A microcap medical technology company is developing a drug-eluting biologic matrix aimed at reducing one of the more costly complications associated with breast reconstruction following mastectomy: post-surgical infection. The technology combines regenerative biologic tissue with localized, extended-release antimicrobial protection, potentially offering an advantage over traditional biologic matrices and synthetic meshes that do not provide the same dual function.

The opportunity is particularly interesting because the underlying technology has already attracted strategic buyers. Related product lines have been sold in multiple transactions, including one approaching $90 million, while management has directed the proceeds toward advancing its remaining breast reconstruction platform. With key regulatory milestones expected over the next year, investors may want to watch whether this becomes the company’s next commercial success, or ultimately attracts another strategic buyer.

Biotech Currents assigns a 59.6% overall probability the company will have a successful product launch.

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Overall Probability: 59.6%

• Market Demand: 54.2%

• Adoption by KOLs: 60.0%

• Clinical Trial Progress: 66.7%

• Experience & Capital: 60.0%

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A beaten-down mRNA platform company may be hiding one of the more compelling asymmetric setups in biotech. While the market has largely written off the story after fading enthusiasm for Covid-related assets, the company has been quietly advancing an inhaled mRNA therapy for cystic fibrosis, starting with Class I patients who are not well served by today’s dominant CFTR modulators.

The potential differentiation is important: rather than simply improving the function of a defective CFTR protein, this approach is designed to help airway cells produce normal, functional CFTR protein. Early clinical data has shown encouraging signals, including reductions in mucus plug number and mucus volume after only 28 days of treatment.

With a market cap near its cash balance, the market appears to be assigning limited value to the cystic fibrosis program. That could change quickly if upcoming Phase II data continues to support safety, tolerability, and early efficacy in a clearly defined patient population. In a multibillion-dollar cystic fibrosis market dominated by one major player, even moderately compelling data could turn this overlooked asset into a strategic priority.

Biotech Currents assigns a 64.4% overall probability the company will report positive data.

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Overall Probability: 64.4%

• Market Demand: 58.3%

• Adoption by KOLs: 80.0%

• Clinical Trial Progress: 63.3%

• Experience & Capital: 60.0%

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A commercial-stage eye care company appears to be turning a fast-growing approved product into a broader dermatology platform, built around a single validated insight: targeting Demodex mites as a contributor to multiple inflammatory conditions around the eyes and skin. With strong early commercial uptake, a path toward international expansion, and a peak-sales target that looks increasingly attainable, the next key catalyst is a mid-stage study in ocular rosacea, where success appears tied to measurable symptom improvement rather than proving a cure or fully validating disease causality.

The edge is that the company may already have physician buy-in, regulatory alignment, and a proven mechanism from its first approved product, while applying that same approach to adjacent conditions with large patient populations and limited treatment options. A recent board addition with deep dermatology and aesthetics experience adds further credibility as the company looks to scale commercially and pursue broader platform opportunities.

Biotech Currents assigns a 56.7% overall probability the company will report positive data.

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Overall Probability: 56.7%

• Market Demand: 45.8%

• Adoption by KOLs: 53.3%

• Clinical Trial Progress: 66.7%

• Experience & Capital: 60.0%

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A company that once looked derailed by a cancer-drug setback has quietly rebuilt its story around relapsing multiple sclerosis, a chronic autoimmune disorder where patients and physicians still value better convenience, lower treatment burden, and durable relapse control.

The current setup centers on whether an already-commercial therapy can extend its momentum into a more patient-friendly formulation, potentially widening its reach in a large MS market where dosing frequency matters. With prior market adoption already established and the clinical hurdle appearing more incremental than exploratory, Biotech Currents assigns a 67.3% overall probability the company will report positive data.

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Overall Probability: 67.3%

• Market Demand: 66.7%

• Adoption by KOLs: 47.0%

• Clinical Trial Progress: 87.0%

• Experience & Capital: 70.0%

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AI has already changed how quickly decisions get made in software and business, but healthcare is where the stakes are higher and the proof has to be stronger. One company is trying to bring that same intelligence layer to cardiovascular care, using a growing database of more than 160 million annotated heart images to help physicians see risk earlier, plan treatment more precisely, and potentially reduce unnecessary invasive procedures. With important one-year validation data expected before year-end, this is a medical AI story where the next update could help determine whether the promise is turning into real clinical evidence.

We assign a 60.6% overall probability the company will report positive data from its trial.

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Given the ongoing uncertainty at the FDA and future NIH funding, large and mid-sized drug companies stepped up M&A deals in early 2026. Among the largest, Biogen, Inc. (NASDAQ:BIIB) agreed to pay $5.6 billion for Apellis Pharmaceuticals (NASDAQ:APLS) and Eli Lilly & Co. (NYSE:LLY) said it would buy Centessa Pharmaceuticals (NASDAQ:CNTA) for $6.3 billion.

A drug candidate’s potential to respond to an unmet medical need remains a driving force behind stock selections by Biotech Currents. While unmet medical need may sound like a standard bullet line in a VC pitch, many “me too” drugs advance by claiming better, faster or stronger efficacy. Both approaches can work.

