Market Cap 5.19B
Revenue (ttm) 806.78M
Net Income (ttm) -363.30M
EPS (ttm) N/A
PE Ratio 8.31
Forward PE 8.21
Profit Margin -45.03%
Debt to Equity Ratio -11.53
Volume 869,000
Avg Vol 1,376,608
Day's Range N/A - N/A
Shares Out 79.44M
Stochastic %K 73%
Beta 0.55
Analysts Strong Sell
Price Target $66.69

Company Profile

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to children and adults living with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spina...

Industry: Biotechnology
Sector: Healthcare
Phone: 908 222 7000
Fax: 908 222 7231
Address:
500 Warren Corporate Center Drive, Warren, United States
Quantumup
Quantumup Oct. 6 at 1:56 PM
Citizens reiterated $LRMR at Market Outperform/$18 after hosting a call w/ the CEO of the Friedreich's Ataxia Research Alliance (FARA), Jennifer Farmer, on Friday, who said, 'The Patient Community Believes Nomlabofusp's Potential Benefits Outweigh Its Risks' $BIIB $LXEO $PTCT $DSGN Citizens JMP said:
0 · Reply
JasonCO
JasonCO Oct. 3 at 3:49 PM
$PTCT beast
1 · Reply
Quantumup
Quantumup Oct. 2 at 2:27 PM
Oppenheimer lowered $LRMR's price target to $21 from $26 to reflect the share count change following Larimar's recent capital raise; reiterated at Outperform. $BIIB $LXEO $PTCT $DSGN Oppenheimer said: Larimar reported positive results from the 50mg OLE cohort of nomla, supporting its use as the potentially first disease-modifying treatment in Friedreich's Ataxia (FA). Data were positive, demonstrating all OLE patients at 6-months reached skin frataxin levels over 50% of healthy volunteers along with improvements in clinical outcomes, though the seven cases of reported anaphylaxis in the OLE drew concern; while a potential concern, anaphylaxis is manageable. OPY sees LRMR's rebound from the previous day's selloff reflecting nomla's favorable benefit-risk profile in FA and still sees a buying opportunity ahead of the Q2 2026 BLA submission, followed by a potential early 2027 launch.
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Quantumup
Quantumup Sep. 29 at 7:02 PM
Leerink reit'd $LRMR Outperform/$25 $BIIB $LXEO $PTCT Leerink said: Bottom Line: The stock is under pressure on the 7 anaphylaxis events that occurred in the open label study. However, we believe that the efficacy and clinical data are quite compelling, especially given Friedreich's ataxia (FA) is a devastating disorder, making the risk/reward profile favorable. This morning, LRMR provided an update on their Phase 2 open label (OL) trial. As anticipated in our preview, key focus areas included safety, frataxin levels, and enrollment progress to assess whether the company remains on track to file their BLA in 2Q26 (here). On skin frataxin, LRMR reported impressive results. 100% (10 out of 10) of patients achieved levels >50% of the median frataxin concentration found in healthy volunteers by day 180 which we believe is striking. For context, heterozygous patients who exhibit no signs of disease typically have frataxin levels at ~50% of those in unaffected healthy individuals. Leerink added:
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JasonCO
JasonCO Sep. 25 at 7:30 PM
$PTCT I buy
0 · Reply
Quantumup
