Conference Call Notes
listened to a bunch of firesides at Citi, Wells Fargo, and Cantor this week. Here are some notes I took.
SNDX - Both products are ramping well. With Revuforj (+43% sequential growth), the ramp is being driven by new patient starts and improved duration. Awareness is also improving. They expect >50% share in KMT2A, with PDUFA in October. Earlier-stage programs in BEAT AML and SAVE AML more than double the market opportunity. With NIKTIMVO, Incyte is the ideal partner, poised for growth due to a high unmet need. Profitable last quarter. OpEx remains flat as revenues increase = high potential for profitability in the coming quarters.
LXEO - The LXEO advantage in safety is (much lower) dosing. Dosing "a full 2 log" lower than DMD programs in the FA program (1e12). PKP2 dose is higher, but they are using a cardio-specific promoter, so they think it will be equally as safe. LXEO manufacturing (SF9 baculovirus production system) results in significantly lower "empty" capsides vs. HEK-based systems (a significant problem for AAV). They called this a "Goldilocks" capsid strategy. High-efficiency manufacturing allows for lower doses, along with the use of specific promoters, which creates an ideal therapeutic window (safety vs. efficacy). The CEO sounded very confident in the pivotal path for AA with the FA program, both on FXN production (any) and LVM (must be > 10%, but so far they are seeing > 25%). Update from the existing Phase 1/2 FA patient study in early 2026.
PTGX - On Rusfertide, the CEO thinks the Phase 3 data are "outstandingly positive" (noted plenary session at ASCO) and won them a breakthrough designation. So far, FDA interactions have been very positive. Also, working well with Takeda, and they think this is a $1-2B peak sales drug. Citi analyst asked about "50/50 vs. Opt-Out" pending decision. The CEO noted that "Opting out" is more front-end loaded and "very incentivizing" on cash payments to the company, whereas picking 50/50 is more back-end loaded. The CEO noted that, "If it's a $1B drug, we opt out, but if it's a $5B drug, we'd want to stay in." However, that decision is 9-12 months away, as it takes effect 4 months after the NDA filing. The CEO discussed opting out to provide $425M in cash to PTGX, enabling them to fund other pipeline assets. I specifically view their decision to opt out as a sign of confidence in the oral IL-17 or obesity programs, which makes me think JNJ might then be more interested in taking out PTGX. Conversely, if PTGX opts in, that suggests more confidence in Rusfertide and (perhaps) less in the pipeline (the oral hepcidin as well). My hunch - they opt out. Turning to Icotrokinra, the CEO noted "outstanding data" and JNJ's focus on "the big-4" indications of UC, CD, PsO, and PsA. JNJ recently guided to $1.4B in sales for Ico in 2027 (yr-1 of the launch = wowza!) The CEO said the oral IL-17 could loop in HS and AnSp. CEO stressed that PTGX can extract more value from these early-stage pipeline drugs (IL-17, GLP-1RA, and Hepcidin) if they advance them farther along in development vs. where they partnered Ico with JNJ - this is what makes me think they opt-out of Rus, get the $425M in cash from Takeda, and then push all these assets forward to create more value then just 50% of Rus commercial.
IDYA - Cantor analyst started off by calling IDYA "one of our highest conviction ideas". The CEO said they will have 9 clinical-stage assets by year-end 2025, with Darov the lead, followed by their efforts in DLL3 and MAT2A. The CEO noted "a sizable dataset" on the DLL3 asset next week. Benchmark for success here would be a cORR rate > 65% (better in the 2L patients) and PFS > 6 months (AMGNs DLL3xCD3 shows 40% cORR & mPFS is 4.3 mo; ZLAB data is 65-70% uORR). IDYA is dosing higher than ZLAB, albeit with a "better" linker (CEO noted its toxin is cleaved internally compared to ZLAB’s internal and external). As I expected, Servier did see the data before signing the OUS deal (and paying $210M to IDYA) on Sep 2nd. Next week, we are looking for the waterfall (tumor shrinkage) data and vision preservation (or improvement) data. Improvement would be a huge win. More data is coming at ESMO in October from the enculeation cohort. The CEO then noted that the FDA already saw these data, after which they awarded breakthrough therapy designation. JNaps Take: Servier saw the PB cohort data and gave them $210M and the FDA saw the EN cohort data and awarded them BTD - seems pretty positive to me!
