Shots:

• The 41^st Annual J.P. Morgan Healthcare Conference took place on 09 – 12 January 2023 in San Francisco, CA, USA. It is the largest and most informative healthcare investment symposium in the industry, bringing together a cohort of industry leaders, emerging fast-growing companies, innovative technology creators, and members of the investment community
•  The major announcement was regarding J&J, where it will spin off its consumer-health business as Kenvue. The new (remaining) J&J will focus on pharmaceuticals and medical technology with an expected $60B drug revenues by 2025 and make up for the patent expiration of the Crohn's disease drug Stelara in 2023. Other announcement was regarding Alkermes, which expects to spin-off its oncology business into a development-stage oncology company
• Our team at PharmaShots summarized the key highlights of various companies, their drug development plans, and business updates with focus areas on Oncology, Neurology, Cardiology, and Rare Diseases. To access the full report email us at connect@pharmashots.com with the Subject line “JPM 2023”

Johnson & Johnson (JNJ)

• In the oncology space, Darzalex, Carvykti, Tecvayli & Talquetamab are predicted to be the main drivers of growth in the multiple myeloma market
• JNJ will accelerate its growth through new product launches and increased sales of its currently marketed products in four areas: Darzalex, Tremfya, Uptravi & Opsumit along with Erleada. Johnson anticipated revenue of >$5B each for Carvykti, nipocalimab, Rybrevant, taris (a drug-eluting device) & milvexian
• Lazertinib + Rybrevant is being studied in the P-III trial (MARIPOSA) against AstraZeneca's Tagrisso for 1L EGFR mutant NSCLC, with expected interim results in 2023 followed by complete results in 2024

Novartis

• Novartis’ CEO Vas Narasimhan is expecting an increase of 4 % in CAGR sales from 2021 to 2027. In a detailed 10-year plan, Novartis expected 50% revenue from biologics, developing radioligand therapy, and moving forward with a “US first” mindset
• Novartis has been narrowing its focus towards innovative medicines, and, more specifically, five therapeutic areas incl. solid tumors and hematology
• Kisqali was approved for HR+/HER2- metastatic breast cancer which showed a significant OS benefit & Pluvicto was approved for prostate cancer. Pluvicto is being studied in a 1L mCRPC setting, the top-line & numerical data are expected later in 2023
• Scemblix had positive early phase data in the 1L setting & expected late phase data in 2024 while Novartis’ goal is to increase presence in rare diseases, focusing on 2023 plans for expected approvals and label expansion

Gilead

• The company highlights its oncology portfolio & expands the use of the ADC Trodelvy (sacituzumab govitecan). The company also predicted that there would be more M&As in the biopharma sector in 2023
• Additionally, six new products with nine approved indications, including six in oncology.Currently ten ongoing trials, six in the P-III trial & 5 in active combination trials. The company intends to fully investigate the oncology candidate magrolimab for several solid tumors and hematological cancers. Gilead also focuses to bring Trodelvy and other drugs to lung cancer patients. In collaboration with Arcus Biosciences, Gilead presented P-II data on domvanalimab in advanced, PD-L1-high NSCLC patients.
• Gilead is also focused on continually growing its cell therapy business

Amgen

• The company highlights its oncology portfolio with a focus on bemarituzumab’s evaluation as 1L treatment in gastric cancer & also highlighted the registration enabling P-II study of tarlatamab in the SCLC
• At ASH, Blincyto, which is being investigated as an SC formulation showed positive survival data. Lumakras continues to perform well on the market

Seagen (SGEN)

•  Seagen’s presentation at JPM focused on oncology, The (ECHELON-1) study OS data of Adcetris were submitted to the US FDA & led to an update to the NCCN guidelines listing it as a Category 1 preferred regimen with an anticipated PDUFA date of June 2023
• Padcev, an SoC in 2L urothelial cancer continues to move up in all lines of bladder cancer & 1L mUC with Keytruda shortly with an expected Priority review in April 2023
  • Padcev is also being studied in the P-III trial for breast cancer & solid tumors with expected results in H1’23
• The company highlighted an ongoing study in DLBCL and solid tumors with expected initial data in H1’23.
  • Tukysa also continues to advance in 2L mCRC with an expected priority review on Jan 19, 2023
  • Tukysa + Kadclya Seagen is being studied in the P-III confirmatory study in 1L mCRC with expected results in H1’23
• Tivdak is the NCCN preferred regimen for 2L recurrent metastatic cervical cancer. Multiple dosing regimens in cancer are being studied with expected results in H2’23