Quantumup Sep. 24 at 6:24 PM
Leerink⬆️ $QURE's PT to $68/⬆️PoS for AMT-130 to 85%/50% in US/EU from 75%/25%: TD Cowen reit'd $QURE at Buy: RBC Capital⬆️the PT on $QURE to $55 from $24, reiterated at an Outperform and said w/b buyers in2💪🏾given its belief $QURE likely has a Tx for a devastating disease that comes w/ 12X higher risk of committing suicide. $PTCT $WVE $IONS $NBIX Leerink and TD Cowen said in their notes to investors:
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Quantumup
Quantumup Sep. 24 at 6:15 PM
Stifel⬆️the PT on $QURE to $65 from $30, reit'd at Buy and said, we're reiterating our Buy rating on QURE following detailed results from the 3yr AMT-130 data. Bottom line: we believe the data suggest a larger market opportunity (we model >$1B US in 2031) and also a higher probability-of-approval (we model 80%). On the regulatory front, in the context of a broader conversation around "how flexible will the new FDA be?", we think AMT-130 sets up well given (1) the severity of Huntington's-and the fact that most patients are adults, (2) the strength of the AMT-130 efficacy data, (3) QURE's attained alignment with FDA ahead of the 3-year data and (4) QURE's receipt of BTD on the AMT-130 2-year data. And on the market opportunity, there does continue to be the relevant question around capacity, but KOL feedback here has been encouraging, AMT-130 pricing should be high, and we think the HD population should be highly motivated for a potential disease-modifying treatment. $PTCT $WVE $IONS $NBIX
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biggercapital
biggercapital Sep. 24 at 11:55 AM
Michael Bigger $CLPT 1 approved and a 95% chance of AMT-130 being approved in 2026. 2 for 2? (Combo Gene Therapy/ Device) in Neuro indications. That is unreal! $QURE $PTCT https://www.biggercapital.com/blog-posts/clearpoint-neuro-inc-unleashing-the-2017-adjacent-possible-into-reality-nownbsp
1 · Reply
Quantumup
Quantumup Sep. 24 at 11:51 AM
Stifel reit'd $QURE Buy-$30: We're reiterating our Buy rating on $QURE as AMT-130 3yr data in Huntington's Disease exceeded our expectations, and in turn should further derisk accelerated approval prospects with FDA. Specifically, AMT-130 showed: (1) 75% disease-slowing on cUHDRS vs. natural history (p=0.003) -- better than our hope for at least ~60%, (2) a statistically significant benefit on TFC (p=0.033) -- an upside surprise, (3) efficacy across various other sub-scales and (4) 3-year neurofilament/NfL levels below baseline. While the Wall Street "bar" for the 3-year data was subjective, this is almost identical to the 2-year data on which $QURE received Breakthrough, and whereas efficacy on TFC (a measure known to be important to FDA) is a bonus. Some caveats here inevitably exist: small N, natural history comparison, and a new FDA who we're still getting to know. That said, this was a prospective analysis -- firmed up after receipt of BTD -- which sets $QURE up well for a 1Q26 BLA. $PTCT
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JasonCO
JasonCO Sep. 18 at 4:18 PM
$PTCT Lfg
0 · Reply
Latest News on PTCT
PTC Therapeutics Stock Is Stuck In A Multi-Year Slump

