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Rakuten Medical, a US-based biotechnology company specializing in cell targeting therapies, announced on Aug. 10, 2023 that it has signed an exclusive licensing and commercialization agreement with Hikma Pharmaceuticals (Hikma), a UK-based pharmaceutical company that serves the Middle East and North Africa (MENA) region as well as Europe.

Under the agreement, Hikma gains an exclusive license to commercialize products in Rakuten Medical’s oncology pipeline to all of Hikma’s MENA markets. Rakuten Medical’s oncology pipeline was developed using its proprietary photoimmunotherapy technology platform, Alluminox. These therapies are not currently approved in MENA for investigational or commercial use. Outside of Japan, they are investigational treatments, according to a company press release.

“We are already developing Alluminox treatment in several countries and regions, including the United States, Japan, Taiwan, and India, and this agreement further accelerates our global expansion. With Hikma’s strong regional footprint and medical expertise, we expect the Alluminox platform to make significant progress in MENA,” said Mickey Mikitani, co-CEO of Rakuten Medical, in the company press release.

“This agreement allows us to work with an excellent global partner to strengthen our growing portfolio in oncology and biotechnology. Most importantly, this allows us to bring a potentially transformative technology to cancer patients in MENA, helping to put better health within reach, everyday,” said Mazen Darwazah, executive vice-chairman and president of MENA, Hikma, in the release.

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Under the exclusive agreement, Hikma will commercialize products in Rakuten Medical’s pipeline for cancer treatment in the Middle East and North Africa region.

Rakuten Medical Signs Exclusive Licensing Agreement with Hikma for Cancer Treatments in MENA

, a biotech start-up focused on regenerative medicines. In this candid discussion with Grant Playter, associate editor, Luis discusses his company’s regenerative bone therapy, how it evolved from his experiences in Iraq, how Theradaptive secured funding in a lukewarm venture capital market, and more.

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Luis Alvarez, PhD, founder of Theradaptive, candidly discusses how his regenerative therapy start-up evolved and secured start-up funding.

Unpacking Regenerative Medicines and Start-Up Funding with Luis Alvarez

The United Kingdom Bioindustry Association recently released new data surrounding UK biotech funding in the second quarter. According to the report, venture capital investments rose from £258 million in the first quarter to £338 million in the second, whereas public funding rose from £37 million to £44 million. This represents an overall quarter-over-quarter uptick of 29%, putting it en route to approximately match the £1.21 billion raised over the course of 2022. However, both years totals are set to lag significantly behind 2021, which saw approximately £2.52 billion in total financing.

According to findings from the report, the average size of venture capital investments in the second quarter was £13 million, the same as the first. The report also noted that there was a “healthy spread” of funding between early- and late-stage companies.

The report summary stated that public markets remain challenging, with no new market launches or lackluster follow-on funding, a trend noted as in-line with international trends; no UK biotech company raised money via initial public offering (IPO) in this quarter, mirroring the first quarter. This is in stark contrast to 2021, which saw approximately $1.3 billion raised via IPO over the course of the year. However, due to the positive momentum of the quarter and overall stability in attracting capital, as well as upcoming UK government policy initiatives set to bolster funding, the overall findings were deemed positive by the members of the UK Bioindustry Association.

“Amidst the current challenges facing the UK’s biotech sector, we are encouraged by the 29% growth in investment quarter-on-quarter, and the positive policy progress from UK Government that bodes well for a rapid rebound.” said Steve Bates, CEO, Bioindustry Association, in the report.

The summarized findings of the report can be read 

A report from the UK Bioindustry Association report indicated that biotech venture and public financing rose from £295 million in the first quarter to £382 million in the second quarter.

United Kingdom Biotech Report Finds 29% Uptick in Funding

Cytomos, a Scottish life science company focused on developing new approaches to analyzing cells, announced on Aug. 9, 2023, that it had secured £4 million (approximately $5 million) to scale-up market testing of its technology platform Cytomos Dielectric Spectroscopy (CDS). According to a company press release, CDS is designed to provide high-speed, scalable, and low-cost cell analysis relative to existing systems.

