Ready to launch

A record 59 NME approvals in 2018 means investors will focus on launches in 2019

The proposed $74 billion takeout of Celgene Corp. by Bristol-Myers Squibb Co. will not shift buysider attention away from upcoming and maturing drug launches heading into 2019, with plenty to track following a record year for NME approvals by FDA.

Several investors expect “shorting the launch” to be an ongoing theme, particularly among small and mid-cap biotechs looking to launch products independently.

Some portfolio managers are turning their attention to companies with more mature launches that have gained momentum. “We try and look at names that aren’t necessarily launching imminently but have been on the market and they seem to be picking up sales and ticking up,” International Biotechnology Trust plc’s Ailsa Craig told BioCentury.

“Once you get to the commercial stage you sell.”

Sven Borho, OrbiMed

Buysiders will also be monitoring reimbursement trends among new modalities, including gene and cell therapies and RNAi, which have seen mixed commercial performance over the last six months (see “Eyes on Launches”).

As for clinical catalysts, a flurry of readouts from late-stage non-alcoholic steatohepatitis (NASH) trials and updates from Biogen Inc.’s aducanumab program in Alzheimer’s disease will take the spotlight, though the latter may be a 2020 event.

Finally, against the backdrop of a poor macro environment and down market, some investors are looking to names they view as being undervalued or unfairly punished on negative news.

“Historically, when valuations have been overblown versus fundamentals, we have stepped back our investment pace,” said Omega Funds’ Otello Stampacchia. “In this environment, we’re stepping back up our deployment pace.”

While BMS’s proposed takeout of Celgene, announced Jan. 3, takes an active acquirer of small to mid-cap biotechs out of the game, buysiders don’t expect the deal to impact their investment strategies. The proposal caps off Celgene’s 15-month decline driven by pipeline discontinuations and commercial headwinds that have made it hard to diversify revenues beyond Revlimid lenalidomide (see “Rocky Road”).

“M&A does not change my investment view for the year. I do not play for takeouts, although I am usually happy when I get them,” said ClearBridge Investments’ Marshall Gordon.

Table: 2019 milestones

Selected products with clinical or regulatory milestones expected in 2019. (A) Bristol-Myers Squibb Co. (NYSE:BMY) is acquiring Celgene Corp. (NASDAQ:CELG); Source: BCIQ: BioCentury Online Intelligence

