biologics. “Additionally, the BIOSECURE Act, introduced in January 2024, aims to prevent ‘foreign adversary biotech companies’ from receiving US federal funding,” the company stated in its press release (1).

Scinai's new US subsidiary aims to serve US-based early stage biotech companies with the goal of providing required CDMO services that are currently in short supply. The US subsidiary will also allow early stage biotech startups, which are supported by US government grant funding, to use their grant money in contract with Scinai, which would allow them to avoid concerns related to the BIOSECURE Act, Scinai stated in its press release.

To put the situation into context, Parviz Shamlou, senior vice-president of Science and Technology, Abzena, spoke to the PharmTech Group during BIO 2024 this past June about the impact the act will have on the bio/pharmaceutical industry and how the industry is preparing for it. “We appreciate that [the BIOSECURE Act] is an important act designed to protect the health of citizens of this country. And we know we must do everything we can to avoid any unintended, potentially harmful use of our services as highlighted in the act. [We] have put in place a robust contingency plan as well as a CMC [chemistry, manufacturing, and controls] strategy that are designed to mitigate any such risk to supply chain and the development of the assets that our clients bring to us,” Shamlou stated in the interview (2).

1. Scinai Immunotherapeutics. Scinai Immunotherapeutics Establishes US Subsidiary for CDMO Business Unit, Scinai Bioservices Inc. Press Release. Dec. 16, 2024.
2. Haigney S. 

Impact of Biosecure Act on the Pharma Industry (BIO 2024)

Scinai Bioservices Inc. has been established in Delaware as the company's new US-based subsidiary, which will serve biotech companies in early stage drug development.

Scinai Immunotherapeutics Establishes US CDMO Business Unit

AbbVie has signed a definitive agreement to acquire Nimble Therapeutics, a biotechnology company specializing in oral peptide therapeutics, which would add Nimble’s lead asset, an investigational oral peptide interleukin 23 receptor (IL23R) inhibitor that is in preclinical development for treating psoriasis. to AbbVie's immunology pipeline. In addition to the lead asset, AbbVie would also acquire a pipeline of other novel oral peptide candidates that show potential across several autoimmune diseases, AbbVie announced in a Dec. 13, 2024 company press release.

With the acquisition, AbbVie also gains Nimble's peptide synthesis, screening, and optimization platform. This platform uses proprietary technology to facilitate rapid discovery and optimization of peptide candidates for a range of targets.

Under the agreement, AbbVie will pay $200 million in cash at the close of transaction, which is subject to certain customary adjustments. AbbVie will also pay certain interim funding payments, and Nimble's shareholders will be eligible to receive potential payment upon the achievement of a development milestone, according to the press release (1).

"The addition of Nimble's pipeline to AbbVie's existing pipeline, combined with our deep clinical and translational expertise in immunology, represents an important growth opportunity," said Jonathon Sedgwick, PhD, senior vice-president and global head of discovery research, AbbVie, in the press release (1). "Together, AbbVie and Nimble have the potential to help address the significant unmet medical need for people living with autoimmune diseases."

Nimble's lead candidate, which targets IL23R, has potential in the treatment of psoriasis and inflammatory bowel disease (IBD). According to AbbVie, IL23R is a clinically validated therapeutic target in certain autoimmune diseases and is known to be a major contributing factor in psoriasis and IBD pathogenesis as well as progression by way of increased inflammation and amplified immune responses (1).

"Nimble Therapeutics is committed to transforming the discovery of oral peptide-based medicines. With AbbVie's … expertise in developing and commercializing medicines on a global scale, Nimble's novel oral therapies will be well-positioned to reach more people living with autoimmune diseases," said Jigar Patel, PhD, founder and chief executive officer, Nimble Therapeutics, in the release. "The talented, passionate, and dedicated team at Nimble has made great progress over the past few years, and we are pleased that AbbVie has recognized the tremendous potential of our proprietary platform and emerging immunology pipeline."

