Your Guide To 2017's Emerging Pharmaceuticals Catalysts

Wedbush’s Liana Moussatos mentioned a slew of potential catalysts that could impact emerging pharmaceutical companies this year.

Aimmune Therapeutics Inc AIMT — Rated Outperform, PT: $42

• First Quarter 2017: Initiate RAMSES trial in the United States.
• March: Q4 FY2016 financials release.
• March 3–6: American Academy of Allergy, Asthma & Immunology.
• Mid-2017: Last patient completes up-dosing in AR101 PALISADE Phase 3 trial.
• Second-Half 2017: Viaskin Peanut Phase 3 (PEPITES & REALISE) data release.
• Mid-2017: Initiate ARTEMIS trial in EU.
• June 17–21: European Academy of Allergy & Clinical Immunology.
• Year-End 2017: Pivotal Phase 3 AR101 results from PALISADE trial.
• Year-End 2017: IND ready.

The company has about $300 million in cash and the AR101 peanut allergy desensitization pivotal trial seems to be on track for “likely positive data release” around yearend 2017. “[W]e anticipate 2017 to be a banner year for the company and recommend buying AIMT,” analyst Moussatos commented.

Ascendis Pharma A/S ASND — Rated Outperform, PT: $31

• March 16: Q4 financials release.
• April 1–4: Endocrine Society 6 abs TC-hGH (TransCon human Growth Hormone), -PTH (Parathyroid Hormone), -CNP.
• April 3: ENDO conference call.
• Second-Quarter 2017: Submit IND for TransCon PTH/hypoparathyroidism.
• Third-Quarter 2017: Initiate Phase 1 SAD/MAD trial for TransCon PTH/hypoparathyroidism.
• Sept.: Pivotal Phase 3 VELOCITY trial data for Somavaratan.
• Fourth-Quarter 2017: Submit IND for TransCon CNP/achondroplasia.
• Fourth-Quarter 2017: Full enrollment for Phase 3 heiGHt trial anticipated.
• 2017: Submit IND for TransCon Ranibizumab for ophthalmology (Roche).
• 2017: Submit IND for TransCon peptide for diabetes (Sanofi SA (ADR) SNY).
• 2017: Potential announcement of US/ex-US partnership for TransCon hGH.

Moussatos considers the stock as “an attractive investment opportunity,” given its “growing pipeline of clinical candidates for endocrine diseases led by weekly dosed TransCon human Growth Hormone (TC-hGH) being tested in a Phase 3 clinical program.” She estimated gross peak annual sales of over $1 billion in 2024, after a potential launch in 2020.

BioMarin Pharmaceutical Inc. BMRN — Rated Neutral, PT: $102

• Feb. 13–17: BMN-250 for MPS IIIB update at 13th WORLDSymposium in San Diego.
• Feb. 23–27: Q4 FY2016 financials release and 2017 guidance.
• First-Quarter 2017: Potential advisory committee meeting for Brineura.
• Second-Quarter 2017: Submit BLA for Pegvaliase/PKU.
• April 27: PDUFA date for Brineura (Cerliponase alfa) for CLN2.
• Second-Quarter 2017: Potential US launch of Brineura.
• Second-Quarter 2017: CHMP recommendation for Brineura approval in EU.
• Third-Quarter 2017: Initiate Phase 2b trial for BMN 270 Factor 8/Hemophilia A.
• Fourth-Quarter 2017: Potential EU launch of Brineura.
• Year-End 2017: Guidance: non-GAAP breakeven or better in 2017.

“BioMarin continues to make pipeline progress and reiterated guidance of attaining non-GAAP breakeven or better by the end of 2017. While we continue believe BMRN is a premier orphan drug company, we remain Neutral on the stock due to its relative valuation,” the analyst wrote.

Catabasis Pharmaceuticals Inc CATB — Rated Outperform, PT: $4

• March 8: Q4 FY2016 Financial results.
• Second-Quarter 2017: Part C interim results Phase 1/2 CAT-1004 in DMD–24 week efficacy.
• Fourth-Quarter 2017: Part C top-line results Phase 1/2 CAT-1004 in DMD–36 week efficacy.
• 2017: Data from the Phase 2a trial of CAT-2054 presented at medical meeting.

Clearside Biomedical Inc CLSD — Rated Outperform, PT: $28

Potential Catalysts

• March: Q4 FY 2016 financials.
• First-Half 2017: File IND and initiate Phase 1/2 for CLS-1002 (SCS-axitinib) for wet AMD.
• First-Half 2017: Initiate Phase 3 for SCS-Zuprata in ME-RVO.
• Second-Half 2017: Potential partnership.
• Fourth-Quarter 2017: Phase 1/2 data releases for SCS-Zuprata in DME.

Moussatos mentioned that 2017 had multiple catalysts for the company. She added that the stock valuation seemed attractive ahead of “multiple clinical and business catalysts anticipated by the end of 2017.”

Intercept Pharmaceuticals Inc ICPT — Rated Outperform, PT: $224

• Feb. 21: Q4 FY2016 financial results.
• First-Half 2017: Complete interim (1,400 patients) enrollment of Phase 3 REGENERATE trial.
• Mid-2017: Data from Phase 2 AESOP (PSC) trial.
• 2017: Data from Phase 2 CONTROL (OCA with statin in NASH) trial.
• 2017: Initiate Phase 3 OCA for NASH with Cirrhosis.
• 2017: EU launch of Ocaliva.
• 2017: INT-767 Phase 1 results to be announced plus initiation of Phase 2 (indication tbd).

“We consider Intercept to be a potential acquisition target. As PBC sales for Ocaliva continue to grow and other companies’ clinical failures continue for NASH, we speculate that large pharmaceutical companies may pursue acquisition of Intercept,” Moussatos stated.