Aug 21, 2025, 10:00 AM EDT - 6 weeks ago

PTC Therapeutics Stock Is Stuck In A Multi-Year Slump


PTC Therapeutics, Inc. (PTCT) Q2 2025 Earnings Call Transcript

Aug 8, 2025, 3:19 PM EDT - 2 months ago

PTC Therapeutics, Inc. (PTCT) Q2 2025 Earnings Call Transcript


These Analysts Increase Their Forecasts On PTC Therapeutics

Jul 29, 2025, 11:23 AM EDT - 2 months ago

These Analysts Increase Their Forecasts On PTC Therapeutics


US FDA approves PTC Therapeutics' metabolic disorder drug

Jul 28, 2025, 4:25 PM EDT - 2 months ago

US FDA approves PTC Therapeutics' metabolic disorder drug


PTC Therapeutics, Inc. (PTCT) Q1 2025 Earnings Call Transcript

May 6, 2025, 11:19 PM EDT - 5 months ago

PTC Therapeutics, Inc. (PTCT) Q1 2025 Earnings Call Transcript


Quantumup
Quantumup Oct. 6 at 1:56 PM
Citizens reiterated $LRMR at Market Outperform/$18 after hosting a call w/ the CEO of the Friedreich's Ataxia Research Alliance (FARA), Jennifer Farmer, on Friday, who said, 'The Patient Community Believes Nomlabofusp's Potential Benefits Outweigh Its Risks' $BIIB $LXEO $PTCT $DSGN Citizens JMP said:
0 · Reply
JasonCO
JasonCO Oct. 3 at 3:49 PM
$PTCT beast
1 · Reply
Quantumup
Quantumup Oct. 2 at 2:27 PM
Oppenheimer lowered $LRMR's price target to $21 from $26 to reflect the share count change following Larimar's recent capital raise; reiterated at Outperform. $BIIB $LXEO $PTCT $DSGN Oppenheimer said: Larimar reported positive results from the 50mg OLE cohort of nomla, supporting its use as the potentially first disease-modifying treatment in Friedreich's Ataxia (FA). Data were positive, demonstrating all OLE patients at 6-months reached skin frataxin levels over 50% of healthy volunteers along with improvements in clinical outcomes, though the seven cases of reported anaphylaxis in the OLE drew concern; while a potential concern, anaphylaxis is manageable. OPY sees LRMR's rebound from the previous day's selloff reflecting nomla's favorable benefit-risk profile in FA and still sees a buying opportunity ahead of the Q2 2026 BLA submission, followed by a potential early 2027 launch.
0 · Reply
Quantumup
Quantumup Sep. 29 at 7:02 PM
Leerink reit'd $LRMR Outperform/$25 $BIIB $LXEO $PTCT Leerink said: Bottom Line: The stock is under pressure on the 7 anaphylaxis events that occurred in the open label study. However, we believe that the efficacy and clinical data are quite compelling, especially given Friedreich's ataxia (FA) is a devastating disorder, making the risk/reward profile favorable. This morning, LRMR provided an update on their Phase 2 open label (OL) trial. As anticipated in our preview, key focus areas included safety, frataxin levels, and enrollment progress to assess whether the company remains on track to file their BLA in 2Q26 (here). On skin frataxin, LRMR reported impressive results. 100% (10 out of 10) of patients achieved levels >50% of the median frataxin concentration found in healthy volunteers by day 180 which we believe is striking. For context, heterozygous patients who exhibit no signs of disease typically have frataxin levels at ~50% of those in unaffected healthy individuals. Leerink added:
0 · Reply
JasonCO
JasonCO Sep. 25 at 7:30 PM
$PTCT I buy
0 · Reply
Quantumup
Quantumup Sep. 24 at 6:24 PM
Leerink⬆️ $QURE's PT to $68/⬆️PoS for AMT-130 to 85%/50% in US/EU from 75%/25%: TD Cowen reit'd $QURE at Buy: RBC Capital⬆️the PT on $QURE to $55 from $24, reiterated at an Outperform and said w/b buyers in2💪🏾given its belief $QURE likely has a Tx for a devastating disease that comes w/ 12X higher risk of committing suicide. $PTCT $WVE $IONS $NBIX Leerink and TD Cowen said in their notes to investors:
0 · Reply
Quantumup
Quantumup Sep. 24 at 6:15 PM
Stifel⬆️the PT on $QURE to $65 from $30, reit'd at Buy and said, we're reiterating our Buy rating on QURE following detailed results from the 3yr AMT-130 data. Bottom line: we believe the data suggest a larger market opportunity (we model >$1B US in 2031) and also a higher probability-of-approval (we model 80%). On the regulatory front, in the context of a broader conversation around "how flexible will the new FDA be?", we think AMT-130 sets up well given (1) the severity of Huntington's-and the fact that most patients are adults, (2) the strength of the AMT-130 efficacy data, (3) QURE's attained alignment with FDA ahead of the 3-year data and (4) QURE's receipt of BTD on the AMT-130 2-year data. And on the market opportunity, there does continue to be the relevant question around capacity, but KOL feedback here has been encouraging, AMT-130 pricing should be high, and we think the HD population should be highly motivated for a potential disease-modifying treatment. $PTCT $WVE $IONS $NBIX
