PoZdroweek 14/2025: A sad epilogue to Sarepta's stubbornness for DMD patients. Women rejoice because Apnimed's good results in OSA therapy offer a chance for their partners' snoring to be less bothersome.

Sarepta Therapeutics finally gave in to the FDA's demands, but at what cost? DMD patients, particularly in America and Europe, suffered the most from this dispute with the regulator. Stock market investors took their toll, as investment firm HC Wainwright deemed the Cambridge-based company unworthy of a penny. Apnimed boasted promising Phase III clinical results for its treatment of obstructive sleep apnea (OSA), giving hope to many women suffering from a partner's snoring. Global healthcare stock indices climbed as one this week. WIGmed is increasingly approaching this year's local peak from May. Ministerial changes have taken place in the government led by Donald Tusk. Jolanta Sobierańska-Grenda, a highly experienced practitioner in the field of medical facility management, has been appointed the new head of the Ministry of Health. We're rooting for her, as we do for any new boss in the ministry, but will her practice emerge victorious from the clash with the theories of the ossified bureaucratic world from Miodowa and political decision-makers who aren't very knowledgeable about health issues?

Finally, we had a week during which all the health care stock indices we analyzed rose. And not just any price. The WIGmed index has been revitalized for the past few weeks. This week, the index rose 1.3% , approaching this year's peak in May. It's about 2 percentage points from reaching that peak. International indices fared even better. The European MSCI Europe Health Care (SPYH) gained 3.0% , and the pan-global MSCI World Health Care (XDWH) gained 2.2%.

Despite this, since the beginning of this year , only WIGmed has been in positive territory , and the remaining measures of the economic situation in the industry are so far only approaching the line, as they are still a few percent short of reaching it.

Source: TradingView

Among the top 20 stocks in the MSCI World Health Care Index , Thermo Fisher (+15.3%), Novo Nordisk (+11.5%), and Danaher (+8.1%) performed best. Only Intuitive Surgical (-4.4%) and United Health Group (-0.6%) bucked the upward trend.

Source: own study based on stooq.pl

In Poland, the most notable performers were Molecure (+38.2%), Selvita (+12.3%), and Medicalghoritmics (+5.5%). The latter's run of success continues, as the medtech's share price is at its highest level since late 2023. New energy has come to Medicalghoritmics shareholders, as the shares have already delivered a 98% rate of return since the beginning of the year.

In the case of Selvita shareholders, the updated recommendation issued by Noble Securities a few days ago may have captured the imagination of investors. The share price increase was accompanied by high trading volumes not seen since 2017. The investment firm reiterated its "buy" recommendation, although it significantly lowered its target price for the company from PLN 70.6 to PLN 51.1.

By contrast, Voxel (-8.9%), NanoGroup (-2.1%), and Ryvu (-1.9%) disappointed. Voxel had gained about 34% since the beginning of January, but this steady upward march was eventually halted. Rumors circulating in the market about a possible tender offer for the Polish diagnostic imaging leader's shares have not yet materialized .

Captor Therapeutics' stock price fell 1%, but the company's management and scientific staff, as well as its shareholders, have reason to be satisfied. The medical industry portal Drug Hunter nominated the company's drug candidate , CT-01, as its Molecule of the Month for June. We'll cover this in more detail later in PoZdroweek.

Source: own study based on stooq.pl

Stubbornly standing your ground sometimes doesn't pay off. Sarepta Therapeutics found this out last Saturday when, contrary to FDA expectations, it declared that it would not suspend deliveries of Elevidys, a drug used to treat people with Duchenne muscular dystrophy (DMD). On Monday, the US regulator halted all clinical trials for limb-girdle muscular dystrophy for the Cambridge-based company. This applies to early-phase trials of its drug candidates: SRP-9003, SRP-9004, SRP-6004, and SRP-9005.

As if that weren't enough, in response to the FDA's decision, investment firm HC Wainwright has reduced its estimate of Sarepta's fair value over the next 12 months to zero. Yes, according to this financial institution, the company's shares aren't worth a penny.

Sarepta has clearly clashed with American regulators. And is suffering the painful consequences, with those unfortunate enough to suffer from the still-incurable Duchenne disease and the caregivers of young boys, most often affected by DMD.

The FDA halted clinical trials for four drugs. A few hours later, the Cambridge-based company tucked its tail between its legs, announcing it would suspend shipments of Elevidys to all US patients. However, the tea is over at this point. The chances that the FDA will suddenly, by some strange coincidence, greenlight drug candidates for SRP-9003, SRP-9004, SRP-6004, and SRP-9005 are truly illusory.