The investment round was led by existing investors Archangels, as well as participation from Old College Capital, Scottish Enterprise, and new investor British Business Bank. According to the release, the new investment will allow Cytomos to significantly advance the development of CDS and expand its team. The company is targeting platform commercialization in 2024.

“On the back of a very successful year for Cytomos, this new funding will now allow us to scale up our engagement with industry partners,” said David Rigterink, CEO, Cytomos, in the release. “We truly believe that, through our CDS technology, we offer a powerful platform which will help the scientific community bring novel therapies to market faster and radically reduce costs by making better informed, game-changing decisions a lot earlier.”

“As a highly innovative, Scottish early-stage life science company with global horizons, Cytomos is an excellent fit for our investment portfolio,” said Sarah Hardy, director and head of new investments, Archangels, in the release.  “Its ambitious vision to improve real time cell analysis in formats to better suit the end users will revolutionize the development and commercialisation of products in the bioprocessing and cell and gene therapies spaces.  We’re looking forward to working with David and the team as they drive the business forward, making smarter, faster and more accurate cell analysis finally more accessible.”

Cytomos will use the £4 million in funding to develop its dielectric spectroscopy technology.

Cytomos Secures £4 Million to Develop Spectroscopy Platform

Astellas Pharma and Poseida Therapeutics announced on Aug. 10, 2023 a strategic investment to further support the development of cell therapies for cancer . According to a press release, Astellas will be investing a total of $50 million and a one-time $25 million payment for a right of exclusive negotiation and first refusal to license one of Poseida’s clinical-stage programs, P-MUC1C-ALLO1, which is an allogeneic chimeric antigen T cell (CAR-T) cell therapy in development for multiple solid tumor indications.

Additionally, Poseida has approved Astellas as a board observer seat, which gives Astellas the right to attend Poseida’s scientific advisory board meetings and certain notice rights related to any potential change of control over Poseida.

“By leveraging our extensive expertise, experience in cancer biology, and unique technologies, we are focused on reinvigorating the immune system’s ability to discover, disarm, and destroy cancers in more patients,” said Adam Pearson, chief strategy officer, Astellas, in a press release. “We believe that this investment fits strategically with our long-term vision of expanding our capability in immuno-oncology and will ultimately lead to the development of new therapeutics for patients in need of cancer immunotherapy.”

"This investment further validates the potential of our proprietary genetic engineering technology platform and cell therapy approach, and we look forward to working with Astellas to advance our shared vision and explore future opportunities for collaboration to further unlock that potential," said Mark Gergen, Poseida's chief executive officer, in a press release.

Astellas and Poseida Therapeutics Plan to Invest in Redefining Cancer Cell Therapy 

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On Aug. 11, 2023, Novartis announced the completion of its acquisition of Chinook Therapeutics, a US-based clinical-stage biopharmaceutical company focused on developing and producing precision medicines for kidney diseases. The transaction included a $3.2 billion upfront payment, with a total deal value of up to $3.5 billion. Stockholders in Chinook are expected to receive up to $44.00 per Chinook share through a contingent value right, which represents a potential added $300 million in aggregate contingent consideration.

In a company press release, Novartis identified two key late-stage assets in development at Chinook: atrasentan and zigakibart (BION-1301). Both assets are being developed to treat Immunoglobulin A nephropathy (IgAN). Atrasentan, an oral endothelin A receptor antagonist, is in Phase III development for IgAN and has shown notable decreases in proteinuria. It is also in early development for other rare kidney diseases. Zigakibart (BION-1301), a subcutaneously administered anti-a proliferation-inducing ligand (APRIL) monoclonal antibody, entered Phase III development for IgAN in July 2023.