For example, Scynexis, Inc. (NASDAQ:SCYX) recorded a 15.4% gain in June 2021 after three months when the FDA approved its antifungal drug ibrexafungerp to treat vulvovaginal candidiasis, which had few specific options at the time. Since trading at ~$8-9 per share in 2021 the stock now trades at $0.99 per share. Conversely, Intra-Cellular Therapies recorded a similar 15.7% gain in March 2024 after positive data in two phase III trials of Caplyta to treat major depressive disorder (MDD); Caplyta already was approved to treat schizophrenia and bipolar disorders. After the phase III data, Johnson & Johnson (NYSE:JNJ) bid $132 per share for Intra-Cellular in January 2025, reflecting a 40% premium to the Intra-Cellular price in early 2025 and also in March 2024.

Small company (Scynexis) versus big company (Intra-Cellular)? Absolutely. Small market versus big market (MDD)? Absolutely. But the continuum of growth, as well as the potential for growth, is relevant in both situations. Sycnexis believed it could expand its antifungal niche and faced stiff challeges as a microcap. Intra-Cellular already was recording $462 million in Caplyta sales and likely would boost its topline exponentially with the MDD indication. Thus, JNJ and others circled Intra-Cellular seeking to build their top and bottom lines with sales from a budding drug franchise.

Among this year’s deals, the Apellis buyout somewhat follows the Scynexis script, albeit at a much higher valuation. Lilly’s buy of Centessa tracks the franchise strategy of Intra-Cellular involving the smaller but growing narcolepsy market.

Biotech Currents first invested in Apellis in September 2019 forecasting a positive outcome for its drug pegcetacoplan to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) when compared to treatment with eculizumab, the current standard of care, marketed by Alexion Pharmaceuticals (NASDAQ:ALXN). Eculizumab was one of the most expensive drugs on the market at the time. Pegcetacoplan, now branded as Empaveli, proved superior in the phase III trial and delivered a 50% return after three months to ~$40 per share after the clinical data.

Apellis’ stock climbed higher for three years as it advanced its drug Syfovre (pegcetacoplan injection) to treat geographic atrophy (GA), touching ~$93 per share in mid-2023. But safety concerns emerged around the injection of Syfovre and Apellis’ shares crashed 70% to the low $20s per share before rebounding to the high $60s per share in February 2024. In the last two years, Apellis’ shares slid to the $16-17 per share range before the Biogen bid on March 30.

The Biogen bid came in at $41 per share, higher than the price of $26.02 per share at the time of the first Biotech Currents report in September 2019. A second Biotech Currents on Apellis in GA was issued in March 2022 when the price was $52.91 per share. The second catalyst report yielded only a 2% gain. Biotech Currents exited each position in Apellis shortly after the catalyst result, begging the question would it have been wiser to just hold on Apellis for six years after buying at $26.02 per share?

The answer, of course, depends on one’s performance and portfolio objectives. Apellis probably took on more than it could handle with the GA indication but the drug’s (pegcetacoplan) mechanism of action proved successful in both PNH and GA. Moreover, Empaveli and Syfovre generated $689 million in sales last year. At some point, Apellis’ board and management decided the growth run of these two drugs had peaked while Apellis’ near-term pipeline was slim. Selling the two drugs to Biogen brought the company’s shares back to the ~$40 per share range. Likely an “acceptable” exit for Apellis after 17 years of work as there was only a slim probability Apellis shares could climb to the $50 per share to $60 per share range in the next year.

Biotech Currents first selected a catalyst from Centessa in September 2023 and then again in September 2024. Both selections involved the company’s lead asset, hemophilia treatment SerpinPC.

In addition to hemophilia, UK-based Centessa was advancing drugs to treat neurological disorders as well as acute myeloid leukemia (AML) and pulmonary arterial hypertension (PAH). Centessa profiled as an indication agnostic drug discovery company or a publicly traded VC/PE firm. Noted in the Biotech Currents September 2024 report was the strong phase I results from ORX750, Centessa’s narcolepsy drug now named cleminorexton. Centessa raised $250 million after the ORX750 phase I results.

Market researchers expect the global narcolepsy market to reach between $4.9 billion and $6.9 billion by 2031-32. Heady growth for a market that stood at $2.53 billion in 2024. While Centessa’s cleminorexton is still years away from a commercial launch, Lilly is applying its hefty weight-loss cash haul to get in on a narcolepsy market expected to ramp up at a compound annual growth rate (CAGR) between 9% to 10% in the next six years.

Lilly paid a 38% premium to Centessa’s share price on March 31. The deal also includes a $1.5 billion contingent value right (CVR) should cleminorexton reach commercial milestones. Cleminorexton appears to demonstrate a best-in-class profile across narcolepsy type 1, narcolepsy type 2, and idiopathic hypersomnia in recent in phase IIa clinical studies.