TGTX - CEO noted they recently completed a $100M buyback (3.5M shares at $28.55/sh). JNap's Take: I believe TG could create more value with $100M than by buying back 2% of its shares (e.g., biz dev), but the stock rose by more than 5% on the news. TG is initiating DTC on Briumvi, so this could help drive uptake in the coming quarters (but true ROI on DTC remains an issue for all biopharma companies). The CEO also noted that Briumvi was priced at a 25% discount to Ocrevus; it sounded like he hinted that there was room to increase this even in the face of a potential biosimilar Ocrevus. CD20 NRx is > 50% of all MS, and TG is capturing (nearly) 1/3rd of the CD20 NRx. The CEO said they have a "pretty high level" of confidence in guidance - said they are the most knowledgeable on their business, so "sell-side consensus" vs. TG guidance seemed to irk Mike a little (after the stock sold off on the Q2 results). TG will provide 2026 guidance early in 2026. Turning to the SubQ, the CEO noted they expect to be a "true SubQ", similar to Kesimpta (relatively painless, at home with an autoinjector) vs. Ocrevus (painful, in clinic with 20 mL). Finally, on the potential for business development, the CEO said they are "actively looking", but it sounds like the best opportunity the CEO sees right now is SubQ, and then ppMS (I don't disagree).
PROK - The CEO walked us through the previously reported top-line data in July. The bilateral vs wait-and-see 2nd injection data suggested bilateral was better, but they will have more data at ASN (Kidney Week) in early Nov 2025 (we knew this already). They are hoping for a "late breaker" presentation along with a full paper (timing remains in question). Nevertheless, for the Phase 3 (PROACT) pivotal study (now enrolling), they shifted focus to later-stage (stage IV) because they believe this is the area with the highest unmet need and where payors are most likely to support uptake. As such, subset data at ASN'25 on these later-stage (eGFR 20-35) is the most important. All patients enrolled who were above 35 eGFR still "count" for the analysis, but all future enrollment will be patients with a 20-35 baseline. The primary endpoint for AA filing is eGFR slope. Data are expected in Q2 2027. The benchmark here would be a 1.5 point improvement vs. sham. The confirmatory endpoint is clinical events (dialysis, transplant, death) - no timeline yet for this read-out (thinking late 2027), and they will not have any of these data when they report the eGFR slope data in Q2 2027.
ETNB - The CEO said, "This is a great time" to be in the MASH space, and that with MDGL and (now, recently) NVO, ETNB thinks awareness will meaningfully increase. But they are confident in their position because Rezdiffra and the GLP1s do not improve fibrosis ("huge unmet need"). Both P3s are enrolling well with "significant clinician" interest, even in the ENLIGHTEN-cirrhosis (F4 population) study. The CEO is confident in the benefits of Pego on top of GLP1RA (including tirzepitide). He also brought up two potential differentiators to AKRO's Efrux: 1) No impact on BMD, and 2) Pego is a liquid, which allows for co-formulation (co-dosing) with GLP1, whereas Efrux is a lyophilized powder. JNap's Take: 1) Maybe, 2) Nah. The ENTRUST P3 in SHTG will read out in Q1 2026. CEO said he still thinks there is an opportunity for Pego in SHTG, but they will need a second P3, and this will likely come after they file the NDA for Pego in MASH. I listened to both presentations at Citi and Cantor. The Cantor analyst asked a lot more questions about the SHTG indication.
ARDX - The CEO reported strong growth with Ibsrela® ($1B peak guidance) and noted some month-over-month growth with Xphozah® ($750M peak guidance), despite the TDAPA/CMS issue. The focus is on communication and awareness, as both products offer an alternative mechanism and serve a significant unmet need. Most of the use is in the refractory/switch market, but they are also seeing some front-line use. JNap's Take: As much as I like to poop on ARDX's CEO for being wildly too bullish, I do agree that both drugs have solid data in big markets, and they have executed well despite the TDAPA/CMS issue.