“We are excited to complete this important transaction and look forward to leveraging our combined resources and expertise to further advance the development of these promising treatments for the benefit of patients with rare, severe chronic kidney diseases,” said Vas Narasimhan, CEO of Novartis, in the press release. “We welcome the Chinook team to Novartis as we expand our renal portfolio and continue our journey to reimagine medicine.”

Novartis has completed its acquisition of biopharma company Chinook Therapeutics.  

Novartis Finalizes Chinook Therapeutics Acquisition in Deal Worth Up to $3.5 Billion

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On Aug. 4, 2023, FDA released a guidance document, 

Recommended Acceptable Intake Limits for Nitrosamine Drug Substance-Related Impurities (NDSRIs)

. The guidance gives both applicants and manufacturers of drugs recommendations for estimating the mutagenic and carcinogenic potential of NDSRIs that could be in drug products and gives acceptable intake (AI) limits for NDSRIs.

NDSRIs are a subcategory of nitrosamine impurities that share structural similarity to the API in drug products; NDSRIs often lack the compound-specific mutagenicity and carcinogenicity data necessary for informed safety assessments. FDA wrote in the guidance document that this guidance “provides a recommended methodology for AI determination that uses structural features of NDSRIs to generate a predicted carcinogenic potency categorization and corresponding recommended AI limit that manufacturers and applicants can apply, in the absence of other FDA-recommended AI limits, in their evaluation of potential impurities in their drug products.”

FDA wrote that it will provide updated NDSRI-specific information on 

 to be viewed in tandem with the guidance. The updated information will include suggested AI limits for specific NDSRIs based on “compound-data or read-across analysis from a surrogate”, suggested interim AI limits for NDSRIs, suggested testing methods for corroborative testing of specific NDSRIs, and suggested safety testing methods for NDSRIs. It will also include suggested AI limits for specific NDSRIs based on “predicted carcinogenicity potency categorization listed by APIs that hypothetically could be at risk of forming such NDSRIs.” 

The guidance can help both applicants and manufacturers limit the mutagenic and carcinogenic potential of NDSRIs.

FDA Releases Guidance on Intake Limits for Nitrosamine Drug Substance-Related Impurities

On Aug. 4, 2023, FDA approved zuranolone (Zurzuvae), an oral medication to treat postpartum depression (PPD) in adults from Sage Therapeutics, a US-based biopharmaceutical company. According to an agency press release, zuranolone is the first oral treatment approved for PPD.

The approval was based on two studies that showed zuranolone’s effectiveness as a treatment for PPD. Patients who took zuranolone showed noticeably more improvement compared to those in the placebo groups, and the effect remained four weeks after the last dose of zuranolone.

PPD is a major depressive episode that usually occurs after childbirth, although it can also start in the later stages of pregnancy. As with other forms of depression, PPD can cause sadness, lethargy, and loss of interest in previously enjoyable activities. Symptoms include feelings of sadness or inadequacy, loss of energy, cognitive impairment, and suicidal ideation. Until this approval, PPD treatment was only available via intravenous injection given by a health care provider in a health care facility. FDA granted the application for Zurzuvae priority review and fast track designation.

“Postpartum depression is a serious and potentially life-threatening condition in which women experience sadness, guilt, worthlessness—even, in severe cases, thoughts of harming themselves or their child. And, because postpartum depression can disrupt the maternal-infant bond, it can also have consequences for the child’s physical and emotional development,” said Tiffany R. Farchione, director of the Division of Psychiatry in the FDA’s Center for Drug Evaluation and Research, in FDA’s press release. “Having access to an oral medication will be a beneficial option for many of these women coping with extreme, and sometimes life-threatening, feelings.”

The new drug could be a powerful tool for patients suffering depressive symptoms after birth.

FDA Approves First Oral Treatment for Postpartum Depression

AGC Biologics announced on Aug. 9, 2023 the completed expansion of its manufacturing space at the Milan Cell & Gene Center of Excellence production. The expansion offers the addition of 1 x 50L, 2 x 200L, and 2x 1000L suspension single-use bioreactors and allows for up to four iCellis500 adherent single-use bioreactors to meet demands for upcoming good manufacturing practice (GMP) viral vector manufacturing projects, according to the company press release.