Alkermes plc (NASDAQ:ALKS) likewise wants to build a franchise in narcolepsy based on alixorexton, an oral, selective orexin 2 receptor (OX2R) agonist, the same target pathway as cleminorexton. Alkermes expects alixorexton can treat the same three forms of sleep disorders as Lilly now is backing via cleminorexton. Not surprisingly, Alkermes’ shares rose following news of the Centessa sale with investors banking on a future bid for Alkermes.

But at what point in the future. In February, Richard Pops said he would step away as CEO of Alkermes after 33 years(!) but remain as board chairman starting August 1. With Pops’ departure, would the Alkermes board be up for another clinical slog with alixorexton should the right offer come its way? Centessa now holds a market cap of $6.1 billion while Alkermes, with drugs on the commercial market and similar orexin drug, has a market cap of $5.5 billion.

Alkermes in 2025 bought Lumryz, an extended release, single-dose formulation of sodium oxybate from Avadel Pharmaceuticals plc. Lumryz was introduced to compete with Jazz Pharmaceuticals’ (NASDAQ:JAZZ) sodium oxybate formulation Xyrem, which had to be administered twice during nighttime hours. Avadel had to fend off litigation from Jazz for several years in order to get Lumryz on the market. And Alkermes had to turn away a surprise bid from H. Lundbeck A/S to buy Avadel. As a result, Alkermes appears to be all in on advancing alixorexton.

Appears is the operative term. When the stars align between an anxious, motivated buyer and a seller like Apellis that can accept a hefty premium, M&A deals happen. With Alkermes, more buyers like Lilly have to gain interest in narcolepsy while Alkermes needs to determine a premium number that will make the board and the shareholders happy. Not out of the question for 2026 as the M&A market heats up.

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Despite missing its key clinical target last October, a small-cap biotech still managed to pull in roughly $400 million earlier this year, capital earmarked to push its lead asset into late-stage studies and begin laying the groundwork for commercialization.

At first glance, that kind of raise looks like a leap of faith. But there are a couple of factors that help explain the appetite. For one, the current treatment landscape for this chronic neurological condition still leaves room for improvement, meaning any therapy with credible signals can carve out meaningful share in a multi-billion-dollar market.

Additionally, the company’s approach targets a well-understood metabolic pathway already validated by existing drugs, while also layering in a potential second mechanism tied to neuronal support and resilience. That dual-action angle is where the story gets more interesting, suggesting the possibility of both disease-modifying and protective effects.

Taken together, investors appear to be underwriting not just a comeback attempt, but a differentiated profile that, if it holds up in late-stage testing, could justify the risk.

We assign a 65.3% overall probability the company will report positive data in the phase III trials.

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The France-based company Abivax SA (NASDAQ:ABVX) recorded the highest stock price gain last year in the biotechnology sector. Vastly unnoticed at the start of 2025, Abivax shares skyrocketed 444% in July 2025 when the company reported topline results from two, 8-week induction trials of obefazimod to treat moderate-to-severe ulcerative colitis (UC).

While the UC market is crowded with multiple treatments from large companies, obefazimod stood out last year because the phase III trial results appeared to demonstrate the drug could control symptoms of the disease without weakening the body’s immune system. In the two trials, named ABTECT-1 and ABTECT-2, obefazimod achieved a placebo-adjusted, 8-week clinical remission rate of 19.3% in ABTECT-1 (p<0.0001) and 13.4% (p=0.0001) in ABTECT-2. Abivax expects to report topline, 44-week maintenance data from the two phase III trials in the second quarter of 2026. We assign a 61.5% overall probability Abivax will report positive data from the two trials.

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Overall Probability: 61.5%

• Market Demand: 58.3%

• Adoption by KOLs: 60.0%

• Clinical Trial Progress: 66.7%

• Experience & Capital: 60.0%

Key catalysts

• Phase III topline maintenance data from obefazimod as a treatment ulcerative colitis (UC)

Key catalyst dates

• 2Q 2026

UC often is diagnosed in late adolescence or early adulthood but onset can occur at any age. UC patients experience inflammation in the large intestines and colon, which brings on chronic abdominal pain, difficulty in passing stools, and on occasion, bleeding stools. UC typically is treated with a combination of drugs, including corticosteroids and biologics such as anti-TNFα blockers and JAK inhibitors, as the condition flares and recedes. Worldwide, the occurrence of UC has increased recently, particularly in Latin America, Asia and Eastern Europe.

In this market, Abivax expects obefazimod initially can be introduced as a second/third line therapy in UC with possible expansion to first-line therapy. Market researchers and analysts project a $4 billion peak sales opportunity for the drug, a heady forecast for a relatively unknown company nine months ago. The clinical success in July, however, enabled Abivax to raise $700 million in capital to navigate the clinical and regulatory landscape ahead.

Why such enthusiasm for obefazimod? About 48% of patients, or 300 total, in the ABTECT trials previously did not respond to advanced UC treatments. The remaining patients in the trial were treatment naïve. Thus, the statistical p values largely reflect obefazimod can improve outcomes for hard-to-treat patients. A game-changing therapy? Possibly.