URGN - The CEO noted that even though Jelmyto® and Zusduri® are very different patient populations, often this is the same urologist, so there is good overlap for the promotion. As such, URGN has expanded from ~55 to ~85 total reps, along with increasing its physician focus. The Wells Fargo analyst asked about the use of Zusduri after a TURBT, and the CEO said, "Well, they can, but that's not our label, so we can't promote for that, and the Doc would also have to be confident in reimbursement". Zusduri is currently reimbursed under a miscellaneous J-code, so it's a little cumbersome (more paperwork) for the community (office) urologist (academic centers don't care about this because they have large admin staffs that handle this stuff). The CEO said, "The demand is there, there's no question about the data, it's all logistics right now." The CEO said there is a lag between PEFs (patient enrollment forms) and actual dosing, and that they are working to reduce this lag and facilitate uptake before the permanent J-code in Jan 2026. The CEO also reminded everyone that the plan is to file for approval of UGN-103 in 2026 and then launch in 2027, at which point they will pull Zusduri off the market and swap in UGN-103. This is all designed to protect and extend the IP to 2041 (Zusduri IP expires in 2031). I listened to URGN at both Citi and Cantor, and the presentations were consistent. The CMO told the same joke to start both chats.
IVVD - COB believes that a "good MAb is going to be very differentiated" from current vaccines, particularly in the immunocompromised population. He then opined that an IM injection for VYD2311 would significantly improve logistics compared to the IV Pemgarda. On the VYD2311 regulatory path: the COB said that the FDA has "acknowledged the totality of the prior work" and suggested that validating the clinical benefit demonstrated with Pemgarda in the P3 CANOPY with VYD23011 would support full BLA approval (not EUA). The Cantor analyst and the COB then discussed ACIP (The Advisory Committee on Immunization Practices) and how guidelines and recommendations can enhance access and uptake for vaccines like VYD2311 compared to other vaccines, touching on Sanofi's Beyfortus® (nirsevimab-alip) for RSV and how it's on a "mega-blockbuster" trajectory in a market that is hundreds of times smaller than COVID-19. JNap's Take: Yes, I agree, but also keep in mind Beyfortus is $500 per and COVID-19 vaccines are only $150-225. That being said, VYD2311 looks pretty analogous to CDTX's CD388 for Flu, and CDTX's market cap (post P2, pre-P3) is $2.7B.
SPRY - The current focus is on expanding coverage, eliminating/reducing prior authorizations, and continuing the "Hello neffy®" DTC campaign (it's all about the jingle). The CEO noted that early adopters are now meaningfully ramping up scripts, and that it "takes time," but awareness and use are increasing. They discontinued the GoodRx coupon ($200 off), which the CEO said was essentially selling the product below cost because some Pharmacists were charging patients the BlinkRx $199 cash price, then applying the $200 off GoodRx coupon to get the cost to $0. However, SPRY was still paying fees to the distributor and GoodRx, resulting in a negative margin on these scripts. Pulling that distribution channel resulted in a dip in scripts that showed up on IQVIA recently (-10% Rx), and this seems to have impacted the stock. The CEO suggested this is a one-time blip, and as that demand transitions to full pay (with coverage) or the BlinkRx $199 cash-pay price, revenues will benefit. The CEO then noted that the average co-pay with coverage is lower for neffy® than the generic EpiPen. The Cantor analyst then asked about the "bolus" expected from the back-to-school sales in the Q3 and the potential for a down quarter (sequentially) in the Q4. The CEO noted that he believes that as they grow market share and coverage, revenues can still grow in Q4 even if scripts are down. JNap's Take: This -10% Rx reported by IQVIA seems to be a bigger issue hitting the stock right now than the Lupin ANDA filing. Expanding (and moving up) formulary coverage (the CEO specifically mentioned PRIME on BlueCross/Blue Shield and Caremark) starting January 2026 should meaningfully improve revenues. There are also non-traditional avenues for neffy® that do not exist with the autoinjector, and they specifically talked about selling to restaurants.