Bristol Myers Squibb (BMS)

• The company introduced nine new medicines b/w 2020 and 2022. In 2022, the company rolled out three FIC therapies in three different disease areas— one being Opdualag for melanoma. BMS considers its new product portfolio and expects the group to deliver $10B-$13B by 2025. Repotrectinib is currently evaluated in the P-I/II trial (TRIDENT-1) trial for advanced ROS1+ NSCLC, US FDA approval is expected in 2023
• BMS acquired repotrectinib through its $4.1B acquisition of Turning Point Therapeutics.

AbbVie (ABBV)

• Due to Imbruvica’s increased competition from the next-gen. BTKi, the oncology revenues of the company are expected to decline to $5.7B in 2023 & will remain flat through 2024-2025
• In 2026, the company expects to launch new oncology products and indications leading to AbbVie’s return to growth
• AbbVie plans to add indications for Venclexta (multiple myeloma & high-risk MDS) and expects additional approval of Venclexta for the treatment of AML
• The following late-phase pipeline drugs are also expected to contribute to the company’s growth

• Epcoritamab: a CD3-CD20 bispecific antibody for DLBCL with initial approval expected in 2023
• Navitoclax: a BCL-2 inhibitor with approval expected in 2024 for myelofibrosis
• Teliso-V: an ADC targeting c-Met with approval expected in 2024 for 2L/2L+ NSCLC

• Additionally, ABBV-647 (an anti-PTK7 ADC) and ABBV-151 (an anti-GARP Ab) achieved PoC for solid tumors, AbbVie plans to initiate P-II clinical trials in 2023

Bayer (BAYN)

• Nubeqa, approved for the treatment of nmCRPC, has been contributing a significant amount to its overall sales
• Recently, Nubeqa has been approved in combination with chemotherapy for the treatment of mHSPC whereas, Nubeqa as monotherapy in mHSPC expected to be approved by 2024 along with a second label expansion for adjuvant prostate cancer setting by 2027
• Bayer expects to generate over a billion-euro in sales of Nubeqa by 2024 with an overall peak sales potential reaching over €3B
• Additionally, Bayer currently has multiple ongoing early-phase trials that are expected to yield positive results and receive approval by the early 2030s

BioNTech (BNTX)

• The company achieved clinical PoC in trials evaluating BNT211 (cell therapy for solid tumors) and BNT312 (a next-generation checkpoint immunomodulator) whereas four additional programs are in first-in-human trials
• Additionally, BioNTech has entered into a multi-year partnership with the UK Government to accelerate clinical trials for personalized mRNA immunotherapies, expected to be provided to ~10,000 patients by YE 2030
• The company also reported its acquisition of InstaDeep intending to enhance its AI and machine learning-based drug development

Genmab (GMAB)

• Genmab started its presentation with a focus on its approved drugs including Tivdak, Darzalex, Rybrevant, Kesimpta, Tepezza, and Tecvayli. The company aims to further develop and expand its pipeline
• Tivdak, approved in Sep 2021 for 2L R/M cervical cancer.
• Epcoritamab, which is developed in collaboration with AbbVie for pre-treated DLBCL received a priority review from the FDA, with a PDUFA date set for May 21, 2023. Both companies are planning to develop the drug in phases for multiple indications
• Additionally, the company also highlighted its collaboration with BioNTech for the development of its P-II immunotherapy, DuoBody-PD-L1x4-1BB in combination with Keytruda for NSCLC. The drug is also being developed in a P-I/II trial in solid tumors
• Genmab is also developing a fully-owned investigational agent with HexaBody technology to add to Darzalex franchise. Darzalex is being developed under an exclusive worldwide license and option agreement with Janssen. The drug targets CD38 and have shown promising data in preclinical models for multiple myeloma, DLBCL, and AML

Immunocore (IMCR)