Unlike the current menu of UC therapy that includes TNF inhibitors, anti-integrins, anti-IL-23, and JAK inhibitors, obefazimod works as a first-in-class, oral miR-124 enhancer. Obefazimod enhances expression of microRNA miR-124. This enhanced expression of miR-124 stabilizes macrophages and T helper 17 (Th17) cells by downregulating pro-inflammatory signaling pathways. Obefazimod is not a messenger RNA (mRNA) drug.

In effect, obefazimod works as a negative regulator of the pro-inflammatory cells that bring on UC and its close cousin, Crohn’s disease, according to NIH publications. The drug lessens the active inflammation in the cells of UC patients and promotes a more quiescent, less pathogenic state for the patient. The ability to broadly tamp down inflammation explains obefazimod’s potential to work across a range of UC patients who either have received treatment or are treatment naïve.

THE EDGE

In addition to the successful results reported in the ABTECT induction trials, obefazimod stands out because it is a small molecule oral drug. For UC patients, taking a once-a-day drug is far preferable to managing the condition than, for example, a subcutaneous injection every 4 to 8 weeks as required with Johnson & Johnson’s (NYSE:JNJ) Tremfya (guselkumab). Tremfya was approved last year.

Moreover, drug makers are well aware of the long-term revenue streams possible for treatments of UC, Crohn’s disease and inflammatory bowel disease (IBD). Obefazimod potentially could emerge as a treatment in all three conditions because of its apparent ability to tamp down inflammation. A buyout rumor already is circling around Abivax.

In December 2025, reports surfaced that Eli Lilly and Company (NYSE:LLY) wanted to bid $15 billion Euros ($17.5 billion) for Abivax. As a comparison, Merck (NYSE:MRK) In April 2023 agreed to pay $10.8 billion for Prometheus Biosciences. Prometheus’ drug Tulisokibart is designed to treat UC and Crohn’s disease; it has yet to gain FDA approval. Merck made the bid following strong phase III results for Tulisokibart in December 2022.

As Abivax plans to release phase III data before July 2026, more bidders may make serious offers following the data, especially as Abivax essentially is a one-drug company. One drug companies often are viewed with skepticism because management often is hesitant or inexperienced. Not so at Abivax.

Abivax CEO Mark de Garidel previously led Corvidia Therapeutics through a successful phase IIb trial and then in June 2020 sold the company for $2.1 billion to Novo Nordisk, including $725 million upfront. Before taking over at Abivax, de Garidel was CEO at Cincor Pharma between July 2021 and February 2023. AstraZeneca bid $1.8 billion for Cincor in February 2023, contingent on Cincor achieving certain milestones.

If the 44-week data is positive, Abivax expects to seek approval for obefazimod as a UC treatment with the FDA and with the European Medicines Agency (EMA). While both agencies have strong guidelines for approval, the EMA is not in the same state of chaos as the FDA. A faster approval by the EMA would not be unexpected, placing higher urgency on potential bidders. As with confirmatory 44-week data, obefazimod approval by the EMA would lift the asking price for Abivax.

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Disclosures:

1. Initiating coverage and a position in ABVX. Biotech Currents may close its position prior to or following the expected catalyst date.

Thanks to recent science and education women now know there are multiple forms of breast cancer and more men recognize the importance of early screening as different types of prostate cancer emerge. Such stratification now extends to the rare condition narcolepsy.

Alkermes plc (NASDAQ:ALKS) wants to build a strong franchise in narcolepsy. To that end, Alkermes last year sought to buy Lumryz, an extended release, single-dose formulation of sodium oxybate from Avadel Pharmaceuticals plc. Lumryz was introduced to compete with Jazz Pharmaceuticals’ (NASDAQ:JAZZ) sodium oxybate formulation Xyrem, which had to be administered twice during nighttime hours. After a surprise bid from H. Lundbeck A/S, Alkermes upped its offer to $2.37 billion and finalized the transaction in November 2025.

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Calendar year 2025, what a year! Whiplash from tariffs to HHS turbulence to FDA dysfunction to a reduction in vaccine administrations. BioPharma held up selectively, especially Big Pharma who set up preferred deals to administer GLP-1 drugs direct to patients. On the growth company level, the result was more topsy turvy.

On the topsy level, Vera Therapeutics, Inc. (NASDAQ:VERA) late last year reported positive phase III results for atacicept in a basket trial to treat extended populations of IgA Nephropathy (IgAN). At the same time the data was reported, Vera disclosed atacicept data was published in The New England Journal of Medicine.

When Biotech Currents issued a report on the atacicept catalyst in November 2025, the company’s stock traded at $29.71 per share. After the data and NEJM publication the stock briefly traded above $50 per share before settling in the $45-50 per share range in January.

