PoZdroweek 19/2025: FDA blocks sale of Valneva's Ixchiq vaccine against the chikungunya virus

Valneva has had a difficult year this year as it grapples with changing decisions regarding its chikungunya vaccine. Nevertheless, the FDA's negative decision to block its sale in the US won't derail the French company's business, as it is developing drug candidates for Lyme disease, Zika, and Shigella. Eli Lilly believes that orforgliprone is ready for registration, as evidenced by the results of the ATTAIN-2 trial. AbbVie acquired the rights to the investigational drug candidate bretisilocin from Gilgamesh Pharmaceuticals for up to $1.2 billion. A significant relief for researchers in the MASH segment will be a change in the method for measuring liver stiffness, supported by the FDA. This involves moving away from invasive biopsy to non-invasive elastography. Biogen and Eisai are pleased that the regulator has allowed a change in the dosage formula for Leqembi in the treatment of Alzheimer's disease. Instead of infusions in medical facilities, patients will be able to switch to weekly injections in the comfort of their home.

The domestic WIGmed index lost a symbolic 0.3%, as it did last week. Foreign healthcare stocks fared slightly worse. The European MSCI Europe Health Care (SPYH) fell 1.1% . The global MSCI World Health Care (XDWH) fell by a similar amount, falling 1.2% .

Looking back from the beginning of this year, the Polish index continued to show strength, remaining marginally positive (+1.6%). The rest were in the red, with the XDWH being a particular case, recording a loss of -10.5%.

Source: TradingView

Among global stocks in the top 20 of the MSCI Global Health Care Index, Eli Lilly performed best (+2.7%). UnitedHealth Group (+0.8%) and Abbott (+0.1%) gained in the tenths of a percent. Pfizer (-4.3%), Merck (-3.7%), and Sanofi (-3.6%) saw the biggest losses.

Source: own study based on stooq.pl

In Poland, Genomtec (+8.6%) performed best, followed by Scope Fluidics (+2.3%) and Synektik (+1.5%). In contrast, the biggest losses were recorded by Enel-Med (-4.9%), PolTREG (-3.3%), and Molecure (-2.7%).

Source: own study based on stooq.pl

The FDA's decision to order the immediate suspension of all deliveries and sales of the Ixchiq vaccine, which is intended to protect against the chikungunya virus, dealt a blow to shares of France's Valneva . The company's shares fell 19% on Monday.

The US regulator cited additional reports of serious adverse events. Four new cases consistent with chikungunya-like illness were reported among vaccine recipients outside the US. Three cases involved people aged 70 to 82, including one involving the hospitalization of an 82-year-old patient who was discharged after two days. The fourth case involved a 55-year-old person.

It's important to note that adverse events mostly affect seniors, not the entire age group. Nevertheless, the FDA's decision effectively restricts access to Ixchiq to those in need, regardless of age.

In its statement, the FDA concluded that a benefit-risk analysis conducted by the Center for Biologics Evaluation and Research (CBER) indicates that the vaccine's risks outweigh its benefits in most scenarios. CBER concluded that the vaccine is not safe and that continued use would pose a health risk.

Valneva maintained that all cases cited by the FDA had symptoms consistent with those documented during clinical trials and post-marketing surveillance, particularly among elderly patients.

Valneva's troubles began in earnest this spring. In April, French authorities suspended the use of the Ixchiq vaccine in adults over 65 years of age following three adverse events, one of which resulted in a death. The European regulatory agency (EMA) suspended its use after 17 adverse events and two deaths, a decision joined by the FDA. However, the EMA and FDA soon lifted their decisions in June. The FDA then reversed its position again in late August.

This raises doubts about Ixchiq's future sales. Even if everything goes according to plan for the Saint-Herblain, France-based company, the confusion we're witnessing will still affect how doctors perceive Ixchiq.

The company is successfully marketing Ixiaro, a vaccine against Japanese encephalitis, and Dukoral, a vaccine to prevent diarrhea caused by Vibrio cholera and/or Escherichia coli bacteria. According to data released on August 12, 2025, during the publication of quarterly results, both drugs accounted for 80% of total sales in the first half of this year, which amounted to €91 million. Ixchiq, in contrast, generated €7.5 million in sales, though its 688% growth rate compared to the first half of 2024 was certainly impressive.