• Bahija Jallal (CEO & BOD), discussed the structure of the company. He highlighted the three main therapeutic areas of the company including oncology, infectious disease, and autoimmune conditions.
  • Focusing on kimmtrak, a novel bispecific protein comprised of a soluble T-cell receptor. The company is expecting the global commercial expansion of kimmtrak from 2023 to 2024
• Immunocore plans to initiate a P-II/III trial to investigate kimmtrak for advanced cutaneous melanoma to address a patient population that is estimated to be 2-4x larger than metastatic uveal melanoma.
  • Immunocore also plans to submit an IND for IMC-R117C (PIWIL1 targeted immunotherapy) in Q4’23

Lantheus (LNTH)

• Lantheus expects its revenue growth in the upcoming years through its drug, Pylarify
• MAA submitted for Pylarify with FDA in June 2022 with approval expected in 2023
• The company also expects good sales growth due to the increasing incidence and prevalence of prostate cancer which escalates the demand for PSMA PET imaging

Arvinas (ARVN)

• Arvinas presented the upcoming milestones for its broad PROTAC degraders pipeline which include ARV-471, an oral, FIC, estrogen receptor (ER)-degrading PROTAC for breast cancer.
• Another focus is on the current recruitment of the P-II (VERITAC-2) trial evaluating ARV-471 of 2L treatment along with the P-Ib (TACTIVE-U) trials in combination with ribociclib and abemaciclib
• Enrolment for P-III (VERITAC-3) assessing combination with palbociclib expected to initiate   in the H2’22 after a discussion with the FDA to update trial protocols
• Bavdegalutamide (ARV-110) and ARV-766, both in P-II development for prostate cancer.
  • The data from the clinical studies of both these drugs are expected in the Q2’23

BridgeBio Pharma (BBIO)

• BBO-8520 is currently under development for KRAS cancers & is currently being studied in IND-enabling studies
• The company plans to initiate P-I studies in 2023 with an expected result in 2024

Iovance Biortherapeutics (IOVA)

• In the conference, the company unveils its 2022 achievements which include the establishment of treatment centers throughout the US, and has been expanding manufacturing capacity at the Iovance Cell Therapy Center (iCTC). Also entered into deal with WuXi AppTec's Advanced Therapies business unit to provide additional cell therapy manufacturing capacity if required
• Also, the company’s expected milestones to be achieved in 2023 includes its plans to obtain Lifileucel’s BLA approval from the US FDA to treat advanced, unresectable or metastatic melanoma for which the rolling submission is already initiated in Aug 2022.
  • Expected price of lifileucel, post approval would be ~$400,000 and
  • Iovance is also planning to expand the combinations in earlier lines of therapy 
• The submission includes P-II study (C-144-01) data that has shown 31.4% [95% CI: 24.1%-39.4%] ORR with 9 CR and 39 PR and durability (median time from lifileucel infusion to best response was 1.5mos., and responses deepened over time)
• Moreover, Iovance is engaged with the CMS and commercial payors to facilitate reimbursement

Deciphera Pharmaceuticals (DCPH)

• Steven Hoerter (CEO) shared P-III (INTRIGUE) study results for 2L GIST and the P-III study (MOTION) results of Vimseltinib in patients with tenosynovial giant cell tumour. The combined potential peak sales of Oncology candidates Qinlock and Vimseltinib could exceed $1B globally
• Qinlock is the first approved TKI designed for GIST, gets a strong momentum by its launch in Germany and the post approval paid-access program in France.
• The company plans to initiate the P-III study (INSIGHT) of Qinlock in 2L GIST patients with mutations in exon 11 and exon 17/18; Vimseltinib in TGCT & DCC-3116 in multiple tumour types

SpringWorks Therapeutics (SWTX)

• Springworks’ CEO Saqib Islam anticipates the first US FDA approval for Nirogacestat in the coming years for the treatment of desmoid tumors
• The 1EPs & 2EPs of nirogacestat were met in the (DeFi) trial & the results were presented at ESMO 2022
• The company highlighted the upcoming expansive opportunities for nirogacestat in ovarian granulosa cell tumors

Alkermes (ALKS)

• In 2023, the company expects to spin off its oncology business into a development-stage oncology company. The investment strategy is coordinated by the medical and economic value of nemvaleukin alfa, a first-in-class IL-2 variant immunotherapy. The drug has received FDA Fast Track Designation for mucosal melanoma and platinum-resistant ovarian cancer with ongoing registration-enabling studies

