Analysts Highlight Notable H1/26 Catalysts in Biotech
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RBC Capital Markets' analysts presented the near-term catalysts for its biotech names under coverage and assigned each one upside and downside projections, in a Jan. 5 equity research note.

"We still see a number of attractive catalyst setups for H1/26, many with differentiated technologies that could spark an inflection in commercial expectations and/or potential takeover interest on positive results," the analysts wrote.

They pointed out that selectivity likely will become more important as the year progresses due to momentum in the sector from mergers and acquisitions (M&A) and capital flow tailwinds potentially starting to ease.

Here are the upcoming catalysts and RBC analysts' take on them:

Xenon Pharmaceuticals Inc. (XENE:NASDAQ)

Target: US$44.49; Rating: Outperform

Given azetukalner's greater-than-appreciated Phase 2 to Phase 3 translatability in the epilepsy space and its market opportunity, meaningful upside remains for Xenon on potentially positive (and expected) derisking Q1/26 results (upside +35%, downside -45%).

Karyopharm Therapeutics Inc. (KPTI:NASDAQ)

Target: US$7.19; Rating: Outperform

Though binary and based on a relatively small Phase 2 data set, the potential for transformative upside on favorable Phase 3 selinexor myelofibrosis data makes for a high-reward set-up (+200%/-75%) for Karyopharm going into March data.

Biogen Inc. (BIIB:NASDAQ)

Target: US$177.82; Rating: Outperform

In the share price range of the US$170s, Biogen does not appear to be baking in much, if any, value for its presymptomatic Alzheimer's opportunity. Positive Eli Lilly & Co. (LLY:NYSE) data would indicate a 10fold increase in long-term total addressable market for the class and could positively impact Biogen's current Leqembi indication, potentially affording a 25% uplift to BIIB's shares (versus -10% to -15% downside).

Mind Medicine (MindMed) Inc. (MNMD:NASDAQ)

Target: US$13.64; Rating: Outperform

Topline data from Mind Medicine's first pivotal trial in H1/26 should recapitulate MM120's impressive Phase 2b efficacy/safety in generalized anxiety disorder (+80%/-40%). This plus data from other late-stage psychedelics should help generate a resurgence of interest in the space and lead to additional upside for MindMed.

Sionna Therapeutics Inc (SION:NASDAQ)

Target: US$39.21; Rating: Underperform

The analysts admit that Sionna's US$2 billion (US$2B) valuation is too optimistic for the risks inherent in developing pre-proof of concept small molecules in a historically challenging cystic fibrosis space with unclear preclinical data translatability. Yet, they predict 75% upside from the biotech's mid-2026 SION-719+Trikafta Phase 2 results (downside -80%).

United Therapeutics Corp. (UTHR:NASDAQ)

Target: US$496.72; Rating: Outperform

This company is a leader in rare lung diseases with a US$2 billion blockbuster franchise with its lead drug Tyvaso. Despite the debates around its potential new opportunity in idiopathic pulmonary fibrosis (IPF), RBC analysts are bullish on it.

"We think Tyvaso addresses a significant unmet need in IPF, and we see 25% upside from current levels if the second pivotal study TETON-1 read outs positively in H1/26," they wrote.

Ocular Therapeutix Inc. (OCUL:NASDAQ)

Target: US$11.82; Rating: Outperform

This biotech is one of RBC's Top Picks in 2026. The first of the company's two pivotal studies in wet age-related macular degeneration (wAMD), SOL-1, is on track to read out in Q1/26. RBC analysts think the superiority study is "highly likely to work" given RBC's Elements analysis shows that a 50% delta between treatment and control is achievable at nine months. The upside into the event skews favorably, added the analysts, who see 40% weighted average upside from current levels.

EyePoint Inc. (EYPT:NASDAQ)

Target: US$17.48; Rating: Outperform

The biotech's first of two Phase 3 trials in wAMD is slated to read out in mid-2026. Favorable results are expected given the historical 78% success rate of non-inferiority trials and prior Phase 2 results. From current levels, RBC analysts forecast 90% upside on Phase 3 success. They think Ocular Therapeutics is positioned to file first and gain approval in the indication but how this might affect EyePoint remains unknown.

Viridian Therapeutics Inc. (VRDN:NASDAQ)

Target: US$30.68; Rating: Outperform

Viridian is poised to become a commercial stage company with acceptance of its recent Biologics License Application for veligrotug in active and chronic thyroid eye disease (TED); its PDUFA date is June 30, 2026. Now, RBC analysts are focused on the Phase 3 readouts pertaining to the biotech's subcutaneous delivery program, the first expected in Q1/26, which could lift the current share price 35%.

"With fewer subcutaneous competitors to VRDN-003, we think the TED market is lining up to become an Amgen Inc. [(AMGN:NASDAQ); Target: US$327.64; Rating: Outperform]-Viridian duopoly for several years to come," they wrote.

Cytokinetics Inc. (CYTK:NASDAQ)

Target: US$61.73; Rating: Outperform

The risk-reward for Cytokinetics' ACACIA study is looking increasingly compelling because it could double the size of the market based on recent epidemiological estimates of obstructive hypertrophic cardiomyopathy (oHCM/nonobstructive hypertrophic cardiomyopathy (nHCM).