AQST - The FDA informed them that an AdCom was not required before the Anaphylm PDUFA on Jan 31, 2026. This is positive news, especially because AQST has shown outstanding disclosure to investors with its regulatory filings. The CEO noted they submitted data on over 300 patients receiving over 900 doses as part of the NDA filing, including PK data, clinical data, and challenge studies. AQST claims they have the fastest pk of all epinephrine products at ~12 minutes (compared to the IN and IM versions at 15-20 minutes). They also seem well-capitalized right now, having raised $85 million in July 2025, with another $75 million in a RIF coming from RTW if Anaphylm gets approved. JNap's Take: Odds of approval seem high (estimated ~80%). I skimmed the clinical data on AQST's website, and it looks fine. I like the concept of AQST Anaphylm (sublingual epi). Do I like it more than SPRY's neffy® (intranasal epi)? I don't know. I believe each likely has its place, and given that over 30 million people in the U.S. are at risk of severe allergic reactions/anaphylaxis, I think the market is substantial enough (> 5 million TRx last year) for both products to succeed. I'm still working through my valuation model with AQST.
LENZ - The CEO said the launch is underway, with a 3-pronged strategy: 1) Getting Docs on board, 2) Building awareness with patients (through DTC to start in Q1 2026), and 3) Maximizing the patient journey (whatever the hell this means). They have an 88-person sales force targeting 15,000 eye care professionals (ECPs, primarily optometrists). Any patient expressing interest will get a 5-day free sample and a script, with e-pharmacy as the preferred fulfillment channel. This is a massive market - 128M presbyopes with 40M optometrist visits annually. The CEO thinks the early adopters are contact lens wearers, post-LASIK patients, and old people (note: I'm all 3 of these). So far, interest is high (>75% of trial users would continue and suggested use 4-7 days per week). Vuity was a commercial bust because it didn't work very well, lasted only 2-3 hours, and actually made distance vision worse. JNap's Take: I'm eager to try this product, but I think they priced it too high ($79 for a 25-day supply or $198 for a 75-day supply). This is a pure cash-pay market (no coverage), so I think the ramp will be very slow, and with DTC not starting until Q1 2026, I think Q3 and Q4 numbers are going to be very underwhelming, with heavy sampling (high costs and virtually no revenues).
TARS - The CEO noted seeing continued great progress for the Xdemvy® launch, with over 20,000 ECPs having written a script to date. Coverage is >90% across commercial and Medicare. The field force will focus on increasing the depth of prescribing, improving education and awareness, and expanding use into new segments (DB, DES, MGD). The CEO noted 9 million patients are seeking treatment, but the TAM is potentially as large as 25 million. The DTC campaign seems to be working, so TARS will continue and plans to spend $70-80M through year-end 2026. Inventory has been stable. Gross-to-net should decline (improve) to low 40s by the end of the year. Retreatment rates have surpassed 10% and could reach 20% steady-state on an annualized basis. Potential upside opportunities include expansion to Europe and Japan, along with TP-04 for ocular rosacea and TP-05 for Lyme disease.
LQDA - The CEO reported being very encouraged so far with the Yutrepia™launch, with over 900 scripts and 550 patient starts in the first 11 weeks. LQDA is seeing uptake from both new and switch patients, with physicians and patients reporting positive feedback relating to differentiation on dose, delivery, and flexibility (to Tyvaso). This is a $3B inhaled market (up to 150,000 PAH and PH-ILD patients), but the CEO also believes they can capture market share from the $2B oral treprostinil market due to its perceived lack of effectiveness. LQDA thinks that "Having two companies promoting inhaled treprostinil products" will help grow the market. JNap's Take: Yes! With respect to L606 (next-generation nebulized treprostinil), the CEO says the goal is to replicate "infusion-like efficacy" with a twice-daily schedule. LQDA does need to complete a P3 study with L606 before they can file. The CEO believes UTHR's positive TETON-2 data with Tyvaso in IPF is "great for patients and good news for inhaled treprostinil," but LQDA will explore the IPF indication with L606 (not Yutrepia). JNap's Take: This is likely because of the: 1) ODD protecting IPF for 3 years at UTHR, and 2) It seems that in IPF, the nebulizer makes more sense.
