Hemophilia B

Six months after launching with a mega-round of financing, Seaport Therapeutics has raised $225 million more to advance a lead drug candidate that could overcome some limitations of currently available treatments for depression. Seaport’s platform technology, called Glyph, designs oral drugs that leverage the lymphatic system to deliver the medicine in a way that makes more of the active pharmaceutical ingredient available in the body to confer its therapeutic effect. The Boston-based company also says its drugs reduce complications such as elevated liver enzyme levels, a sign of drug toxicity that can limit the use of some molecules. Lead Seaport program SPT-300 is a prodrug, a compound that converts inside the body into the active drug allopregnanolone. This neurosteroid modulates the GABA-A receptor to spark effects on the central nervous system. Seaport is advancing the drug into Phase 2b testing as a potential treatment for major depressive disorder with or without anxious distress. Seaport says the new capital will go toward advancing its pipeline through important clinical milestones. The company also plans to further advance the capabilities of its Glyph technology platform. presented by Health IT Accelerating Claim Processing: Strategies to Shorten the Life of a Claim These strategies and practices can significantly shorten the life cycle of claims, leading to quicker resolutions and improved financial outcomes. By Greenway Health Seaport was spun out of PureTech Health, a company that forms startups around platform technologies. In April, Seaport launched, revealing its science, its lead drug candidate, and $100 million in funding. That Series A round was led by Arch Venture Partners and Sofinnova Investments. General Atlantic led Seaport’s new Series B financing. Other participants in the latest round included T. Rowe Price Associates, Foresite Capital, Invus, Goldman Sachs Alternatives, CPP Investments, and other unnamed new investors. Founding investors Arch and Sofinnova also joined the round, as did Third Rock Ventures and PureTech Health. Seaport is part of a bevy of life science companies that have secured new capital. Here’s a recap of recent biotech financing news: —Radiopharmaceuticals developer Alpha-9 Oncology closed a $175 million Series C round to support its pipeline, including A9-3202, which began a Phase 1 test earlier this year in advanced melanoma. Lightspeed Venture Partners and Ascenta Capital led Alpha-9’s new financing. —AvenCell Therapeutics has $112 million in Series B funding to continue Phase 1/2 testing of AVC-101, a CAR T-therapy that can be switched “on” or “off” to address safety and efficacy challenges of currently available cell therapies, which are always “on.” AvenCell was formed in 2021 by Intellia Therapeutics and Cellex Cell Professionals. The company’s latest financing was led by Novo Holdings. Sponsored Post The Future of Hospitals and Pharma Companies Will Depend on Strength of Healthcare Analytics Insights PurpleLab® stands out from others in this sector by providing its data analytics services to several different groups of users across healthcare and pharma companies. By Stephanie Baum —Tolerance Bio launched with $17.2 million to support development of allogeneic stem cell-based cell therapies. These therapies are intended to preserve the thymus, the organ that trains T cells to defend against threats and preserve autoimmunity. The Philadelphia-based biotech’s seed financing, led by Columbus Venture Partners, will be used to advance toward the clinic with potential therapies for immune diseases. —March Biosciences, spun out of the Center for Cell and Gene Therapy, unveiled $28.4 million in Series A financing. Lead program MB-105 is a CAR T-therapy designed to target CD5, a protein expressed on cancerous T cells. A Phase 1 test is underway enrolling patients with refractory T cell lymphoma and leukemia. Mission BioCapital and 4BIO Capital led March Bio’s financing. —Be Biopharma closed $82 million in financing as the cell therapy developer moves into the clinic with BE-101, a potential treatment for hemophilia B. The biotech’s therapies are made by engineering B cells to produce therapeutic proteins. Be Bio last raised money in 2022, a Series B financing that totaled $130 million. —Terray Therapeutics, a biotech that uses generative AI to discover and develop small molecule drugs, unveiled a $120 million Series B financing. Los Angeles-based Terray will use the capital to advance internal programs into clinical trials for indications that were not disclosed. The financing was led by new investor Bedford Ridge Capital and earlier investor NVenture, the investment arm of NVIDIA. Here’s more about Terray and its technology platform, tNova, which it unveiled in 2022. —Genetic medicines have been difficult to deliver to the kidney. Judo Bio is developing oligonucleotide drugs that target a particular receptor to reach the organ, and the Cambridge-based startup unveiled $100 million in seed and Series A financing to advance its research toward the clinic. The Series A round was co-led by Atlas Venture, TCG, and Droia Venture. —Purespring Therapeutics, a developer of gene therapies for kidney disease, raised £80 million (about $105 million). The London-based company’s therapies target the podocyte, a special cell implicated in 