Acceleron Pharma Inc. (NASDAQ:XLRN) / Celgene Corp. (NASDAQ:CELG) (A)LuspaterceptMyelodysplastic syndrome (MDS)-associated anemia and transfusion-dependent β-thalassemiaSubmit BLA and MAA1H19
Acer Therapeutics Inc. (NASDAQ:ACER)ACER-001Urea cycle disorderSubmit NDA4Q19
Aimmune Therapeutics Inc. (NASDAQ:AIMT)AR101Peanut allergies in patients ages 4-17Submit MAA1H19
AIT Therapeutics Inc. (Pink:AITB)AIT-PPHNPersistent pulmonary hypertension in the newbornSubmit PMA2Q19
Alder BioPharmaceuticals Inc. (NASDAQ:ALDR)EptinezumabPrevent episodic and chronic migraineSubmit BLA1Q19
Aldeyra Therapeutics Inc. (NASDAQ:ALDX)Topical dermal reproxalapIchthyosis in patients with Sjogren-Larsson syndromePhase III data2H19
Aldeyra Therapeutics Inc. (NASDAQ:ALDX) / Ligand Pharmaceuticals Inc. (NASDAQ:LGND)Ocular reproxalapAcute allergic conjunctivitisPhase III dataEarly 2019
Aldeyra Therapeutics Inc. (NASDAQ:ALDX) / Ligand Pharmaceuticals Inc. (NASDAQ:LGND)Ocular reproxalapNon-infectious anterior uveitisPhase III data2H19
Alexion Pharmaceuticals Inc. (NASDAQ:ALXN)Soliris eculizumabNeuromyelitis optica spectrum disorderSubmit regulatory applications in EU, Japan and U.S.Early 2019
Alexion Pharmaceuticals Inc. (NASDAQ:ALXN)Ultomiris ravulizumabAtypical hemolytic uremic syndromePhase III dataEarly 2019
Alkermes plc (NASDAQ:ALKS)ALKS 3831 (olanzapine/samidorphan)SchizophreniaSubmit NDAMid-2019
Allergan plc (NYSE:AGN)Bimatoprost srGlaucomaPhase III additional data; submit NDA1H19; 2H19
Allergan plc (NYSE:AGN)RapastinelMajor depressive disorder (MDD)Phase III data2019
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY)GivosiranAcute hepatic porphyriasPhase III data; submit NDA and MAAEarly 2019; mid-2019
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) / Sanofi (Euronext:SAN; NASDAQ:SNY)FitusiranHemophilia A and BPhase III data2019
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) / Sanofi (Euronext:SAN; NASDAQ:SNY)LumasiranPrimary hyperoxaluria type 1Phase III dataYE19
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) / The Medicines Co. (NASDAQ:MDCO)InclisiranHypercholesterolemiaPhase III data; submit NDAMid-2019; YE19
Amryt Pharma plc (LSE:AMYT; ISE:AYP)Episalvan (AP101)Epidermolysis bullosaPhase III data2Q19
AnaptysBio Inc. (NASDAQ:ANAB)EtokimabAtopic dermatitisPhase IIb data2H19
Apellis Pharmaceuticals Inc. (NASDAQ:APLS)APL-2 Paroxysmal nocturnal hemoglobinuria Phase III data2019
Ardelyx Inc. (NASDAQ:ARDX)TenapanorHyperphosphatemia in end-stage renal disease patients on hemodialysisPhase III data2019
Ascendis Pharma A/S (NASDAQ:ASND)TransCon Growth Hormone (ACP-001)Growth hormone deficiencyPhase III data1Q19
AstraZeneca plc (LSE:AZN; NYSE:AZN)PT010Moderate to very severe chronic obstructive pulmonary diseasePhase III data; submit MAA and NDA2H19
AstraZeneca plc (LSE:AZN; NYSE:AZN) / Celgene Corp. (NASDAQ:CELG) (A)Imfinzi durvalumabFirst-line non-small cell lung cancer (NSCLC)Phase III data1H19; 2H19
Atara Biotherapeutics Inc. (NASDAQ:ATRA)TabelecleucelEpstein-Barr virus-associated post-transplant lymphoproliferative diseasePhase III data; submit conditional MAA1H19; 2H19
Aurinia Pharmaceuticals Inc. (TSX:AUP; NASDAQ:AUPH) / 3SBio Inc. (HKSE:1530)Voclosporin Lupus nephritisPhase III dataYE19
Bayer AG (Xetra:BAYN) / Syndax Pharmaceuticals Inc. (NASDAQ:SNDX)EntinostatHormone receptor-positive, HER2-negative breast cancerPhase III interim data2Q19
BeiGene Ltd. (NASDAQ:BGNE; HKSE:6160)Zanubrutinib (BGB-3111)Waldenstrom macroglobulinemiaSubmit NDAYE19 - early 2020
Bellicum Pharmaceuticals Inc. (NASDAQ:BLCM)RivogenlecleucelPrevent graft-vs-host disease (GvHD) and leukemia recurrence in pediatric patients receiving hematopoietic stem cell transplantatSubmit MAA2019
BeyondSpring Inc. (NASDAQ:BYSI)PlinabulinPrevent chemotherapy-induced neutropeniaPhase III final data; submit Chinese NDA; submit U.S. NDA1H19; early 2019; 2H19
BeyondSpring Inc. (NASDAQ:BYSI)PlinabulinNSCLCSubmit Chinese NDA1H19
BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX)BCX7353Prevent attacks of hereditary angioedemaPhase III data; submit NDA2Q19; 4Q19
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)Valoctocogene roxaparvovecHemophilia APhase III data; submit BLA2019; 2H19
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)VosoritideAchondroplasiaPhase III dataYE19
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) / Catalyst Pharmaceuticals Inc. (NASDAQ:CPRX)Firdapse amifampridineCongenital myasthenic syndromesPhase III data2H19
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) / Catalyst Pharmaceuticals Inc. (NASDAQ:CPRX)Firdapse amifampridineMyasthenia gravis in patients with anti-muscle associated receptor tyrosine kinase (MUSK) antibodiesPhase III data2H19
Blueprint Medicines Corp. (NASDAQ:BPMC)AvapritinibGastrointestinal stromal tumors (GIST)Submit NDA1H19
Bristol-Myers Squibb Co. (NYSE:BMY) / AstraZeneca plc (LSE:AZN; NYSE:AZN)AnifrolumabSystemic lupus erythematosusSubmit MAA and NDA; submit Japanese NDA1H19; 2H19

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