Johnson & Johnson (JNJ)

• JNJ’s Spravato, an NMDA agonist showed a significant efficacy as early as 24hrs. after administration for treatment-resistant depression

Alnylam Pharmaceuticals (ALNY)

• The company highlighted the P-I results of zilebesiran in hypertension while the results from the P-II study & the combination study (KARDIA-2) are expected in 2023
• The major highlight for 2023 will be the first clinical data for an RNAi in the CNS, ALN-PP which targets APP production to treat Alzheimer's disease and/or cerebral amyloid angiopathy

Xenon Pharmaceuticals (XENE)

• Xenon highlighted its focus to differentiate the therapy from other seizure medicines by novel mechanisms and its ambitions to treat epilepsy & MDD
• XEN1101 is being studied in an ongoing P-II PoC trial for MDD & P-II trial (X-NOVA) in MDD (initiated in May 2022), results are expected in Q3’23
• The company plans to initiate a 2^nd P-III trial (X-TOLE3) of XEN1101 for focal-onset seizures and also intends to advance XEN1101 in FOS and primary generalized tonic-clonic seizures.
• The presentation also highlights the company’s other activities incl. upcoming P-II data on NBI-921352 for adult focal onset from its joint program with Neurocrine Biosciences, expected in H2’23

Anavex Life Sciences (AVXL)

• The company’s lead asset ANAVEX 2-73 (blarcamesine) restores neuronal homeostasis by re-establishing the SIGMAR1 levels of the body. Following is the information provided by the company related to ANAVEX 2-73’s clinical data:

• Release of positive topline data from P-III (AVATAR) trial for Rett Syndrome and P-IIb/III (AD-004) trial for Alzheimer’s disease
• Completion of positive P-II studies for Rett Syndrome and PDD
• Continuation of the OLE study (ATTENTION-AD) over 96 weeks
• Expected release of data from P-II 48-week OLE study in PDD
• Initiation of P-II imaging-focused trial and P-II/III trial assessing the drug in PDD and fragile X syndrome respectively
• The enrolment of patients in the P-II/III paediatric (EXCELLENCE) study for Rett Syndrome is near completion

• The company mentioned that following the full data set from ANAVEX 2-73 placebo-controlled study for Alzheimer’s disease, it will discuss the data with the respective regulatory authorities to commercialize the drug in Europe, Asia-Pacific, and the US
• The company also talked about its other lead asset ANAVEX 3-71 targeting the SIGMAR1 receptor (in different affinity levels) and one additional target. The P-II study evaluating the drug in schizophrenia treatment is expected to be initiated in the near term. Anavex is also evaluating ANAVEX 3-71 in the treatment of Alzheimer’s and Frontotemporal Dementia

Arvinas (ARVN)

• The company expects to advance with an IND or CTA submission for in the H2’23 the BCL6 degrader for DLBCL and LRRK2 degrader in Alzheimer’s disease in the H2’23

AxoGen (AXGN)

• The company is focused on peripheral nerve repair providing 4 unique solutions to improve the current standard procedure. These 4 solutions are:

• Axoguard Nerve Cap – it helps patients avoid long-term pain management by reducing the neuromas formation from amputations and other surgeries like tumour removal
• Axoguard Nerve Protector – it guards injured nerves and reinforces repairs
• Advance Nerve Graft – it is the flagship offering of the company aiding in reconnecting severed or damaged nerves
•  Axoguard Nerve Connector – it aids in reconnecting severed or damaged nerves

• Avance Nerve Graft is a biologically active allograft, presently used as a tissue product for surgical repair of nerve discontinuities. The clinical data from the (RECON) trial reveal that Avance aids in providing more chemical support for regrowth in case the gaps between the severed nerve endings get larger, lowers the likelihood of neuromas, and reduces the recovery time by up to 3 months
• Axogen plans to submit BLA to classify Avance Nerve Graft as a biologic by YE 2023
• Axogen has constructed a $65M production facility in Dayton, Ohio to help with the filing of this BLA. The construction is expected to be completed by H1’2023 along with manufacturing operations transfer to the new location

Bayer (BAYN)

• Bayer is currently investigating new gene and cell technologies intending to develop products in the CNS therapy area incl. Parkinson’s Disease and Huntington’s Disease
• The company will invest 25% of its R&D budget in 2023 toward the early-phase clinical trials investigating products for the treatment of these diseases