The FDA's decision won't shake Valneva's business. At most, it will stall development somewhat. The company has three promising drug candidates in its research pipeline. Starting with the least advanced substances, the company is currently testing VLA 1601 (Phase I), a vaccine against the Zika virus . S4V2, a drug against Shigella bacteria, responsible for bacillary dysentery, commonly known as "dirty hands disease," is in Phase II clinical trials, the results of which will be known later this year.

And the real hit could be VLA115, developed with Pfizer, which targets Lyme disease (the potential of this drug is presented in the infographic below). VLA115 is in the Phase III VALOR clinical trial, the results of which will be published in late 2025.

And the €180-190 million that Valneva's management expects this year looks modest compared to the potential of over $1 billion for the commercialization of a drug against Lyme borreliosis (tick-borne spirochetosis).

Source: Valneva

Eli Lilly announced that once-daily oral orforgliprone led to significant weight loss after 72 weeks in obese/overweight adults with type 2 diabetes in the ATTAIN-2 study, allowing the initiation of regulatory proceedings to approve the drug candidate for marketing.

The ATTAIN-2 study involved more than 1,600 participants, randomly assigned to receive one of three doses of orforgliprone or a placebo. The Indianapolis, Indiana-based company said all doses met baseline requirements, with the highest dose of 36 mg reducing weight by an average of 10.5% compared with 2.2% for placebo.

In addition to meeting the primary objective of weight loss, orforgliprone at all doses also met key secondary objectives of the study. A1C levels decreased by an average of 1.8%, compared with 0.1% in the placebo group. The drug also demonstrated clinically meaningful benefits on key cardiovascular risk factors, including non-HDL cholesterol, systolic blood pressure, and triglycerides.

Kenneth Custer, president of Lilly Cardiometabolic Health, noted that the population studied tends to have greater difficulty losing weight, and he called the data from ATTAIN-2 truly encouraging.

Eli Lilly's stock price rose 5.9% on Tuesday in response to data from its latest Phase III clinical trial. This contrasts with investor reaction to the ATTAIN-1 trial (in non-diabetic patients) published earlier this month, which led to a 14% decline in the world's largest biopharmaceutical company's shares. The company reported then that a 36 mg dose of orforgliprone resulted in an 11.5% weight loss, but that wasn't what seemed to interest investors most. They also noted that the discontinuation rate due to adverse events in the ATTAIN-1 trial ranged from 5% to 10% (depending on the dose administered), while Novo Nordisk's competing version of oral semaglutide had a 7% drop.

In the ATTAIN-2 trial, discontinuation rates due to adverse events ranged from 6.1% to 10.6% (compared to 4.6% in the placebo cohort).

Tuesday (26/08/2025)

NanoGroup received a PLN 17 million capital injection through a share issue in exchange for cash contributions. Fourteen investors participated in the transaction, purchasing shares at PLN 2.50 each.

The company plans to use the acquired funds for purposes related to the expansion of the capital group and to finance projects carried out by portfolio companies.

Wednesday (27/08/2025)

- Pure Biologics allocated a total of 4,081,572 P1 series shares to investors for PLN 4.08 million . This occurred following the decision of the company's general meeting of June 30 of this year to increase the share capital through the issue of new shares.

Synthaverse announced that the investor acquired 5 million series W shares at an issue price of PLN 4.60 each, totaling PLN 23 million . The funds will be used to implement strategic investment projects.

Thursday (28/08/2025)

The government has adopted a preliminary draft budget for 2026, which stipulates that healthcare spending will amount to PLN 247.8 billion (6.8% of GDP), representing a PLN 25 billion increase compared to the last budget. Higher funding for the in vitro fertilization program is also planned for the coming year – PLN 600 million (PLN 100 million more than in 2025).

Neuca reported PLN 19.9 million in consolidated net profit attributable to shareholders of the parent company in Q2 2025. A year earlier, the company recorded PLN 30.6 million in net profit ( a 35% decrease ). Operating profit amounted to PLN 43.2 million, compared to PLN 62.4 million in profit a year earlier. Consolidated sales revenue reached PLN 3,268.0 million, compared to PLN 3,043.4 million a year earlier.

The company announced that the decrease in net profit was influenced by the increase in remuneration (+11%) and the weakening of the dollar, which influenced the amount of negative temporary exchange rate differences (an increase in costs by PLN 6.6 million).

- PZU Zdrowie plans to recapitalize to support its growth, which will include, among others, 11 modernizations and openings of medical facilities by the end of 2026.

Currently, there are over 40 PZU Zdrowie Diagnostyka Imaging laboratories operating throughout Poland, as stated in the results presentation.