60% of renal diseases. Proceeds will be used to advance lead program PS-002 into a Phase 1/2 clinical trial for immunoglobulin A nephropathy, a rare disease currently treated with chronically administered therapies. Sofinnova Partners led Purespring’s Series B financing. —Artificial intelligence biotech company Basecamp Research revealed a $60 million Series B financing to scale its collection of data and strengthen its AI capabilities. The London-based startup also announced a multi-year collaboration with David Liu of the Broad Institute of MIT and Harvard. Basecamp said this partnership will research new approaches to programmable genetic medicines with potential applications in a range of diseases. —City Therapeutics launched with $135 million to develop next-generation RNA interference (RNAi) drugs. The company says its engineering platform can design therapies in smaller sizes that have novel cellular mechanisms and improved potency, efficiency, and specificity. The Cambridge-based biotech, co-founded by scientists involved in the first generation of RNAi therapies, expects to enter the clinic by the end of 2025. Arch Venture Partners led City’s Series A round. —Resolution Therapeutics raised £63.5 million to advance to Phase 1/2 testing with RTX001, an engineered autologous macrophage cell therapy that could offer anti-fibrotic and anti-inflammatory effects in patients with end-stage liver disease. The company, which maintains operations in London and Edinburgh, Scotland, said this trial will be conducted in the United Kingdom and Spain. Syncona led the Series B round, which will also support research in other inflammatory fibrotic conditions such as graft-versus-host disease and lung fibrosis. —Triveni Bio raised $152 million to support a pipeline whose most advanced program, TRIV-573, is a bispecific antibody that blocks two targets associated with atopic dermatitis. The Watertown, Massachusetts-based biotech aims to file an investigational new drug application for this program in the first quarter of 2025. Triveni launched last year supported by a $92 million Series A financing. Goldman Sachs Alternatives led the company’s Series B. —Kailera Therapeutics launched with $400 million to support its GLP-1 and GIP receptor agonist drugs for metabolic indications, including obesity. Kailera’s four programs were licensed from China-based Jiangsu Hengrui Pharmaceuticals. The Series A financing was co-led by Atlas Venture, Bain Capital Life Sciences, and RTW Investments. —Radiopharmaceutical developer Aktis Oncology unveiled $175 million in financing to support development of a pipeline led by AKY-1189, an alpha radiotherapy that targets Nectin-4, a tumor-associated antigen found in a range of solid tumors. The Series B round was led by RA Capital Management and co-led by RTW Investments and Janus Henderson Investors. Boston-based Aktis emerged from stealth in 2021 backed by $72 million in Series A financing. —Genespire raised €46.6 million (about $52 million) to advance to Phase 1/2 testing with GENE202, an in vivo gene therapy in development for methylmalonic acidemia, a rare inherited disorder that impairs the metabolism of certain amino acids and fats. The Milan, Italy-based startup’s Series B round was co-led by Sofinnova Partners, XGEN Venture, and CDP Venture Capital. —Vaccines developer Vicebio unveiled $100 million in financing as it begins a Phase 1 test of VXB-241, a bivalent vaccine designed to address both respiratory syncytial virus (RSV) )and human metapneumovirus (hMPV). Data are expected in mid-2025. The next Vicebio program is VXB-251, a trivalent vaccine for RSV, hMPV, and parainfluenza virus 3. The London-based company’s Series B round was led by TCGX. —GC Therapeutics launched with $65 million in financing. The biotech’s next-generation cell therapies come from a platform technology called TFome, which programs induced pluripotent stem cells. GC’s initial therapeutic areas of focus include gastrointestinal, neurological, and immunological diseases. Cormorant Asset Management led Cambridge, Massachusetts-based biotech’s Series A financing. —Neuroscience startup Nura Bio extended its Series A financing by $68 million to proceed to Phase 2 testing with an oral small molecule designed to block SARM1, an enzyme associated with axonal damage. It faces potential competition from Eli Lilly, which is in early clinical development with a drug that goes after the same target. Nura Bio’s founding investor The Column Group led the startup’s new financing. —F2G raised $100 million for olorofim, which is in late-stage development to treat aspergillosis and other invasive fungal infections. Olorofim is from a novel class of antifungal agents that work by inhibiting the pyrimidine synthesis pathway. The cash will enable the Manchester, U.K.