Abbvie (ABBV)

• AbbVie has highlighted its products for the treatment of migraine, psychiatric conditions, and neurodegeneration
• Migraine:
  • AbbVie’s Ubrelvy (oral CGRP treatment for acute migraine) & Qulipta (oral CGRP treatment approved for preventative treatment of episodic migraine) are under regulatory review for the preventative treatment of chronic migraine
  • Both Ubrelvy & Qulipta are expected to achieve sales of over $1B each by 2023
  • Additionally, Botox, approved for the prevention of chronic migraine, is also expected to add to the overall revenue of the company’s migraine portfolio
• Psychiatric Conditions:
  • Previously approved for the treatment of bipolar disorder & schizophrenia, Vraylar has now been approved as an adjunctive treatment for MDD
  • The total revenue of Vraylar is expected to reach ~$5B by 2023
  • Additionally, AbbVie’s ABBV951 is expected to receive approval for the treatment of advanced Parkinson’s disease in 2023 & is expected to touch a revenue of ~$1B

Alkermes (ALKS)

• Alkermes outlined its areas of focus which included neuroscience.
  • Launch of Lybalvi which is an oral atypical antipsychotic composed of olanzapine and samidorphan is planned as a treatment option for adults with schizophrenia or bipolar I disorder
  • The company also revealed the plan to launch LYBALVI direct-to-consumer advertising (DCA) campaign for campaign through digital and broadcast channels
• Additionally, Alkermes is looking forward to advance its orexin 2 receptor agonist (ALKS 2680) for orexin dysfunction in narcolepsy and other sleep disorders by targeting clinical PoC data YE 2023. The P-Ib PoC study is anticipated for H1 2023

SpringWorks Therapeutics (SWTX)

• The approval of mirdametinib (MEK inhibitor) for the treatment of NF1-PN patients is expected by 2025 & P-IIb trial (ReNeu) trial is expected in 2023

Vertex Pharmaceuticals (VRTX)

• The company has launched four approved medicines in cystic fibrosi & has eight programs in mid or late-stage development & anticipates five potential new launches in the next five years
• Trikafta and its three other cystic fibrosis drugs (Orkambi, Symdeko, and Kalydeco) received approvals in the younger patient population
• VX-522, an mRNA therapy is currently in P-I study and has received FTD from the US FDA
• Exa-cel is being studied in the P-II/III study for sickle cell disease and beta-thalassemia
• VX-548 is in P-III study for acute pain with expected completion of development at YE 2023 or early 2024

Insmed (INSM)

• Insmed highlighted P-II study results of Brensocastib for Cystic Fibrosis which showed avg. inhibition on NSP activity was dose-dependent, and the degree of inhibition appeared to be CATG>NE>Pr3 & the therapy was safe and well-tolerated, with no new safety signals

Beam Therapeutics (BEAM)

• The company focuses to advance its pipeline where BEAM-101, a clinical base editing program used to treat Sickle cell disease & the results from the (BEACON) trial are expected in 2024
• The regulatory filing of BEAM-301, a base editing technology targeting the liver is expected in late 2023 to early 2024 & for BEAM-302 (focus on E342K mutation) in early 2024
• The company also provided an update on the BEAM-201 program to bring the therapy into clinical trial

Ascendis Pharma (ASND)

• The company primarily focuses on the parent drugs & provides the potential benefits to patients.
• Skytrofa provides benefits in body composition, cardiovascular disease, fractures, and mental health on top of increase in height for PGHD with an expected launch in the US in 2023
• TransCon CNP showed positive data in the P-II trial (ACcomplisH) for children with achondroplasia & also received priority review from the US FDA, enrolment is open for an expanded access program in the US while NDA submitted to the US FDA for hypoparathyroidism, approval expected in April 2023

BridgeBio Pharma (BBIO)

• The company highlighted the cohort 5 of the P-II trial (PROPEL 2) for low-dose infigratinib to treat achondroplasia & the results are expected in early March 2023
• Ribitol is currently being studied in the P-III trial for the treatment of muscular entropy

Related Post: Insights+: Key Takeaways of J.P. Morgan Healthcare Conference 2022 Based on Therapeutic Areas