Friday (29/08/2025)

- Neuca expects to achieve a positive operating profit in the clinical trials segment , concentrated in Humaneva , in the second half of this year.

Neuca is conducting several due diligence processes for potential acquisitions and expects to finalize at least one transaction in the coming quarters.

Monday (25/08/2025)

- AbbVie has decided to acquire the rights to the investigational drug candidate bretisilocin , which is in Phase II clinical trials, from a private (unlisted) company, Gilgamesh Pharmaceuticals, for up to $1.2 billion.

It is a 5-HT2A serotonin receptor agonist and 5-HT releaser that, unlike psilocybin or LSD, can keep patients in a psychoactive state for 6-10 hours. The parties to the transaction reported that bretysilocin has a shorter duration of the psychoactive experience while maintaining extended therapeutic benefits .

These are the conclusions of the Phase IIa clinical trial. In patients with moderate to severe major depressive disorder, a single 10 mg dose led to a reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) score from baseline of 21.6 points after two weeks , compared with a reduction of 12.1 points in patients receiving a low-dose active comparator.

The antidepressant effect occurred within 24 hours and was sustained after just two treatment sessions. The drug candidate was also well tolerated (no serious adverse events were reported).

Brian Barnett, clinical director of the Psychiatric Treatment Resistance Program at the Cleveland Clinic, suggested that bretysilocine could be a "nice alternative" to Johnson & Johnson's Spravato (esketamine) , with more lasting benefits compared to it.

Tuesday (26/08/2025)

Regeneron Pharmaceuticals aims to file for U.S. approval of cemdisiran in the first quarter of 2026, as its experimental siRNA therapy successfully completed a Phase III clinical trial in generalized myasthenia gravis (gMG) . The stock rose 2.7% on Tuesday.

Myasthenia gravis is an autoimmune neuromuscular disease. It is rare, affecting 3 in 10,000 people. Approximately 9,000 people in Poland suffer from it. Pathological processes in the human body disrupt the transmission of impulses from motor nerve endings to skeletal muscles.

The primary symptoms of myasthenia gravis are weakness and fatigue of various skeletal muscle groups. Drooping eyelids are the first symptom of the disease in approximately 70% of affected individuals. Within two years, approximately 80% of patients experience generalized disease, affecting additional muscle groups.

Myasthenia gravis has two age peaks. Young women between 20 and 40 years of age and men between 60 and 80 are most commonly affected. Women are 2-3 times more likely to develop myasthenia than men.

Source: PTChNM

Regeneron Pharmaceuticals' NIMBLE study involved 288 patients and showed that quarterly subcutaneous injections of cemdsiran improved patient function compared with a placebo control group.

Cemdisiran, a drug being developed under an agreement with Alnylam Pharmaceuticals, was tested as monotherapy (600 mg every 12 weeks) and as combination therapy (with Regeneron's C5 antibody).

After 24 weeks, the NIMBLE study showed that patients receiving monotherapy experienced a placebo-adjusted improvement from baseline in the myasthenia gravis scale (MG-ADL) of 2.3 points, the study's primary endpoint. In the combination group, the improvement was 1.7 points. In the key secondary NIMBLE endpoint, the quantitative myasthenia gravis scale (QMG), placebo-adjusted improvements were 2.8 and 1.9 points, respectively.

No meningococcal infections were observed. There were no treatment discontinuations due to adverse events in the cemdisiran group through 24 weeks.

Treatment-related adverse events occurred in 69% of patients receiving monotherapy, 81% of patients receiving the combination of cemdisiran and C5, and 77% of those receiving placebo. Serious adverse events were least common in the cemdisiran group (3%) and in the combination group (9%) (compared to 14% in placebo).

There were two deaths in the extended phase (one case of pneumonia and the other of septic shock) among patients who were also receiving immunosuppressive drugs.

Under the agreement signed last year, Regeneron obtained a worldwide license for cemdisiran as a single agent in addition to the license in combination with C5 antibodies, while Alnylam is entitled to receive milestone payments and royalties – up to 15% for sales of the single agent and a lower fixed royalty and milestones of up to $325 million for the combination use.

Akeso reported that its lung cancer drug ivonescimab significantly extended overall survival (OS) in the pivotal Phase III HARMONi-A clinical trial. Investors reacted this week by selling off the Hong Kong-based company's shares, which fell 12% .

This may have been due to discounting the facts, as speculation about Akeso's business development has fueled a 166% increase in the company's share price since the beginning of this year. The company's strong financial results for the second half of this year also didn't help the company close its positions – sales increased by 49% compared to the first half of 2024. The growth rate doubled, compared to 24% a year earlier.