0 · Reply
biggercapital
biggercapital Sep. 24 at 11:55 AM
Michael Bigger $CLPT 1 approved and a 95% chance of AMT-130 being approved in 2026. 2 for 2? (Combo Gene Therapy/ Device) in Neuro indications. That is unreal! $QURE $PTCT https://www.biggercapital.com/blog-posts/clearpoint-neuro-inc-unleashing-the-2017-adjacent-possible-into-reality-nownbsp
1 · Reply
Quantumup
Quantumup Sep. 24 at 11:51 AM
Stifel reit'd $QURE Buy-$30: We're reiterating our Buy rating on $QURE as AMT-130 3yr data in Huntington's Disease exceeded our expectations, and in turn should further derisk accelerated approval prospects with FDA. Specifically, AMT-130 showed: (1) 75% disease-slowing on cUHDRS vs. natural history (p=0.003) -- better than our hope for at least ~60%, (2) a statistically significant benefit on TFC (p=0.033) -- an upside surprise, (3) efficacy across various other sub-scales and (4) 3-year neurofilament/NfL levels below baseline. While the Wall Street "bar" for the 3-year data was subjective, this is almost identical to the 2-year data on which $QURE received Breakthrough, and whereas efficacy on TFC (a measure known to be important to FDA) is a bonus. Some caveats here inevitably exist: small N, natural history comparison, and a new FDA who we're still getting to know. That said, this was a prospective analysis -- firmed up after receipt of BTD -- which sets $QURE up well for a 1Q26 BLA. $PTCT
0 · Reply
JasonCO
JasonCO Sep. 18 at 4:18 PM
$PTCT Lfg
0 · Reply
Thelonius_Stonk
Thelonius_Stonk Sep. 17 at 6:22 PM
$PTCT we dgaf
0 · Reply
JasonCO
JasonCO Sep. 17 at 4:11 PM
$PTCT very
0 · Reply
Quantumup
Quantumup Sep. 17 at 11:43 AM
Goldman Sachs initiated coverage of $TRDA at an Early-Stage Biotech rating. [ $SRPT $EWTX $PTCT SLDB $RGNX PEPG RNA] Goldman Sachs said: We initiate coverage of Entrada Therapeutics ( $TRDA) with an Early-Stage Biotech (ESB) designation. In our view, $TRDA's proprietary Endosomal Escape Vehicle (EEV) platform is differentiated from other delivery peptide approaches, and as such, we see best-in-class potential driven by key properties including: 1) enhanced cellular uptake, 2) a wider therapeutic index, and most notably 3) a better safety profile. Within the broader Duchenne muscular dystrophy (DMD) treatment paradigm, we continue to evaluate the positioning of gene therapies relative to exon-skipping therapeutics following recent safety events observed with $SRPT's Elevidys. With the barriers to entry for next generation DMD therapeutics lowered, we are cautiously positive on the efficacy/safety profile of $TRDA's burgeoning clinical-stage portfolio and translation of data into the clinic.
1 · Reply
Quantumup
Quantumup Sep. 17 at 11:01 AM
Barclays y'day after the close initiated coverage of $SRRK at an Overweight rating and a $45 PT. Barclays said: Scholar Rock is a biopharmaceutical company that focuses on the development of antibody therapeutics for the treatment of neuromuscular disorders as well as other rare diseases and oncology. Lead asset apitegromab is an anti-latent myostatin antibody that has successfully completed a Phase 3 trial in spinal muscular atrophy (SMA) and a US FDA approval decision is expected in September 2025. $SRRK's R&D pipeline includes SRK-439 (anti-latent myostatin) that the company plans to submit an IND for exploration in rare neuromuscular disorders in 2H25. We see potential near- and medium-term upside to $SRRK shares on approval and commercial launch based on our assumptions and the historical performances of new launches in SMA. $RHHBY - $PTCT $BIIB - $IONS
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Quantumup
Quantumup Sep. 16 at 7:23 PM
Guggenheim reiterated $LRMR at a Buy rating and a $26 PT after its FDA Expert (fmr. Director of the Division of Pharmacometrics at the FDA) Expressed High Conviction in $LRMR's Regulatory Case and Noted $BIIB's Qalsody as One of Several of His Case Points. [ $PTCT $LXEO $DSGN] Guggenheim said in its note:
1 · Reply