Lexicon Pharmaceuticals, Inc. LXRX — Rated Outperform, PT: $33

• Feb. 27: Q4 FY2016 financials release/call and pipeline update.
• Feb. 28: FDA PDUFA date for telotristat ethyl.
• First-Quarter 2017: Potential U.S. launch of telotristat ethyl.
• June 9–13: InTandem1&2 pivotal presentations at ADA in San Diego.
• Mid-Third-Quarter 2017: Results from 52-week pivotal extension (InTandem1,2) and safety (InTandem3).
• Third-Quarter 2017: Potential early indication that FDA may consider reviewing T1D in 2018.
• Second-Half 2017: Additional data from InTandem1,2,3 sub-analyses presented at medical meetings.
• 2017: Potential EU approval of telotristat ethyl.
• 2017: Potential Phase 1 data release for LX2761 (local GI SGLT1 inh).
• 2017: LX9211 (neuropathic pain with BMS) IND filing.
• Mid-2017: Phase 1 start.

Omeros Corporation OMER — Rated Outperform, PT: $47

• Feb. 22–26: OMS721/Stem Cell Transplant-Associated TMA at BMT Tandem.
• March 6: Q4 FY16 Financials and pipeline update.
• April 21–25: OMS721 data in aHUS at ISN World Congress of Nephology.
• June 3–6: OMS721 data in Renal Diseases at ERA-EDTA.
• 2017: Additional data from Phase 2 trial for OMS721/aHUS and TMAs.
• 2017: Presentations on the use of OMIDRIA at medical meetings.
• 2017: Publications on OMS527/PDE7 MOA.
• 2017: Potential partnership(s).
• 2017: Update on program/restart Phase 2 study of OMS824 for Huntington’s.
• 2017: Initiate Phase 2 study of OMS824 in Schizophrenia.
• 2017: File IND/CTA and initiate Phase 1 for OMS527/PDE7 inhibitor.
• 2017: File IND/CTA and initiate Phase 1 for OMS616/Plasmin.
• 2017: Phase 1 results for OMS616/Plasmin.
• 2017: Initiate clinical testing of OMS906.
• 2017: Initiate pivotal trial for OMS721 in HSCT-TMA.

“With an emerging pipeline of rare disease focused products and OMIDRIA sales offsetting the company’s burn, we believe OMER to be an attractive investment for investors with a 2017-2018 horizon,” the analyst wrote.

Pacira Pharmaceuticals Inc PCRX — Rated Outperform, PT: $89

• Feb. 22: Symphony Health estimate for January.
• Feb. 23–27: Q4 FY2016 Financial Results.
• First-Quarter 2017: IR-RCT¥ EXPAREL TKA active comparator top-line Phase 4 data release.
• March 14: IR-RCT¥ EXPAREL TKA Phase 4 data presentation.
• First-Half 2017: IR-RCT¥ EXPAREL knee active comparator Phase 4 data publication.
• Second-Quarter 2017: Top-line Phase 2 data release for DepoTXA in TKA.
• Second-Quarter 2017: Data releases from two Phase 3 EXPAREL Nerve Block Trials.
• Third-Quarter 2017: Submit sNDA for EXPAREL/Nerve Block.
• Second-Half 2017: Top-line Phase 4 EXPAREL/spine data release.
• Fourth-Quarter 2017: Submit MAA for EXPAREL approval in EU.
• 2017: Potential regional exUS EXPAREL partnership(s).
• Year-End 2017: Publication of EXPAREL RCT in spine clinical results.

The company’s recent initiatives could result in a reacceleration of sales from Q2 2017 onward, Moussatos mentioned.

Regulus Therapeutics Inc RGLS — Rated Outperform, PT: $8

• First-Quarter 2017: Estimated release of Q4 FY2016 financial results.
• First-Half 2017: Results from MAD study for RG-012 in HV.
• Second-Half 2017: Phase 2 HERA study for Alport resumes.
• Second-Half 2017: File IND for RGLS5040 for cholestatic diseases.
• Second-Half 2017: File IND for RGLS4326 for ADPKD.
• Second-Half 2017: Nomination of additional clinical candidates.
• Fourth-Quarter 2017: Potential POC data release from RG-125 (AZN T2D/NASH program)

“We see progress with multiple other pipeline candidates in 2017… We believe RG-012 for Alport Syndrome (AS) is an underappreciated jewel in RGLS’ clinical pipeline,” Moussatos commented.

Sangamo Therapeutics Inc SGMO — Rated Neutral, PT: $4

• Feb.: Q4 FY2016 Financials release and call.
• 2017: Initiate Phase 1/2 AAV-FVIII SB-525/Hemophilia A study.
• 2017: Updated data from SB-728-1101 Cohort 3 HIV study.
• 2017: Data from Phase 1 SB-728-mR-HSPC HIV program.
• 2017: Initiate Phase 1/2 trial s for Hunter and Hurler syndrome.

Sangamo’s new management had “positively reorganized” the company for the longer term with “clear goals” and “reliable success,” Moussatos noted. She added, however, that the pipeline overhaul had created a lack of material clinical catalysts until early 2018.

United Therapeutics Corporation UTHR — Rated Outperform, PT: $229

• Feb. 23: Q4 FY2016 financials release and call.
• 2017: Potential U.S. sNDA approval for RemoSync.
• 2017: Launch of RemoSync (Remodulin implantable pump).
• 2017: Initiate Phase 2/3 trial of eNOS gene therapy.
• 2017: FREEDOM-EV potentially completes enrollment.

“We continue to believe shares of UTHR are undervalued as we believe investors have overemphasized the impact of generic treprostinil and undervalue potential growth from new delivery mechanisms and new indications for approved products to maintain historical growth into the future,” Moussatos stated.