GPCR - The CEO remains optimistic about oral smole GLP-1 drugs despite some recent below-expectation data from LLY's orforglipron. GPCR believes that orforglipron will still be well-received by patients due to its oral convenience, and they are excited about their data coming in December because they believe aleniglipron to be "best-in-class." In December, we will get data from the Phase 2b ACCESS-I and ACCESS-II studies. The benchmark for success with these studies is 8-10% pbo-adjusted weight loss at week 36. JNap's Take: This has come down recently after seeing LLY's P3 ATTAIN-1 data that showed only -11.5% (pbo-adjusted) at week 72. VKTX's oral showed -10.9% (pbo-adjusted) at week 13, but this was with the 120 mg qd dose and likely not what VKTX pushes forward into P3. It's more likely that VKTX pushes the 30-60 mg into P3, which showed 6-8% (pbo-adjusted) weight loss at week 13. We will see the whole "curve" from LLY's P3 ATTAIN-1 at EADV on Sep 17th, so this should help us narrow in on the benchmark even more. GPCR also spoke about their amylin and DACRA smoles, which the market doesn't seem to be paying much attention to at this stage. The CEO noted they are thinking about various combinations and paths forward here. They are also "in dialogue" with potential partners because they envision a rather extensive P3 program to maximize the aleni value (assuming the P2b data looks good in December 2025).
MTSR - The CEO called obesity a "mega-market" but with challenges of scale, and he thinks the company's edge is in its peptide manufacturing. And with respect to MET-097i, the differentiation is on potency (they are doing 1/10th tirzepitide) and durability (~15 days of half-life vs. ~5 for tirzep). MTSR is focused on moving as quickly as possible into P3 (goal is by year-end 2025), so that's why they are aligning timelines on the P2b VESPER read-outs. Benchmarks for VESPER-1 and VESPER-3 data later in September are to match tirzepitide at week 28, which would be ~13% pbo-adjusted weight loss with similar tolerability (< 15% vomiting, single-digit severe nausea, < 25% diarrhea). Beyond the data from week 28, MTSR's goal is to identify a maintenance dose, potentially a low-dose oral MET-097o or a monthly subq MET-097i. They are also exploring MET-233i (subq amylin) as both a monotherapy and in combination with MET-097. JNap's Take: I found it interesting when the CEO discussed the subq vs. oral debate, noting that "people don't seem to mind the weekly subq and some may actually prefer it vs. a daily oral pill." I agree 100%. Finally, the MTSR CEO, like GPCR, reported being "in talks" with partners, but sounded far less pressured/urgent/anxious than the GPCR CEO.
SKYE - Topline data from the P2a CBeyond are expected in the coming weeks. Success is a 5-8% pbo-adjusted weight loss at week 26 for nimacimab (but mind the slope). SKYE would also like to see "additive" weight loss on top of semaglutide, which they threw out as 17-20% total weight loss. JNap's Take: This is a 4-arm study: Pbo, SKYE's nima, semaglutide, and then a nima-sema combo arm. I think success for nima is to match (or at least come close) to sema, with better GI tolerability and absolutely no CNS side-effects. For the combo arm, I don't think it needs to be 1+1 > 2, but it should be at least 1+1 > 1.5, with no additional tolerability issues.
RCKT - Recently shifted to focus exclusively on cardiovascular programs, including PKP2, Danon, and BAG3. In total, these 3 programs target > 100,000 patients in the US/EU. The CEO spoke about the prior use of a C3 inhibitor and how that negatively impacted AAV GTx safety. So, they corrected (removed) that from the dosing and aligned a path forward with the FDA on a new, lower (half) dose. CEO still thinks this is going to be efficacious based on previous data and better patient selection going forward. JNap's Take: Perhaps, but enrollment here has been slowed so that the next 3 will be one-at-a-time with 4-week follow-ups in between each. So, it's unlikely we get much new here until mid-2026.
ABEO - The CEO said "momentum is building" for the Zevaskyn® launch. ABEO has two qualified treatment centers (QTCs) up and running and has already identified about 50 patients. ABEO said these patients are the most severe (with large and open wounds), and many are treatment naive. JNap's Take: This seems a little contradictory to me - if these are the most severe patients with the highest need, why haven't they at least tried KRYS's Vyjuvek®? Nevertheless, ABEO believes that there are ~750 RDEB patients in the U.S. who might be eligible for Zevaskyn. What could slow the uptake is the logistics/ administrative step, which the company said is taking 3-4 months. They think they can improve this in the coming months. Guidance is still to treat 10-14 patients in 2025 and then get up to potentially as many as 10 per month once the current targeted five QTCs come online. ABEO thinks they can break even at 2-3 per month. So far, all patients who have sought Zevaskyn have been reimbursed. JNap's Take: This all sounded pretty bullish, but execution has been an issue for these guys, so color me skeptical for now. I think ABEO (stock) is likely stuck in the "prove it" category until they actually demonstrate they are achieving these bullish forecasts. Cash seems fine for now.