Zenas BioPharma, Inc. (NASDAQ:ZBIO) appeared headed for a similar upward trajectory with a phase III trial of its autoimmune drug obexelimab. Two funding deals in the autumn of 2025 plus promising obexelimab data in a phase II multiple sclerosis trial pushed the company’s stock price to ~$43 per share in late October. Then on January 2, 2026, Zenas reported phase III obexelimab results to treat IgG4-related disease (IgG4-RD). Though the drug met the primary endpoint with a statistically significant and clinically meaningful ~56% decline in the risk of IgG4-RD flare compared to placebo, analysts discounted the results because of a cross-trial comparison with Amgen Inc.’s (AMGN) Uplinza (inebilizumab-cdon). Zenas’ stock crashed more than 50% on the data release on January 2 to $16.61 per share.

When announcing the data, Zenas remained confident about obexelimab’s prospects, declaring it would file a BLA for the IgG4-RD indication by the second quarter of 2026. Some investors appear to accept Zenas’ claim that obexelimab has a different mechanism of action than Uplinza as the stock has climbed higher than $20 per share in the past three weeks.

While Biotech Currents expects the differentiation of obexelimab will hold up and the drug will reach the market, it is closing its coverage of Zenas at this time. Biotech Currents plans to exit part of its position in Zenas. When Biotech Currents first reported on the Zenas catalyst in September 2024, the company’s stock traded at $19 per share.

On the turvy side, Rezolute, Inc.’s (NASDAQ:RZLT) dependence on RZ358 (ersodetug) to treat congenital hyperinsulinism led the company to a position, as forecast by Biotech Currents, where it likely would seek capital of the last resort. Rezolute’s phase III trial of the drug to treat hyperinsulinism did not meet the primary endpoint nor key secondary endpoints, sending the stock down 90% to $1.40 per share on December 11. When Biotech Currents first reported on the Rezolute catalyst in July 2025, the company’s stock traded at $6.38 per share. Though the stock has rebounded in the past month to ~$2.73 per share, Biotech Currents is closing its coverage and exiting most of its position in Rezolute.

CRISPR Therapeutics AG (NASDAQ:CRSP) reported phase I/II data on December 22 for CTX112 drug sending the stock up 3.6% from the previous day’s close. The early data was not expected to be a strong catalyst for CRISPR but it did provide a peek into the company’s future pipeline.

CRISPR’s stock rose higher in the fall of 2025, partly due to rumors of an impending buyout. The rumors remain reasonably strong but with no deal imminent CRISPR shares fell ~17% from the time of the Biotech Currents report on October 6, 2025. Biotech Currents is exiting part of its position in CRISPR but will place part of its holdings in a pullback portfolio as the buyout prospects of CRISPR likely will remain strong throughout 2026.

The FDA approved Kura Oncology, Inc.’s (NASDAQ:KURA) ziftomenib on November 13, 2025. After the approval, the company’s stock rose 18% from the time of the Biotech Currents report to the close of the catalyst on November 28.

Disclosures

1. Biotech Currents is discontinuing coverage and exiting part of its positions in VERA, ZBIO, RZLT, CRSP and KURA.

2. Proceeds from the closing of this position likely will be distributed into investments in other pending catalysts, unless explicitly stated otherwise.

3. Should any pending catalysts for other companies in Biotech Currents‘ coverage be realized, proceeds from the closing of those positions will likely be distributed into investments in all other pending catalysts, unless explicitly stated otherwise.

4. Should Biotech Currents decide to make any interim buy or sell decisions, or any suspensions for any pending catalysts, you may be notified of any such moves via email.

A little-followed oncology developer vaulted into the spotlight after posting standout Phase III results in a rare tumor indication, igniting a sharp move in the stock and resetting investor expectations. The asset, acquired through a recent corporate reshuffling, now sits at the center of a well-capitalized push toward a regulatory filing, backed by a leadership team with a deep track record of advancing late-stage therapies and executing high-profile transactions. Fresh funding secured after the data readout positions the program for a near-term submission as confidence builds around its path to approval.

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A late-stage kidney disease program just cleared a trio of important milestones, putting it on track for a potential commercial debut next year. The therapy delivered strong Phase III results in a rare autoimmune condition that progressively damages the kidneys, with findings published in a top-tier medical journal and a regulatory submission now filed under the Accelerated Approval pathway. Developers are also pushing the platform into additional glomerular diseases through a mid-stage study designed to expand its reach beyond the initial patient population.

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The race to develop next-generation treatments for acute myeloid leukemia is tightening as one recently approved therapy faces safety questions, while another nears its own FDA decision. With a competing candidate now under review and showing cleaner cardiac safety data, the odds of regulatory success appear favorable, setting the stage for a pivotal inflection point in this high-stakes therapeutic niche.

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Companies with mature science such as kinase inhibitors and “zombie” biotechs – public companies that remain listed despite numerous failures – rarely thrive in the biopharma M&A market. Bidders typically prefer a company with an approved drug or a drug near approval, which can be tucked neatly into revenue and profit streams. A promising, specific kinase inhibitor for a certain cancer may attract some attention. More often, the drug is destined to become a lonely JAK (just another kinase) because the revenue potential is too low.