The initially intransigent stance of Doug Ingram, the company's CEO, simply didn't pay off. As a result, Elevidys will not be sold in the US, and its approval in the EU is currently a distant dream.

On Friday, the European regulatory body, the EMA, joined the FDA in its skepticism about Elevidys. The EU regulatory body announced its decision not to recommend conditional approval of the substance.

Levi Garraway, Roche's chief medical officer, who is helping Sarepta secure European market approval for Elevidys, expressed disappointment with the EMA's decision. He also noted that the Swiss pharmaceutical giant plans to collaborate with the regulator to explore a potential path forward to resolve the approval impasse for a drug eagerly awaited by many DMD patients who are not concerned about potentially serious liver complications.

The CHMP, an advisory body to the EMA, explained that the main study submitted by Roche to support its application for approval for the treatment of DMD in people aged 3 to 7 years who were able to walk, did not show that Elevidys had an impact on motor skills after 12 months of treatment. Improvements in movement, as measured by the North Star Ambulatory Assessment (NSAA), were observed in both patients taking Elevidys and those taking placebo, but the difference of 0.65 points between the two test cohorts on a 34-point scale was not statistically significant.

The suspension of Elevidys supplies to American patients with DMD took effect on Tuesday. At the same time, the FDA required the company to place a black box warning on the drug due to the risk of acute liver injury and liver failure. This is another blow, as clinicians, patients, and their caregivers will think twice about using the remaining drug. And this will likely be a hellish conundrum, as with the suspension of Elevidys supplies, medical supplies will inevitably run out at some point.

The result of all these problems is that the survival of the American company has become a great unknown.

All that's left are the somewhat archaic exon-skipping therapies Amondys 45, Vyondys 53, and Exondys 51, which are already in a fairly advanced sales phase. And their slow and inexorable decline is likely. Furthermore, the EMA recently approved the competing drug Duvyzat (givinostat). Therefore, wherever you turn and look, you see a picture of misery and despair. And by your own choice.

In my opinion, the risk has increased that Sarepta, in its current form, may not survive until the end of this year , unless it somehow miraculously straightens out its strained relationship with the FDA, which is doubtful, especially since Trump appointee Marty Makary holds the reins of oversight. And the US president seems to be quite critical of the biopharmaceutical industry, which inevitably affects the decisions made by lower-level officials.

The FDA's response to the Sarepta case clearly stated that there's no point in playing games with the company, as it could end badly. This also sends a clear message to other companies in the sector that it's better to reach an agreement with the FDA than to go to war with it.

The high level of experience and competence of the company's scientists working on these discoveries is undeniable. They are Sarepta's most valuable asset, not the management team, which is making schoolboy errors. I assume they will soon be leaving the company, as I haven't seen such a sequence of mishaps in the industry in years. In this entire chain of events surrounding the company's gene therapies, the greatest victims are primarily DMD patients, followed by capital market investors.

In my opinion, given the current circumstances, Sarepta has no business existing in its current configuration. I suspect that, given the extensive know-how it has developed over the years, it could be a tempting prospect for BigTech representatives. I've even identified three candidates who could take over the company, which wouldn't require a significant investment, but we'll see what the future holds.

UnitedHealth Group (UHG) shares fell nearly 5% on Thursday after the company confirmed that the Department of Justice (DOJ) is conducting criminal and civil investigations into its Medicare Advantage benefit practices.

In May of this year, the Wall Street Journal (WSJ), citing its sources within the federal government, revealed the existence of a criminal investigation. At the time, UHG denied this, condemning the WSJ's conduct as deeply irresponsible. The company stated that it had not been notified of the DOJ investigation. How, then, was it able to responsibly determine whether or not an investigation was underway? Such a baffling pyramid of logic is the subject of ridicule among healthcare analysts.

UnitedHealth stated that it "proactively" contacted the Department of Justice following media reports about the investigations in May. If obtaining confirmation of the ongoing investigations after two months constitutes proactivity, then investors and the company likely have different understandings of the concept. It's frightening to think what would have happened if the company hadn't taken a "proactive" stance.

UHG emphasized on Thursday that it does not expect to be charged with any wrongdoing. The company noted its long history of responsible conduct and effective compliance. It cited independent audits conducted by the Centers for Medicare & Medicaid Services, which confirmed that its practices are among the most thorough in the healthcare provider industry.

UnitedHealth Group reiterated that it has initiated an independent review of its performance, processes, policies and practices related to risk assessment coding, healthcare benefits management practices and pharmacy services.

The level of quality involved in the Minnetonka, Minnesota-based company's investigation is apparently not acceptable to some investors, as UHG's share price has fallen 44% since the beginning of the year.