DSGN - DSGN is developing small molecules that are designed to "dial up or knock down" the expression of target genes. The lead candidate is DT216P2, which dials up expression of the frataxin (FXN) gene in patients with Friedreich's ataxia (FA). The "P2" after the DT216 indicates this is the second-generation attempt using this strategy to dial up endogenous frataxin expression. JNap's Take: This is an entirely different strategy from LRMR, which is attempting to deliver exogenous FXN into the mitochondria, or LXEO, which is trying to drive FXN expression through an AAV gene therapy. DSGN's first-gen candidate increased FXN levels, but its too short half-life and dose-limiting injection site reactions (ISRs) prevented an effective therapeutic window. The CEO believes they have addressed both these limitations with the gen-2 version. A P2 study called RESTORE-FA is ongoing, with initial data expected in 2026. The second clinical candidate is designed to knock down a toxin form of the TCF4 gene that results in Fuchs' endothelial corneal dystrophy. DSGN has formulated this candidate, DT168, into convenient eye drops. A P2 study is planned to start shortly. JNap's Take: DSGN seems interesting along similar lines to FULC, but it doesn't appear cheap and lacks near-term catalysts.
TARA - The CEO walked us through the Dec 2024 and April 2025 data for TARA-002 in NMIBC, noting they have since enrolled "many many more" patients since that time and reiterated a big update coming in the Q1 2026 from the ADVANCE-2 P2b study (guidance is ASCO-GU in February 2026). The benchmark for CRR at 6 months (per the CEO) is: Must be > 50-55% to be viable, above 65% is competitive with JNJ/CGON, and then >75-80% is practice-changing. At 12 months, these numbers change to > 35%, 45%, and > 55%. JNap's Take: I agree, but I also think the convenience of administration for TARA-002 (especially compared to JNJ/CGON) and safety shown to date, positions TARA well, even with comparable data. They will have a presentation at SUO in early December 2025, but this will be from the completed Phase 1 ADVANCE-1 study. The CEO remains confident in the pathway for approval (single-arm study) even if JNJ and/or CGON get approved ahead of them. Moving to the IV choline product, the CEO said they are "frustrated" with the slow enrollment in the P3 TRIVE-3 study, which has been hampered by the requirement for an overnight hospital stay. He expects the vast majority of enrollees will come from Europe, where there are dedicated "beds" for these patients, and that the U.S. will likely remain challenging. Nevertheless, the CEO thinks they will complete enrollment in Q2 2026 and have data in the summer of 2026. Nothing with respect to TARA-002 in lymphatic malformations (LM) was discussed, which baffles me.
FULC - FULC describes its platform along the same lines as DSGN - smoles to modify gene expression. The significant difference between FULC and DSGN, at least at this stage, is that FULC's pociredir is oral. The CEO then walked through the 12 mg update in July 2026, which showed mean absolute HbF improved from 7.6% at baseline to 16.2% at 12 weeks (+8.6%), with all 16 patients showing an increase and 56% (9/16) showing a ≥ 20% increase. Mean % F-cells increased from 34% to 67% at 12 weeks. Total hemoglobin increased 0.9 g/dL to 8.7 g/dL. They observed a 50% reduction in VOCs over the 12-week study. JNap's Take: This came close to home run data, and I'm optimistic for the 20 mg update coming at year end 2025. What I'd like to see from the 20 mg cohort is the % F-cells increase to > 70%, and the mean % total HbF ≥ 21% (with >75% over 20%). At this level, FULC can claim "pan-cellular" protection, which is a level where pociredir will likely demonstrate a significant and clinically meaningful reduction in VOCs. If it does that, it's practice-changing for SCD patients and a blockbuster drug.