And even though a zombie may hold twice the cash of its market cap, few bidders immediately circle for untapped science potential after a drug crashes badly in the clinic. Reverse mergers are the norm, when managements and boards finally are forced to relent. This year’s Biotech Currents selections reflect both ends of the spectrum.

Genmab A/S’s (NASDAQ:GMAB) recent $8 billion cash bid for Merus N.V. (NASDAQ:MRUS) reflects the desire to pull in a new revenue stream via Merus’s cancer drug petosemtamab. Biotech Currents issued a report on a petosemtamab trial in May 2025, forecasting a positive outcome for the petosemtamab plus Keytruda (pembrolizumab) to treat in head & neck cancer.

The data were positive and Merus’s shares climbed 31.5% on the news. Genmab then bid for Merus on September 29, 2025, for $97 per share. The companies expect the deal to close early next year. Upon closing, the return from the original May 2025 Merus stake will be 115%.

Biotech Currents also issued a report on the Genmab’s internal drug, BNT312/GEN1042, to treat head & neck cancer on September 16. As Genmab appears to be placing an emphasis on petosemtamab, Biotech Currents is exiting its position in Genmab.

Two other companies recently attracted offers after clinical success. Novartis AG (NYSE:NVS) bid $1.4 billion for Tourmaline Bio, Inc. on September 9, following Tourmaline’s successful data from its phase II trial of pacibekitug to treat atherosclerotic cardiovascular disease (ASCVD). Tourmaline announced the results in June 2025 and Novartis bid three months later. The Novartis offer at $48 per share represents a 172% increase from the time of the Biotech Currents original report in March 2024.

Johnson & Johnson (NYSE:JNJ) reportedly is in talks to buy Protagonist Therapeutics (NASDAQ:PTGX) to add Protagonist’s icotrokinra, a drug to treat immune diseases, including plaque psoriasis and ulcerative colitis. The two companies are in a partnership to develop icotrokinra. J&J reportedly owns the exclusive rights to commercialize the product. Protagonist also is developing rusfertide (PTG-300) to treat the rare blood cancer polycythemia vera (PV) with Takeda Pharmaceutical (NYSE:TAK). The companies reported positive data from a rusfertide trial in March 2025. Protagonist’s stock has climbed 66% since the original rusfertide report one year ago.

In contrast to rich buyout offers, MoonLake Immunotherapeutics (NASDAQ:MLTX) and aTyr Pharma, Inc. (NASDAQ:ATYR) expect to rely on significant cash holdings to regroup following devastating late-stage clinical failures.

MoonLake, with a current market cap of $639 million, held $425 million in cash and short-term securities as of June 30, 2025. The company’s stock cratered on October 17, falling 76.8% after the company reported very disappointing data from two phase III trials of sonelokimab to treat the rare skin disorder Hidradenitis suppurativa. The company is evaluating a clinical path forward for sonelokimab, its primary asset.

aTyr Pharma likewise targeted a rare disease, pulmonary sacrodiosis. Its phase III trial testing efzofitimod to treat pulmonary sacrodiosis did not meet the primary endpoint of change from baseline in mean daily oral corticosteroid (OCS) dose at week 48. The company claims clinical benefit for efzofitimod was observed across multiple study parameters. The company’s shares fell 78% on the news. aTyr held $101 million in cash and available-for-sale investments as of June 30. Following the phase III data, aTyr’s market cap has ranged between $90 million and $95 million. The company expects to hold a meeting with the FDA within the next few months to determine if it can advance efzofitimod without conducting another phase III trial.

Glaukos Corporation (NYSE:GKOS) on October 20 announced the FDA had approved its Epioxa HD/Epioxa New Drug Application (NDA) to treat keratoconus, a rare eye disease that affects the structure of the cornea. Unfortunately, an analyst report surfaced on the same day that Glaukos management will meet with five Medicare administrator contractors on November 12. Though the analyst characterized the meeting only as a sharing of information, investors had doubts, sending the stock down ~10%.

Biotech Currents issued a report on the Epioxa decision in June 2025.

Glaukos shares have slid ~25% since the time of the report.

Disclosures

1. Biotech Currents is discontinuing coverage and its positions in GMAB, GKOS, ATYR and MLTX.

2. Proceeds from the closing of this position likely will be distributed into investments in other pending catalysts, unless explicitly stated otherwise.

Considering the long list of drug trial failures to treat HD, the spike is not surprising. HD is inherited from a patient’s parents and there are no direct treatments for HD or its symptoms. Adult onset of HD typically begins between the ages of 30 and 50; most HD patients live 15 to 20 years after signs and symptoms appear.

Given the many failures in HD as well in gene therapy drugs in general, the likelihood of success for the gene therapy AMT-130 was equal to the expectation of failure. So how did uniQure overcome the 50/50 odds?

Look first at the clinical consensus that there is no direct treatment for HD. With no approved treatments, any hint of success would be embraced by researchers, physicians and regulators. The history of HD efforts is dismal.