The drug candidate Captor Therapeutics did not escape the attention of industry professionals at Drug Hunter (DH). The portal nominated ABS-752 as one of its molecules of the month for June 2025. This is noteworthy because it marks the first time in the history of drugs developed in Poland that anyone has received such a nomination from DH.

In his opinion, what caught the interest of Drug Hunter experts were several unique elements and features of the ABS-752 molecule.

Secondly, the drug candidate being investigated by Captor Therapeutics is a currently very popular degrader, a molecule that removes disease-causing proteins.

Michał Walczak shares his knowledge about experimental processes conducted by biopharmaceutical corporations from around the world on his blog at https://adastra71.blog/ .

Synektik has announced details of its plan to split the company into two independent entities. The newly formed Syn2bio will receive assets related to research on new molecules , including the cardiotracer—an innovative radiopharmaceutical with global sales potential. The remaining business will remain with Synektik (primarily sales of Da Vinci surgical robots, training in their use, and maintenance services).

The company intends to have both separated entities listed on the Warsaw Stock Exchange in the first half of 2026.

On behalf of mInvestment Banking, he performed a valuation of both companies.

The company's existing shareholders will be allocated Syn2bio shares on a 1-to-1 basis.

Monday (21/07/2025)

Katarzyna Kosiorek, an analyst at Trigon DM, lowered the target price for Bioceltix shares from PLN 134.50 to PLN 124.30 in a July 21 report, while maintaining a "buy" recommendation. The recommendation was issued at a market price of PLN 101.

Tuesday (22/07/2025)

- Adamed Technology is among the investors who supported the French biotechnology company One Biosciences with €15 million in a Series A financing round. The value of Adamed's involvement remains unknown.

One Biosciences focuses on precision oncology, providing clinical-grade transcriptome profiling of single-cell tumors.

In addition to Adamed Technology, the latest financing round also included Galion.exe, Invus, Sofinnova Partners, Polytechnique Ventures and Kima Ventures.

Wednesday (23/07/2025)

Medicalgorithmics has completed an integration for a key client in the US. The company estimates that total contract revenue for the first 24 months after integration approval and the commencement of commercial trials will range from approximately $7.7 million to approximately $11.5 million. The contractually guaranteed minimum contract revenue (for sessions and integration) will exceed $1.7 million.

- According to PEX PharmaSequence calculations, the value of sales in pharmacies in Poland in June 2025 increased by 10.7% year-on-year to approximately PLN 4.68 billion. This represented a 3.8% decrease compared to the previous month.

Average pharmacy sales totaled approximately PLN 380,000 (+12.4% year-on-year). The average retail price of a drug was PLN 33.82, up 10.8% year-on-year. Pharmacy margins improved significantly, reaching 26.5% (+5.6 percentage points year-on-year).

Friday (25/07/2025)

Urteste has signed an agreement with the German company Biotype, under which the foreign partner will produce Panuri and Panuri Control kits, which will be used to conduct clinical trials in Europe. The publicly traded company will pay approximately €770,000 for the kits. Urteste's share price rose 5.1% on Friday.

The Polish Financial Supervision Authority has approved the delisting of EMC Instytut Medyczny shares from trading on the Warsaw Stock Exchange. The delisting date has been set for August 8, 2025.

Monday (21/07/2025)

AstraZeneca plans to allocate $50 billion to expand its US operations by 2030. At the heart of the plan is a new manufacturing facility in Virginia, which will focus on producing active ingredients for a new generation of weight-loss and metabolic drugs. These include the orally administered GLP-1 molecule currently in development and the experimental hypertension drug baxdrostat.

This initiative complements a previously announced $3.5 billion investment (November 2024) for expansion in several US states, including research and development centers in Gaithersburg, Maryland, and Cambridge, Massachusetts; next-generation cell therapy production in Rockville, Maryland, and Tarzana, California; and specialty manufacturing in Coppell, Texas.

According to Astra's managers, these initiatives will enable this Big Pharma leader to achieve its ambitious goal of generating $80 billion annually by 2030 , half of which is expected to come from America. In the last four quarters, this British-Swedish company generated $55 billion in sales.

The company's announcement aligns well with the investment trend announced by various biopharmaceutical companies in recent months. According to estimates by politykazdrowia.com, the pressure exerted by President Donald Trump on the industry has led to new investment announcements totaling at least $225 billion. In addition to Astra, the investment group includes Roche, Eli Lilly, Novartis, Sanofi, Johnson & Johnson, AbbVie, Bristol Myers Squibb, Gilead Sciences, UCB, and Biogen.