Additionally, the project included the build-out of more process development lab space, a new automated filling line, and the addition of a new fully dedicated 1000-square-meter warehouse space.

“This is another important milestone for our site and for the AGC Biologics global Cell and Gene network as we prepare to serve more developers,” said Luca Alberici, General Manager, AGC Biologics Milan, in a press release. “Thanks to this expansion we have the flexibility to support companies from clinical to commercial stages for 

 gene therapy applications and can manufacture adeno-associated and lentiviral vectors from medium 50L scales to as large as 2000L. When you combine this with our development, analytical and fill/finish services, this site can offer cell and gene developers any type of support they need throughout their product’s lifecycle.”

The site was acquired by AGC Biologics in July 2020, and the Milan location is the first cell and gene therapy site approved in Europe for GMP manufacturing of clinical and commercial supplies, according to the press release. Further, the site has brought multiple products from the pre-clinical stage through commercial phases, such as developing a custom plug-and-play systems for vector manufacturing.

The two cell and gene facilities from AGC Biologics, in Milan and Longmont, US, receive support from the Biologics Heidelberg site, which produces proprietary plasmid starting materials for advanced therapies in high-quality and GMP grades, according to the press release.

The site was acquired by AGC Biologics in July 2020, and the Milan location is the first cell and gene therapy site approved in Europe for GMP manufacturing of clinical and commercial supplies.

AGC Biologics Completes Expansion at CGT Manufacturing Site in Milan

Regeneron Pharmaceuticals and Decibel Therapeutics announced a definitive agreement on August 9, 2023, for the acquisition of Decibel by Regeneron at $4 per share of Decibel common stock payable in cash at closing.

“We at Decibel are deeply committed to discovering and advancing innovative new therapies with the potential to be transformative for people with severe forms of hearing loss. We have built a pipeline of gene therapy product candidates for the ear that we believe have such potential. After full consideration, the Decibel Board has determined that this transaction is the best way to maximize shareholder value and ultimately benefit patients,” said Laurence E. Reid, PhD, president and chief executive officer of Decibel, in a press release. “We have collaborated with our colleagues at Regeneron for the past six years and have huge respect for their research and development capabilities. We have full confidence that with Regeneron’s expertise and resources the Decibel pipeline can be optimally developed, and our team is committed to enabling that long-term success.”

The initial collaboration between the two companies began in 2017 with an extension announced in 2021, and they are now developing three gene therapy programs targeting different forms of congenital, monogenic hearing loss, according to the press release. Decibel’s lead investigational candidate, DB-OTO, is currently in the global Phase I/II CHORDTM clinical trial, which is an investigational cell-selective, adeno-associated virus gene therapy. The objective of the study is to provide physiological hearing to individuals with congenital hearing loss caused by mutations of the otoferlin gene.

“We are delighted to announce the planned acquisition of Decibel, who have been long-standing collaborators, notable for their deep scientific knowledge and commitment to people with hearing loss,” said George D. Yancopoulos, MD, PhD, Board co-Chair, chief scientific officer and president of Regeneron, in a press release. “DB-OTO, our shared lead investigational gene therapy, will soon reach patients in its first clinical trial, offering new promise to children with this rare form of congenital hearing loss, as well as potential proof-of-concept for future gene therapies addressing more common forms of genetic hearing loss. We believe that Decibel’s assets and specialized team will further strengthen our genetic medicines portfolio, enabling Regeneron to accelerate the development of innovative genetic therapies and a rich pipeline of hearing loss treatments.”

The transaction is expected to close in the third quarter of 2023, according to the press release.

The initial collaboration between the two companies began in 2017 with an extension announced in 2021.

Featured Content

Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Behind the Headlines Episode 28

Ask the Expert

Drug Digest December 2025

A “Plan, Prevent, Prove” Approach