-based company to complete late-stage clinical development, submit regulatory applications, and prepare for U.S. commercialization of the drug. The new financing was led by new investor AMR Action Fund and co-led by ICG. —T cell engagers first reached patients as cancer treatments. Candid Therapeutics aims to bring such drugs to autoimmune disorders, and its efforts are backed by more than $370 million in financing. Candid’s drug candidates came from acquiring and merging two biotechs, Vignette Bio and TRC 2004. The Candid pipeline includes two T cell engagers for cancer now being repositioned for development in autoimmune diseases. These therapies are designed to selectively deplete B cells that drive disease. —Startup creator Flagship Pioneering unveiled its newest company, Mirai Bio. Mirai’s platform technology is applied to the development of genetic medicines for the company’s partners working across a range of therapeutic areas and therapeutic modalities. It says its platform technology unlocks delivery of a drug to any tissue and cell type, optimize design of the drug cargo, and facilitates manufacturing. Like other startups that come from Flagship, Mirai is backed by the customary $50 million in financing. —Superluminal Medicines, a company developing small molecule drugs that target elusive G protein-coupled receptors (GPCRs), closed $120 million in financing. The Boston-based startup says its drugs target membrane receptors that mediate cell signaling and response processes fundamental to human physiology. These small molecule come from a generative biology and chemistry platform technology. The new capital will be used to advance toward the clinic in undisclosed indications. RA Capital Management led the Series A financing. —Riding strong investor interest in metabolic disorder drugs, OrsoBio closed $67 million to advance a pipeline that includes programs for type 2 diabetes, obesity, and the fatty liver disease MASH. The Menlo Park, California-based startup’s drugs address pathways associated with energy metabolism. The Series B round, led by Ascenta Capital and Woodline Partners, comes less than a year after the company unveiled $60 million in Series A financing. Photo by Flickr user Massachusetts Office of Travel & Tourism via a Creative Commons license

Wikimedia Commons, Abhiram Juvvadi Pfizer CEO Albert Bourla is in a tough spot as activist investor Starboard Value continues to call for a change in the company’s leadership. However, analysts are supportive of the embattled executive. After taking a $1 billion stake in Pfizer earlier this month, activist investor Starboard Value has put a target on the back of CEO Albert Bourla. At an investor summit on Tuesday, Starboard laid out its case against Bourla and senior management in a presentation that included 74 slides on how the once-mighty company “has dramatically underperformed peers and the market since 2019.” While Starboard CEO Jeff Smith told CNBC this week that Pfizer’s management did a “great job” during the COVID-19 pandemic, he said “the growth they’re producing from their investments is worst in class” and that the company’s board needs to hold management “accountable” for achieving sufficient revenue returns on R&D and M&A. “In similar situations where a company has underperformed like this, you may see a board make a change at CEO,” Smith said. “It wouldn’t be unheard of, it wouldn’t be strange, it could make sense.” No one can deny that Bourla is in a tough spot. In 2022, Pfizer was the biopharma industry’s top revenue generator, bringing in more than $100 billion. However, the company’s revenue nosedived more than 40% to $58.5 billion in 2023 as sales of its COVID-19 products Comirnaty and Paxlovid cratered. Making matters worse, Pfizer is “facing various challenges or uncertainties with their nine largest revenue generators,” Guggenheim Securities analysts wrote in a note to investors, pointing out that several medications that are expected to lose patent exclusivity over the next four years. However, analysts have rallied around Bourla in response to the Starboard challenge. Support From Analysts BMO Capital Markets analysts in a note to investors said their view “remains that an activist could do little to change the story near term,” and while a senior management change may “feel good in the moment,” it “is also likely to do little to change the narrative.” BMO’s Evan Seigerman opined in a separate note that “this seems like the ideal story” for an activist investor like Starboard. However, he said solving the company’s “many woes” will likely take time instead of a “quick fix.” Likewise, Guggenheim analysts say Starboard’s recent $1 billion stake in Pfizer “has spurred discussions, but we view their potential influence as limited given the company’s scale.” Indeed, Starboard’s stake represents just 0.6% ownership of the company. Seigerman acknowledged that Pfizer is faced with a very challenging business environment but contends the activist assault on Bourla seems short-sighted. “While placing blame on one person may seem easy, rarely will it result in a quick turnaround,” he wrote to investors. Bourla has taken steps to turn things around at Pfizer through cost-cutting programs and pipeline investments to help return the company to growth, including an RSV vaccine and a potential approval in obesity. Guggenheim credits Pfizer’s current management team for its “aggressive” cost-saving efforts, with $4 billion being removed from the company’s operating expenses by the end of 2024. “The first step in optimizing their manufacturing efforts is expected to deliver another $1.5B in savings over the next three years, with the CFO already suggesting there are more steps they can take on that front, all leaving investors to wonder what additional steps can be taken from an activist perspective,” according to Guggenheim. Despite Starboard’s criticism of