Source: own work based on public domain

Wednesday (23/07/2025)

- Roche has halted all new orders for Elevidys (delandistrogen moxeparvovec) for outpatients with Duchenne muscular dystrophy (DMD) in markets that reference the FDA as the basis for their local approval.

After concluding an agreement with Sarepta as the inventor of the molecule, Roche obtained approval for gene therapy in 8 countries, including Bahrain, Brazil, Israel, Japan, Kuwait, Oman, Qatar and the United Arab Emirates.

The Swiss company revealed that to date, approximately 760 DMD patients have benefited from outpatient treatment with Elevidys, and there have been no treatment-related deaths. According to the Basel-based company's management, the benefit-risk ratio of Elevidys remains positive in the outpatient population.

Apnimed , whose shares are not publicly traded, is celebrating another Phase III clinical success for its drug candidate, AD109, for the treatment of obstructive sleep apnea (OSA). OSA is a sleep disorder characterized by repeated episodes of cessation or shallow breathing during sleep, caused by a blockage of the upper airway.

The LunAIRo study has met its primary endpoint in late-stage clinical trials. It enrolled 660 adults with mild, moderate, and severe OSA who were intolerant of or refused continuous positive airway pressure therapy. Patients were randomly assigned to receive AD109 ( a combination of aroxbutynin and atomoxetine , a selective norepinephrine reuptake inhibitor) or placebo, and the primary endpoint was assessed 26 weeks after treatment initiation.

The study demonstrated a significant and statistically significant reduction in airway obstruction. After 26 weeks of treatment, the mean reduction in the Apnea-Hypopnea Index (AHI) was 46.8% compared to baseline. For the placebo cohort, the reduction in AHI was 6.8%.

AD109 was well tolerated as no serious adverse events were reported.

The latest findings from the Phase III clinical trial strengthen Apnimed's management's plans to submit an application to the FDA for approval of this once-daily oral formulation in early 2026 .

In May of this year, Apnimed reported that the first Phase III study – SynAIRgy – also met its primary endpoint.

There is a chance that many women will feel relief at night when their partners finally reduce their snoring, and men will also feel an improvement in their comfort in everyday life.

Friday (25/07/2025)

Sarepta again. The FDA announced it was investigating the death of an 8-year-old boy who received Elevidys. In response, Sarepta stated that the death was deemed unrelated to treatment.

The FDA said the boy died on June 7, 2025. Sarepta revealed that the case, which occurred in Brazil, was reported to regulators there on June 18 through the FDA Adverse Event Reporting System (FAERS).

But why were DMD patients and capital market investors completely unaware of this? Could it really be such a trivial matter that it's not worth bothering them with?

Bayer has learned that the FDA has extended its pre-approval review of elinzanetant , a non-hormonal therapy for the treatment of vasomotor symptoms (VMS) associated with menopause (primarily associated with hot flashes). This will likely delay the approval decision for the drug candidate by up to a quarter.

The Germans revealed that the FDA justified its move by saying it needed more time to conduct a full review of the notification, adding that the regulator did not cite any issues with the general acceptability of elinzanetant.

If the molecule is approved for marketing, Bayer will become a formidable rival to Japan's Astellas , which successfully markets fezolinetant under the Veozah brand in the US and Veoza in Europe. Naoki Okamura, head of the Japanese company, who has been with the company since April 1986, likely has plenty to think about with his team.

The Japanese consider fezolinetant a strategic molecule. On April 25th of this year, Astellas, during the publication of its financial results, showed that Veozah, alongside Izervay, were the brands with the most dynamic year-on-year growth, with growth significantly exceeding 300%. Furthermore, according to the company's management , Veozah is the third most important brand in terms of expected peak sales (after Padceva and Izervay).

Investors are closely watching how the Japanese-German menopause rivalry will unfold. Since the beginning of this year, Astellas's share price has performed quite similarly to the sector's MSCI World Health Care index. The Tokyo-based company's share price (in dark blue in the upper right corner of the infographic below) has fallen 3%, while Bayer has risen 51% in the same period (in dark yellow in the upper right corner of the infographic below).

Source: own study based on Astellas and TradingView

Bayer's conclusion is based on Phase III clinical results from OASIS 1 and OASIS 2. These showed a significant reduction in the frequency and severity of vasomotor symptoms within 12 weeks of treatment compared with placebo. More than 80% of treated participants experienced a reduction in the frequency of VMS by at least half by week 26.

In addition, the long-term phase III OASIS 3 study demonstrated that elinzanetant resulted in a sustained significant reduction in the incidence of moderate to severe vasomotor symptoms and was free from hepatotoxicity.