ZURA - ZURA has three candidates, but right now only tibulizumab, a bispecific IL-17 x BAFF, is in the clinic. There is an ongoing P2 (TibuShield) for hidradenitis suppurative (HS) with data expected Q3 2026, and an ongoing P2 (TibuSure) in systemic sclerosis (SSc) with data expected in Q4 2026. The Cantor analyst was Josh Schimmer, a very knowledgeable analyst, so much of the conversation was about the mechanistic validation (IL-17) and rationale (BAFF) for tibulizumab. JNap's Take: We've been flooded with HS data lately, so the benchmark for success here in HS is pretty well understood. I'm not sure this can truly differentiate (MLTX's IL-17 data pending), but I do think it will get a lot of attention ahead of that Q3 2026 data. In SSc, it's a bit of a development graveyard, so a wide open opportunity, but I need to do more work here for that Q4 2026 data. Since it comes after the HS data, it plays second fiddle in my mind right now. Torudokimab (IL-33) seems interesting, but ZURA has yet to advance it into Phase 2. Given the failure of QTTB's bempikibart, ZURA's crebankitug (IL-7Rα x TSLP bispecific) has been deprioritized. The current market cap (at $2.05/sh) is $190 million, and ZURA held $155 million in the bank as of June 30, 2025. ZURA will burn $55 million over the next year, so cash will be around $100 million when the P2 TibuShield HS study reads out. That's ~$1 per share (93 million basic out). So, for me, ZURA only gets interesting at this level.
ASTX - The CEO noted that the Phase 3 ALPHA-ORBIT with navenibart in HAE is enrolling and data are expected in early 2027. The Cantor analyst seemed very optimistic about navenibart, both on efficacy and tolerability. For STAR-0310, the OX40 asset for atopic derm, ASTX will have initial data at EADV on September 17th. They got a late-breaking oral presentation spot, so that seems interesting. Sanofi's amlitelimab "worked" in the P3 COAST-1 study (reported Sep 4th), but with very disappointing results. SNY's drug is an OX40L vs. ASTX's targeting OX40 (same as AMGN's rocatinlimab). The CEO believes it makes more sense to target the receptor (on T cells) rather than the ligand. JNap's Take: I agree. She also noted that the OX40 mechanism has a slower onset of action, so despite the lower-than-expected SNY data, she remained optimistic about STAR-0310.
IOVA - The CFO highlighted the positives: the drug is effective, they are manufacturing it successfully, it's being reimbursed, and logistics are improving. However, she acknowledged that the prior guidance was too high. She did express confidence in the current guidance of $250 to $300 million for 2025. IOVA also expects that IL-2 revenues will smooth out by the end of the year, although distributor ordering patterns remain unpredictable. IOVA is "right-sizing" the business, but still thinks that Amtagvi® has > $1 billion peak potential. JNap's Take: This is unrealistic without a label or international expansion. There are pathways for both, but IOVA has a lot of work to do before I'd agree with that peak guidance. The CFO said they are exploring ways to shore up the balance sheet, which might include non-dilutive options. However, it's too early to guide when they can achieve breakeven.
DYN - The CEO believes they have the real potential to commercialize two best-in-class products for neuromuscular diseases, DMD and DM1, in 2027. DYNE-251 topline data in exon-51 DMD will come before the end of the year, followed by DYNE-101 data in DM1 around the middle of 2026. The CEO thinks they are in a good place with both products on AA pathway. I'm still a bit unclear about DYN's pursuit of AA in DM1 versus RNA's full approval. The CFO stated that they have sufficient cash into Q3 2027, enabling them to file both BLAs and potentially reach decisions on both. JNap's Take: Yes, but they will certainly raise again beforehand. DYN seems really interesting, but not cheap at $2B.
CGON - The company reported 24-month data from the P3 BOND-003 study in HR-NMIBC this morning, showing a 42% CRR. To put that number in perspective, the 24-month CRR numbers for Keytruda, Adstiladrin, and Anktiva are 9%, 19%, and 24%. So, the cretostimogene data are outstanding (we do not yet know the JNJ number at 24 months). The P3 PIVOT-006 study in IR-NMIBC completed enrollment earlier this week. The CEO noted they are going for both grades, and if they get both, they would have the widest label and (potentially) best data. CGON is also running a study in the BCG-naive setting, with data later this year. The rolling BLA will start in Q4 2025. Josh Schimmer asked about the CMC process, and the CEO's answers seemed solid, with Josh saying, “That was a remarkably convincing answer to a question that is really hard to give a remarkably convincing answer to.” JNap’s Take: If Josh is happy, I’m happy!
Check the Bio5C Model for position disclosures!!
Cheers,
JNap