Roche tested its antisense drug tominersen to treat HD, while two separate antisense drugs from Wave Life Sciences failed in phase III and phase II/III trials, respectively. Tominersen is a snippet of DNA researchers believed would block production of the mutant protein, huntingtin, which causes HD. Roche halted a phase III tominersen trial in 2021 because of concerns about the drug’s risk/benefit profile. Wave’s drugs likewise demonstrated no significant benefit in early-stage trials, influencing the company to discontinue its efforts.

Sage Therapeutics, which focused on drugs that act on the brain’s natural signaling mechanisms, attempted to advance dalzanemdor to treat cognitive impairment in HD. But after the drug failed to meet primary and secondary endpoints in a phase II trial, Sage halted development.

Accordingly, uniQure’s AMT-130 gene therapy still was regarded as a long shot, even though uniQure succeeded in introducing the gene therapy Hemgenix (etranacogene dezaparvovec) as an approved treatment for hemophilia B. Because HD is a genetic disease, gene therapy would appear to be the appropriate modality to treat HD. Yet AMT-130 stumbled. The FDA issued a hold on the phase I/II AMT-130 trial because of one case of hepatocellular carcinoma in an early study. In 2023, the phase I/II trial data pointed to a temporary increase in a biomarker for neurodegeneration. Both red flag events delayed the progress of the trial and cast doubt on the prospects for AMT-130.

uniQure, however, persisted and one year ago the company released 24-month data that demonstrated a statistically significant, dose-dependent slowing in disease progression measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) in patients receiving the high dose of AMT-130. The slowing in disease progression was compared to a propensity score-weighted external control (p=0.007).

Notably, the external control data was culled from the Enroll-HD data set. Enroll-HD collects natural history data from HD individuals and control patients. uniQure, as with other companies researching HD drugs, compared AMT-130 to biosamples from Enroll-HD. Specifically, the comparisons involved 940 samples for high-dose AMT-130 and 626 for the low dose of the drug.

In the most recent data, uniQure analyzed clinical outcomes for 29 patients treated with AMT-130. Seventeen received the high dose, while 12 received the low dose. Twelve patients per dose group had attained 36 months of follow up and topline data were reported on the 24. The primary endpoint of slowing disease progression was met as measured by cUHDRS (p=0.003). The key secondary endpoint of Total Functional Capacity also was met at p=0.033.

As a result, the 36-month data reads out better than the 24-month data reported one year ago, prompting investors to rush in. One London physician declared AMT-130 likely can add four years to the life expectancy of an HD patient. That form of clinical conviction, as well as the data, led investors to overcome justifiable skepticism.

Biotech Currents maintained a speculative forecast on the AMT-130 catalyst because of the risk from past HD drug efforts. Many clinical signals pointed to success, including a meeting with the FDA in November 2024 in which the agency informed uniQure the 36-month data could serve as the primary basis for a BLA submission for AMT-130 under the accelerated approval pathway. uniQure’s stock shot up on the news but caution flags still fluttered around the progress of AMT-130. Even a minor setback with the 36-month data likely would have held back AMT-130 and sent uniQure’s shares downward.

Now uniQure expects it can receive the necessary regulatory approvals to launch AMT-130 in the US and Europe next year.

Approximately 75,000 people in the US, the European Union and the United Kingdom have HD, according to uniQure. HD afflicts an estimated 3 to 7 persons per 100,000 people of European ancestry.

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IDEAYA Biosciences, Inc. (NASDAQ:IDYA) said on September 8 that 76% (16/21) of uveal melanoma (UM) patients who were treated with the company’s drug darovasertib achieved a ≥20% ocular tumor shrinkage by product of diameters in a phase II trial. The tumor shrinkage endpoint is the response definition proposed for the IDEAYA’s phase III registration-enabling OptimUM-10 trial.

IDEAYA presented the preliminary data at the company’s 10-year anniversary day. Additional data from the phase II OptimUM-09 trial will be presented at a mid- October scientific conference in Berlin.

UM is a rare, aggressive, poorly diagnosed form of melanoma cancer, which typically manifests in the eyes of adults. Unfortunately, about half of new patients diagnosed will develop metastatic UV melanoma (MUM). When the cancer spreads beyond the eye to other organs, the MUM survival rate is only one year.

In January 2022, Immunocore Holdings plc (NASDAQ:IMCR) introduced tebentafusp, branded as KIMMTRAK, as the first approved drug to treat MUM.

Tebentafusp, however, is restricted to patients with the HLA-A*02:01 allele. In spite of the very narrow MUM population eligible for treatment with tebentafusp, the drug recorded $238.7 million in sales in 2023 and $310 million in 2024.

In contrast to tebentafusp, IDEAYA expects it can place darovasertib first in the neoadjuvant setting, where the drug could be used prior to surgery. This indication potentially positions darovasertib for use by estimated 12,000 patients annually in North America, Europe, and Australia. Tebentafusp may be suitable for perhaps 10% of that total once they develop MUM.

IDEAYA started the registration-enabling OptimUM-10 trial of darovasertib in the second quarter of 2025.