Source: own study based on Akeso and TradingView

Wednesday (27/08/2025)

- The FDA Office of New Drugs (CDER) has accepted a letter of intent to qualify liver stiffness measurement using vibration-controlled transient elastography as a rational plausible surrogate endpoint in clinical trials in adults with liver disease associated with fatty metabolic dysfunction (MASH).

Evaluating new therapies for MASH without the need for repeated liver biopsies could aid in study recruitment, enable evaluation of representative patient populations, help overcome challenges in conducting adequately powered clinical trials, and accelerate drug development for patients with this liver disease.

Eli Lilly revealed that Verzenio (abemaciclib) significantly improved overall survival (OS), when combined with hormonal therapy, in patients with high-risk early breast cancer .

The results come from a new analysis of the phase III monarchE study, which included more than 5,600 adults across two cohorts. Participants were randomly assigned to receive Verzenio twice daily in combination with standard adjuvant endocrine therapy or adjuvant endocrine therapy alone as standard of care.

monarchE showed that two years of treatment with Verzenio in combination with endocrine therapy showed a statistically significant and clinically meaningful improvement in OS compared with endocrine therapy alone.

Thursday (28/08/2025)

Johnson & Johnson has halted clinical trials of Imaava (nipocalimab) in combination with the anti-TNFα therapy Cimzia (certolizumab pegol) for the treatment of rheumatoid arthritis . The Big Pharma giant concluded that the results of the Phase IIa DAISY clinical trial were a developmental dead end. They did not demonstrate that the combination therapy provided additional benefit compared with Cimzia monotherapy after 12 weeks of treatment.

Imaavy, which the company acquired in 2020 through its $6.5 billion acquisition of Momenta Pharmaceuticals, received FDA approval earlier this year for the treatment of generalized myasthenia gravis in adults and adolescents. Johnson & Johnson is also investigating the molecule in other autoimmune diseases, including fetal and neonatal hemolytic disease, heat-induced autoimmune hemolytic anemia, and Sjögren's disease.

Friday (29/08/2025)

Israel's Teva has launched the first generic weight-loss drug in the United States . The generic is equivalent to Novo Nordisk's Saxenda (liraglutide). Teva has not disclosed the price it will charge patients for the injectable drug.

This is a sign of continued market expansion. Last year, the Israeli company introduced a generic version of Novo Nordisk's Victoza, which is based on the same active ingredient but is indicated for the treatment of type 2 diabetes.

Biogen and Eisai triumph after securing FDA approval to market a subcutaneous maintenance formulation for Leqembi (lecanemab), which is used to treat Alzheimer's disease.

Until now, Leqembi was administered as an infusion every two weeks, a procedure that took an hour. Following the FDA's decision , patients will be able to transition to weekly injections at home after 18 months of conventional treatment.

This is a significant step forward, as 82% of US neurologists believed that the current dosing regimen for Leqembi poses a significant barrier for patients. The drug will be launched on October 6th , which could provide a sales boost in the face of Eli Lilly's competing drug Kisunli (donanemab-azbt).

In July 2025, Biogen/Eisai reported that patients who received Leqembi for 4 years experienced a 1.75-point reduction in cognitive decline compared to the expected decline observed in the Alzheimer's Disease Neuroimaging Initiative (ADNI) cohort.

Sanofi has received the green light from the FDA for Wayrilzu (rilzabrutinib) for the treatment of adults with persistent or chronic immune thrombocytopenia who have had an inadequate response to prior therapy.

The regulatory approval was based on results from the Phase III LUNA 3 clinical trial. In this trial, 202 patients were treated with oral Wayrilz twice daily or placebo. The drug candidate met the study's primary endpoint of sustained platelet response , with 23% of patients who received treatment achieving platelet counts at or above 50,000/μL for at least 8 of the last 12 weeks of the 24-week treatment period, compared with 0% of patients in the placebo group.

Wayrilz reduced the need for rescue therapy by 52% compared with placebo and also led to significant and clinically meaningful improvements in physical fatigue and bleeding. The most common adverse events were diarrhea (23%), nausea (17%), headache (8%), and abdominal pain (6%).

Rilzabrutinib is expected to become a second-line option after thrombopoietin receptor agonists, with the potential for rapid adoption given physicians' familiarity with similar inhibitors in blood cancers (Imbruvica). Sanofi has requested regulatory review of Wayrilz in the EU and China.