Bourla, the activist investor has provided no recommendations on how Pfizer’s management could improve the company’s business performance. While BMO analysts said they “understand why an activist might think that Pfizer is the right story to get involved in,” their assessment is that “management appears to actually be undertaking many of the corrective actions to right the ship.” “[C]hange takes time,” they continued, “and replacing the CEO (while satisfying to some) is unlikely to fix the story and immediately re-rate shares.” Q3 Outlook for Pfizer BMO analysts last week said it’s been a “tumultuous few weeks” heading into Q3 earnings season for Pfizer. In addition to this month’s Starboard challenge, Pfizer pulled sickle cell drug Oxbryta off the global market in late September, while on the positive side of the ledger the company earned the FDA’s approval of Hympavzi for hemophilia A and B earlier this month. Pfizer is slated to report its third-quarter results on Oct. 29. Jefferies analysts said they believe the company will beat Wall Street Q3 expectations “on both topline and bottom line.” Guggenheim analysts also said they see Pfizer having “significant upside potential” based on increased demand for its COVID-19 products in Q3. The group forecast Q3 sales of $16.95 billion and earnings per share (EPS) of $0.78, significantly above the current FactSet consensus sales of $14.74 billion and EPS of $0.59, respectively. Guggenheim analysts’ projections also suggest “favorable trends” for full year 2024 and 2025, with higher estimates than current consensus. “Despite market skepticism about the long-term demand for COVID-19 products, we believe that concerns are somewhat misplaced, especially for Paxlovid, which we expect to maintain demand even if vaccination uptake slows,” the analysts wrote. “Paxlovid’s high margin and significant share of commercial insurance coverage will continue to enhance Pfizer’s profitability.” BMO was similarly positive, with an overall message to investors to sit tight for now. “Staying the course and focus is usually the right answer in the long run.” The analysts pointed out that Eli Lilly “did not undergo a management change despite a challenging decade, only recently becoming the darling of pharma.”

Eli Lilly’s Alzheimer’s drug Kisunla has secured regulatory approval in the UK, but has been rejected for widespread use in NHS. Credit: Michael Vi via Shutterstock. Eli Lilly’s Kisunla (donanemab) will not yet be made available to Alzheimer’s patients in the UK after the National Institute for Health and Care Excellence (NICE) said it doesn’t demonstrate good value for UK’s NHS. On 23 October, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granted approval to the therapy for the treatment of mild cognitive impairment or mild dementia due to Alzheimer’s disease in some adults. This follows on from the US Food and Drug Administration (FDA)’s green light in July 2024 . However, NICE, which acts as the UK’s price watchdog, said that although the monthly injection slows disease progression by four to seven months, as demonstrated in the Phase III TRAILBLAZER-ALZ 2 (NCT04437511) study, there are “significant uncertainties about how much benefit donanemab provides, and how long this lasts for after stopping treatment”. It also said that more evidence is needed on the clinical and cost-effectiveness of Kisunla. A NICE committee also explained that one-third of patients in the trial had signs of brain swelling or brain bleeding, as determined by amyloid-related imagining abnormalities (ARIAs). Similar concerns were raised in the FDA’s June 2024 advisory committee convened to discuss the drug’s approval. Patients in Lilly’s trial were closely monitored due to this, with MRIs done at four, 12, 24, 52, and 76 weeks in addition to allowing for unscheduled MRIs at investigator discretion. This frequent monitoring can rack up increased costs for payers , as well as bringing up accessibility issues. In the announcement accompanying the decision, Helen Knight, director of medicines evaluation at NICE said: “The cost-effectiveness estimate for donanemab is 5 to 6 times above what NICE normally considers an acceptable use of NHS resources.” See Also: TreeFrog and University of Pittsburgh probe TLS role in immuno-oncology Be Bio raises $82m to advance haemophilia B gene therapy to Phase I/II trial In the US, Kisunla has a price tag $695.65 per vial. This means that the cost of treatment for a six and 12 month-plan would amount to $12,522 and $32,000, respectively. Kisunla isn’t the only Alzheimer’s therapy on the market. Eisai and Biogen ’s Leqembi (lecanemab) secured UK approval in August 2024, but NICE published a separate draft guidance that said it leans against reimbursing the drug. Leqembi has an annual cost of $26,500 per year in the US, and its infusions are administered every two weeks for a minimum of 18 months. In July, Leqembi faced rejection from the European Medicines Agency (EMA) , while the agency is continuing to review Kisunla. Kisunla and Leqembi are monoclonal antibodies that work by targeting and clearing amyloid beta plaques from the brain, aiming to slow early cognitive decline by reducing toxic buildup linked to the disease. It is estimated that around 70,000 adults in England would have been eligible for treatment with Kisunla, according to NICE.