Nimble is the latest in a series of acquisitions that AbbVie has conducted this year. Also in December 2024, AbbVie completed its 

$1.4 billion acquisition of Aliada Therapeutics

$8.7 billion acquisition of Cerevel Therapeutics

, both of which strengthen AbbVie’s neuroscience pipeline (2,3). In June 2024, AbbVie 

acquired Celsius Therapeutics for $250 million in cash

, which included an antibody drug candidate in clinical development for IBD (4). In May 2024, AbbVie completed its acquisition of Landos Biopharma for approximately $137.5 million in cash, which included that company’s lead asset, NX-13, an oral nucleotide oligomerization domain-like receptor family member X1 agonist in clinical development for treating ulcerative colitis and Crohn's disease (5).

1. AbbVie. AbbVie to Acquire Nimble Therapeutics, Further Strengthening Immunology Pipeline. Press Release. Dec. 13, 2024.
2. AbbVie. AbbVie Completes Acquisition of Aliada Therapeutics. Press Release. Dec. 11, 2024.
3. AbbVie. AbbVie Completes Acquisition of Cerevel Therapeutics. Press Release. Aug. 1, 2024.
4. AbbVie. AbbVie Acquires Celsius Therapeutics. Press Release. June 27, 2024.
5. AbbVie. AbbVie Completes Acquisition of Landos Biopharma. Press Release. May 23, 2024.

AbbVie's latest acquisition target is Nimble Therapeutics, which will give AbbVie a lead asset, an oral peptide for treating psoriasis.

AbbVie to Boost Immunology Pipeline with Acquisition of Nimble Therapeutics

Part two Colin McKinlay Nutcracker Therapeutic trends future mRNA LNP development.

Talking mRNA–LNP Formulation Trends (Part Two)

One of the exciting things in messenger RNA (mRNA) development is the fact that not everyone involved in this field is necessarily going in the same direction, says Colin McKinlay, PhD, senior director, Chemistry and Delivery Technologies, Nutcracker Therapeutics, in part two of this video interview. “There [are many] different places that mRNA can be applied, and, in some cases, moving past the early stages of this field, one of the things that people are going to have to do is start specializing in different areas,” he says.

“Vaccines is obviously an area that mRNA has really demonstrated itself already, and so, I think making sure that the platform technology doesn't get pigeonholed into only being something that people think of as a vaccine technology is … important for the growth of the field, and moving beyond infectious disease vaccines, or even COVID[-19] vaccines,” McKinlay explains.

“In terms of the LNP [lipid nanoparticle] side of things, where I spend [much] of my time, I think there [are many] lipid nanoparticle technologies that will focus on expression in the liver and are directed towards the liver. One of the things that [many] folks in the field are really focusing on is, how do we actually direct these lipid nanoparticles to other organs in the body so that we can address other types of disease areas? Those are things where there hasn't been … [much] large-scale clinical successes,” McKinlay adds.

LNPs for RNA delivery, in terms of non-viral RNA delivery, have dominated the field in terms of clinical approvals, but McKinlay expresses concern about the industry possibly getting stuck in a kind of local maxima, “where the field does [much] optimization and [may] climb in efficacy, but then … reach a point where there's not too much that [one] can really optimize on anymore. [At Nutcracker Therapeutics], we're able to branch away from traditional LNP type particles, and have particles that are a little bit more unique to the chemistry of … peptoid molecules, and we think that that's pushing us over a little bit away from where the rest of the field is working, so that there's maybe a little bit more room to grow and a little bit more room to improve,” McKinlay states.

Click above for part two of this video interview. Click 

Colin McKinlay, PhD, Senior Director, Chemistry and Delivery Technologies, Nutcracker Therapeutics

Colin McKinlay is the senior director of Chemistry and Delivery Technologies at Nutcracker Therapeutics. He has over 10 years of expertise in nucleic acid delivery through his current position at Nutcracker Therapeutics and his doctoral research at Stanford University with Professors Paul Wender and Robert Waymouth. This research involved preparation of synthetic delivery vehicles through organocatalytic ring-opening polymerization, and their characterization and 

evaluation for the delivery of DNA, RNA, and other biological macromolecules. He holds over five patents and nine peer-reviewed publications on chemical delivery technologies. McKinlay has led the delivery efforts at Nutcracker Therapeutics since 2018, including the establishment of its unique peptoid-based Nutshell delivery platform.

In the second part of a video interview, Colin McKinlay, PhD, senior director, Chemistry and Delivery Technologies, at Nutcracker Therapeutics, discusses trends and the future direction of mRNA–LNP development.