stu4
stu4 Sep. 16 at 2:27 PM
$IONS $NBIX $PTCT $QURE $WVE they have too,,lol They JAMMED IT into high net worth clients accounts , that they manage , hahahah …
0 · Reply
Quantumup
Quantumup Sep. 16 at 12:08 PM
Mizuho y'day after the close reiterated $QURE at an Outperform rating and a $30 price target. $PTCT $WVE $IONS $NBIX Mizuho said: $QURE was down 10% (vs -0.7% XBI) at the close. We believe some investors expected 3-year data from the Phase 1/2 trial of AMT-130 in Huntington's disease and became concerned due to the absence of data release today. Management noted today that the 3-year readout was not positioned as a "back-to-school" catalyst and is still on track for September. Based on historical timeline, we estimate the readout is more likely to occur in latter half of September (see Table 1 below). We see the weakness as a buying opportunity, as we expect positive 3-year Phase 1/2 data for AMT-130.
1 · Reply
Night_Owl_Biotech
Night_Owl_Biotech Sep. 14 at 1:10 PM
The attachment compiles certain valuation related data points on 9 mature commercial-stage bios (3 in oncology & 6 in non-oncology). For the more mature bios in this peer group, we consider enterprise value related multiples the best predictor of value potential (since all 9 are long past traditional M&A exit timelines). $OGN appears to be the most leveraged. Should they be able to service the debt (not considered in EPS) then... We understand $PTCT first meaningful FDA approval came in 2017 so they are the youngest in this peer group. They are the only bio of the 9 still not profitable. $BMRN is hovering near 52-week lows yet still appears to trade at revenue multiples consistent with others in this peer group. $ACAD & $ALKS have almost identical multiples. ACAD just got a major IP extension. This is not investment advice as we do not follow the non-oncology bios in this peer group in any detail. We'd otherwise appreciate genuine feedback. Are we missing a data point to consider?
1 · Reply
Night_Owl_Biotech
Night_Owl_Biotech Sep. 13 at 3:55 PM
Change in share price for the week for all 57 non-BP comm'l-stage non-oncology focused bios with MCs over ~$1B. 37 of the 57 were down which was consistent with oncology. There were 3 HC related investor conferences last week $CORT was a winner after the FDA accepted Relacorilant's filing for certain ovarian cancers. Perhaps we’ll track CORT with our oncology peer group in the future like JAZZ. $PTCT appeared at 3 investor conferences over the last 2 weeks, as did $APLS $SLNO was down after a 17-year-old patient taking Vykat tragically died. The doctor treating the subject patient said the death had nothing to do with Vykat, as did SLNO management. Baird & HC Wainwright maintained their targets. Investors should check out SRPT for risks to further valuation changes. TARS was down, we suspect, due to an insider sale. See our other TARS post $HRMY is a head scratcher to us. If any HRMY investor can attribute its fall to a specific inflection point please note so This is not investment advice
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Thelonius_Stonk
Thelonius_Stonk Sep. 12 at 4:41 PM
$PTCT red days are illegal here
1 · Reply
Quantumup
Quantumup Sep. 12 at 12:01 PM
H.C. Wainwright⬆️ $MAZE $50/said, Yesterday, Maze's Phase 1 HV data from the MZE782, a SLC6A19 small molecule inhibitor program, showed a robust PD signal, far exceeding JNT-517's PD benchmark ( $OTSKY; Not rated) in HVs, reinforcing best-in-class potential of '782 in PKU, in our opinion. However, the major surprise was the data from an exploratory analysis showing a dose dependent, reversible eGFR dip, indicating for the first time that MZE782 might play a therapeutic role in CKD similar to the SGLT2 inhibitors, in our opinion. Based on the KOL feedback we think that the eGFR dip pattern is consistent with a benign hemodynamic mechanism seen in ACEi/ARB/SGLT2i but believe that Phase 2 must confirm this with injury biomarkers and link it to proteinuria/UACR reduction. Therefore, given the compelling PoC data that now potentially supports efficacy in both PKU and CKD, we have increased our PoS assumptions for both the programs which increases our PT from $34 to $50. $AGIO BMRN $PTCT $TVTX
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JasonCO
JasonCO Sep. 12 at 3:35 AM
$PTCT boss
0 · Reply