In June 2025, Biotech Currents issued a report on the phase II darovasertib catalyst. At the time of the report, IDEAYA’s stock traded in the mid $22 range. At the end of trading on September 8, IDEAYA’s median price for the day was $25.46 per share, reflecting an approximate 13% increase for the darovasertib catalyst.

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Disclosures

1. Biotech Currents is discontinuing coverage and exiting part of its position in IDYA. The remaining position will be placed in a pullback portfolio, likely until the additional data is released in mid-October 2025.

The FDA issued a complete response letter (CRL) on August 28 for Telix Pharmaceuticals Ltd.’s (OTCPK:TLPPF) TLX250-CDx (Zircaix), an imaging agent designed to non-invasively diagnose and characterize clear cell renal cell carcinoma (ccRCC), one of the most aggressive subtypes of kidney cancer. Telix had submitted a biologic license application (BLA) for TLX250-CDx.

The CRL cited deficiencies relating to the chemistry, manufacturing, and control (CMC) processes for TLX250-CDx. The FDA also requested additional data to establish comparability between the drug product used in the Telix’s phase III clinical trial and the scaled-up manufacturing process intended for commercial use. In addition, the FDA documented notices of deficiency (Form 483) issued to two third-party manufacturing and supply chain partners that will require remediation prior to resubmission.

Australia-based Telix said it believes the FDA concerns are addressable and the company will begin to answer the FDA immediately. Telix also requested a Type A meeting with the FDA as soon as possible.

While several deficiencies were cited by the FDA, the CMC issue frequently is a red herring put out by the agency, often when the agency did not have enough time or resources to evaluate the BLA properly. Notably, Telix’s Illuccix is an approved prostate cancer imaging agent and has been marketed in the US for several years. The FDA also issued a CRL in April 2025 for the company’s application to market TLX101-CDx (floretyrosine F18; Pixclara), an imaging agent designed to diagnose the brain cancer glioma.

Telix appears to have been caught in the FDA crosshairs as an ex-US company seeking to expand aggressively in the US and the looming, unresolved tariff predicament affecting all ex-US biopharma and medical technology companies. The FDA’s recent CRL decisions signal a clear delay-or-stall strategy to prevent allowing new treatments to enter the marketplace, particularly if the applicant is not US-based. Once – or if – the tariff policy is resolved, the FDA decisions may become more predictable.

At the same time, Telix did not distinguish itself when in July 2025 it said it received a subpoena from the US SEC seeking information related to disclosures regarding the development of its prostate cancer therapeutic candidates. The subpoena request did not extend to Telix’s commercial and late-stage products including Illuccix, Gozellix, Zircaix, Pixclara and Scintimun.

Biotech Currents is discontinuing coverage and exiting its position in TLPPF. At the time of Biotech Currents’ May 2025 report on the TLX250-CDx catalyst, Telix’s stock traded at ~$18.52 per share.

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At the end of trading on August 28, Telix’s median price for the day was $10.40 per share, reflecting an approximate 43% decline for the TLX250-CDx catalyst.

Disclosures

1. Biotech Currents is discontinuing coverage and exiting its position in TLPPF.

The FDA issued a complete response letter (CRL) on August 19 for PTC Therapeutics, Inc.’s (PTCT) vatiquinone to treat the rare neuromuscular disorder Friedreich's ataxia (FA).

In line with recent FDA responses, the reason cited in the CRL was non-specific. The CRL stated, “substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission,” according to PTC Thera.

In February 2025, the FDA awarded vatiquinone a Priority Review for its new drug application (NDA), seemingly acknowledging the drug’s efficacy demonstrated in a phase III trial that had designated a pre-specified endpoint known as the mFARS upright stability subscale. mFARS stands for modified Friedreich’s Ataxia Rating Scale. While vatiquinone did not statistically meet the primary endpoint of overall mFARS score (p=0.14), the FDA did not have to grant a Priority Review to vatiquinone.

A Priority Review is granted to a drug that is intended to treat a serious condition and would provide a significant improvement in safety or effectiveness over currently available treatments. Biogen Inc.’s (NASDAQ:BIIB) Skyclarys (omaveloxolone) is the only other drug available to treat FA. Skyclarys was introduced to the market in February 2023.

Notably, Vinay Prasad resigned in late July 2025 as director of the FDA’s Center for Biologics Evaluation and Research (CBER). Prasad was in the job for only three months and appeared to run astray from the “right to try” effort advanced by many supporters of President Trump. The “right to try” agenda appears to support moving drugs with potential, as well as certain risks, into the market to treat rare and debilitating conditions. Prasad then curiously returned to the CBER post in mid-August and the vatiquinone CRL was issued days later.

At the time of BioTech Currents’ early August report on the vatiquinone catalyst, PTC’s stock traded at ~$52 per share.

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Today, PTC is trading near $51 per share, essentially a near break-even outcome for the vatiquinone catalyst.

Disclosures

1. BioTech Currents is discontinuing coverage and exiting its position in PTCT.