"With the commercial opportunity in oHCM providing downside protection, we see as much as 40% upside on a statistically significant win in ACACIA," noted the analysts.

REVOLUTION Medicines Inc. (RVMD:NASDAQ)

Target: US$79.02; Rating: Outperform

RBC analysts have high conviction in REVOLUTION's clinical and regulatory paths this year, they wrote. This is due to the impressive roughly twofold overall survival and progression-free survival benefits over historical standard of care in pancreatic cancer and the many favorable regulatory designations. Analysts see potential for 25% upside on success.

Compass Pathways Plc (CMPS:NASDAQ)

Target: US$6.55; Rating: Outperform

The probability of success with the COMP006 trial in treatment-resistant depression (TRD) is high (80%), the analysts wrote, because previous COMP005 and Phase 2 data showed clear evidence the drug is active. Because the market is underappreciating the TRD opportunity, there is potential to double on strong data. Success could derisk the regulatory route forward and enable a path to commercialization.

Axsome Therapeutics Inc. (AXSM:NASDAQ)

Target: US$178.69; Rating: Outperform

Axsome had a strong run in 2025, but upside remains on the potential approval of AXS-05 in Alzheimer's disease agitation; AXSM could rise to the US$180 range or higher on this US$1.5–3B opportunity.

"The recent priority review could enable seven to eight months of launch in 2026, and other pipeline programs (binge eating, shift work, etc.) may deliver upside optionality," the analysts wrote.

WAVE Life Sciences Ltd. (WVE:NASDAQ)

Target: US$15.95; Rating: Outperform

Arrowhead Pharmaceuticals Inc. (ARWR:NASDAQ)

Target: US$67.79; Rating: Outperform

If upcoming updates on the biotechs' Activin E programs show even modest weight loss, both companies, particularly Wave, could become potential M&A candidates. Genetics for the target are clear. Preclinical data indicate weight loss should be just a matter of time and/or higher doses and/or enrolling patients with higher body mass indexes.

Other companies the RBC analysts are bullish on include:

Ascendis Pharma A/S (ASND:NASDAQ)

Target: US$213.29; Rating: Outperform

The RBC analysts like Ascendis, citing potential from Yorvipath in chronic hypoparathyroidism, the TransCon CNP launch within weeks and "another name that we think may offer M&A optionality," they wrote.

Dyne Therapeutics Inc. (DYN:NASDAQ)

Target: USUS$18.50; Rating: Outperform

DYN is trading at a portion of what Novartis AG (NVS:NYSE) paid for RNA technology.

Ionis Pharmaceuticals Inc. (IONS:NASDAQ)

Target: US$79.67; Rating: Outperform

Ionis has several catalysts ahead, including the potential approval and launch of Olezarsen for severe hypertriglyceridemia program and executing its Phase 3 trial in TTR-cardiomyopathy.

Ultragenyx Pharmaceutical Inc. (RARE:NASDAQ)

Target: US$23.60; Rating: Outperform

The biotech expects to release topline data in H2/26 from the pivotal Phase 3 Aspire trial in Angelman syndrome.

Bausch Health Companies Inc. (BHC:NYSE)

Target: US$7.21; Rating: Sector Perform

Topline data from the Phase 3 RED-C trial for the rifaximin soluble solid dispersion formulation are due out in Q1/26. With strong results, 25–30% upside is possible if the all-cause mortality data show clinical relevance and statistical significance. If the trial fails, downside could be -5% to -10%.

Ligand Pharmaceuticals Inc. (LGND:NASDAQ)

Target: US$190.11; Rating: Outperform                                  

A possible decision on the Filspari supplemental new drug application for focal segmental glomerulosclerosis (FSGS) could come on the PDUFA date, Jan. 13, 2026. If greenlighted, Filspari would become the first and only U.S. Food and Drug Administration-approved FSGS treatment. An approval would be significant for Ligand because it is entitled to 9% royalties on net Filspari sales. An approval delay carries a possible downside of -3%. If the market prices in a removal of the FSGS indication, downside could be as much as -15%.

Highlights and Lowlights

The RBC analysts highlighted six catalysts that they believe have favorable set-ups. They are readouts for these trials: Compass' Phase 3 in TRD, MindMed's Phase 3 in generalized anxiety disorder, EyePoint's Phase 3 in wAMD, Karyopharm's Phase 3 in first-line myelofibrosis, Ocular's Phase 3 in wAMD, and Xenon's topline in focal onset seizures.

Conversely, the analysts presented four upcoming catalysts they deem worth avoiding because their set-ups are "less favorable." These catalysts are: Sionna's Phase 2 sweat chloride data readout in cystic fibrosis; Lexeo Therapeutics Inc.'s [(LXEO:NASDAQ); Target USUS$9.61; Rating: Outperform] update on LX2020; Wave Life Sciences' six-month update on WVE-007; and Sarepta Therapeutics Inc.'s [(SRPT:NASDAQ; Target: USUS$21.31; Rating: Sector Perform] update on myotonic dystrophy type 1/facioscapulohumeral muscular dystrophy.

These catalysts are for companies whose valuations have appreciated significantly and now are "baking in much more optimism for success," added the analysts.